Dr. Scott Gottlieb on the impact of Trump administration's cuts to health research funding

Key Concepts

• NIH Funding Cuts & Spending
• Impoundment Act of 1974
• Presidential Authority to Impound Funds
• Reconciliation Bill
• DEI Programs & NIH Grants
• Gene Therapy Approvals
• Sarepta & Duchenne Muscular Dystrophy
• FDA Approval Process & Fast Track
• Vinay Prasad (New Head of CBER)
• Real-World Evidence & Rare Disease Drugs

NIH Funding & Spending

• Proposed Cuts: The Trump administration proposed a substantial $17 billion cut in the president's budget for 2026.
• Current Spending: The NIH is currently $2.3 billion behind its spending goals for 2025. This discrepancy could lead to either a surge in grant funding towards the end of the year or a legal challenge regarding the impoundment of funds.
• Impoundment Act: The Office of Management and Budget (OMB) may attempt to test the president's authority to impound funds, referencing the Impoundment Act of 1974 and a 1975 Supreme Court case involving Nixon and the EPA.
• Test Case: The speaker believes NIH will not be used as a test case for impounding funds. Instead, the administration will likely target a less critical area of government spending.
• Spending Surge: The speaker anticipates that the NIH will ultimately spend the allocated funds, potentially leading to a surge in spending during the latter half of the year, although possibly not in the originally intended manner.

Impact on Research

• Research Cuts: There are existing cuts to research, some of which are politically motivated, targeting specific institutions or programs.
• DEI Programs: A court case in Boston ruled against cutting NIH grants earmarked for DEI programs, deeming it overtly political and discriminatory.
• Spending Alternatives: The NIH will need to find ways to spend the allocated money, either through new grants or alternative programs.

Sarepta & Gene Therapy Concerns

• Sarepta Case: A second patient death due to liver failure following gene editing treatment from Sarepta has raised concerns. The drug received fast-track approval from the FDA for Duchenne muscular dystrophy, a disease that typically affects young males.
• FDA Approval Process: The fast-track approval by Dr. Peter Marks at the FDA, despite some staff objections, has led to questions about the approval process.
• Potential Freeze: The Sarepta case, combined with changes at the FDA, has raised concerns about a potential slowdown in gene therapy advancements.

FDA Leadership & Regulatory Scrutiny

• Vinay Prasad: The appointment of Vinay Prasad as the new head of CBER (Center for Biologics Evaluation and Research) is notable because he is a political appointee, which is unusual for this role.
• Review of Approvals: Prasad has expressed interest in reviewing past approvals, including the Sarepta case, which he has previously criticized.
• Unpredictability: The speaker warns that arbitrary intervention by the CBER director could create unpredictability in the development space, affecting investment decisions.
• Exceptional Cases: The speaker acknowledges that center directors sometimes take an active role in approvals that establish new paradigms, as was the case with early gene therapy approvals.
• Past Scrutiny: The Sarepta approval has faced significant scrutiny, with some arguing that the FDA was too permissive in its approval of gene therapy technologies.

FDA Approval Process & Real-World Evidence

• Broader Approval: Dr. Marks broadened the approval for the Sarepta drug to include older patients who were no longer walking, which has contributed to the questions surrounding the drug.
• Real-World Setting: The FDA has made efforts to get potentially transformative gene therapies to patients sooner, while evaluating their benefits in a real-world setting.
• Marty Makary: The FDA commissioner has discussed broadening policies to enable rare disease drugs to reach patients more quickly.
• Test Case: The Sarepta case may serve as a test case for how much the new FDA leadership will intervene in past decisions and potentially relitigate them.

Synthesis/Conclusion

The discussion revolves around potential cuts to NIH funding, the implications for research, and concerns surrounding the FDA's approval process for gene therapies, particularly in light of the Sarepta case and changes in FDA leadership. The speaker anticipates that the NIH will ultimately spend its allocated funds, albeit potentially in a less targeted manner. The appointment of Vinay Prasad at CBER and his expressed interest in reviewing past approvals introduces uncertainty into the regulatory landscape. The Sarepta case highlights the tension between expediting access to potentially life-saving treatments and ensuring thorough evaluation of safety and efficacy. The speaker suggests that the Sarepta case could be a test case for the new FDA leadership's approach to past regulatory decisions.