21st Century Cures Act—An Intrepid Redesign and Commitment to Clinical Research
On December 7th, a revamped 21st Century Cures Act (“Cures”)—with its bold funding of large scale health innovations projects and dramatic charge to speed FDA approvals while maintaining current standards for safety and effectiveness—abruptly, and somewhat remarkably, completed its two-year journey through Congress.
Cures soon will be on its way to the President, who has credited it with having the ability to “end cancer as we know it” and to “[unlock] cures for diseases like Alzheimer’s,” and who has promised to sign it into law the moment it hits his desk.
With such lofty proclamations, the pressure is on for Cures to make real change. The big picture goals of ending certain human diseases are far off in the distance, but Cures will result in an immediate series of discrete actions that will usher in a period of dramatic changes to biomedical innovation and clinical research. It is these more discrete actions that will be our focus today.
Modernizing Trial Design
A key action throughout Cures is to make legal, regulatory, or policy changes that serve to loosen the traditional understanding of what constitutes the “adequate and well-controlled studies” necessary for supporting or sustaining market approval for drugs, devices, and biological products.
One such example is the addition of a provision to the New Drugs section of the Federal Food, Drug, and Cosmetic Act (FFDCA) requiring the US Food and Drug Administration (FDA) to develop a program that allows for the use of “real world evidence” in support of a new indication for an approved drug or to help with any post-approval study requirements. The FDA has already warmed to the idea of using less controlled data for medical devices, but to date has been reluctant to extend this flexibility to drugs or biologics.
Accordingly, Cures also revises the FFDCA, for both drugs and devices, to allow investigators the ability to forgo obtaining prior legally effective informed consent for certain types of clinical research. This flexibility is limited to only research that may pose no more than a minimal risk to participants and in which there are appropriate safeguards to protect the rights, safety, and welfare of such participants. This paves the way for the FDA Human Subject Regulations to include a waiver of informed consent provision much like the Health and Human Services (HHS) Human Subject Regulations and will presumably throw the door open wide for utilizing real world evidence in marketing applications.
Increased Access to Investigational Products and Information
A second key action throughout Cures is to provide greater access to existing investigational products or data that may be useful to both patients and researchers.
With regard to product availability, Cures further formalizes a Breakthrough Devices program to provide priority review for certain high need devices, creates an accelerated approval process for regenerative advanced therapies and requires FDA to establish clear standards for their marketing consideration, and expands the number of individual cases that can occur for a device to be considered for Humanitarian Use Device status.
Cures also builds on the progress that FDA has recently made in improving the process for Expanded Access (EA) to investigational medicines. Many, if not all, drug manufacturers will be required to make publicly available a policy for evaluating and responding to EA requests—even if that policy is to not grant any such request.
With regards to information availability, Cures once again looks to build on the recent progress from National Institutes for Health (NIH) to require clinical trials and results information be posted in a public form. Cures goes further and allows the NIH to require award recipients to share scientific data and gives NIH further abilities to ensure that summary results data are in fact being posted as currently required by law. Interestingly, Cures also recognizes that access to data remains a persistent problem for researchers due to privacy related concerns. Cures’ solution is to require HHS to release discrete and actionable guidance to help improve understanding around the access, sharing, and use of health data for future unspecified research purposes under the Health Insurance Portability and Accountability Act (HIPAA).
Removing Administrative Barriers for Research
A third key action throughout Cures is to remove perceived administrative barriers that serve to prohibit, as opposed to facilitate, the conduct of safe and meaningful research.
Cures requires the Secretary of HHS to harmonize and, if necessary, modify the HHS and the FDA Human Subject Regulations to avoid regulatory duplication and unnecessary delays. This charge specifically identified the need for improvement in multi-site research projects and the ability to use a central or single Institutional Review Board. In truth, there is no barrier to this other than institutional hesitation, but HHS could now be emboldened to mimic the recent change in NIH policy and mandate single IRB review as opposed to simply encouraging it.
Cures also seeks to provide relief to researchers and research institutions, who recently voiced significant concerns in the Public Comments on the Common Rule NPRM and the National Academies Report for Optimizing the Nation’s Investment in Academic Research, regarding the burden from disparate regulations, policies, and procedures endemic to funding agencies within the federal government.
Specifically, Cures addresses the complexities created by non-unified conflicts-of-interest policies and various non-complimentary animal care use standards. But it does not stop there: Cures goes so far as to require creation of a special “Research Policy Board,” similar to that proposed by the National Academies, who will be responsible for leading a widespread harmonization and minimization of effort for federally funded researchers.
Cures as the Proverbial Tip of the Iceberg
Once the dust settles and Cures is signed into law, the real work will begin. Big expectations have be placed on HHS, NIH, and FDA to work together to bring greater clarity, efficiency, and access to biomedical innovation and clinical research.
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