The FasterCures Track: January 2014 - The top 10 medical research issues and trends to watch in 2014

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Each year, your FasterCures team examines the landscape of issues in medical research and identifies the 10 trends to watch in 2014. This year, we will be tracking a wide range of issues ranging from the BRAIN initiative to the quantified self movement, collaboration and how to make it work, and this notion of who exactly defines value. Below is a summary; be sure to check out our full top 10 list.

  1. image of FasterCures' Top 10 list of 2014BRAINiacs: Having decoded the human genome and begun to crack the code of cancer cells in some small measure, medicine is turning its attention to perhaps the greatest puzzle of all – the functioning of the human brain.
  2. Open-sourcing life science: Are we finally beginning to crack the code of how to make life sciences as productive an ecosystem as computer sciences?
  3. Enrolling the quantified self in research: Who are the entrepreneurs who will figure out how to make meaningful use of the torrents of real-world data for research and improved health?
  4. Collaborative capital: We're hearing a lot more talk these days about blending or staging different sources of capital to support early-stage R&D.
  5. Playing nice in the sandbox: We expect increasing focus on research-by-consortium as these efforts proliferate and organizations must evaluate the potential benefits of devoting their scarce resources to them.
  6. Regulation in the personal genomics era: We hope the issues in this area get more airtime, such as the lack of public literacy about genomics, consumer rights, the power of the crowd in research, the big business of genomics, big data, and regulatory protections.
  7. Breaking through at the Food and Drug Administration (FDA): Can FDA continue to support the "high-touch" approach of breakthrough therapy designations for all these reviews, especially with continuing strains on its budget?
  8. Healthcare reform: Just as we see the FDA pursuing a new model for integrating meaningful patient input into the approval process, we hope to see 2014 bring new models for doing the same in reimbursement decisions, and we plan to help lead them.
  9. Democratizing science: One of the most inspiring panel discussions we heard at Partnering for Cures in 2013 featured five young innovators who are breaking the mold in medical research – not so much on the scientific front (though they are doing that as well) as on the process and business model fronts.
  10. Putting the patients in "patient-centeredness": How are policymakers, innovators, payers, and researchers going to take into account patient needs and preferences, patient-reported outcomes, patients' perceptions of risk and value, in a meaningful way, particularly as healthcare reform continues to unfold?
  • Want to know more about the Patient-Centered Outcomes Research Institute's network of activated individuals willing to provide their own patient-reported data for comparative effectiveness research? Check out more info below about our Jan. 29 Webinar on this topic.

This list is a reminder that, while there has been significant progress in medical research and we’ve seen exciting new drug approvals, the challenges in bringing important new treatments from discovery to patients remain. In 2014, we will continue to find effective and efficient ways to break down the barriers that are standing in the way of progress.

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Margaret Anderson
Executive Director

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Register today for Webinar on PCORnet: The National Patient-Centered Clinical Research Network
PCORI logoThe Patient-Centered Outcomes Research Institute (PCORI) announced in December an investment of more than $100 million in the development of PCORnet: The National Patient-Centered Clinical Research Network to improve the nation's capacity to conduct comparative clinical effectiveness research rapidly and efficiently and provide needed evidence to help patients and their caregivers make better informed decisions. In this Webinar, you will hear from PCORI's leadership about the vision for the network and its potential value for not only outcomes research but also new therapy development. This free Webinar is part of FasterCures' Webinar series designed to spotlight innovative approaches to disease research. Register now.

FasterCures Program Updates

photos from FasterCures Track January newsletter

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Patients and Policy

Our goal: Increase patient engagement in research and improve research processes and develop policies that will better support efficient development and approval of new therapies.

FasterCures releases workshop report on value and coverage
Value and Coverage report cover"Value and Coverage: How reimbursement decisions impact innovations needed to improve health" articulates findings from a workshop convened on the topic and key actionable recommendations for patient-driven organizations to consider. In this report, FasterCures notes that when it comes to the issue of value and coverage for medical innovations, the medical research community is in search of a better understanding of the evidentiary standards that payers consider when reimbursing treatment and care. The report states that establishing safety and efficacy is no longer sufficient when developing a new therapy; innovators must also prove value to patients, providers, and payers in order to generate a revenue stream that covers their R&D costs and provides adequate profits to fuel the next discovery's translation and commercialization. The report outlines a series of recommendations specifically for patient organizations to consider. Recommendations fall under three major themes: collaboration; leveraging data; and deploying smarter, better care.

FasterCures supports investment in NIH through United for Medical Research
United for Medical Research logoFasterCures
continues to support National Institutes of Health (NIH) appropriations through United for Medical Research (UMR), a consortium of patient, provider, and research organizations, as an active member and serving on the steering committee. Recent articles have brought attention to NIH funding, including an article in the Washington Post in which Carrie Wolinetz, president of UMR, said the proposed spending bill “won’t adequately reverse the damage done by last year’s budget sequester and ensure the nation’s biomedical research enterprise makes continued progress in lifesaving research and development.”

R&D Policy page offers resources about protecting federal investment in medical research
R&D policyFasterCures' R&D Policy page includes letters, reports, and more that demonstrate how the federal investment in medical research saves lives, creates jobs, and maintains U.S. leadership in the global economy. The page also includes coverage of the federal funding impact on states and the federal investment in innovation.

Blog: The Future of Medicine
photo of Bernard MunosWe already have the technology to monitor dozens of body parameters in real time, and much more is on the way, writes Bernard Munos, FasterCures senior fellow, in a blog post. The problem is that today all these data end up in different places, in different formats and are hardly exploitable, he says. But that could easily change.

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collaboration

Our goal: Stimulate innovative collaborations across all sectors - academia, government, industry, investors, and nonprofits - to speed up the time it takes to get new therapies from the lab to patients.

Rock Health’s top 50 digital health entrepreneurs
Margaret Anderson at Partnering for CuresBusiness accelerator and health IT seed fund Rock Health recently named the top 50 people with the most significant impact in digital health technology in 2013, a list that features FasterCures Executive Director Margaret Anderson. The list also included startup co-founders and CEOs, venture investors, and nonprofit and university officials. According to the article, "These are the people who inspire and motivate us by working to change the world for the healthier."

Anderson speaks about ‘Digital Health Manifesto’
Consumer Electronic Show logoAt the Consumer Electronics Show in Las Vegas on Jan. 9, Margaret Anderson joined a panel of healthcare leaders to identify and resolve barriers that impede marketplace success in order to optimize the health of the nation. The panel was moderated by Reed Tuckson of Tuckson Health Connections and included speakers from New Atlantic Ventures, Qualcomm Life, "The Doctors"/MD LIVE, Audax Health, and Kaiser Permanente.

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capital

Our goal: Facilitate greater access to and more strategic allocation of capital to support results-driven medical research.

Highlighting novel R&D funding models
Fixes in Financing case studiesBe sure to check out case studies on 10 novel approaches to financing early-stage drug development, including an orphan disease drug development accelerator, a precompetitive alliance to create shared cancer model platforms, a biotechnology company that experimented with crowdfunding, and more.

Tools you can use

Did you know that 9 out of 10 PhD scientists rely on NIH to support their research training at some point in their careers? View more helpful slides and graphics in the R&D Policy section of our Web site.
NIH impact graphic

Until next issue

If you have questions or would like more information about any of the items above, please contact info@fastercures.org.

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