When politics and science mix, we all hold our breath.
Several pieces of legislation have been floated in Congress over the past year with the shared objective of encouraging biomedical research and therapeutics.
These follow in the footsteps of other recent
legislation designed to speed advances in medicine by modernizing regulatory
processes. Even many skeptics of the Washington political process hail some of
these efforts, such as Breakthrough Therapy Designation (BTD) and the Generating
Antibiotics Incentives Now (GAIN) Act, both implemented in connection with
Food and Drug Administration Safety and Innovation Act (FDASIA), as great successes. In 2015 alone, the FDA approved 45 new molecular entities, which included 10 with BTD and 16 drugs with novel mechanisms of action. Almost half were drugs for rare diseases.
Critical to their success, these initiatives were designed through a collaborative process with buy-in from multiple stakeholders – including FDA, patient groups, and industry.
More recently, H.R.6., known as the 21st Century Cures Act, was overwhelmingly passed by the House in July 2015. Its goal is “to accelerate the discovery, development and delivery of 21st century cures.” This legislation grew out of a bipartisan process, championed by Representatives Fred Upton (R, MI) and Diana DeGette (D, CO), that carefully constructed something about as close to a consensus as is possible in Washington today. Patient groups, industry, academia, FDA, NIH and other stakeholders all had a voice in constructing a balanced package of reforms designed to enhance the medical innovation ecosystem.
Following in the footsteps of the House, Senate leaders from both parties are advancing the Senate’s so-called “innovation legislation,” a suite of bills that collectively are that Congressional body’s companion to the House’s 21st Century Cures. Those bills were discussed across three Senate Health Committee meetings in the first half of 2016, with a total of 19 bills approved by the committee as of April 2016.
Like FDASIA in 2012, these bills are the product of efforts to build consensus and alignment across stakeholders, including FDA, and they have the potential to further the advances that grew out of FDASIA.
CDER New Molecular Entity (NME) and New Biologic License Application (BLA) Filings and Approvals
At the same time, it is possible to push too far and too fast in trying to speed new therapies to market. Earlier this year, legislation known as the Reliable and Effective Growth for Regenerative Health Options that Improve Wellness (REGROW) Act was introduced into both houses of Congress. REGROW is a bill “to amend the Federal Food, Drug, and Cosmetic Act with respect to cellular therapies” with the aim of supporting regenerative medicines.
A major criticism of REGROW is that it would introduce significant changes that challenge the basic architecture of the new drug approval process. As one example, REGROW would allow for the sale of stem-cell therapies that have been shown to be safe but have not yet been proved effective.
It is perhaps telling that multiple advocacy organizations in the field are opposed to REGROW – these include the Alliance for Regenerative Medicine (ARM), the International Society for Stem Cell Research, and most recently, a coalition of 10 patient advocacy groups that wrote a letter to Senator Mark Kirk (R, IL), the bill’s sponsor, expressing their shared concerns.
ARM noted “We continue to believe the proposal does not contain critical statutory protections for patients,” with the patient advocacy coalition similarly noting patient safety could be compromised with the conditional approval pathway stipulated by REGROW, and further, that it would be difficult for FDA to withdraw such products should it subsequently identify safety issues.
The need for new drugs to demonstrate not only safety but also efficacy has been the cornerstone of FDA regulation since the 1962 Harris-Kefauver Amendments to the Federal Food, Drug, and Cosmetic Act, passed in response to the agency’s experience with thalidomide. Critics, including FDA officials, have expressed concern that REGROW would upend over 50 years of standard practice at the agency.
Most everyone agrees that there is room to continue to optimize the FDA approval process, and that regenerative medicine represents an area of enormous promise. Efforts to enhance FDA regulation, like the 21st Century Cures process, should be welcomed. But any discussion of eliminating the requirement that new drugs be shown to be efficacious prior to FDA approval should be undertaken only with careful thought and buy-in from key stakeholders across the spectrum, including most importantly FDA itself.
Recent history has shown that Washington can act to advance biomedical innovation. But efforts such as REGROW remind us of the dangers of mixing politics with science.