December 13 marked the one-year anniversary of the 21st Century Cures Act. While the law holds tremendous promise for enabling treatments and even cures for disease, multi-sector collaboration – and federal funding – is likely essential if the law is to reach its potential.
The three-year journey that culminated in the passage and enactment of the law was filled with idea generation and questions. What steps could be taken to accelerate the progress of biomedical research? What levers could be pulled to advance the science and the regulatory paradigms so patients can benefit sooner? As head of FasterCures, a nonprofit devoted to this notion, I participated in the first convening that the House Energy and Commerce Committee held and urged lawmakers to put the patient at the center of the legislation.
The notion of patient-centered care is not new. Janet Woodcock, director of the Center for Drug Evaluation and Research at the U.S. Food and Drug Administration (FDA), was recently given an award by the Reagan-Udall Foundation. In her acceptance remarks, she noted that patients are beginning to take drug development into their own hands. Numerous non-profit disease foundations have accelerated the pace of progress in their own spaces by creating patient registries, clinical trial consortia, and by providing research funds that can help evaluate high-risk ideas.
That concept of patient involvement was woven into the fabric of the Cures legislation. Ultimately, the final package included changes at the National Institutes of Health (NIH) and the FDA that highlight the science of patient input and patient-focused drug development, real-word evidence, data sharing, precision medicine, OCE oncology center of excellence efforts, opioid addiction, and the next generation of researchers.
How is implementation going after the first year? On November 30, Congress held a hearing to discuss the implementation of Cures. At the hearing, both FDA Commissioner Scott Gottlieb and NIH Director Francis Collins praised the legislation. However, they both had furrowed brows when discussing financial resources. While funding was included in the bill, it must still be approved through the appropriations process. The law allocates $6.3 billion in funding for NIH and FDA over the next 10 years, beginning in fiscal year 2017. Without proper funding for FDA and NIH, Cures could lose momentum.
What do stakeholders think? I asked several patient advocates and representatives of disease research organizations how they felt about the Cures effort as they reflected on the past year. There was a general sense of pride and enthusiasm for the way the community provided ideas and energy. Several people cited the bipartisan nature of the legislative process, which was led by Representatives Fred Upton (R-Mich.), former chairman of the House Energy & Commerce Committee, and Diana DeGette (D-Colo). Biomedical research is clearly something that can unify both parties.
From my conversations, four themes emerged:
- Resources matter: Federal staffers told me the legislation created a tailwind that helped push some initiatives forward. But many advocates worry that agencies need dedicated staff to implement this work – and resources are tight. Some industry observers have expressed a concern that some of the big science work could compete with basic science. One advocate was concerned that the pace of changes in the external environment (e.g., health reform and tax reform) is dividing the attention among patient advocates so they cannot focus on agency resources the way they would like.
- Patient centricity continues to propel the law forward: Many advocates are positive about FDA’s work to fulfill Cures requirements around the science of patient input. Forthcoming guidance is expected to provide more regulatory clarity that can benefit industry and the nonprofit community. Many nonprofits that were not part of the initial FDA ‘Voice of the Patient’ series have begun working with the agency to develop their own patient perspectives to enhance regulatory understanding. While FDA continues on its path forward, numerous pharmaceutical and biotechnology companies are clearly continuing to analyze their current efforts and examine the return on investment (ROI) based on regulatory clarity to date.
- Innovative approaches to implementation are welcome: Some people told me they were concerned that while agencies are implementing the intent of the bill, new requests for proposals (RFPs) might create a premature sense of complacency that the task is complete. While pursuing the implementation milestones, advocates cited the need for agencies to think out of the box as they implement solutions. Should agencies create some type of feedback loop to encourage further dialogue on the problems that were addressed in Cures? Should there be yearly convenings to discuss progress, and to allow external groups to help iterate the solutions?
- Big, audacious ideas are (still) needed: I recently attended a global philanthropy summit where a major philanthropist talked about the need for philanthropists to collaborate and place big bets to help foster big ideas. Other philanthropists cited the need for optimism to combat the “we couldn’t ever do that” spirit that often occurs when we are defeated by big challenges. The ideas and excitement generated during the crafting of the legislation was partially due to multi-sector involvement. While last year’s signing was an enormous step forward, the job is far from complete. Factors continue to trend in a positive direction for the future outputs of biomedical research, but we likely still need new approaches to drug discovery, more effective methods for seeking patient experience data, and greater focus on the scientific workforce pipeline.
The 21st Century Cures legislation was a bold, ambitious, and important effort. It was exciting to have played a part in its passage, but the work is not done yet. There has been a rise in the role of the consortia to problem solve pre-competitive challenges, but there are still things that should be tackled. The Cancer Moonshot, for example, was created to do some of this creative problem-solving, and there is much still to be done in that arena.
I am passionate about this field because the promise is so great, but the solutions are complex and require multi-sector collaboration. We should continue to help ensure that while we focus on implementing Cures, we don’t forget to imagine big, audacious solutions that include patients, industry, academia, government, and other important collaborators.