fastercures

[fastercures]

In Case You Missed It: FasterCures Webinar on Using Innovative Financial & Business Models to Speed Science to Patients

by Rachel Tunis, Associate 

Overview: Aligning Economic Incentives

On February 20, FasterCures, a center of the Milken Institute, held a webinar exploring innovative financial and business models that could speed therapies to patients and catalyze future investments in biomedical R&D.

Speakers include:

Tanisha Carino, Executive Director, FasterCures, a center of the Milken Institute

Freda Lewis-Hall, Chief Patient Officer and Executive Vice President, Pfizer

Dan Liljenquist, Senior Vice President and Chief Strategy Officer at Intermountain Healthcare, Chair of Civica Rx

Colleen Rye, Director of the Aligning Incentives Program, FasterCures, a center of the Milken Institute

Rye provided an overview of FasterCures’ work in this area. The core issue, as Rye described, is that there are several channels for developing promising therapeutics that may not be a strategic fit for an organization or have uncertain commercial value—such as those with unclear reimbursement, weak patent protection, or small patient populations—even if there is promising science. Though these therapies could impact lives, they struggle to garner the needed investments, are cut from industry pipelines, and are fated to sit on a shelf unused.

FasterCures and the Center for Financial Markets, two centers of the Milken Institute, are collaborating to identify new pathways to capitalize the development of these underinvested therapeutics. They are investigating what an “ideal” market for these compounds might look like, how society could unlock fresh capital for innovation, and how new business models can be scaled to the ecosystem.

To open the market for new forms of capital, compounds must be out-licensed between organizations more freely. Traditionally, out-licensing has been a pharma-to-pharma transaction. But other transactions are possible: pharma to nonprofits, to spin-outs, to government agencies, or to virtual drug developers. New players bring new capital sources which could lower the cost of developing new therapeutics.

Dr. Freda Lewis-Hall: Pfizer; Board Member of SpringWorks Therapeutics

Lewis-Hall spoke about her experience with the Pfizer portfolio and the creation of SpringWorks Therapeutics, a drug development company that was spun-out from Pfizer.

Pfizer’s portfolio is large and includes internal R&D, in-licensed compounds, integrations, and collaborations with academic researchers. Such a large portfolio needs to be concentrated on funded, innovative, and strategic assets. Inevitably, many compounds fall outside of Pfizer’s strategic focus and remain unfunded. But, if these unfunded compounds are pharmacologically active, do not present safety concerns, fail their original indication, and have the potential for “off-strategy” indications, then they can be available for strategic partnerships. Since Pfizer has many ongoing partnerships, including collaborations with foundations, NGOs, academia, and venture philanthropists, its executives began to ask if Pfizer could create a fit-for-purpose model to advance deprioritized compounds.

SpringWorks was launched in September of 2017, with a portfolio of compounds in-licensed from Pfizer across several therapeutic areas. It was founded with financial backing from life sciences investors and is led by an experienced team of accomplished drug developers. As Lewis-Hall phrased it, when you put the right assets and the right people in the right place, you create a “sweet spot” for advancing therapeutics. SpringWorks expects to start to pivotal trials with two compounds from its portfolio in the first half of 2019. Both compounds are for rare oncology indications—neither of which currently have an approved therapy. According to Lewis-Hall, SpringWorks is “the beginning of a proof-point that is putting the financial and human resource investments in a novel mechanism, giving an opportunity to speed these therapies to patients with unmet need.”

Dan Liljenquist: Civica Rx

Dan Liljenquist provided an overview of Civica Rx and its efforts to address the shortages and monopolies in the generic drug market. Medication shortages, largely driven by consolidation, has affected many hospitals around the country by disrupting the supply chain for key drugs and created instability. Civica Rx, which Liljenquist describes as a democratized utility company for the manufacturing of generic drugs, will have the important role “to bring competition to the generic drug market focusing on value, price, and quality, and specifically to bring competition to areas… where there is a dominant player that has essentially cornered the market for a particular [generic] compound that we rely on for patient care.” Civica’s three chief objectives are:

Focusing on value and invigorating competition in the market for generic drugs

Ensuring a stable and predictable supply of essential generic drugs to correct shortages

Serving as a check against the aggressive pricing behavior of some generic drug manufacturers

According to Liljenquist, Civica Rx approach will be to establish fair, sustainable, and transparent pricing at all times, with no fees or rebates. The company also intends to become an FDA-licensed manufacturer, offering long-term, guaranteed contracts to hospital systems, with redundant manufacturing and safety stocks to avoid shortages and ensure the continuity of patient care. Finally, Civica Rx will implement a labeling system that, for transparency, indicates where a drug was manufactured.

Since the initiative was announced in January 2018, Civica Rx has received generous amounts of interest, news coverage, and support. In fact, one-quarter of the U.S. hospital market has signed agreements with the company. Liljenquist expects that the company will have its first 14 drugs on the market this year. Furthermore, Liljenquist believes that Civica Rx’s democratized utility model for the manufacturing of generic drugs can be scaled and replicated to other parts of the market.

Q&A

Partnering with patients

When asked how SpringWorks has worked with patient organizations, Lewis-Hall explained that patient input was helpful for the process of choosing compounds and therapeutic areas to advance. There are a variety of considerations in this process aside from just commercial potential, such as the regulatory environment, how to successfully measure outcomes in clinical trials, and how to identify the patient population, especially in the case of rare diseases. Patient organizations have been integral for SpringWorks to prioritize and balance such considerations.

Liljenquist addressed a question about whether the Civica Rx model passes on savings to patients. He said that Civica Rx would publish its market prices with the goal of driving transparency. Patients will be able to see if their health system has marked up the price of a drug.

Partnering with philanthropy

Lewis-Hall and Liljenquist both addressed the role that philanthropy has played for their organizations. In Lewis-Hall’s view, philanthropic organizations and foundations bring value not just for their financial contributions, but for their subject matter expertise, convening power, and relationships with patient groups and other stakeholders. As the system shifts from traditional models of collaboration to a more thoughtful “all hands on deck” model, the deep knowledge and expansive networks of philanthropic organizations will help advance therapies.

Liljenquist said that Civica has incorporated philanthropists onto their board for many reasons, but in particular, to ensure that in the future the capacity being built at Civica Rx will not be monetized by its members.  

The path forward

Responding to a question about what is needed to scale current efforts and create more innovative models, Lewis-Hall noted three things that she would like to see accomplished:

A network or system for facilitating knowledge sharing and connecting different stakeholders so that potential investors would know what assets might be available for investment

A cultural shift in the way we think about creating collaboration in what has traditionally been a go it alone environment. Scientists and industry may be reluctant to release their work to other parties for collaboration and further advancement, but this needs to change.

A recipe or algorithm to discover what types of models fit specific development needs. Variables that might be included, for example, include information on the patient community, unmet therapeutic needs, potential investors, and available assets. The most effective collaboration structure would output a series of opportunities and models that could be a good fit.

Conclusion

The issue, in both the development and manufacturing of therapeutics, is that patients often can’t get the therapeutics they need either because they don’t exist, they aren’t available, or they aren’t affordable. Rye provided a trenchant diagnosis of this issue; Lewis-Hall and Liljenquist gave prescriptions. SpringWorks Therapeutics is a replicable model for funding compounds that are deprioritized by pharmaceutical companies. Civica Rx is a disruptive business model for building and bracing a supply chain of affordable generic drugs. If these companies are successful as case studies, they may offer the biomedical system fresh models for getting patients the products they need—faster and more affordably.

[fastercures]

Access New Resources for Hosting Externally Led Patient-Focused Drug Development Meetings

by Julien Rashid, Associate

The U.S. Food and Drug Administration (FDA) and patient organizations use patient-focused drug development (PFDD) meetings as a tool to listen to patients and gather their perspectives. During these meetings, patients give their views on living with their conditions and current and future therapies. In 2013, FDA launched the PFDD initiative with plans to host 20 condition-specific meetings, a goal they have since surpassed. In 2015, after two years of demonstrated success, FDA shared the mantle and gave patient organizations the chance to host PFDD meetings (often called “externally led PFDD meetings”). Since then, patient organizations have successfully amplified the voices of patients through nearly 20 additional meetings.

Perhaps you’ve heard about the value of these meetings, or you’re considering or currently planning one. The success of previous meetings may make it look easy, but don’t be mistaken: PFDD meetings are not cut-and-paste. Each event is unique and requires months of planning, but there have been enough meetings that common practices and lessons learned can be shared, and organizations don’t have to start from scratch. For this reason, FasterCures is excited to announce that we are unveiling three new resources based on lessons from the field, specifically designed for those who are interested in hosting externally led PFDD meetings: the PFDD Readiness Assessment, the Smart Practices from Patient Community Leaders planning guide, and the PFDD Community Toolbox.

These tools are described below. Before continuing, we recommend that you read our background blog on PFDD meetings and FDA resources and visit our PFDD Resources webpage if you haven’t already.

The PFDD Readiness Assessment

How do you know when your organization and your community are ready for a PFDD meeting? This self-guided PFDD Readiness Assessment is designed to help you answer that question by asking you to reflect on the knowledge and capacity of your organization and the context of therapy development for your community. To use the Assessment, answer each question and tally your points as you go. At the end, evaluate your status using the scoring key. We’ll continue to update the Assessment with links to new resources.

PFDD Meetings: Smart Practices from Patient Community Leaders

This extensive guide details common practices, lessons learned, and considerations for planning PFDD meetings. The guide is based on interviews conducted with community leaders that have planned and hosted externally led PFDD meetings, and it has been checked for accuracy by FDA. It is organized chronologically into three planning sections: Section 1: Steps to Take Before Submitting a Letter of Intent, Section 2: Planning Your PFDD Meeting, and Section 3: Preparing for Post-Meeting Outcomes. Each section includes topics relevant to that planning period depending on where you are in your planning.

