The future of health care in the United States requires modernization of clinical trial regulations and processes. On July 9, I had the opportunity to deliver that message and stress its importance in testimony on behalf of Mayo Clinic before the U.S. House of Representatives Energy and Commerce Subcommittee on Health. As part of the full Committee’s 21st Century Cures Initiative, the Health Subcommittee held a hearing focused on policy issues surrounding clinical trials. I applaud the Committee’s interest in this issue and urge continued focus on the need for reform to reduce the length of time from target discovery to approval of new drugs and treatments.
As I reported to the Committee, the average length of time from target discovery to approval of a new drug currently averages approximately 14 years, the failure rate exceeds 95 percent, and the cost per successful drug exceeds $2 billion, after adjusting for all of the failures. This situation must change.
I strongly commended to the Committee’s attention the work of the National Center for Advancing Translational Sciences (NCATS), a program under the National Institutes of Health (NIH), which was created in 2011 to speed up the translational process of science, or in other words to accelerate the speed of research from “bench to bedside.” NCATS is pursuing this goal at multiple levels, including facilitating new drug discovery, providing the tools to better understand human physiology and disease, discovering new biomarkers, and enhancing the conduct of clinical trials. NCATS currently funds 62 Clinical and Translational Science Award (CTSA) sites around the country, creating a network of potential clinical trial sites. The NCATS vision is very exciting for research, creating, among other things, the potential that high priority clinical trials funded either by NIH or by industry could be run very efficiently through all or part of this network.
To assist in this necessary effort to modernize the clinical trial process, I advanced a number of policy areas for the Committee to consider. In short, I offered four potential areas of policy focus for the Congress to consider, which could assist in modernizing clinical trials:
- Provide greater harmonization of regulations both within and between federal and state government agencies.
- Develop policies that encourage the Institutional Review Board (IRB) to have greater reciprocity with other institutions, thereby avoiding duplicative efforts, multiple times for a given clinical trial.
- Provide sufficient privacy protections to leverage the immense value of electronic health records for the benefit of clinical trials and critical outcomes research, including comparative-effectiveness research.
- Strengthen governmental and private sector support for discovery science. Similarly, provide incentives to enhance the ability of the medical centers to streamline their clinical trial process.
However, reform of the clinical trial process is not the sole domain of policy makers; both academia and industry have significant capability and responsibility to improve the processes.
I offered that academic medical centers might consider:
- Shortening the contract negotiation process with the increased use of “master agreements” and IRB reciprocity between academic medical centers and companies
- Enhancing the participation of minorities and women in clinical trials
- Creating a streamlined biospecimens policy that is broadly accepted across sites and companies
- Initiating a greater feasibility assessment for subject recruitment at each site in order to avoid initiating studies that are doomed to fail at that site
- Improving electronic capabilities to enhance recruitment, screening, enrollment and tracking of study participants
- Ensuring a robust pipeline of future clinical trialists with increased education and training of young clinicians in the conduct of clinical trials
Finally, the role of private industry in developing more creative, scientifically rigorous and efficient methods to conduct clinical trials cannot be underestimated and is certainly a critical success factor in modernizing the process. For example, private industry’s review of the efficacy of alternative clinical trial designs, such as “adaptive trials,” which aim to use the information generated in the trial as it emerges, not simply when the study has been completed, would be invaluable going forward.
Modernizing the clinical trial process is critical in capturing the promise of discovery for the future of health care. The path to this reform requires study, analysis and policy change on the part of all the stakeholders — government, academic medical centers and private industry. It is only through this collaboration that we’ll be able to deliver the necessary discoveries to our patients.
Editor's note: Sundeep Khosla, M.D., is the Dean for Clinical and Translational Science, principal investigator and director of the Mayo Clinic Center for Clinical and Translational Science (CCaTS)