01/15/2015 10:42 am ET Updated Mar 17, 2015

Top 10 Medical Research Issues and Trends to Watch in 2015

At FasterCures, we are always thinking about ways to innovate the medical research system so that better treatments and cures get to patients faster. Read below to find out the top 10 issues and trends that we are watching this year:

  1. Crossing the aisle for cures. Kicking off 2015 will likely be the release of a draft of the 21st Century Cures legislation the U.S. House Energy & Commerce Committee has been developing under the leadership of Chairman Fred Upton and Representative Diana DeGette. The process itself has been a rare bright spot of comity and bipartisanship in the Congress, and a positive undertaking by members and staff to address a number of the entrenched systemic challenges in medical research that have become status quo. Will it make it through the wringer that it takes for an idea to become a bill and then a law? Perhaps, if patient groups and advocates like what they see and encourage their members to move it along.
  2. PDUFA VI: Regulations for patients? Also kicking off in 2015 will be the process of discussions leading to a new Prescription Drug User Fee Act (PDUFA), due to be passed in 2017. PDUFA V introduced some important initiatives to improve the regulatory process; what add-ons will the new round of negotiations bring? We'll be looking for continued improvement in the patient-focused drug development process that began with PDUFA V (specifically the inclusion of patient perspective in the assessment of desired benefits and tolerable risks, not just the understanding of disease and adequacy of existing treatments), a commitment to the sustainability of existing accelerated approval mechanisms like "breakthrough therapy designation," and more focus on the Food and Drug Administration's (FDA) regulatory science needs.
  3. Making the value case for new therapies. There was plenty of good news in FDA's unofficial year-end report, beginning with the fact that FDA approved a record 41 new drugs and biologics in 2014, the most since 1996, giving new options and hope to millions of patients. This included the highest number of orphan drugs and new biologics ever approved in one year, and 43 percent of new approvals were first-in-class drugs. While these numbers seem to bode well for the state of innovation - FasterCures' Senior Fellow Bernard Munos suggested earlier this month that we are on the cusp of a hyper-innovation age - how will the payer marketplace react? There may be a lot of very costly new drugs coming at us. Forbes's Matt Herper noted that the most important immunotherapy drugs just approved will carry price tags of $150,000 per patient per year. How will the value these new therapies deliver to patients be defined and evaluated? There is wide agreement that the current paradigm of uncertainty about how innovation will be paid for cannot be sustained, but will stakeholders be willing to align their interests with those of patients?
  4. Advancing the science of patient input. Will 2015 be the year we all move beyond lip-service to "patient-centeredness" and start building the science around patient-reported outcomes, benefit-risk, and value and coverage - in partnership with patients rather than on their behalf? More and more people are paying attention now, from regulators to industry to payers to academics. The Patient-Centered Outcomes Research Institute and FDA's Center for Devices and Radiological Health are leading the way to incentivize patient-centered approaches to clinical effectiveness research and medical device design and regulation. Capacity-building in the patient community is needed too, so that patients and patient organizations are prepared to generate the right data and provide robust, meaningful input other stakeholders need to inform their decision-making. A vehicle is needed for moving this agenda forward; who will lead the charge?
  5. Venture philanthropy really pays off. Undoubtedly there will be a frenzy of new or intensified interest among foundations in "venture philanthropy" with the Cystic Fibrosis Foundation's $3.3 billion payout from its investment in Vertex Pharmaceuticals - a game-changing return that the foundation will continue to invest in the future of cystic fibrosis research. This is proof-of-concept on a mammoth scale of the patient-driven R&D model, in which patients and patient groups provide not only funding but also patient data, insight into patient populations, patient participation in trials, and more to incentivize and drive the process in academia and industry. The philosophy - and commitment to it - goes way beyond inserting revenue-sharing provisions in funding agreements. Dozens of other foundations - from the Leukemia & Lymphoma Society to the Michael J. Fox Foundation for Parkinson's Research to the Alpha-1 Foundation and many more - are doing whatever it takes to move good ideas from the lab bench to the patients they care about and bringing all the partners they need to the table to make it happen. We expect to see more foundations pursuing that model in earnest in 2015 and more researchers and companies being very willing to partner.