The PFDD Community Toolbox

Many organizations developed tools and resources for PFDD meetings during their planning process. The PFDD Community Toolbox is a collection of materials that were donated by organizations that have hosted meetings. You’re encouraged to reference or adapt these tools for your PFDD planning purposes.

A Note on These Tools

FasterCures developed these three tools based on internal expertise and interviews with patient advocacy groups that have hosted PFDD meetings. While FDA provided input on the accuracy of the content in the Smart Practices guide, all of these tools represent the perspective of FasterCures alone and do not necessarily reflect the perspectives of the organizations involved or the FDA. These tools do not set standards and will continue to evolve along with patient-focused drug development.

Direct all questions related to externally led PFDD meetings to the FDA’s PFDD Program Staff.

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The 21st Century Cures Act: 2-Year Update

Dec. 13, 2018, marked two years since the signing of the 21st Century Cures Act (P.L. 114-255). In its first two years, the Cures Act has contributed to significant advances in research, changed how stakeholders share health data, and helped modernize the biomedical ecosystem through far-reaching studies such as the All of Us Research Program and the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative. These advances have reduced obstacles to data sharing and encouraged more patient engagement and the use of real-world data in product development and approval. In addition, the Cures Act provided research funding, prioritized increased support for early-career researchers and high-risk, high-reward research, and provided new abilities for federal agencies to more competitively hire the skilled workforce needed in a 21st Century health-care system.

Through FasterCures’ 21st Century Cures tracker, we continue to monitor the implementation of Cures Act provisions and applaud the U.S. Department of Health and Human Services (HHS) for completing requirements that are improving the biomedical innovation system to the benefit of patients. New for 2019, FasterCures will issue a series of blog posts digging deeper into the impact the Cures Act has had on specific issues of interest. Stay tuned throughout 2019!

In Progress: Some requirements of the section have yet to be completed

Complete: All requirements of the section have all been met

Monitoring: FasterCures is monitoring the use of selected (completed) sections that establish new procedures or processes  

Two Years of Impact: Implementation Highlights

Since its enactment in 2016, the Cures Act has driven improvements in how research is conducted and incentivized, how products are developed and approved, and how health care can more effectively benefit patients. Below are a few key achievements marking these first two years of the Cures Act, as well as updates to watch for in 2019.

National Institutes of Health (NIH)

Sec. 2002: EUREKA prize competitions

The Cures Act required the NIH to support prize competitions to improve health outcomes for serious diseases that represent a significant burden in the U.S. as well as to advance biomedical science. On Sept. 11, 2018, the National Institute on Aging announced its Alzheimer’s prize challenge to improve care navigation and dementia care coordination for older adults. It is accepting submissions until June 30, 2019, and will announce winners in September 2019.

Sec. 2011: Precision Medicine Initiative

On May 6, 2018, the NIH launched the All of Us Research Program, a study aimed to advance the science of precision medicine by involving 1 million participants living in the U.S. So far, the program has enrolled over 150,000 participants, about 75 percent of which are from historically underrepresented groups in biomedical research.

Sec. 2041: Task force on research specific to pregnant women and lactating women

On March 21, 2017, the NIH established the Task Force on Research Specific to Pregnant Women and Lactating Women, which held many meetings throughout 2017-2018.

On Oct. 1, 2018, the task force submitted final recommendations for research on therapies used by pregnant and lactating women.

Food and Drug Administration (FDA)

Sec. 3002: Patient-focused drug development guidance

In May 2017, the FDA released its Plan for Issuance of Patient-Focused Drug Development Guidance under 21st Century Cures Act, Title III, Section 3002.

On Dec. 18, 2017, FDA held a public workshop on “Patient-Focused Drug Development: Guidance 1- Collecting Comprehensive and Representative Input,” including issuing discussion documents. On June 12, 2018, the FDA released the draft guidance.

On Oct. 15-16, 2018, the FDA held a public hearing, “Patient-Focused Drug Development Guidance: Methods to Identify What is Important to Patients and Select, Develop or Modify Fit-for-Purpose Clinical Outcome Assessments.”

On March 19, 2018, the FDA held a public workshop entitled “Patient-Focused Drug Development: Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data.” On Dec. 21, 2018, the FDA issued the draft guidance.

Sec. 3011: Qualification of drug development tools

On July 25, 2018, the FDA published its Surrogate Endpoint Table, listing surrogate endpoints that have supported approval of drugs and biologics.

On Dec. 11, 2018, the FDA held a public meeting, “Drug Development Tool Process under the 21st Century Cures Act and [Prescription Drug User Fee Act] PDUFA VI,” to discuss the drug development tools qualification pathway for animal models, biomarkers, and clinical outcome assessments.

On Dec. 11, 2018, the FDA issued draft guidance, “Biomarker Qualification: Evidentiary Framework.”

Sec. 3021: Novel clinical trial designs

On March 20, 2018, the FDA held a public workshop, “Promoting the Use of Complex Innovative Designs in Clinical Trials,” to inform the development of draft guidance on using complex trial designs to support product approval.

On Aug. 29, 2018, the FDA launched the “Complex Innovative Designs Pilot Meeting Program,” which allows selected drug and biologic companies to discuss the use of these trial designs in their product development programs.

Sec. 3022: Real-world evidence

On Sept. 13, 2017, the Duke-Margolis Center for Health Policy, supported by a cooperative agreement with the FDA, held a public workshop, “A Framework for Regulatory Use of Real-World Evidence,” to discuss the use of real-world data and real-world evidence in drug development and regulatory decision making.

On Dec. 6, 2018, the FDA released its strategic framework to evaluate the use of real-world evidence to support the approval of a new indication for a previously approved drug or to support or satisfy post-approval study requirements.

Sec. 3042: Limited population pathway

The Cures Act established a limited population approval pathway for antibacterial and antifungal drugs. On June 13, 2018, the FDA issued its draft guidance, Limited Population Approval Pathway for Antibacterial and Antifungal Drugs (LPAD).

On Sept. 28, 2018, Arikayce® became the first FDA-approved product under LPAD.

Sec. 3060: Clarifying medical software regulation

On Dec. 13, 2018, the FDA released a report, “Report on Non-Device Software Functions: Impact to Health and Best Practices.”

Sec. 3072: Hiring authority for scientific, technical, and professional personnel

In June 2018, the FDA provided its “FDA 21st Century Cures Workforce Planning Report to Congress,” outlining the agency’s needs and progress in recruiting and retaining scientific, technical, and professional staff.

Government Accountability Office

Sec. 4008: GAO study on patient access to health information

On May 5, 2018, the GAO issued its study on patient access to health information (GAO-18-386).

Centers for Medicare and Medicaid Services (CMS)

Sec. 4011: Medicare site-of-service price transparency

On July 31, 2018, the CMS announced that it would make available a website that provides comparison information between the outpatient prospective payment system and ambulatory surgical center payment and copayment rates.

Sec. 4012: Telehealth services in Medicare

In February 2018, the CMS published an updated Medicare Learning network booklet on telehealth services.

Looking Ahead to 2019

Below we list a few of the actions we are anticipating for 2019.

Sec. 2031: NIH Strategic Plan

We expect the NIH to release its strategic plan for advancing biomedical research and funding in 2019. According to a presentation by the NIH detailing its strategic planning process, the institute is currently seeking input to inform its plan.

Sec. 2041: Task force on research specific to pregnant women and lactating women

The NIH’s Task Force on Research Specific to Pregnant Women and Lactating Women automatically terminates on March 21, 2019 (i.e., two years after establishment), and the HHS Secretary may extend its term for one additional two-year period.

Sec. 3002: Patient-focused drug development guidance

We expect at least two more draft guidances on PFDD from FDA in 2019—one “describing processes and methodological approaches to development of holistic sets of impacts that are most important to patients” and the other “describing approaches to identifying and developing measures for an identified set of impacts (e.g., burden of disease and treatment), which may facilitate collection of meaningful patient input in clinical trials,” both informed by the public meeting held on Oct. 15-16, 2018.

We anticipate that the FDA will hold a number of public meetings in 2019, likely including one to inform draft guidance on “methodologies, standards, and technologies to collect and analyze clinical outcome assessments for purposes of regulatory decision making” and one to inform guidance on “how the Secretary, if appropriate, anticipates using relevant patient experience data and related information, including with respect to the structured risk-benefit assessment framework…to inform regulatory decision making.”  

Sec. 3021: Novel clinical trial designs

The Cures Act requires the FDA to issue draft guidance addressing the use of complex adaptive and other novel trial design in the development of new drugs or biologicals by Dec. 13, 2019. (Relatedly, in September 2018, the FDA issued draft guidance on “Adaptive Designs for Clinical Trials of Drugs and Biologics Guidance for Industry,” meeting a PDUFA requirement.)

Sec. 3051: Breakthrough devices

The Cures Act requires the FDA to issue a report to Congress on the breakthrough device review pathway and opportunities for improvement to better meet patient needs by Jan. 1, 2019. We have yet to see the report, but the FDA will likely share it publicly at some point this year.

Sec. 4004: Information blocking

We expect the Office of the National Coordinator for Health IT to issue its proposed rule on information blocking (as well as other topics, such as interoperability) in 2019 (the proposed rule is currently at the Office of Management and Budget).

21st Century Cures Tracker

For more information on upcoming statutory deadlines and completed actions, we invite you to visit our 21st Century Cures Tracker, which monitors the implementation of the 114 sections in Division A, including the key provisions relevant to biomedical research and innovation. The tracker seeks to highlight progress, measure impact, and identify areas where more resources or action may be needed.

21st Century Cures Impact Blog Series

In 2019 we are excited to enhance the 21st Century Cures Tracker by bringing you a series of resources to more specifically investigate the impact of selected Cures Act provisions on the biomedical and research ecosystems. Stay tuned for additional updates in this series.

