Drug Industry Shrugs Off Accelerated Approval Reforms
The drug industry lobbying arm Pharmaceutical Research and Manufacturers of America told a House Energy and Commerce Committee hearing 3/17 that proposed reforms to the accelerated approval program are not needed and that it is concerned with any FDA-related policy proposals and legislative riders being added to negotiated user fee agreements because it would “undermine and threaten passage of the important agreements” which have to be signed into law before the user fee programs expire on 9/30.
PhRMA vice president Lucy Vereshchagina told the committee that accelerated approval drug are “approved under the same rigorous standards of safety and efficacy as traditional approvals,” and the new drug user fee agreement “contains efforts aimed at modernizing the U.S. regulatory and drug development paradigm and improving efficiencies in drug review. For example, FDA will update their review processes to provide timelier discussions and ensure agreement with sponsors on postmarketing requirements, including for drugs and biologics approved under the accelerated approval pathway.”
Vereshchagina reminded the hearing that the longstanding accelerated approval pathway has helped expedite more than 200 treatments for patients with serious or life-threatening diseases. “It is a critical tool for patients and regulators, and the biopharmaceutical industry continues to support the pathway in its current form,” she said.
In opening remarks, committee chairman Frank Pallone (D-NJ) pitched his recently introduced Accelerated Approval Integrity Act, which would give FDA greater authority to ensure drug companies that have been granted accelerated approval conduct a well-controlled clinical trial to confirm that an actual clinical benefit exists for patients.
“Unfortunately, however, under the current system, some sponsors have failed to conduct trials in a timely manner,” Pallone said. “For example, take Aduhelm, the Alzheimer’s drug that was approved by FDA last June. Here we are nine months later, and the sponsor has not screened a single patient for its required confirmatory trial. Other drugs have stayed on the market for eight or nine years without proving a clinical benefit, and as [CDER director] Dr. Cavazzoni testified last month, the process for removing these drugs from the market is cumbersome and can take months or even years.”
He noted that his bill would require FDA and a sponsor to agree to a confirmatory study clinical trial protocol before a drug is granted accelerated approval. It would also allow FDA to require that the trials are underway prior to approving the drug. “The bill would also improve transparency and streamline the process for withdrawing approval when clinical trials are not conducted with due diligence or no clinical benefit is shown,” he said. “These reforms will strengthen the accelerated approval program and help facilitate additional medical discoveries and product development.”
Biotechnology Innovation Organization chief scientific officer Cartier Esham suggested that the current accelerated approval program along with enhancements the user fee reauthorization agreement is proposing will negate the need for any added legislative rider offered by the committee. “To ensure that the design and implementation of confirmatory trials are more effective, the PDUFA VII Commitment Letter provides avenues for earlier and timely discussions on the design of post-market requirements (PMRs), which are critical to confirming the clinical benefits of products receiving accelerated approval,” she said. “PDUFA VII aligns processes to ensure necessary early engagement and discussions are occurring in a manner that allow for more effective assessments of PMR needs and enable more thoughtful PMR study designs.”
Esham said that the revised user fee program will also establish “improved processes for the continued evaluation of PMRs post-approval to ensure requirements are being met and/or remain scientifically valid.” She said it will also “advance regulatory understandings” about when and how real-world evidence may be used to support post-marketing requirements that may significantly improve the ability to complete these requirements in a “more effective and efficient manner.”