First, we need a more nuanced review process that balances the needs of consumers and patients. For example, it is important to recognize that patients and consumers have varying tolerance levels for risk depending on their health status and the number of treatment options currently available. Someone taking an allergy medicine would be less likely to use the drug if there are serious side-effects; however, someone with pancreatic cancer and few treatment options must balance the risk of side effects against the hope of a longer life. A drug benefit-risk analysis is not a “one size fits all” decision. It must address both the condition the medication is designed to treat and the intended user, such as a consumer who moves in and out of the health care system or a patient who is dependent on medications for the chance to live a healthier life – or the chance to stay alive at all.
Second, with today’s deficits, the FDA needs these user fees in order to accomplish their ever-increasing workload. We also believe the FDA needs to use the fees more wisely. To fulfill its objective to accelerate the delivery of new treatments, the FDA should evaluate enhancements to its approval process, such as considering new frameworks for approval, including an adaptive system that would allow for early access to promising drugs for unmet needs.
Under an adaptive system, biomarkers would be used to target new compounds to specific patient populations. When drug candidates demonstrate the potential to address an unmet medical need, such as Alzheimer’s or ALS, an approval based on a biomarker, together with a risk management system, could be granted. This process would allow for patients to access promising drugs expeditiously. Safety and efficacy outcomes would be closely monitored and would inform whether the drug should be restricted, withdrawn, or continue to be approved.
In sum, the PDUFA reauthorization agreement must address both the needs of consumers who use prescription drugs only when needed and, as a result, have a low tolerance for serious side effects with the concerns of patients who face life-threatening diseases and need ongoing treatments to manage their lives. The proposed PDUFA agreement released last week addresses the competing needs of our respective communities, and we encourage Congress to enact the PDUFA reauthorization legislation.
Sally Greenberg is Executive Director of The National Consumers League, America’s oldest consumer organization, and Myrl Weinberg is President of the National Health Council, a patient advocacy organization comprised of more than 100 national health-related organizations.