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Voicing Patient Perspectives on FDA’s Digital Health Software Pre-Cert Pilot Program

by Brenda Huneycutt, Director, and Julien Rashid, Associate

One of the ways FasterCures helps bring patient perspectives into product research, development, and regulatory review is by convening groups of stakeholders to voice their perspectives on various topics. We recently brought together a small group of patients and patient representatives to explore their thoughts on the U.S. Food and Drug Administration’s (FDA) Digital Health Software Precertification (Pre-Cert) Pilot Program to help the agency shape the program.

This innovative pilot program shakes up the old paradigm of how FDA regulates certain medical devices. With this new approach, patient perspectives, needs, and opinions should be considered. Below we outline the Pre-Cert pilot program and describe some of what we heard from participants at our convening. For those wanting to offer their opinions on this pilot program to FDA, the agency is accepting public comments through its open docket until March 8, 2019. FasterCures will continue to provide updates as this program develops.

Regulating software products

FDA regulates thousands of medical devices—everything from popsicle-stick tongue depressors to electrical brain implants. The swell of digital health is creating waves of new and complex software technologies, such as mobile medical applications. However, the FDA’s current regulatory process is not built to accommodate the fast-paced, iterative development that software products go through. Imagine if the weekly updates for the apps on your phone had to each go through the FDA approval process—app developers would be discouraged, and the FDA overwhelmed.

To avoid delays in getting new software products to patients while ensuring the application of the same standards of safety and effectiveness to all devices, FDA is developing a new regulatory approach that will first be tested on one type of software called software as a medical device, or SaMD. SaMD is software used for a medical purpose—to treat, diagnose, drive, or inform clinical management that operates independently of hardware. For example, the software internal to an infusion pump that controls the motors and the dispensing of medication is not SaMD because it is a part of and necessary for the pump to work. However, the software of a mobile app on a smartphone that analyzes images from an MRI to diagnose a patient is SaMD since that same software could also operate on your laptop.

General wellness apps are not SaMD since they are used to maintain or encourage a healthy lifestyle and not used to treat, diagnose, drive, or inform clinical management.

FDA launched the Digital Health Software Precertification (Pre-Cert) Pilot Program in mid-2017 and revised the model based on public feedback, with a second version, v0.2, released in June 2018. To ensure that patient perspectives inform the development of the program, on Nov. 27, 2018, FasterCures convened a small group of patient representatives to provide their perspectives on this pilot program to FDA staff. On January 7, 2019, FDA released a new working model 1.0, a test plan describing how FDA intends to iterate the model, and regulatory framework for conducting the pilot program.

About the Pre-Cert Program

The Pre-Cert Program evaluates software developers on whether they meet certain standards of quality and organizational excellence. Software developers are pre-certified if they demonstrate excellence in five principles: (1) product quality, (2) patient safety, (3) clinical responsibility, (4) cybersecurity responsibility, and (5) proactive culture. Pre-certified developers must also have a robust method for collecting and analyzing real-world data, and they must commit to monitoring these data to assure product safety and effectiveness.

Pre-certified developers can bring their SaMD products to market either without pre-market review or through a streamlined pre-market review. Which pathway a product takes depends on (1) the risk category of the product, (2) the level of pre-certification of the developer, and (3) whether the product is a new device or an iteration of an existing device. After entering the market, developers must follow the product’s real-world performance by tracking (and sharing with FDA): (1) real-world health analytics (e.g., clinical safety and health benefits), (2) user experience analytics (e.g., user satisfaction), and (3) product performance analytics (e.g., cybersecurity).

Comments from Patient Representatives

During FasterCures’ convening, patient representatives described their individual views on the pre-cert program; the meeting was not meant to elicit a consensus of views. Below is a selection of comments we heard during the discussion about elements of the Pre-Cert Program:

Excellence Certification Assessment:

Companies should be evaluated on their patient engagement efforts and should be encouraged to engage patients earlier in the development pathway for medical devices. FDA should include guidance for companies and patients on what specific actions meet the excellence standard for patient engagement.

The proposed organizational elements listed in Appendix B (of working model v0.2) to demonstrate excellence resonate but may be difficult for a lay audience to understand.

FDA should consider the disease state when creating measures to establish excellence. For example, for patients facing a decline in function, holding steady or even a small decline in function could be a benefit, so the measure should not require improvement.

Healthcare provider understanding of the product is important, especially for some diseases where the healthcare provider may be a user as well.

FDA should make it clear to patients and other users if only a part of a company is pre-certified or different parts of the company have different pre-certification ratings. The pre-certification could be related to each product to try to make this clear.

FDA should give guidance to developers on what the company can and can’t say about its pre-certification status (which might differ among products), to avoid the public assuming all products from a pre-certified company are pre-certified.

Excellence appraisal should include whether the company proactively engages with users to identify issues and how it reports (including to users), abets, and resolves product issues to them.

Real-World Data Monitoring:

User experience is important in assessing safety. System downtime and customer satisfaction are not the same things; clinical safety and accuracy are not the same things.

Patients want companies to reach out proactively to users regarding known issues.

Many device errors currently go unreported because of the burden of reporting on users, so companies should try to reduce the burden on patients to report errors. Companies should tailor error reporting for different patient populations (e.g., reflect any physical limitations of that population).

Patients expressed that cybersecurity is a top concern given that the correct performance of these devices and the protection of user privacy is very important.

Product performance is the most important of the three listed real-world data domains (the other two are user experience and real-world health).

FDA should develop monitoring requirements that are flexible enough to allow a product on the market (and stay on the market) even if it is only helpful to a subset of the patient population (i.e., it doesn’t meet the needs of all users).

 Transparency:

FDA should define transparency and encourage companies to share data with patients in a format they can use. FDA should also consider data access granted to healthcare providers and the complex webs of data flow between SaMD products and electronic health records.

Algorithmic transparency is important; patients should be able to know the inputs of an algorithm (in a way that does not compromise intellectual property protection).

Companies should tell users when they recognize inaccuracies or flaws in their products and that they are working to fix them. It is also important for companies to relay to users why they make changes to the product.

FDA should make sure that the description of its pre-certification approach and review process (on its website and other materials) should be easily located and described in lay terms for the public. The description should balance simplicity and depth: simple explanations and labels should build awareness for all consumers, while comprehensive information should be easily obtainable for consumers who want it.

How to Voice Your Opinion to FDA

FDA will continue to develop and refine the pre-certification program throughout its pilot based on experience and comments from the public. Anyone is welcome to submit comments to FDA by March 8, 2019 (docket # FDA-2017-N-4301 Fostering Medical Innovation: A Plan for Digital Health Devices; Software Precertification Pilot Program).

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The Evolution of Venture Philanthropy in the Quest for Cures

by Kristin Schneeman, director

When is philanthropy no longer philanthropy? This and other existential questions—as well as more practical ones—were asked in a small-group dialogue that took place at the Milken Institute’s Future of Health Summit on “Venture Philanthropy 2.0.” More than 40 leaders from public charities and private foundations, investors of various stripes, biomedical product developers, and other interested parties heard about established and emerging models of philanthropic investment to accelerate R&D in the interest of patients. They considered issues such as:

What makes philanthropy unique as a source of capital?

What are the financing gaps where philanthropy can make the greatest difference?

What are the most significant challenges for venture philanthropy right now?

How can these financing approaches be scaled? What’s needed to build capacity and partnerships?  

This dialogue will inform FasterCures’ continuing work to provide thought leadership and capacity-building to support the growth of venture philanthropy in biomedical R&D.  We share some of the insights gleaned from the discussion in this blog.  

Background

FasterCures, a center of the Milken Institute, has had a longstanding interest in philanthropy as a source of funding for innovation in medical research. People sometimes wonder why, since philanthropy regularly represents only 2-3 percent of the U.S. investment in medical research every year. However, while philanthropy may be small in size, it can be unique in its role as a source of capital. Like venture capitalists, venture philanthropists are inclined to fund novel, high-risk research. They view philanthropy as a form of capital that can afford to take risk—particularly in the early stages of R&D where costs are relatively low but risks are relatively high.  

For the last dozen years, FasterCures has convened a network of patient-driven foundations that fund medical research and share a desire to be strategic and outcomes-driven with their funding and other resources. Our TRAIN (The Research Acceleration and Innovation Network) program has been a platform for these organizations to share information and best practices and for networking with other stakeholder groups. It also provides insights into the systemic challenges and unmet needs in the biomedical innovation ecosystem that foundations are trying to understand how to address with scarce resources.  

Private Session Discussion

Participants in the conversation at the Future of Health Summit heard an overview of existing philanthropic investment models that are working. . For example, over the last 11 years, the Leukemia and Lymphoma Society’s Therapy Acceleration Program has sought to de-risk the path for young companies by investing $120 million in 60 programs, with two U.S. Food and Drug Administration (FDA) approved products and a third on the way. Funding opportunities go through a very rigorous due diligence process, and terms are flexible depending on the company (e.g., convertible notes, equity, collaborative licensing).

Baiju Shah, CEO of BioMotiv, part of the Harrington Project for Discovery and Development started by the philanthropic Harrington family, provided a different perspective. Harrington features a nonprofit academic granting arm and a for-profit accelerator arm (BioMotiv) and is not disease-specific. It brings not just financial investment, but also development expertise and partnerships. This approach is useful in getting the assets that it invests in across the stages of translational research, where is there a high risk of failure, and into clinical development, which Shah described as “an effective machine for moving really exciting academic discoveries to the point where patients are benefitting from them.”

Participants also discussed several emerging models. Efforts to create hybrid funds, pooling nonprofit and for-profit capital, or staging different types of capital, have been tried in the past but now appear to be gaining traction. For example, Ed Walters and his partner, David Bates, of Tamarisc Ventures have been advising a family office on the creation of a philanthropic entity focused on mental and behavioral health. It is structured to maximize flexibility as a charitable trust with a for-profit LLC and private foundation built in. The foundation can be funded annually, as well as the investment capital for the LLC, and everything eventually flows back into the charitable trust. During the session, Walters explained, “We spent a lot of time asking our network and other experts out there for advice on what the optimal structure was and, to be honest, we're still looking,”, which produced knowing nods from many around the table.

