The Prescription Drug User Fee Act (PDUFA), enacted in 1992, has transformed the U.S. drug approval process by providing the FDA with essential funding from biopharmaceutical companies. Critics often argue that this funding creates conflicts of interest, claiming it “corrupts” the FDA. One such narrative is presented in another book published this week, which misrepresents the intent and impact of PDUFA. In reality, without this program, life-saving drugs would face catastrophic delays, putting patient lives at risk and stifling medical innovation.
Before PDUFA, FDA drug reviews were mired in delays due to chronic underfunding and limited staffing. It wasn’t uncommon for drug applications to take over two years for a decision. The process was bogged down, not by scientific rigor, but by bureaucratic constraints. Patients—especially those with rare, degenerative, or terminal conditions—were often forced to wait while treatments languished in regulatory limbo.
PDUFA changed that. It allowed the FDA to collect fees from pharmaceutical companies to fund additional reviewers, streamline regulatory processes, and improve review timelines. In return, the FDA committed to performance goals, ensuring transparency and accountability. Critics who argue that this “pay-to-play” system compromises safety ignore the rigorous scientific standards that the FDA continues to uphold regardless of funding source.
To appreciate the real-world impact of PDUFA, consider the case of Keytruda (pembrolizumab), a breakthrough immunotherapy for cancers like melanoma and non-small cell lung cancer. Keytruda’s expedited approval in 2014 was a direct result of PDUFA’s structured timelines and resources that supported priority reviews. Imagine if that approval had been delayed by years—thousands of patients who benefited from early access to this drug might have died waiting.
Another poignant example is Spinraza (nusinersen), approved in 2016 for spinal muscular atrophy (SMA), a deadly genetic disorder affecting infants and children. Under a sluggish pre-PDUFA regime, Spinraza might have been delayed indefinitely. Thanks to PDUFA, the FDA had the capacity to prioritize this drug and bring it to patients in record time.
The biggest criticism of the PDUFA program is the fees paid by the applicants to the FDA for review, which the authors claim have corrupted the FDA reviewers. This is no different from a fee that you pay to any government agency for services. Would you say that paying the Motor Vehicle Administration (MVA) for a driving license corrupts the MVA to issue driving licenses to people who can’t drive? Or would you consider your doctor to be corrupted by the fee you pay for medical services? Far from being a source of corruption, PDUFA provides predictability, transparency, and accountability in a resource-strained regulatory system. It ensures the FDA can keep pace with scientific advancements and rising submission volumes. Importantly, the fees make up only a portion of the FDA’s budget and are strictly regulated, with Congress overseeing reauthorization every five years. Each cycle includes robust public and stakeholder input to refine the process.
Critics often forget the real-world consequences of longer drug reviews: people die. It’s not theoretical—it’s measurable. Diseases don’t wait for bureaucracy to catch up. Delays in approval mean delays in access, in treatment, and in hope.
Books that argue PDUFA has “corrupted” the FDA ignore the data, the science, and the lives saved. Instead of dismantling the program, we should be refining it—ensuring it remains robust, transparent, and above all, patient-focused. In the end, the goal is not just faster approvals—it’s better, safer, faster access to the therapies patients desperately need.
