As members of the Patient, Consumer, and Public Health Coalition, which includes nonprofit organizations that represent patients, consumers, scientists, and researchers, we want to provide our comments regarding the results of the Prescription Drug User Fee Act (PDUFA VII) negotiations between the Food and Drug Administration (FDA) and pharmaceutical industry. We appreciate the opportunity to review the Commitment Letter for PDUFA VII upon conclusion of these negotiations. After reviewing the Commitment Letter, we offer our comments on the agreement as it stands at this stage of the reauthorization process.
We have 2 major concerns:
1) This letter includes numerous policy changes. Policy changes are the purview of the Congress and should not be negotiated by FDA and the industry it regulates. Therefore, policy and regulatory changes should be deleted from the Commitment Letter.
2) The performance goals reflect the priorities of industry, and do not adequately include safeguards regarding the safety and efficacy of pharmaceutical products that are being reviewed and approved by the FDA.
The policy provisions in the Commitment Letter were a surprise to us, and not explicitly shared in meetings that patients and consumers had with the FDA. In contrast, throughout the process, we have expressed concerns that the performance goals being negotiated by the FDA and industry are focused largely on the speed of the review and approval process, with a lack of attention to the safety and effectiveness of the drugs approved. We recognize that some of the goals have the potential to benefit patients by speeding up the approval process and improving the chances that drugs will be available when patients need them. There is too little emphasis, however, on performance goals aimed at improving the safety and efficacy of drugs. Drugs should only be approved with adequate evidence to support their safety and effectiveness; moreover, a robust post-market surveillance system must be in place to ensure that drugs found to be dangerous are removed from the market as quickly as possible.
It is critical that there be adequate evidence to support claims of safety and effectiveness and this evidence must include diverse populations. Unfortunately, FDA is currently approving most new drugs on the basis of preliminary data, often based on just one pivotal study instead of two, relatively small numbers of patients that are not diverse, and on surrogate endpoints rather than outcomes that are meaningful to patients (such as improved health or living longer). It is only with the inclusion of diverse patient populations, including children (when appropriate), older adults, racial and ethnic minorities, and people with disabilities that we can truly ensure that approved drugs are safe for all those likely to use them. These trials must include subgroup analysis to examine effects across the different patient populations. Post-market confirmatory trials are often required but the requirements are not always enforced and monitored closely to keep patients safe. Such enforcement should be required as one of the performance goals
The recent FDA meeting regarding ineffective cancer drugs that had been approved for inappropriate indications under an accelerated pathway is just one example of the dangers that can occur when speed is prioritized over safety. These ineffective drugs were found to cost Medicare more than half a billion dollars.
FDA points to the Sentinel program as a post-market surveillance system; however, it remains ineffective at quickly removing dangerous or ineffective drugs from the market. Among other problems, the Sentinel program lacks transparency: Descriptive data should be reported publicly on the number of drugs removed from the market, the type of approval granted to the drug, the number and type of adverse events reported, and the length of time the drug remained on the market after these safety signals were first studied. This information will help the medical decision-making process for patients, consumers, and physicians.
Finally, there is a need for improved accessibility throughout the PDUFA negotiations for all groups who are potentially receiving the drugs; this includes older adults, children, and people with disabilities. The FDA should work to ensure information is presented in multiple formats to meet the needs of diverse populations. This includes multiple languages, website content that can be accessed by a screen reader, and the use of plain language. Videos and virtual meetings should have the option for closed-captioning and American Sign Language translation. These changes will help to create a more inclusive process of PDUFA negotiations and will give a voice to a broader group of people affected by these decisions.
In conclusion, we believe that the proposed recommendations must do more to ensure the safety of patients and consumers and the scientific integrity of the drug review process. We appreciate the efforts of the agency to work toward those ends, but as long as patients and consumers are excluded from the PDUFA negotiations, the concerns and priorities of these principal stakeholders will also be excluded. We ask the Biden Administration to stand up for these excluded interests and improve the PDUFA VII proposals by supporting the topics discussed in this letter.
MRSA Survivors Network
National Center for Health Research
National Organization for Women
The TMJ Association
Washington Advocates for Patient Safety