Advances in scientific discovery are converging with unparalleled policy and regulatory action for medical research. At FasterCures, we have surveyed the landscape and interviewed subject matter experts to come up with our recommendations of the top 10 issues to keep an eye on in 2016:
1. Patient engagement: Put a ring on it (with apologies to Beyoncé)
Patient engagement was the "hot date" of 2015, and it's about time, right? Now, 2016 must be about turning it into a lasting commitment before the passion fades. Anecdotal evidence is building about the positive impact of patient input on R&D efficiency and effectiveness, but this needs to become a discipline that is scalable and actionable. Methods for collecting and applying patient perspective data must be created and consensus built around them. Skills and capacities need to be built in organizations, from industry to patient groups to regulators and, yes, even payers. Concerns about conflict-of-interest must be addressed. The U.S. Food and Drug Administration (FDA) is leading the way by offering guidance on incorporating patient preferences into clinical trial designs and using those data to inform approval decisions; its new Patient Engagement Advisory Committee will convene in 2016.
2. Value frameworks: The time has come to put patients in the picture
2015 saw the introduction of multiple frameworks for determining the value of new medical products, including those from American Society of Clinical Oncology, Institute for Clinical and Economic Review, Memorial Sloan Kettering Cancer Center and National Comprehensive Cancer Network. These tools will continue to proliferate, with varying audiences and objectives. While all claim to have patients' interests at heart, none have included patient participation and insight in any significant way. And all this at a time when patients are paying an increasing load for their health care. What are the critical elements of value from patients' perspective? How can and should they be considered in reimbursement decisions? What's the value proposition for payers in particular to engage with patient groups? Look for patients to get more engaged with these exercises in 2016 as they realize decisions are being made "about them without them." It's time.
3. Drug pricing debate: Fasten your seatbelts as there will be no rest for the weary
The debate over drug pricing is not going away as we enter a presidential election year, much as the biopharma industry might hope it will blow over, or that it can "focus the dialogue on value," or refocus attention (even if justified) on other high-cost elements of health care. In 2015, industry allowed others to define the value of its products (see #2 above). In 2016, it needs to offer something more concrete than talking points and gauzy patient testimonials. Companies hold the keys to the kingdom and we all want them to create better and faster treatments and cures, and we know this work is not for the faint of heart, but this responsibility includes honest discussion. They need to build trust with policymakers, citizens and payers. Experiments with alternative payment models are one way companies are doing that, but more strategies and, frankly, more dialogue are needed. They may need to seek policy changes to enable communicating health outcomes that extend beyond a product's traditional labeling information.
4. Precision Medicine Initiative lifts off: To infinity and beyond?
The planning for the Precision Medicine Initiative, led by the National Institutes of Health (NIH), has proceeded at warp speed since President Obama announced it in January 2015. There seems to be widespread agreement that it has the potential to be a transformative initiative for research, on the scale of the Human Genome Project, which has changed how we think about medicine and science dramatically. The devil, of course, is in the details. Enrollment for the million-person research cohort is set to begin in 2016; can we ensure that it is appropriately representative of Americans from all walks of life, and that trust will be built in the government's ability to handle sensitive health information? Funding opportunities are on the street, and collaboration and sharing will be required on an unprecedented scale. Two-hundred million dollars has been appropriated for the initiative; is this enough? Can all the stakeholders play nice and lean in to realize the vision?
5. FDA: Help wanted
FDA has been approving record numbers of new drugs and has lots of tools in its toolkit now to get important new treatments onto the market. Recent gains in regulatory efficiency cannot be sustained, however, if the agency's persistent challenge in building and maintaining its workforce isn't addressed pronto. The 2016 to-do list enumerated by the FDA's Center for Drug Evaluation and Research Director Janet Woodcock in a recent presentation is exciting yet overwhelming, and her center alone is short more than 600 staff. Changes in federal hiring policies and practices are urgently needed to allow FDA to effectively compete for the highly specialized staff it needs. The drumbeat of concern about this issue began almost a decade ago; time is now of the essence as we expect more innovation like the recent spate of activity in immunotherapy to come barreling through the pipeline at FDA. This team needs more players on its roster, a point the likely incoming FDA Commissioner Dr. Rob Califf - a Duke basketball fan - will surely appreciate.
