The law guiding U.S. drug approval was created to meet the demands of patient advocates, and the patient community still shapes the process.
Stakeholders discussed their latest opportunity to impact the Prescription Drug User Fee Act (PDUFA), which regulates America’s gold-standard drug approval process, at BIO’s Patient Advocacy Changemakers Event (PACE) in Washington, DC, on Oct. 22-23.
PDUFA has roots in the 1980s, when ACT UP activists fought for AIDS patients, according to Steve Berman, VP for Science and Regulatory Affairs Strategy at the Biotechnology Innovation Organization (BIO), who led the PDUFA discussion at PACE.
“At that time, drug development in the U.S. was very different than it is today. Seventy percent of all new medicines were available outside the U.S. before they were available here,” and drug review often took three years, Berman said. “At the height of the AIDS crisis, patients couldn’t get drugs to save their lives. Patient advocates said this was unacceptable.”
Congress responded with PDUFA. First passed in 1992 and reauthorized every five years, PDUFA requires drug makers to pay fees to help fund the Food and Drug Administration (FDA). In return, FDA is required to provide a professional, efficient approval process. Before the act, FDA drug review averaged close to 29 months, and today the average is down to 10 months, according to a report.
“It is absolutely critical to have a functional FDA, to have a well-funded FDA, to have an FDA that has processes and the infrastructure allowing for more regulatory certainty,” said panelist Adora Ndu, Chief Regulatory Officer and EVP of Portfolio Strategy at BridgeBio Pharma. Along with Berman, Ndu represented the biotech industry during recent negotiations with FDA to create a proposal for the contents of PDUFA VIII.
Why PDUFA reauthorization matters to patients
The reauthorization process, which provides an excellent opportunity for patient input into FDA operations, is currently underway.
The process began with a public meeting, including many speakers from the patient advocacy community. This transitioned to the biopharma industry, represented by BIO and others, negotiating with FDA officials about the fees industry will pay and the services and initiatives the agency will provide in return. The six-month-long negotiations ended in August with the PDUFA VIII commitment letter.
Throughout negotiations, FDA had monthly check-ins with stakeholder groups consisting of 50-60 patient advocates. That input was crucial, according to Ndu.
“[FDA] would pull from the stakeholder meetings to say, ‘This is what we’re hearing, how does it inform the direction that we should take?’ ” she said.
Patient input continues as Congress uses the commitment letter’s proposals to create PDUFA legislation, which must be reauthorized by September 2027.
“Patient advocacy groups play an important role in helping educate Congress members about the importance of user fees overall and the details of this particular agreement, and also in shaping any policies that may come along as riders,” said Berman.
As panelist Jeff Allen, President and CEO of Friends of Cancer Research, told the session at PACE, PDUFA updates are essential.
“Science and technology are evolving rapidly,” Allen said. “This five-year process gives us all the opportunity to check in and make sure policies are in place to keep pace with the science.”
Along with adding necessary updates, Congress members often use the “must-pass” nature of the act to add riders that are not central to PDUFA’s mission. As Congress increasingly delays “must-pass” legislation, that strategy is risky, Allen said.
“I’m sure we will see some good ideas from various policy proposals that could appropriately ride along this PDUFA package,” Allen said. “But I think we have to keep this relatively narrow, in order to make sure that we don’t inadvertently derail an important process.”
Increasing patient voice in drug development
The proposal in the commitment letter aimed at achieving the basic, but challenging, goal of ensuring FDA operates efficiently, according to Ndu. This resulted in a PDUFA VIII commitment letter that contains improvements to increase patient participation, she said.
Ndu highlighted some provisions benefiting the rare disease community, including continuing the successful Rare Disease Innovation, Science, and Exploration (RISE) workshop series. The PDUFA VIII commitment letter envisions 10 RISE workshops.
RISE workshops “enable a discussion around the barriers in drug development, bringing all the stakeholders together to ensure conversation can happen and can actually translate into enabling more treatments,” Ndu said.
The entire patient community can benefit from “continuation of patient-focused drug development activities from prior PDUFAs,” proposed in PDUFA VIII, according to Berman.
“FDA would be required to hold multiple public meetings talking about how they consider patient-focused drug development and patient preferences in their regulatory decision making,” he said.
Ideally, FDA should include data from patient experiences in their drug labelling, and this is a step in that direction, Ndu said, noting that “FDA will share some of the case studies of how they have considered patient experience data.”
Berman maintained that PDUFA VIII’s improved patient engagement opportunities will result in better drug development.
“One of the reasons why drug development is so slow and expensive is that science is challenging,” he explained. “And the best way to address scientific challenges is as a community.”
