The History of PDUFA

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I talk a lot about the Prescription Drug User Fee Act (PDUFA) and how it improves and impacts patients' lives. As we head toward the sixth authorization of PDUFA, I'd like to look at how we've come this far and where it all started. I've discussed the history of the bill before, but since October is Health Literacy Month, it's the perfect time to take a deeper dive and learn even more about a law that has left a lasting impression on public health.

Prior to PDUFA, patients who needed cutting-edge treatments endured lengthy review times for new medicines while manufacturers and researchers, who worked diligently to fight disease and serious illness, saw their products tied up in complicated approval processes. Average approval time was just shy of 2 years -- far too long of a wait for patients looking for new and innovative treatments.

This frustration reached a tipping point during the height of the AIDS epidemic, a crisis that served as a stark example of the effects of the delayed review of medicines. As the disease spread rapidly and treatments remained widely unavailable, the patient community grew increasingly vocal. In response, the FDA accelerated the approval of AZT, the first HIV treatment. Even this process took over two years to complete, but it was still the fastest approval in recent memory.

The initial success of AZT sparked the motivation to pursue further improvement to the review process. Those close to the situation came to the realization that a faster turnaround on new medicines could tangibly and dramatically improve public health. Patient activists and pharmaceutical industry representatives, along with the FDA itself, knew they had to make a lasting change in this regard, and this prompted lawmakers to draft corresponding legislation. Rep. John Dingell proposed the bill on October 6, 1992 and within 23 days it became law.

In an effort to stay current and effective, PDUFA includes a provision that requires Congressional reauthorization every 5 years. This way, new features can be added to improve and modernize the program to best suit the changing health care landscape. Below is a timeline of how the law has grown in its first 23 years:

  • PDUFA I permitted the FDA to charge a fee from pharmaceutical companies to review their products. This extra money supplemented Congressional funding and expedited the review process to allow groundbreaking cures to hit the market faster.
  • PDUFA II (1997) implemented stricter performance expectations to ensure the FDA completed reviews in a timely and efficient manner. It also brought the investigational phase of review -- the period when trial participants test new medicines -- under the scope of the law.
  • Congress passed PDUFA III in 2002 as a part of the Public Health and Bioterrorism Preparedness Act. This version of the bill supplied the FDA with additional funding to track the performance of drugs once they became available to consumers. The FDA also used funding to hire additional skilled employees to further expedite the review process.
  • In 2007, PDUFA IV helped the FDA expand its operations to accommodate the growing number of applications it received under the already thriving user fee program. Increased fees allowed the FDA to improve the post-market review initiated in PDUFA III.
  • PDUFA V went into effect in 2012 and included important tools such as benefit/risk analysis, patient recorded outcomes and the Patient Focused Drug Development (PFDD) program. These components allow the FDA to incorporate feedback from the patient community and meet the needs of each individual looking for treatment.

While the means have changed and expanded throughout the years, the ends have always remained the same: increasing resources and enhancing the review process. Before 1992, the standards set by PDUFA would have seemed unimaginable - total funding at the time was less than $150 million. In 2014 alone, the FDA collected $796 million in user fees which aided the approval of well over 100 new medicines.

Congress will review the next iteration of PDUFA for reauthorization in 2017, and the planning has already started. The FDA, patients, advocacy organizations and the pharmaceutical industry periodically gather for negotiation sessions and stakeholder meetings to keep everyone active and engaged in the process.

As PDUFA VI begins to take shape, be sure to check our blog for updates and new developments. To learn more about what we envision for the new bill, click here.