By Ken Mills, Sara Berl and Nina Hunter
Ken Mills is president and CEO, Sara Berl is senior vice president of Law and Policy and Nina Hunter is vice president of Regulatory and Science Policy at REGENXBIO Inc., a Rockville-based biotech company.
In the coming weeks, Congress has an opportunity to pass legislation that is intended to improve the lives of millions of patients. The time is at hand for Congress to renew the law creating the FDA prescription drug user fee program, known as “PDUFA.” The reauthorization of this law, which is referred to as PDUFA VII, will help ensure the continued funding of the FDA’s product review activities that enable the approval of the next generation of medicines.
Chief among the Marylanders who stand to benefit from PDUFA VII are patients who need new treatments, as well as those employed in our state’s burgeoning life science sector who work at the FDA, biotechnology companies and government-supported labs.
While to date, there has been positive collaboration among Congress, patient organizations, innovators of medicines, and FDA leaders in crafting this legislation, time is becoming our enemy. If the bill is not enacted before the August congressional recess, the FDA may soon need to issue pink slips to thousands of product reviewers because the legal authority to collect user fee funds to pay them will expire at the end of September.
Substantial layoffs at the FDA would be detrimental to millions of families in Maryland and across the country because proper staffing and prompt review of drug applications are necessary for patients to gain expeditious access to new drugs. For Maryland’s biotechnology companies, major interruptions in FDA operations would add yet another barrier to the challenges of developing FDA-approved therapies.
For the FDA staff, to whom we owe a debt of gratitude for meeting the increased workload and challenges presented by the pandemic, layoff notices would send a demoralizing signal and have negative long-term consequences for the FDA’s ability to recruit and retain a high-quality team.
REGENXBIO is a gene therapy company located in Rockville, Maryland, with approximately 400 employees. Our product development pipeline focuses on debilitating conditions, including prevalent diseases such as wet age-related macular degeneration and diabetic retinopathy and rare diseases such as Hunter Syndrome and Duchenne Muscular Dystrophy.
The legislation pending in Congress is of particular importance to Maryland companies such as REGENXBIO, which are developing therapies in the rapidly evolving field of genetic and cellular medicine. There are nearly 2,000 gene therapies under development – over 500 of which have progressed to various stages of human clinical trials.
PDUFA VII would provide funding for an additional 550 full time employees on the FDA’s human drug review program staff – about two-thirds of whom would be in the Center for Biologics Evaluation and Research, the division of the agency that regulates cell and gene therapy products.
This legislation creates a vehicle for making improvements in the FDA drug approval system. One such improvement of PDUFA VII is aimed at increasing enrollment of minorities in clinical trials.
PDUFA VII also includes proposals to make refinements in the FDA’s accelerated approval program to help ensure that post-approval clinical trials will be initiated and completed in a timely fashion. The accelerated approval pathway is especially important to the more than 30 million Americans suffering from over 7,000 rare diseases, most of which lack FDA-approved treatments. For developers of drugs for rare diseases, the rules for this vital approval pathway must be clear, fair and consistently applied. It is also imperative that Congress not adopt any new provisions that unintentionally act as unnecessary barriers to access innovative therapies.
PDUFA VII represents the 30th anniversary and sixth renewal of this groundbreaking user fee law. The law has always been a product of strong bipartisan cooperation. A bill has already been approved in the House of Representatives by an overwhelming 392-28 vote, and a Senate committee recently endorsed its version. As the legislation moves forward in these final stages, members of Congress should avoid adding controversial amendments that could derail timely enactment to the detriment of patients and their families.
While Congress has proceeded constructively on a bipartisan, bicameral basis so far this year on this legislation, there is more work to be done before the user fee program lapses at the end of September. Please join us in urging the Maryland congressional delegation to vote in support of this critically important legislation which will help ensure the health of countless Marylanders and bolster our State’s growing life science economy. Patients are waiting for new medicines, and we join them in calling on Congress to pass PDUFA VII before the August recess so that the FDA’s drug review program activities continue without disruption.