PDUFA VI Is Accomplishing:
PDUFA VI is strengthening the FDA’s human drug review program and helping the Agency remain the global “gold-standard” of medicine review through:
- Real-World Evidence (RWE): By employing the data uncovered in the “real-world” practice of medicine – whether through electronic health records, payer administrative claims, or patient registries – the FDA can harness valuable information about the safety and efficacy of a medicine in a broader population than a clinical trial. PDUFA VI provides additional resources to the FDA to help develop a better understanding of how RWE can be used in regulatory decision-making.
- Patient-Focused Drug Development: PDUFA VI boosts the FDA’s expertise and capacity to advance the science of patient input and to incorporate patient perspectives in the drug development and review processes. Understanding how medicines improve how a patient feels and functions is just as important as clinical benefit. Patients, family members, and caregivers can provide their unique and valuable perspectives on their disease condition and available treatment options to advance this understanding. These perspectives can help inform evaluations of a medicine’s benefits and risks, provide additional context for FDA’s regulatory decision-making, and help researchers better identify clinical outcomes that matter to patients and their caregivers.
- Innovative Clinical Trial Design: Randomized controlled clinical trials (RCTs) have long been the industry-standard for assessing efficacy and safety of new medicines. However, as the drug development process adapts to bring innovative new treatments to patients faster, the clinical trial process must become nimbler. PDUFA VI enhances drug development by establishing processes to facilitate appropriate use of innovative clinical trial designs and novel statistical methods which will help to enhance their review and acceptance in the drug review process.