Importance of Reauthorizing PDUFA for Rare Disease Patients

Congress still has to reauthorize the Prescription Drug User Fee Act, and it can have big consequences for rare disease patients if there is a delay.

Every 5 years, Congress has to reauthorize the Prescription Drug User Fee Act (PDUFA). This is a lengthy process that is at least a year in the making after negotiations between industry and the FDA. However, September is coming up fast, and there hasn’t been much mention of PDUFA as the House of Representatives spent months on the American Health Care Act, and the Senate now works on its own Obamacare replacement bill.

PDUFA is important because the fees generated from this agreement with industry accounts for a majority (nearly 70%) of the FDA’s drug review budget. The National Health Council (NHC) and the National Organization for Rare Disorders (NORD) wrote to Congress earlier this month, asking them to act swiftly on PDUFA to reauthorize the user fees.

“We request the Committees of jurisdiction and Congress as a whole keep the user fee reauthorization process timely, bipartisan, uncontentious, and focused on the patients FDA serves every day,” they wrote.

The groups stand behind the negotiated user fee agreements, which had been developed with input from the patient advocacy community. NORD and NHC are concerned that without the user fees, review of innovative therapies will be slowed down or stopped and a delay to reauthorize the agreement could impact the FDA’s ability, they argue.

“The chronic and rare disease and disability patient communities rely on FDA to ensure that innovative, safe, and effective treatments reach those in need,” NHC and NORD wrote. “We thank Congress for moving forward with these critical funding mechanisms, and look forward to their swift and unimpeded passage.”