July 14, 2025
Good morning. I’m Dr. Amanda Berhaupt, Health Policy Director at the National Center for Health Research. I appreciate the opportunity to speak on behalf of our nonprofit think tank. We scrutinize the safety and effectiveness of medical products and do not accept funding from companies that make those products or entities with a financial interest in our work.
Prior to my current position, I worked at the FDA and for the United States Senate.
The appropriated funds for FDA are not sufficient to support all of its critical work, so the agency needs user fees to get safe and effective medical products to market in a timely manner. User fees are vital to ensure that reviewers have subject matter expertise, institutional knowledge, adequate time and uninterrupted access to the FDA library with peer-reviewed research, among other scientific resources.
User fees have mainly supported faster reviews and more frequent meetings between the FDA and sponsors to address concerns about their applications. I want to emphasize that this is not what’s most important for most patients. Their greatest concern is to have access to safe and effective medical products to treat, maintain, and promote their health.
With a renewed focus on transparency at FDA, will patients and healthcare professionals be represented at user fee negotiations? At minimum, they deserve to watch the negotiations virtually, and in real-time, if they are not participating. In the past, summaries of negotiations have been too vague, which has prevented key stakeholders from providing input.
To date, the performance goals in the Commitment letters have outlined metrics for meetings and timelines in premarket reviews. These goals benefit industry, and may indirectly benefit patients, but are not patient-centered and do not focus on safety or efficacy.
We urge the agency to include performance goals with metrics on quality post-market surveillance including confirmatory studies with clinically meaningful outcomes. This is especially important when drugs are approved based on data from short-term studies with a small sample size, or based on surrogate endpoints instead of measures for how a patient feels, functions, or their overall survival.
Patient advocates, public health researchers and professionals without industry ties need representation during negotiations to ensure there are performance goals that directly benefit patients and consumers.
The public’s trust in the FDA has eroded. PDUFA needs to show that user fees will do more to ensure that drugs are safe and effective in ways that matter to patients. Speed should be secondary, because when drugs are ineffective or unsafe, patients lose confidence in their doctors and the FDA, and look for advice from other sources like social media where they may find erroneous and harmful advice.