  6. The emergence of debt as a financing mechanism for medical R&D. Medical research has lagged behind other sectors in the creation and application of novel financing methods to address market failures. However, new energy and focus around advancing different types of debt instruments are bringing new sources of capital to the table, particularly in the early stages of R&D. Social impact bonds, which are being employed in other fields of social need, are beginning to be discussed and deployed in the health space as well (with the leading edge in prevention and wellness, more so than new treatments). New York state has proposed issuing bonds to support Alzheimer's research, and some disease foundations are proposing doing the same in their own fields, such as the Foundation Fighting Blindness' Eye-Bond that would raise funding for blindness disorders through debt with a limited federal guarantee. There is also a debt component to the "megafund" concept proposed by MIT professor and Milken Institute Senior Fellow Andrew Lo, which has been attracting much attention over the last year or so. In 2013, the U.S. bond market was $40 trillion compared to $193 billion of venture capital investment, so there could be significant opportunity to tap into this investor base for life sciences R&D. We'll be following closely to see how these tools find a place in the flow of funds.
  7. Measuring effective collaboration. Collaboration has always been a focus for us at FasterCures because the complexities and risks of medical research require cross-sector, cross-disciplinary, cross-institutional partnerships to advance progress for all. The rapid growth over the last few years of collaborative research consortia would seem to indicate that stakeholders see value in these initiatives despite the challenges of creating, managing, and sustaining them. The good news is opportunities for such collaboration are growing. The bad news is that it's becoming increasingly difficult to know which are the best opportunities to take. With research-by-consortia taking off, watch for efforts to develop new frameworks and metrics for establishing and evaluating these efforts.
  8. Future of academic research. Academic research institutions are undeniably under duress. While some of it may be due to overbuilding research infrastructure during the years of doubling the National Institutes of Health budget, they are now dealing with a new normal of having to do more with less. There have been blue-ribbon commissions and white papers paying homage to the value these institutions create in terms of health and economic well-being. With pressures on tuition, grants, and reimbursement for healthcare delivery, it feels like time for a serious analysis of how they will be able to sustain their critical mission of research, alongside equally critical missions of education, training, and care going forward. Efforts to increase funding for NIH still matter greatly, the question is how that case will be made effectively on Capitol Hill in the years ahead.
  9. Personal health data in R&D. It finally feels like we're at a tipping point with regards to the ability of personal and mobile health data to be transformational in healthcare and research. (Just check out the Consumer Electronics Show in Vegas.) The technology is there, entrepreneurs are less scared off by the regulated nature of the industry, and FDA is getting more comfortable with the technologies. A recent initiative between GlaxoSmithKline and Medidata generated 18 million FDA-compliant data points, and a wave of mobile-enabled trials seems imminent. Could this also mark a turning point in patient/consumer empowerment? As individuals generate, possess, and contribute this valuable asset, will they demand access to the rest of their personal health information as well? If so, how will it be used by the individual and the health systems they interact with to deliver on the promise of better health?
  10. Understanding what's really escalating drug development costs. The Tufts Center for Drug Development has updated its estimate of the cost of developing a single approved drug to almost $2.6 billion. Whether or not you completely buy into their analysis, suffice it to say the costs are not going down but continue to increase dramatically. We were interested to note that in their analysis the time of development has actually declined and is having a mitigating effect on cost increases. We'd like to see in 2015 some further analysis of what the cost drivers actually are and whether industry and policymakers are targeting efforts at tackling those drivers directly. Both the 21st Century Cures initiative and PDUFA VI discussions are good steps toward identifying the policy fixes needed.