Lisa Ryerson of AARP explained the well-known advocacy organization’s interest in supporting ventures that benefit the aging population, including as a significant investor in the Dementia Discovery Fund, as well as other social impact enterprises. This led to a discussion of the availability of new players to help scale venture philanthropy approaches. Finally, Garen Staglin, founder and chairman of One Mind, gave an overview of a new effort to create a large-scale social impact bond in collaboration with stakeholders such as the World Bank and the Society for Neuroscience. This endeavor will put more resources behind R&D in the high-risk area of neuroscience with the aim of alleviating the enormous economic and social costs of mental health disorders around the globe.  

As individuals in the private session made clear, philanthropy is playing an important role in high-risk therapeutic areas such as neuroscience and rare disease, as well as in particular stages of R&D such as translational science and early-stage clinical research. Later-stage clinical development is also an area in which new hybrid business models are incorporating elements of philanthropy or social responsibility. The group heard from Freda Lewis-Hall, Chief Medical Officer at Pfizer and a member of FasterCures’ advisory board, about a new business model that harnesses socially responsible investing to develop compounds that have been deprioritized by companies but that may still have the potential to be developed into new treatments to address unmet patient needs. “The question was, can you build a fit-for-purpose business model that allowed you to get these late-phase compounds developed as quickly as possible and into the hands of the patients that need them?” One approach described by Lewis-Hall was the creation of SpringWorks Therapeutics, a benefit corporation, which was initially funded by a group of other socially responsible investors, including Pfizer, and which was launched with rights to four compounds licensed from Pfizer. SpringWorks also has partnerships with patient organizations around individual assets.  

Following these discussions, several critical points on the key questions raised at the session’s outset were discussed:

1. Why philanthropy?

It can play a unique role in de-risking promising research for further investment.

Philanthropies can provide value other than financial resources, such as access to key opinion leaders, development expertise, and commercial partnerships.

Philanthropies tend to take a systemic approach and look for levers of change.

By nature, philanthropy can have a lower expectation of financial return than other investor types.

Two interesting questions that surfaced were:

Is there an important distinction between “venture philanthropy” and “impact investing”?

Does investing philanthropic capital potentially dissuade other types of market capital from coming in early or introduce undesirable conditions into the equation?

2. What are the challenges of venture philanthropy? 

Identifying optimal deal structures is hard.

Identifying/sourcing opportunities for investment can be challenging, especially in non-disease-specific contexts.

Philanthropy has a natural interest in openness and sharing, which can come into conflict with intellectual property and other professional/commercial considerations.

Financial and organizational capacity at philanthropies to do this kind of deal-making is usually limited – though success helps build capacity.

As health-care costs continue to rise, many patient-driven organizations are feeling pressure to try to exert influence on pricing and access.

There is little agreement on what reasonable and appropriate return on investment is.

Interest areas of foundations/philanthropists can be narrow.

3. How can these approaches be scaled?

Existing models and best practices should be more widely shared—there is no need to reinvent the wheel.

New players such as AARP could turn their attention and resources to investing in medical research.

A mega-fund of research projects could be securitized for investment.

Looking ahead, FasterCures will be exploring a variety of ways to use innovative finance to catalyze future investments in research. We and our colleagues at the Milken Institute’s Center for Financial Markets are investigating new channels to develop therapies stuck in the pipeline—and venture philanthropy is certainly part of the solution. Successes have mobilized more capital, and greater experience is promoting more sophisticated deal structures and organizational models. We look forward to further dialogue and collaborative work to harness the increasing power of philanthropic capital to create new channels for investment and development.  

[fastercures]

In Case You Missed It: Webinar on Master Protocol Trials and their Impact on the Future of Clinical Trials

by Anna DeGarmo, Associate

On December 10, FasterCures, a center of the Milken Institute, hosted a webinar titled “So You Want to Start a Master Protocol Trial.” Master protocols are an innovative model for conducting clinical trials, and they are gaining traction with regulators, product developers, and patients. These trials have the benefit of being patient-focused, time-efficient, cost-efficient, and highly collaborative. However, this model also poses challenges in the initial stages, requiring thoughtful planning and up-front collaboration and investment. The discussion focused on the advantages of conducting master protocol trials, current trials successfully using this framework, and lessons learned through implementing this trial design.

The need for a new model

FasterCures Senior Fellow Anna Barker, co-director of Complex Adaptive Systems, director of the National Biomarker Development Alliance, and professor at Arizona State University, moderated the webinar and began with an overview of master protocol trials, highlighting their significance and advantages in the research landscape.

Currently, clinical trials follow a “one-one-one” approach, where one trial seeks to answer one question about one drug. While such trials have long been the “gold standard” for clinical research and regulatory approval, Barker noted that “we are entering an era where this will no longer be sustainable”—in the era of precision medicine. One solution is conducting studies in which a single infrastructure and study design can answer multiple research questions about multiple agents more quickly and cost effectively. In other words, master protocol trials.

Lisa LaVange, professor and associate chair, and director, Collaborative Studies Coordinating Center, Department of Biostatistics, Gillings School of Public Health, University of North Carolina, followed Barker’s introduction by explaining the three different types of master protocols: basket, umbrella, and platform. Umbrella and platform trials involve researching multiple treatments for a single disease, whereas basket trials involve researching one drug in multiple disease cohorts.

In addition to the benefits associated with the multi-tasking abilities of master protocols, LaVange noted that multiple investigations being conducted under the same umbrella can mitigate the issue of finding and potentially sharing patients for trials and can allow for more coordination in challenging research efforts. Master protocols are proving attractive in both precision medicine and rare disease trials, as they can effectively deploy scarce eligible participants, at the same time giving patients the greatest opportunity to be placed in a trial that is most likely to benefit them.

With these benefits come initial challenges. LaVange noted that these are complex and expensive endeavors to get up and running, and she said patient advocacy groups have often been important catalysts. The U.S. Food and Drug Administration (FDA)’s vocal support (embodied in a recent guidance) has also been critical in engaging partners.

Master protocol success stories

While still relatively new, master protocol trials have already been successfully implemented and executed. Barker cited the I-SPY trials for breast cancer, one of the longest-standing master protocols, as an example of a successfully executed protocol. Since the I-SPY trial’s implementation, 1,300 patients have been enrolled in the study, 17 agents have been tested, and seven of these agents have moved to smaller phase III trials than would otherwise have been required. One drug has received accelerated approval by the FDA, while another has been given breakthrough designation. Additionally, the study has been able to validate a highly valuable surrogate endpoint that is able to be employed regardless of the drug being tested.

Adding to the master protocol success stories, Louis DeGennaro, president and CEO of The Leukemia and Lymphoma Society (LLS), told the story of “Beat AML,” a current umbrella study for adults recently diagnosed with acute myeloid leukemia (AML), an extremely lethal blood cancer that has seen no advances in treatment in 40 years. This patient-focused trial sought to determine the feasibility of implementing an umbrella study in patients with AML, the procession of patients from the master protocol to sub-studies, and the clinical efficacy of the treatments.

In the case of Beat AML, LLS holds the Investigational New Drug application, which allows it to convene all of the stakeholders needed for the study, of which there are many. Data are just now becoming available, but thus far, the trial is achieving its objectives of getting almost all patients assigned to therapy within seven days and moving patients to appropriate sub-studies. Additionally, the efficacy of the treatments observed appear to be promising.

Best practices in establishing master protocol trials

Following DeGennaro, the Pancreatic Cancer Action Network’s (PanCAN) chief medical officer, Victoria Manax, discussed her organization’s adaptive trial platform for studying pancreatic cancer. The platform, “Precision Promise,” is built with multiple study and control arms, includes patient-reported outcomes (in addition to clinical and molecular), and involves many stakeholders.

While successfully advancing the clinical trials available to patients with pancreatic cancer, Manax noted that the creation and implementation of Precision Promise faced challenges, including:

·         Infrastructure development: Multiple resources are required in developing an infrastructure, and it takes time to understand how it will best achieve the needs addressed in a study, as well as how it will fit the trial design. Strong governance and control are also necessary in determining an appropriate infrastructure in order to function on a daily basis.

·         Internal expertise: Master protocols are not like typical clinical trial designs, though having knowledge from internal staff who are familiar with clinical trials can be a useful foundation.

·         Time: There is a need to work quickly to meet the needs of the patient community, though designing and implementing master protocols require a lot of time up front. Such time includes designing a regulatory strategy plan, engaging pharmaceutical companies, and adapting to the evolving field.

Manax finalized her Precision Promise analysis and lessons learned by noting that “developing master protocols takes longer than you think, but there is no doubt that it is well worth it in the end.” Such feedback is positive, implying a promising outlook for the future of master protocol trials despite any initial challenges.

Conclusion

Concluding the webinar with key takeaways, Barker stressed the impact master protocol trials have on driving research and cures, as well as the importance of patient advocacy organizations in facilitating master protocol trials. With their ability to reduce study time, involve more patients, decrease costs, and better support precision medicine initiatives, master protocol trials create the option of a more sustainable structure for quickly driving science toward cures.

Related resources:

·         Watch a recording of the webinar

·         View the slides

[fastercures]

In Case You Missed It: Exploring the Frontiers of Data and Analytics for Precision Medicine, a Webinar in Partnership with the Kraft Precision Medicine Accelerator

On Oct. 3, FasterCures, a center of the Milken Institute, partnered with the Kraft Precision Medicine Accelerator at the Harvard Business School to present a webinar entitled “Exploring the Frontiers of Data and Analytics for Precision Medicine.”

The goal of the webinar was not only to introduce attendees to the Accelerator but also to highlight its landscape of organizations working in real-world data and advanced analytics to support precision medicine, including artificial intelligence and machine learning.

