FasterCures' top 10 medical research issues and trends to watch in 2014

January 10, 2014

1. BRAINiacs. 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. The White House has made this a priority with NIH and DARPA leading the charge (though with few new resources attached), academia is organizing interdisciplinary efforts to tackle the science, venture philanthropy is weighing in, and after years of high-profile failures, industryaEUR(TM)s interest is being re-engaged. According to National Institute of Mental Health Director Tom Insel, diseases of the central nervous system, from AlzheimeraEUR(TM)s to autism to mental illnesses, are now the focus of the greatest number of start-ups and mergers and acquisition activity, surpassing interest in oncology. "The smart money is moving into the CNS area and industry will soon follow as we start to better understand the biology of the brain." The question is, how quickly can better understanding translate into better treatments?

2. Open-sourcing life science. FasterCures Fellow John Wilbanks has started a fascinating series of blog posts on how to apply the lessons learned from the open source movement in information technology to life sciences discovery and development. He fervently believes open source principles hold the promise of accelerating the R&D process, describing the foundation of open source thus: aEURoea loose collection of individuals, connected by technology, coming together for a variety of reasons to collectively create a product that is larger than the sum of its parts, distributed through computer networks at costs far lower than traditional commercial products.aEUR? While acknowledging that the two fields are fundamentally different in many ways, he notes that aEURoeopen source would have sounded insane in 1970,aEUR? and yet has become a critical foundation for computing (and hence almost everything we do) today."When three industry titans like Merck, J&J, and Sanofi are driving [data] sharing, itaEUR(TM)s fair to say the idea has traction." Are we finally beginning to crack the code of how to make life sciences as productive an ecosystem as computer sciences?

aEUR?3. Enrolling the quantified self in research. The concept of self-tracking using smartphones and wearable sensors isnaEUR(TM)t new, but there has been exponential growth in the number of devices, apps, tracking sites, and people using them. And these tools are moving from lifestyle applications to aid health care and medical research (see this recent blog post by FasterCures Fellow Bernard Munos). The Chronic Collaborative Care Network is using smartphones to collect passive and active data from patients with inflammatory bowel disease, merging measures into a behavioral index that helps patients and providers track how patients are doing in real time and triggers contact or interventions when certain cautionary patterns emerge. The Michael J. Fox Foundation sponsored a challenge to identify aEURoedryaEUR? biomarkers from cell phone data collected from ParkinsonaEUR(TM)s patients and healthy controls. TheyaEUR(TM)re exploring how this data can be used to better understand disease progression between doctor visits. Who are the entrepreneurs who will figure out how to make meaningful use of these torrents of real-world data for research and improved health? Probably not the ones who built the ginormous electronic health records systems that currently have our health data under lock and key.

aEUR?4. Collaborative capital. WeaEUR(TM)re hearing a lot more talk these days about blending or staging different sources of capital to support early-stage R&D. Pure Tech Ventures and JDRF announced in 2013 a new venture, T1D Innovations, a company-creation vehicle that will provide seed-funding to start-ups; JDRF is investing $5 million and the goal is to raise an additional $25 million from for-profit and not-for-profit investors. Family offices are exhibiting increasing interest in how to make the best use of both their for-profit and philanthropic investments to advance research in disease areas of personal interest. Broadview Ventures is a good example of innovative efforts in mission-driven investing in medical research, a field where that concept has lagged behind other fields of social endeavor (except in global health - see the Gates/JPMorgan-initiated Global Health Investment Fund, however, as a new example of blended capital in that space, with philanthropy providing loss protection). Securities crowdfunding will open up significant new sources of capital for emerging life sciences companies. WeaEUR(TM)re excited to see this trend grow and develop in 2014.

aEUR?5. Playing nice in the sandbox. Research-by-consortium is becoming an important part of the R&D pipeline; as part of FasterCuresaEUR(TM) Consortia-pedia project we discovered that in 2012 there were a record 51 new consortia launched. But thereaEUR(TM)s a growing sense of aEURoeconsortium fatigueaEUR? and confusion thataEUR(TM)s stemming from an unmapped landscape and unclear value proposition. WeaEUR(TM)ve made a first effort at defining the parameters of multi-stakeholder consortia, dissecting their characteristics, and categorizing existing efforts to help bring clarity, to be followed by an online database of existing consortia. Other organizations, such as MITaEUR(TM)s Center for Biomedical Innovation, are also seeking to bring some discipline to the aEURoescience of collaboration.aEUR? We expect increasing focus in this area as these efforts proliferate and organizations must evaluate the potential benefits of devoting their scarce resources to them.

