As members of the Patient, Consumer, and Public Health Coalition, we are writing to share our views on needed improvements to make PDUFA VIII a more patient-centered, evidence-based program that improves the timely access to safer and more effective medical products. The focus on speedy reviews in previous PDUFA legislation has resulted in many newly approved medical treatments; however, research shows that many of those treatments are not better than previously approved treatments and, in some cases, are less safe or less effective. [i],[ii] Despite those shortcomings, they almost always cost significantly more than comparable drugs already on the market. [iii]
We understand that user fees were designed to pay for faster reviews, but that original focus has shifted, resulting in many other requirements that CDER and CBER are required to meet in order to receive those user fees. These include new pilot programs and ‘flexibility’ in approval standards requested by industry and many meetings between FDA staff and companies applying for approval. Those meetings help companies get the information they need to improve the chances that their drug will be approved. Ultimately, these meetings make the review and approval process more efficient for the companies, but require enormous time commitments on the part of FDA staff, taking them away from other important duties that are needed to ensure that all new drugs are safe and effective.
Patients appreciate access to new treatments, but only if the new treatments are better than the older, less expensive treatments. User fees should help achieve that goal, but because of the focus on speedy reviews, many new treatments are not safer or more effective.1,2
The PDUFA VII commitment letter was 71 pages long and included many requirements for the FDA. All PDUFA Commitment letters have been negotiated behind closed doors; patients, consumers, public health advocates, and health professionals have had no access. We can neither attend the meetings nor watch them. We have “PDUFA Stakeholder meetings” with FDA staff and that provides an opportunity for us to express our views, but the Commitment letters are focused on what industry wants, not on what other stakeholders need.
For example, although the PDUFA VII Commitment letter had a section entitled “Enhancing the Incorporation of the Patient’s Voice in Drug Development and Decision-Making,” the FDA’s efforts to be more patient-centered have not adequately represented the safety concerns expressed by patients who were harmed by medical products. Instead, the focus is on patient groups that have been funded by industry. These industry-funded patient groups are trained to ask for earlier access to medications rather than emphasizing the quality of the new medications or their evidence regarding safety or efficacy.
PDUFA VII’s lengthy commitment letter included many issues that would have been strengthened and improved by our perspectives and our voices. Even relatively small changes in wording would have made an important difference in PDUFA VII. We strongly urge the FDA to make sure that the transparency and scientific evidence promised by FDA leadership this year is reflected in these and other changes in PDUFA VIII.
Sincerely,
Cancer Prevention and Treatment Fund
Doctors for America
Generation Patient
Jacob’s Institute of Women’s Health
Healthy Americas Foundation
Mothers Against Medical Error
MRSA Survivors Network
National Alliance for Hispanic Health
Patient Safety Action Network
SCAD Alliance
The TMJ Association
USA Patient Network
Washington Advocates for Patient Safety
Woody Matters
1 Vokinger, K. N., wang, T. J., Glaus, C. E. G., & Kesselheim, A. S. (2022). Therapeutic Value Assessments of Novel Medicines in the US and Europe, 2018-2019. JAMA Network Open, 5(4), e226479–e226479. https://doi.org/10.1001/jamanetworkopen.2022.6479
2 Rupp, T., & Zuckerman, D. (2017). Quality of Life, Overall Survival, and Costs of Cancer Drugs Approved Based on Surrogate Endpoints. JAMA internal medicine, 177(2), 276–277. https://doi.org/10.1001/jamainternmed.2016.7761
3 Gellad, W. F., & Kesselheim, A. S. (2017). Accelerated Approval and Expensive Drugs—A Challenging Combination. New England Journal of Medicine, 376(21), 2001–2004. https://doi.org/10.1056/NEJMp1700446