With so many changes and innovations occurring in the space in recent years, the webinar provided an opportunity for reflection on where the field of data for precision medicine has been and where it needs to go in order to meet the needs of patients across disease areas.

Setting the stage for data and precision medicine

FasterCures Executive Director Tanisha Carino opened the discussion by explaining why the work of the Accelerator is significant to FasterCures and the health-care field at large.

The role of personal health data is critical to any effort to advance precision medicine. Over the last year, FasterCures has introduced Health Data Basics, a project developed to engage patients with their health information and to help enable the two-way flow of health data between patients and the health-care system. Despite the numerous sources of health information being gathered at regular intervals, there are currently many challenges for patients and caregivers; the majority of individuals still do not have the resources to fully utilize their data in meaningful ways.

Carino introduced webinar speakers Kathy Giusti (Executive Chairman, Board of Directors, Multiple Myeloma Research Foundation [MMRF]; Senior Fellow and Co-Chair, the Kraft Precision Medicine Accelerator at the Harvard Business School) and Gabriel Eichler (Founder and Managing Director, Oak Health Partners; Data & Analytics Workstream Lead, Kraft Precision Medicine Accelerator at the Harvard Business School).

Giusti has been a friend of FasterCures for over a decade. The MMRF, which Giusti founded, is a pioneer in the field of venture philanthropy and has been involved in FasterCures’ network of organizations innovating in that space.  

Eichler has spent the last 15 years working to apply health-care technologies to problems in the pharmaceutical and biotech industry and the delivery of health care. He was formerly the vice president of products at GNS Healthcare. Prior to that, he managed several global client programs at PatientsLikeMe, focusing on patient-centered research and real-world outcomes.

An introduction to the Kraft Precision Medicine Accelerator

The overall goal of the Accelerator is to focus on how new incentives can drive great models in precision medicine. Giusti and her team started their work by looking at the greatest roadblocks facing precision medicine. Their hope is to devise sustainable solutions to these issues and disseminate them through impactful groups such as FasterCures in order to build a strong landscape of organizations innovating in precision medicine.

After many meetings with leaders in precision medicine to decide where they should focus their time, the Accelerator identified four highly integrated work streams: Direct to Patient, Data & Analytics, Innovative Trials, and Investment/Venture. Giusti explained that all four work streams are highly integrated. The Accelerator seeks to bring together many voices to focus on these four areas of need and collectively push precision medicine forward.

Analyzing the Accelerator’s real-world data landscape in oncology  

Eichler’s presentation was broken into two parts: 1) an overview of the Accelerator’s real-world data landscape and 2) a look at data analytics companies doing innovative work in the space.

To set the stage for his work in the Data & Analytics workstream of the Accelerator, Eichler made note that a useful recipe for anyone looking to create data-driven insights is: a well-defined question + a rich data source + an aptly applied, innovative analytical method. What comes out of this model is not answers, but hypotheses. Eichler stated that these hypotheses are useful in and of themselves and that no one walks away from these projects with all the answers, but rather a new place to start.

In 2016, the Accelerator looked at sources of real-world data across the landscape of oncology. Since then, many things have changed in the space—new data assets have been made available, new registries and platform trials have been started, and massive investments have been made in oncology. As such, the Accelerator decided to look at the landscape again in 2018.  

They focused a little differently while conducting the 2018 landscape and were interested in who is integrating molecular, clinical, and longitudinal data assets in the oncology. While talking to Academic medical centers (AMCs) and organizations collecting data, Eichler had an insight. Many people think of AMCs as having neat, accessible, clean, and ready-to go data assets. The reality is that we should think of AMC data more like a field of wild flowers rather than a pre-made bouquet—disbursed, complex, and often inaccessible. Eichler noted that funding is sometimes necessary on the AMC side to reap and harvest this data to be able to answer real research questions.

Overall, the Accelerator team noted several reflections while comparing the 2016 landscape to 2018’s:

Real-world assets are growing in size and sophistication: And patient registries have some of the richest data on specific diseases.

Sharing and linking models are winning: Acknowledging the need to link is a real positive in the field.

Willingness to share and link data are rising: While there may be technical and privacy issues to address, many companies are moving to this methodology.

The frontiers of real-world data are advancing: There are improved data standards and patient re-contact rights and mechanisms that are enabling additional research opportunities.

Leaders among real-world data analytics companies

Data analytics companies have been thrilling to watch in recent years. There is tremendous promise for what AI can do in health care, but Eichler warned that despite the barrage of headlines indicating an exciting future for the space, we should be careful not to get ahead of ourselves. To get an accurate take on what these companies are working on and indicate the emerging leaders in the field, the Accelerator put together a landscape of the top data analytics companies.  

The objective in building the landscape was to reach stakeholders (investors, researchers, non-profits, hospitals/care centers, payers, and patients) with key questions (Where are advanced analytics being most applied? Whom should I invest in? Is it best to build vs. buy vs. partner? When may these technologies be ready for real-world applications?).

They broke down this space into three key areas where AI is being applied (pre-clinical R&D, clinical research, and clinical care) and looked at 46 companies that met their criteria.

After creating this list, they looked at how these organizations were experiencing or demonstrating recent acceleration in their business through financing, job growth, and open positions. They also looked at the establishment of the organizations including publications, partnerships, and overall financing.  

While looking at how established these organizations were and how they were accelerating, Kraft noted the diversity in competencies of companies at different levels of maturity and development. When segmented out on the two-by-two matrix above, it was clear that some companies are industry-standard, highly established organizations, but are not accelerating rapidly. Others are just emerging, having tremendous growth but without much establishment.

Among these patterns, the Accelerator was most interested in companies that reside in the top right-hand quadrant of the above matrix: Hot Companies. These organizations have size and acceleration. Eichler commented that they are the most interesting companies to watch because they are breaking out of the mold and innovating in exciting ways.

Eichler detailed their key findings from this landscape:

Tremendous investments: These companies have raised more than $1.4 billion.

Diverse platform capabilities: Many companies are applying technologies to multiple use-cases.

Paradigm-shifting potential: These companies are young and will take a few years to mature; they have the potential to define the type of stories that will be use-cases for the future of health care.

Where precision medicine is going next

The webinar closed with some time for questions and answers, primarily revolving around the actionable next steps for those funding, conducting, or investing in research and development.

Giusti reiterated that the Accelerator is a huge believer in the data space and where it is going. In order to validate what they want to keep working on, they have to know what other data sets are still out there and whether AI companies have the resources to work with them. Giusti said that the field is in constant need of new data and new AI companies.

Eichler commented that all organizations, especially philanthropies, are trying to figure out their role and how they can bring real-world data into the mix. There is no shortage of challenges in trying to define the right questions to ask. The work that MMRF and others are doing to define, prioritize, and ask the key questions in their disease are going to unlock the most progress in helping patients.

In closing, Carino reminded the audience that precision medicine is where all diseases are headed. The Accelerator and FasterCures will continue their work to catalyze innovation in this space and look forward to collaborating on future endeavors to benefit patients everywhere.

Additional 

 Visit FasterCures’ Health Data Basics project

Learn more about the Kraft Precision Medicine Accelerator

Watch an archived recording of the event and view presentation slides

Register for the next FasterCures webinar, “So You Want to Start a Master Protocol Trial…” on December 10 

[fastercures]

Drive Patient Engagement by Pledging to Patients

by Cynthia Grossman, Director

In today’s health-care environment, you would be hard-pressed to find someone who would disagree that patients should be at the center of everything we do. Despite this widely accepted belief, there is still much work to be done to make this sentiment a reality. When it comes to patient engagement, we are still learning to walk the walk instead of simply talk the talk.

The good news is that there has been an expansion over the past few years in efforts to directly engage patients across the continuum of medical product development and delivery. Now we must build upon this foundation by encouraging more organizations and individuals to add their voices and action to further accelerate patient engagement in medical R&D.  

This is why FasterCures is excited to partner with Patient Focused Medicines Development (PFMD), a Brussels-based non-profit coalition focused on lifting up the patient voice across the lifecycle of medicine, to expand the suite of patient engagement initiatives contained in its SYNaPsE platform as well as to share its Pledge to Patients.  

Take the Pledge

The Pledge to Patients is an easy but significant step that all individuals, no matter their role in the health-care ecosystem, can take to acknowledge their commitment to patient engagement and join an accountable, global community that is driving this work forward. The pledge was created and endorsed by PFMD members. Members of PFMD are institutions or organizations that are eager to see a better future for patients and are working to drive impactful patient engagement worldwide. The Pledge to Patients was founded on the belief that small, individual action can lead to long-lasting, widespread change.

Through this pledge campaign, our goal at FasterCures is to help PFMD get to 1,000 pledges by October 5. PFMD’s goal is to engage the entire biopharma ecosystem worldwide to support the advancement of patient engagement and bring the reality of better solutions and outcomes to patients.

We encourage you to take the pledge and commit to joining the cause of advancing patient engagement and changing the health-care system for the better. Visit the pledge records page to see how others in the field have committed to the movement, and feel free to share with your networks. We are eager to see how this group of like-minded pledge-takers, who understand the power of patients, will shape the future of how medical products are discovered, developed and delivered.

Expanding the Patients Count Resource Library

In addition to sharing the Pledge to Patients, our partnership with PFMD will allow us to expand our Patients Count Resource Library through PFMD’s SYNaPsE platform. This platform is an incredible resource that gathers patient engagement initiatives in a searchable database, allowing individuals and organizations to connect as they seek to push the field forward. In the coming months, FasterCures will transition our resource library into the SYNaPsE platform; PFMD will host the library moving forward.

We are excited about this partnership with PFMD and the opportunity to multiply our patient engagement efforts on a global level. Stay tuned for updates about the resource library transition and the outcome of our Pledge to Patients campaign!  