6. Academic research: The Next Generation or final frontier?
The state of the academic research workforce is another issue that has been the subject of concern for some time but seems to be reaching a state of crisis. Many are familiar with the statistic that NIH grantees are now on average 42 years old at the time of their first R01 grant. Nature published a stunning graph in 2015 showing that researchers under age 35 received more than 20 percent of R01 grants in 1980 but receive fewer than 5 percent today. Bruce Alberts and colleagues claimed in a PNAS article in 2014 that we've produced an "unsustainable, hypercompetitive" system and that we're producing too many researchers for the number of positions available. Conversely, many senior academic researchers are concerned that they don't have junior faculty to whom to "pass the baton." Forums like Future of Research are springing up to give young researchers a voice in their own futures. Could 2016 be the year we begin to see some serious experimentation at academic institutions with "the way we've always done things" - upending the system of eternal post-docs, publication, tenure, the stigma of spending even part of one's career outside of academia? We certainly hope so.
7. Weaving the new evidence fabric: Quilting bees, anyone?
Another idea whose time has come seems to be recognizing the value and defining the appropriate role of different types of data and evidence at different points in the R&D process - as Arizona State University's Ken Buetow has put it, "how the new evidence fabric can be knit from all the emerging sources (Big Data, PRO data, new infrastructures like CancerLinQ, PCORNet, etc.) that complement the traditional RCT [randomized controlled trial] framework." FDA's Califf - an experienced clinical trialist - has exhibited strong interest in modernizing clinical evidence development with electronic health data and other "real-world evidence" (including patient-reported outcomes), as well as in the appropriate role and conduct of pragmatic trials. FDA's Sentinel post-market surveillance network and PCORNet's comparative effectiveness research infrastructure are exploring ways to integrate their data networks. As Califf has noted, most clinical trials fail to provide the evidence needed to inform medical decision-making in the real world. It's time to build consensus around what data we need to answer important questions throughout the R&D process.
8. Navigating the shoals of election 2016: Find your life jacket
It's hard to believe we have another almost full year of electioneering ahead of us. Can the gridlock-busting momentum behind the House's 21st Century Cures legislation be sustained, or will it fizzle in the Senate? Will the Senate manage to do anything to improve electronic health record interoperability? Might any unfinished business find its way into the "UFA" (user fee agreements) processes? Will drug pricing dominate the presidential platforms, or will others follow Hillary Clinton's lead (on Alzheimer's) and put investment in innovation front and center? The rest of the world has read our playbook on life sciences, and they are steamrolling ahead - will we read the memo we wrote in time? What leadership turnover might be in store at NIH, FDA and Health and Human Services? Lots to keep track of in the coming year, and much to make your voice heard on.
9. "Liking" the future of philanthropy
In 2015 Priscilla Chan and Mark Zuckerberg captured headlines when they announced they would donate 99 percent of their Facebook shares, currently valued at $45 billion, to charity, identifying curing disease as one of their top priorities. Former New York mayor and fellow philanthropist Michael Bloomberg noted, "When it comes to philanthropy, 30 is the new 70." Receiving almost as much attention as the dollar figure was the structure of the "Chan Zuckerberg Initiative" - an LLC, providing more flexibility for and control by the donors and possibly less transparency. As details about the initiative begin to trickle out in 2016, the spotlight will intensify on a conversation that has been going on for some time about the evolution of donors as passive supporters into more active problem-solvers, and about what the right investment tools are to achieve one's philanthropic goals.
10. The research-industrial complex is ripe for disruption
Ten years into FasterCures' existence, it finally feels like this is not a fanciful statement. Science and technology are driving it, patients are driving it, data are driving it and big federal priorities like the Precision Medicine Initiative will require it. Silicon Valley is driving it (finally): Google (Verily, Calico), Apple, Intel and 23andMe are significant players in R&D now, and philanthropists like Zuckerberg, Sergey Brin, et.al., are asserting their influence and bringing tech industry ideas and approaches to the party. Even Samsung is making biosimilars. Now what can we do to harness the potential and fly like an eagle towards cures? This is where the excitement will be in 2016. Can the research establishment keep up? What's your next move so you aren't left behind?