Biden Budget ‘Wish List’ Maps FDA, Healthcare Priorities

In laying out its formal budget proposal for fiscal year 2022, the Biden administration supports a notable increase in funding for public health and research as a lead priority for the coming year. While Congress will reject or modify many of the specifics, the federal government’s remarkable success in combatting the COVID-19 pandemic continues to generate bipartisan support for advancing biomedical research and public health and safety.

FDA’s oversight and guidance in speeding the development of critical COVID vaccines, diagnostics and therapies is rewarded with additional funding to continue and strengthen those capabilities. The new spending plan requests $6.5 billion for the agency, up 8%, with $3.6 billion in public appropriations and $2.9 billion in user fees paid by industry. The added $344 million, according to the Alliance for a Stronger FDA, is allocated primarily to three main areas: critical public health infrastructure, including data modernization and expanded laboratories, facilities and staff; core safety programs for food and medical products and global supply chains; and ensuring public health by increasing secure inspections, promoting health equity, and addressing the opioid crisis.

Among the specifics, FDA seeks $19 million to enable its field inspection program to carry out site visits delayed by the pandemic and to avoid a reduced frequency in both domestic and foreign inspections going forward. To help end the opioid crisis, FDA would gain $38 million to support research for developing overdose reversal treatments and new non-opioid pain medications and to devise systems to better track and manage opioid use.

For several years now, FDA has emphasized the need to modernize its data and communications systems for medical products and food, and the new budget may finally advance its Enterprise Technology and Data modernization efforts. An additional $45 million would support wider use of AI and advanced IT systems to enable data sharing across product centers and ease communications among agency offices, streamline core business processes, and better track drug manufacturing to better anticipate shortages.

While the National Institutes of Health (NIH) would see a hefty 22% rise in funding for a $50.5 billion 2022 budget, $6.5 billion of that increase would fund the new Advanced Research Projects Agency for Health (ARPA-H) within NIH, a hot-button proposal that has raised questions. Designed as a parallel to the Defense Department’s Defense Advanced Research Projects Agency, stakeholders already are debating if ARPA-H should reside within NIH or be independent, how it could advance “transformational” research issues for multiple diseases, and how its projects would involve private sector R&D and technology transfer.

The accelerated approval debate

FDA’s surprise decision to approve Biogen’s treatment for Alzheimer’s disease has raised serious questions about the validity and value of the agency’s accelerated approval process. FDA cleared Aduhelm based on data from one clinical trial that shows some effect on reducing amyloid beta plaques in patients with mild cognitive impairment. Unlike most surrogate markers supporting early approval, that effect has not been linked to clinical benefit, challenging its validity for anticipating patient gains. The broad labeling approved, moreover, has generated heated debate among scientists, researchers, patients, and payers as to the long-term impact of the approval decision on the credibility and independence of FDA decisions.

The high price tag set by Biogen also is heightening the debate over drug costs, reimbursement, and prescribing. In setting a price of $56,000 a year, way above the cost-effective threshold of $2,500 to $8,000 calculated by the Institute for Clinical and Economic Review, Biogen stands to earn some $17 billion a year from the therapy, according to Wall Street analysts, particularly with a label that opens prescribing to early and severely debilitated Alzheimer’s patients. All eyes are on how Medicare and health and drug plans will set coverage parameters and how doctors and medical authorities will identify those individuals most suitable for treatment.

FDA’s accelerated approval process was launched in the 1980s to provide early access to treatment for dying AIDS patients with no available option. Since then, it has been used to speed approvals of new therapies, primarily for AIDS and cancer where validated surrogate markers have provided evidence that a drug has potential for curbing disease. Some consumer groups and healthcare providers, though, complain of long delays in confirming benefit and have questioned the often high cost of paying for unproven drugs.

Jill Wechsler is Pharm Exec’s Washington Correspondent. She can be reached at jillwechsler7@gmail.com.