Sign the Pledge to Patients and commit to driving patient engagement forward

[fastercures]

Advancing Models of Partnership between Patient Organizations and Shared Data Networks

Kristin Schneeman, Director, FasterCures

FasterCures, a center of the Milken Institute, the People-Centered Research Foundation (PCRF), and the National Evaluation System for Health Technology Coordinating Center (NESTcc) have embarked on a collaboration to better integrate patient perspective and participation, as well as patient-generated health data, into the growing landscape of shared data networks. This collaboration—the first of FasterCures’ new series of Acceleration Labs—will identify and describe models of partnership between patient organizations and research networks that are supporting the creation, use, and diffusion of evidence generated in disease-specific data platforms and in large, disease-agnostic data networks. The effort will also clarify the value of such partnership models in bringing patient perspective, experience, and data into the research process.

Clinical innovation—the development, approval, dissemination, and monitoring of new drugs, biologics, and medical devices—is in a period of rapid change. Researchers, product developers, government, patient organizations, and other stakeholders aim to accelerate medical research and innovation using all available data. While data resulting from randomized controlled trials remain the gold standard for efficacy research, there is now an increased emphasis on effectiveness and new opportunities for use of real-world data from electronic health records, mobile health devices, claims, and other sources. As part of this change, there is an emerging focus on the importance of engaging with patients in the definition of research questions and meaningful outcomes, as well as a re-examination of the role of patient-supplied data within the expanding yet still fragmented data ecosystem.

A vital actor in supporting the goal of a patient-centered medical research and innovation system is patient organizations. Patient organizations have a wide range of goals, capabilities, and resources, but at their core they have been established to help current and future patients by raising awareness, ensuring robust investment in medical research, and supporting innovation and access to effective preventive and treatment interventions. These patient organizations have funded discovery research, preclinical and clinical studies, and in some cases created new data repositories to support and advance research in their respective areas.

With the emergence of shared data networks and the investment patient organizations are making to advance medical research, it is an important time to develop models for what mutually beneficial and productive partnership can look like between patient organizations and shared data networks.

FasterCures Acceleration Labs deploy our unique capabilities to convene traditional and non-traditional partners to advance promising ideas in biomedical research and development to real-world implementation. Acceleration Labs are miniature think tanks in action, designed to devise new business models, capital structures, policy frameworks, or medical and financial technologies with the goal to move from research to results.

This initiative will be guided by an advisory group that includes, in addition to the three collaborating organizations, patient-driven organizations that have a significant investment in supporting research and creating data resources. Project advisors include:

Gina Agiostratidou, Director of the Type I Diabetes Program at the Leona M. and Harry B. Helmsley Charitable Trust;

Rachel Fleurence, Executive Director of NESTcc;

Kathy Hudson, Executive Director of PCRF;

Todd Sherer, CEO of the Michael J. Fox Foundation for Parkinson’s Research; and

John Wilbanks, Chief Commons Officer at Sage Bionetworks and a FasterCures Senior Fellow.  

As part of this project, FasterCures will create a typology of patient partnership models that incorporate the wide range of mutual needs and competencies across both patient organizations and shared data networks. We hope that the creation of generalizable frameworks and a shared vision of successful partnership will benefit many in the R&D ecosystem seeking to use a broad array of types of data to get to better answers to questions that matter to patients.

[fastercures]

Celebrating the Accomplishments of FasterCures’ Consortia-pedia Catalogue

by Taylor Cusher, Associate Director

Five years ago, FasterCures turned our attention toward the emergence of consortia in medical research. With some collaborations just starting and others having worked together for years, there were several fundamental questions on our mind: How many consortia are there in the medical research space? Who is involved in them? What are they working on? Are these collaborations really spurring medical research to happen more quickly and efficiently?

From our unique vantage point within the ecosystem, we set out to understand the landscape, share best practices, and highlight the essential questions to answer for anyone interested in establishing new collaborative efforts, expanding existing ones, or re-orienting early-stage programs. This resulted in a 2013 Science Translational Medicine paper, Consortium Sandbox: Building and Sharing Resources, and FasterCures’ Consortia-pedia report. In researching, understanding, and writing about these collaborations, we collected information about nearly 500 consortia around the globe and made them public through the Consortia-pedia Catalogue.

Since its debut four years ago, the Consortia-pedia Catalogue has had more than 16,000 unique visitors from 124 countries.

The most popular profiles (and their areas of focus) have been:

1.     Beta Cell Biology Consortium (Type 1 diabetes)

2.     Molecular Diagnosis and Risk Stratification of Sepsis Consortium (Sepsis)

3.     Accelerating Medicines Partnership (Alzheimer’s disease)

4.     Clinical Decision Support Consortium (electronic health records)

5.     TB Drug Accelerator Program (tuberculosis)

The top terms users have searched for have been:

1.     Alzheimer’s

2.     Cancer

3.     Pfizer

4.     Biomarker

5.     Multiple sclerosis

We are thrilled and thankful for the attention and support of all who have used the resource, promoted it, and learned from it. As the field has changed in the last five years, it is also time for FasterCures’ focus to adjust once again to emerging topics. The Consortia-pedia Catalogue will no longer be updated as of August 2018. While the site will remain available through the end of the year, we will not be refreshing or adding any new profiles. If you are interested in the details behind the Catalogue, please reach out to info@fastercures.org.

Thank you again for your interest, support, and use of Consortia-pedia throughout the years. Stay tuned for our next efforts and new directions!

[fastercures]

Your One-Stop Shop to Track Patient-Focused Drug Development Meetings

by Julien Rashid, Associate

Today, we’re unveiling a new resource: our PFDD Meeting Tracker.

In 2012, the U.S. Food and Drug Administration (FDA) launched its Patient-Focused Drug Development (PFDD) initiative. PFDD serves as a venue for patients to inform the FDA’s Center for Drug Evaluation and Research’s (CDER) assessments of benefit-risk for drugs and treatments.

While the FDA has listed every internally led and some externally led PFDD meetings, we saw an opportunity for a complete, collated list of past and future PFDD meetings. If you plan to host a PFDD meeting or if you are just interested in patient-focused drug development, use our tracker to locate resources from past PFDD meetings and to identify future meetings you might want to attend.

You can download a PDF version of our tracker (updated July 6, 2018). It includes all past and upcoming meetings; host organization(s); links to meeting reports, agendas, and video recordings; and information on attendance, public comments, surveys, and consultants. You can also find a more limited version of this tracker online.

On our tracker, you’ll notice there are missing links; those web pages are no longer available. If you have information about an upcoming or past meeting that could be included in this tracker, please contact Julien Rashid. We will be updating it as new information about meetings become available.

Our next blogs on this topic will feature best practices and lessons learned from organizations that have hosted PFDD meetings. Follow us to keep ahead of PFDD news, updates, and resources.

Related Resource:

·Introducing the Externally Led PFDD Blog series: Getting familiar with FDA Resources

[fastercures]

In Case You Missed it: Webinar on the Game-Changing Impact of Empowering Patients with Health Data

by Mark Williams, Associate 

The world of health data exists all around us, with apps, wearables, electronic health records, and other devices constantly gathering information about our lives. Clinical care, the focus of our health systems, only account for 11% of our overall health, meaning that there exists a goldmine of information gathered from diverse sources across an individual’s life. This information could not only benefit the biomedical R&D system but also could allow everyone to live happier, healthier lives.  

This environment is how moderator Tanisha Carino, executive director, FasterCures, a center of the Milken Institute, set the stage for the July 19 FasterCures webinar, “The Game-Changing Impact of Empowering Patients with Health Data.” In an R&D system that increasingly relies on data, patients are often unaware of where their gathered data are held and may not understand how to utilize and access their information. However, surveys have shown that when patients do access their data, they often find it useful.

Webinar panelists Lucia Savage, chief privacy and regulatory officer, Omada Health, Erin Mackay associate director, health information technology programs, National Partnership for Women & Families, and Taylor Cusher, associate director, FasterCures, discussed these challenges facing stakeholders across the health-care spectrum, in addition to highlighting the potential benefits of more fully utilizing health data as a point of connection within patient communities. 

System and Patient Responsibilities Regarding Health Data

Savage began by sharing that policymakers have only recently tried to understand privacy issues that may support or impede clinical information exchange. However, patients and their caregivers today have a much higher awareness of the differences between the data collected in a clinical setting and the information collected about our health every day through the use of apps and digital tools.

As Savage noted, if we want patients to be engaged in using their data, then the information displayed from these tools must provide real-time insights that are digestible, meaningful, and actionable.

During her time as chief privacy officer at the U.S. Department of Health and Human Services Office of the National Coordinator for Health IT, Savage heard research volunteers tell policymakers what they wanted regarding data. This included wanting to sign a single consent valid across institutions, have their input respected, know how their participation helped, and secure their data from the “Bad Guys” and not from each other. Research participants want institutions to collaborate and allow participants’ data to be used where needed.  

Savage argues that since researchers are asking patients to read and sign complicated documents for their participation in research, we should give them more credit as we return results back to them. As Savage posted in a tweet on this issue, “I am super sympathetic to results being misunderstood, but I would hypothesize that if the people described are competent to #consent, they are competent to get their results back.”

Consumer Pain Points and Tools to Help

Following Savage’s presentation, Mackay discussed the pain points currently facing the community of patients she serves through the National Partnership for Women & Families, as well as some tools that the organization has found useful in its efforts to engage individuals with their health data.

She highlighted that health IT and data are so important to the National Partnership for Women & Families because 82 percent of women make health-care decisions for themselves and their families, as well as providing two-thirds of family caregiving. Efficient and convenient access to online medical records and technology helps women in their role as “Care Coordinator-in-Chief” for their families.

In a 2017 survey, 53 percent of Americans who had been offered online access to their medical records were logging in and viewing their data. But for the 47 percent who were not, the top reasons cited were their desire to speak to a provider directly and their perceived lack of need for their health data. For those viewing their data, 85 percent used it to view test results, and 62 percent for “convenience” tasks — requesting prescription refills, completing paperwork, or scheduling appointments.