aEUR?6. Regulation in the personal genomics era. ThereaEUR(TM)s been a lot of kerfuffle over the late-2013 standoff between personal genomics pioneer 23andMe and the FDA. Whatever course that particular case takes in 2014, we hope the larger issues at stake will get more airtime as a result, 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.

aEUR?7. Breaking through at FDA. FDAaEUR(TM)s new aEURoebreakthrough therapyaEUR? designation was the talk of the town in 2013. At the beginning of the year everyone wondered what on earth it meant and by the end of the year everyone wanted to have oneaEUR"and it seemed most of them did, with 34 granted, far exceeding anyoneaEUR(TM)s expectations, including FDAaEUR(TM)s. Setting aside the question of where all these breakthroughs have been hiding until now, the question for 2014 will be, can FDA continue to support this aEURoehigh-touchaEUR? approach for all these reviews, especially with continuing strains on its budget? Having mandated this program in FDASIA, will Congress put its money where its mouth is?

aEUR?8. Healthcare reform: aEURoeValueaEUR? for whom? ItaEUR(TM)s been a big year for health reform, but leaving behind issues related to implementation, weaEUR(TM)ve been observing the creep of concerns about reimbursement for new treatments up into the earliest stages of the R&D pipeline. WeaEUR(TM)ve felt strongly that patients need to be represented in a substantive way as debates about the definition of aEURoevalueaEUR? unfold among policymakers, payers, providers, and innovator companies (all of whom tend to claim the proxy of the patient). FasterCures held a workshop in July 2013 to begin to educate patient organizations about the importance of being involved in these issues and to educate other stakeholders about what patient groups can do to contribute to the evidentiary needs of payers and the methods used to capture patient preferences; stay tuned for a full report from that workshop right after the new year. Just as we see the FDA pursuing a new model for integrating meaningful patient input into the approval process (see below), 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 year 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. A theme that emerged from several of them was the importance of democratizing science, of opening it up, of questioning why things are the way they are. They are opening scientific inquiry up to citizen scientists, to crowdsourcing solutions to scientific problems, to young entrepreneurs from outside the field, to individual investors. They gave us a strong sense of how much untapped energy, intellectual horsepower, and resources are out there that could be turned toward solving our greatest health challenges. They are thinking about whether non-scientists can be trained to do Ph.D.-level science via online games, and about how citizen scientists can not only solve puzzles but can propose them. We hope aEURoethe academyaEUR? and the biopharma industry will sit up and take notice of such efforts in 2014 and find ways to help them grow and flourish.

10. Putting the patients in aEURoepatient-centeredness.aEUR? WeaEUR(TM)re certainly not the first to notice that absolutely everyone these days is proclaiming the importance of putting patients at the center of things - medical innovation, healthcare decisions, outcomes research etc. etc. etc. The problem is there are very rarely patients or patient representatives at the table for the discussions that matter about these issues. ItaEUR(TM)s time we all grappled with what those words mean in practice. How are policymakers, innovators, payers, and researchers going to take into account patient needs and preferences, patient-reported outcomes, patientsaEUR(TM) perceptions of risk and value, in a meaningful way, particularly as healthcare reform continues to unfold?

  • We would like to give a shout-out to the Patient-Centered Outcomes Research Institute, which seems to be putting its money where its mouth (or name) is. In December 2013 the new agency launched 18 grants totalling12 million a year each to form Patient-Powered Research Networks of activated individuals willing to provide their own patient-reported data for comparative effectiveness research. WeaEUR(TM)ll get a window into what those will look like in 2014.
  • On last yearaEUR(TM)s Top 10 list, we featured FDAaEUR(TM)s new Patient-Focused Drug Development Initiative. A year later, FDA has conducted five of the twenty planned disease-specific consultations on benefit-risk and has issued a series of reports summarizing patient community input for sponsors and innovators to learn from. Advocates for diseases not on the list of 20 are starting to apply the FDA template to gather similar information on their conditions. The new framework will be put to the test with new molecular entities reviewed in the coming fiscal year - stay tuned.