Mackay shared that her favorite finding from the survey was that patients encouraged by their provider to use their online record were twice as likely to do so. It is not enough to make portals available; providers need to help patients understand that their information is available to them and educate them about how it can be beneficial.

Several other pain points emerged from Mackay’s presentation:

Patients are still unaware of their rights in accessing their data

Requesting data can be confusing

Inconvenient delivery methods exist

Data are still designed primarily for the doctor

There are cultural issues around providers guarding the data and not sharing it

There is limited usability of the data provided

Data access should not be a replacement, but a complement, to the patient-provider relationship. As digital access is evolving, education is going to be vital in helping stakeholders understand that patients have a right to their data. But it is also important that people understand their own responsibilities to protect their health data as it becomes easier to access and manage it on their phones and devices.  

Breaking Down Barriers to Patient Engagement with Data

Given all of the current challenges and benefits in the world of health data, Cusher closed out the panelist presentations by discussing the Health Data Basics project that she has been leading for FasterCures.

The issue of health literacy among users has shown to be a barrier — only 12 percent of adults in 2003 could be considered “proficient” in understanding their data. If they cannot understand their health generally, then how can they understand their health data? Cusher also agreed that many patients find their data useful, but they often cannot track down where their data is shared. Furthermore, they often do not use their data to help them make decisions around their health.

Cusher went on to note that the potential future uses of data that participants identified are enormous, but we need to work on creating more systems to leverage them. Patients want to see their data put to actual use.

Given these findings, how do we get to a place where patients are able to not only access their data, but also understand and use the data that they are accessing?

Cusher identified a number of ways to achieve this:

Educate patients to improve their health literacy and understand data literacy

Remove technological barriers to data, yet maintain privacy and security

Build trust, be transparent, return results, and tell patients how their data is being used

Keep patients and their caregivers at the center

Collaborate, as no one can do all of this alone

Confronting Data Challenges

To kick off the Q&A portion of the webinar, Carino asked participants how do deal with possible privacy loopholes in platforms holding personal patient data, as has been recently highlighted in the media.

Savage answered that peer support is an important part of helping patients deal with illnesses, but we need to ensure that this technology is used without abusing privileges. While we do not regulate what individuals do with their data once they have it, they are responsible for securing it.

Mackay added that people will always have different thresholds regarding their comfort with data sharing and their privacy expectations. Some are willing to share “any and all of their data” in order to find a cure, while others are more stringent. As there is no one-size-fits-all solution, we need to educate people about their data to enhance their data literacy.

Carino went on to ask participants about how major cohort studies, outside of clinical studies, are changing engagement with the research process. Cusher shared that major cohort studies provide an enormous opportunity to do the research with all of the technological innovations that we have now, and that they lay the ground work now for studies in the future. However, there are concerns around establishing trust. Savage added that underserved communities can provide a benefit to their whole community by participating, but we need to keep in mind the ethical considerations of inviting people in and the conclusions reached from their participation.

Finally, Carino asked participants if the emergence of data technology necessitates an overhaul of HIPAA. Mackay answered that we need to educate people on what data sharing HIPAA already allows. Savage responded that HIPAA was an effort to digitize health care and is actually flexible and timeless, agreeing with Mackay that people misunderstand it. Savage further added that consumers are often confused that the protections that they expect in their physician’s office may not also apply in non-health-care settings, and this should be a bigger area of focus and concern.  

Navigating the Future of Health Data

Changes within the health-care system are necessary in order to reap benefits from health data across biomedical research and development. There is a certain level of responsibility on both sides —the patient and the system itself — to break down barriers to access, education, and the utilization of health information.

However, through evaluating current practices and addressing the influx of data that exists all around us, patients may be able to live healthier lives in which they can make informed decisions about their health, and interact with healthcare and biomedical R&D systems that are better equipped to truly meet their needs.  

Related resources:

Watch a recording of the webinar

View the slides

Check out FasterCures’ Health Data Basics project

[fastercures]

Continuing the Value Conversation

by Tanisha Carino, Executive Director

Today the Institute for Clinical and Economic Review (ICER) announced its governance board elected me as one of three new members, along with Mark Skinner, president and CEO of the Institute for Policy Advancement, and Anya Rader Wallack, associate director of the Center for Evidence Synthesis in Health within Brown University’s School of Public Health.

The goal of FasterCures has long been to ensure an environment that delivers more, better, faster, and cheaper cures and treatments to patients who need them. In 2015, we partnered with Avalere Health and over 23 organizations representing a wide range of health care to create the Patient Perspectives in Value Framework (link).  The PPVF’s goal was to incorporate measures of benefits and costs in the context of patients’ personal goals and preferences to assess the value of different health-care treatment options.  Today, the PPVF serves as a guiding light in designing decision support tools and informing the work of value-framework developers.  

In addition to advancing the work of the PPVF, it is critical to engage with organizations such as ICER to create that environment and to continue putting patients at the center of the conversation around value.  I am eager to work with the ICER leadership and Board to ensure that the signals the market sends to innovators reflect the perspectives of patients and continue to reward the innovation that the patients so desperately need.

[fastercures]

Creating Connections during the Patient and Caregiver Experience

Visiting the doctor’s office or local pharmacy is a common experience for almost everyone, and these visits can encompass a variety of hopes, fears, and curiosities for patients and caregivers alike. Whether you are getting your braces off or waiting on test results that can alter your life, these can be powerful moments throughout your health journey. However, are these moments in which the health-care system itself could – or should – reach out to educate and connect with you?

As part of Health Data Basics, a FasterCures project funded by a Patient-Centered Outcomes Research Institute (PCORI) Eugene Washington PCORI Engagement Award (#4202-MI), we sought to better understand what patient and caregivers experience in clinic and pharmacy settings. This information was crucial to answering a key question about connecting with individuals within the health-care system: when is the best time to engage with patients and caregivers to provide meaningful moments of education? Since Health Data Basics aims to increase patient and caregiver knowledge related to health data, ensuring that information is presented at a time and location where they are ready and open to receive it is vital.

To gain this understanding, our partners at GoInvo observed patients and caregivers in the clinic and pharmacy setting and created the following visualizations. They focus on what each person is doing, thinking, and feeling throughout the patient or caregiver experience, as well as the goals that they hope to achieve with each visit.

For example, during a clinic visit, a patient may feel sick and is likely concerned about the implications of his or her illness. At the same time, the patient’s caregiver is thinking about how to best comfort them, while trying to be quiet, so as not to disturb the others in the waiting room. Both the patient and the caregiver may be anxious and would like to get out of the visit as soon as possible.

This mix of feelings and actions indicates that the clinic waiting room might not be an ideal time to share messages related to health data. Whether because of not wanting to touch materials and pick up others’ germs or because of a caregiver’s role in comforting and distracting an ill patient.

We observed a similar experience among patients at the pharmacy. The consensus was that patients often view their trips to pick up their prescriptions as a chore and want to leave as soon as possible. With the additional distractions of other areas of the pharmacy for browsing or purchasing non-health related items, we discovered that the pharmacy is also not an ideal time or place to help individuals begin to understand their health data.

This observational data gathered through the Health Data Basics project provided insight into our question of when to engage patients and caregivers with education, specifically about their health data. Our ultimate conclusion is that those seeking to educate patients and caregivers should examine other opportunities outside of these settings in which to create points of connection and engagement.

Understanding how patients and caregivers feel during clinic and pharmacy visits is becoming increasingly important as medicine begins to take a more patient-centered approach. Future research should focus on what can be done to shift the attitudes of patients and caregivers to improve their overall experiences, as well as on how the clinic or pharmacy environment can be more considerate of what individuals are doing, thinking, and feeling to better meet their goals.

To learn more about how to engage patients and caregivers about their health data, visit the Health Data Basics website.

[fastercures]

How Patient-Reported Outcomes are Rocketing Patient Organizations to the Next Level

by Taylor Cusher, Associate Director

At FasterCures, we see great potential in more effectively using patient-reported outcomes (PROs) to advance the science of patient input. Patient organizations could play an important role in supporting that effort.

In theory, many agree that PROs used in research and clinical care should be based on what patients report as being most important to their disease management, health, and well-being, but this has often not been adequately considered as part of routine practice. Ensuring that PROs themselves are truly patient-centered is one element of the challenge ahead. Another challenge is aligning various stakeholders’ interest in, and their use of, PROs. 

There are many groups of stakeholders, each with their own goals for using PROs:

o Patients and caregivers hope that using patient-centered PROs will advance medical products that present better treatment options and/or can be used to inform care decision-making.

o Drug and device companies use PROs to demonstrate impacts of medical products that matter to patients and/or to differentiate their products from others in the same market.

o Clinicians frequently use PROs to assess how well individual patients feel or function as a basis for shared decision-making about care.

o Hospitals and clinics use PROs and other tools to understand clinical care outcomes or the way particular care approaches affect patient health and health-care utilization at the population level.

o The Food and Drug Administration (FDA) can use PROs, as long as they meet certain data requirements, as part of its benefit-risk determination and as evidence of unmet medical need.

o Payers use comparative and cost-effectiveness data, which can include PROs, to make reimbursement decisions.

At the end of 2017, FasterCures surveyed organizations that are using patient-centered PROs in their patient registries. The goal was to better understand the role patient organizations are playing in advancing the use of PROs.

Methods

We conducted the survey through the Patients Count Network, a digital directory of patient organizations maintained by FasterCures. Patients Count Network involves nearly 150 patient organizations of all sizes and areas of focus. The network serves as a one-stop shop to find patient organizations and as a mechanism for these organizations to showcase their programmatic and research resources.

We sent a survey on patient-centered measurement to 142 current members in late 2017. The goal was to better understand the types of work that they are doing related to PROs, who they are partnering with, and the purpose of their efforts. Thirty percent of organizations (42) responded. Most of the organizations are small, with 10 or fewer staff.

 Conclusions

Three-fourths of patient organizations surveyed are working on PROs.

Patient organizations are using their resources in multiple ways to advance the development and use of PROs. They indicated many uses for PROs and patient-perspective data and are partnering with different organizations and stakeholders to accomplish their work. Patient organizations are positioned to work within and possibly across diseases and conditions and may be an underappreciated partner in the development and use of patient-centered measurement.

Based on our findings and our prior work in this area, we recommend three key actions to drive patient-centered PROs forward:

1. Collaborate: Find the patient organizations that represent your disease area(s) of interest. They may already be doing great work, but if not, work with them early and often to make their needs and perspectives a starting point.

2. Find success and build on it: Where have PROs been successfully aligned with patients and effective for decision-making in R&D or care delivery? Don’t recreate the wheel, but do make sure existing PROs are aligned with what patters to patients. 

3. Consider technology: Electronic delivery of PROs and technology to manage and make use of the data are going to be critical aspects of the future viability and success of PROs.

For more of FasterCures’ work on patient-reported outcomes, check out the following resources:

· Webinar: Patient-Centered Measurement: How Close Are We?

· White paper: Patient-Reported Outcomes: Design with the End in Mind

· Blog post: Patient-Reported Outcomes and the Elephant in the Conference Room

· Blog post: What the Pros Know about PROs: Top 3 Lessons Learned from Implementing Patient-Reported Outcomes

[fastercures]

Meeting the Needs of the Rare Disease Community

by Tanisha Carino, Executive Director

I had an opportunity last week to be part of the Spotlight Health conference, the opening segment of the Aspen Ideas Festival held in beautiful Colorado, as a Spotlight Health Scholar.

This event brings together over 1,000 attendees to grapple with the most pressing issues facing all aspects of health – public health, global health, U.S. health care, and the future of biomedical innovation and affordability.  

Dual themes emerged for me throughout the conference – the great potential of science and new types or analytics applied to health, and the pressing needs that exist for society and individual patients. Nowhere is this duality more present than for patients and their families suffering from a rare disease.  

Rare or orphan diseases are those that affect less than 200,000 patients nationwide. You may not have direct experience with someone suffering from a rare disease. However, there are nearly 7,000 known rare diseases – hemophilia, cystic fibrosis, muscular dystrophy, and Friedreich’s ataxia, to name a few – and today, an estimated 25-30 million (or 1 in 10) Americans live with a rare disease. This means that in our communities where we live, work, and play, there is someone who is waiting for the next cure or effective treatment.  

FasterCures has a long history of supporting efforts to accelerate medical research overall, and especially for rare diseases. I was encouraged to meet so many more colleagues in Aspen who have joined in this mission.

Medicine’s “Platinum Age”

One presenter posited that we are no longer in the “golden age” of medicine, but because of the investment in genomics, we are now entering a “platinum” age. Based on the insights that have been accumulated about our genes and their link to medical conditions such as blindness and hemophilia, we are beginning to see the start of Food and Drug Administration-approved gene therapies. The science brings hope, but it may also bring hype. There was an acknowledgment and engaging discussion at the conference about how to manage the expectations of patients and the public surrounding these advancements.

Blockchain isn’t Just for Bitcoins

This new era of medicine is also made possible by the availability of data and the application of advanced analytics. A Facebook Live interview that I hosted with Dr. Shao-Lee Lin, Horizon Pharma’s head of research and development and chief scientific officer, and Ronald Bartek, president and co-founder of the Friedreich’s Ataxia Research Alliance, recognized that new data structures are needed to accelerate research on a wide range of rare diseases. Our discussion also highlighted the importance of partnership between industry and patient organizations to invest in the knowledge needed to turn science into treatments and cures.

One of the most thought-provoking ideas about blockchain came from Shada Alsalamah, a visiting scholar at MIT Media Lab during a session titled “Unlocking the Power of Blockchain in Healthcare.” She showcased two case studies for how blockchain was transforming the patient experience and enabling more collaborative, patient-centered care – an important need for patients with rare conditions who may have to seek care from a wide range of physicians and providers.

Innovation and Affordability, a False Choice

Treatments for rare diseases can be costly. Spark Therapeutics’ new treatment for blindness, LUXTURNA™, costs $425,000 per eye. During a breakout session at the conference, Spark Therapeutics’ CEO Jeff Marrazzo talked about the various approaches it was taking to work with payers and patients to ensure access.

For patients with rare disease, access to affordable insurance is critical. Atul Gawande reminded attendees that “life is the accumulation of pre-existing conditions.” Children make up over 50 percent of those newly diagnosed with a rare disease. For them, their pre-existing condition starts at a young age.

Joining the Call

Despite modern advancements within the biomedical R&D system, my time at Spotlight Health made clear to me that there is work to do to deliver more, better, faster, and cheaper treatments and cures to patients suffering from rare diseases.

As we look forward to the road ahead, it is encouraging that we at FasterCures will not be alone in these important efforts. In reflecting on the conference, I am inspired by the many wonderful colleagues I interacted with who have joined the call to serve rare disease communities. I am hopeful that continued collaboration among all parties, most especially the patients themselves, will lead to bright futures for those suffering from rare disease.

[fastercures]

21st Century Cures Act: 2018 Mid-Year Update

June 13, 2018, marked 18 months since the signing of the 21st Century Cures Act (P.L. 114-255). FasterCures continues to assess its effect on the biomedical innovation system, and we are pleased to see progress on implementing Cures Act provisions. At FasterCures we talk about saving lives by saving time, and the Cures Act continues to succeed in that mission.  

2018 YTD Implementation Highlights

There are several recent deadlines that we’ve eyed with excitement. We would like to congratulate the U.S. Department of Health and Human Services (HHS) for completing and sharing key deliverables with the community.

Sec. 3021. Novel clinical trial designs.

The U.S. Food and Drug Administration (FDA) was required to host a public meeting by June 11, 2018, to discuss the incorporation of complex adaptive and other novel trial designs into clinical protocols and new drug applications. The FDA hosted this public meeting, "Promoting the Use of Complex Innovative Designs in Clinical Trials," on March 20, 2018.

Sec. 3002. Patient-focused drug development guidance.

The FDA is directed to release at least one draft guidance within 18 months of enactment and a plan for issuance of the PFDD guidances.

On June 12, 2018, the FDA released draft guidance on “Patient-Focused Drug Development Collecting Comprehensive and Representative Input.” Concerning the public docket for the draft guidance, electronic or written comments can be submitted by September 11, 2018.

Additionally, FDA held a public workshop on Dec. 18, 2017, on "Patient-Focused Drug Development: Guidance 1 – Collecting Comprehensive and Representative Input” including discussion documents. FDA held an additional workshop on March 19, 2018, “Patient-Focused Drug Development: Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data.”

Sec. 3072. Hiring authority for scientific, technical, and professional personnel.

The FDA submitted a required report on workforce needs and planning to Congress on June 12, 2018.

Sec. 4008. Government Accountability Office (GAO) study on patient access to health information.

The GAO was required within 18 months of enactment to publish a study on patient access to health information. The GAO published GAO-18-386 on May 5, 2018 (full report available here).

Sec. 4012. Telehealth services in Medicare.

The Centers for Medicare & Medicaid Services (CMS) was required by Dec. 13, 2017, to provide information to Congress on use of telehealth services by Medicare. CMS built upon its 2017 work by publishing an updated Medicare Learning Network Booklet on Telehealth Services. Of note, Congress also included landmark support for telemedicine in Medicare in the Bipartisan Budget Act, with specific sections on the topic:

Sec. 50323. Increasing convenience for Medicare Advantage enrollees through telehealth.

Sec. 50324. Providing accountable care organizations the ability to expand the use of telehealth.

Sec. 50325. Expanding the use of telehealth for individuals with stroke.

Sec. 3086. Encouraging treatments for agents that present a national security threat.

The section establishes a Priority Review Voucher program at FDA to incentivize the development of medical countermeasures. FDA issued draft guidance for industry, “Material Threat Medical Countermeasure Priority Review Vouchers," on Jan. 17, 2018.

Looking Ahead to Other 2018 Deadlines

National Institutes of Health (NIH)

Sec. 2031. National Institutes of Health strategic plan.

Develop and publish the NIH Strategic Plan.

Sec. 2038. Collaboration and coordination to enhance research

Develop and disseminate appropriate measures related to reporting health information about sexual and gender minority populations.

Release of triennial report for inclusion of information regarding number of women and minorities included as research subjects.

FDA

Sec. 3011. Qualification of drug development tools.

Host a public meeting to solicit input on a new qualification process for biomarkers and other drug development tools.

Publish taxonomy on drug development tools for public comment.

Sec. 3022. Real world evidence.

Develop and begin to implement a framework to evaluate the use of real-world evidence to support the approval of a new indication for a previously approved drug or post-approval study requirements.

Office of the National Coordinator for Health Information Technology

Sec. 4001. Assisting doctors and hospitals in improving quality of care for patients.

Adopt certification criteria for a voluntary program for health IT use in pediatric settings.

Issue a strategy and recommendations for improving patient care and reducing the administrative burden of electronic health record use by medical professionals.

Sec. 4004. Information blocking.

Determine activities that do not constitute information blocking and therefore are not subject to penalties established by this section.

21st Century Cures Tracker

For more information on upcoming statutory deadlines and completed products, we invite you to visit our 21st Century Cures Tracker, which monitors the implementation of the 114 sections in Division A, which include the key provisions relevant to biomedical research and innovation. The tracker seeks to measure impact, highlight progress, and identify areas where more resources may be needed. While we are pleased with Cures Act implementation to date, we know that there is much still to be done and look forward to seeing more progress this year.

 For more information and to view the tracker, visit http://www.fastercures.org/21CC