EveryLife Optimistic for Rare Disease Priorities Ahead of Senate PDUFA Hearing

As the U.S. Senate Health, Education, Labor & Pensions (HELP) Committee completes work on its version of the 2022 user fee agreement reauthorization for drugs, biosimilars and medical devices the EveryLife Foundation for Rare Diseases is encouraged by language representing key priorities of the rare disease community. If the existing provisions within the Senate and House drafts of the User Fee Agreement are finalized, this legislation will signify tremendous momentum for the rare disease community.

Introduced in the Senate as the Food and Drug Administration Safety and Landmark Advancements (FDASLA) Act, but commonly referred to as the Prescription Drug User Fee Authorization (PDUFA) the bill will be considered by the HELP Committee during a hearing tomorrow, Tuesday, June 14, 2022, at 10:00 AM ET. The House completed work on its package of user fee reauthorizations last week. Following Senate passage, leaders from both the House and the Senate will negotiate a final bill.

“We are optimistic that when the House and Senate align their respective versions of the legislation, we will have a reauthorization package that includes important commitments to the rare disease community.” said Annie Kennedy, chief of policy, advocacy and patient engagement. “It is evident that the tireless work of patient advocates is resonating with lawmakers.”

Among elements of the Senate User Fees Agreement that reflect the rare disease community priorities is language that would enhance FDA’s accelerated approval pathway. On the House side, the bill includes a focus on funding regulatory science for therapies to treat ultra-rare diseases; it also features language allowing companies developing therapies to communicate with payors earlier in the regulatory process to reduce delays, affordability and accessibility issues when therapies are ready for market.

Absent from both proposed packages is the creation of a Rare Disease Center of Excellence at FDA to advise on rare disease therapy development and discussions on trial design, patient preferences, patient outcomes and regulatory flexibilities. According to Kennedy, the community will continue to ensure that remaining priorities, including the establishment of the Center of Excellence for Rare Diseases, will move forward within other legislative packages.

“Focused attention to creating and strengthening regulatory policy that can bring rare and ultra-rare disease therapies to patients who need them is central to resolving the annual, trillion-dollar public health crisis happening in our country today,” Kennedy said.

PDUFA was enacted in 1992 to reduce what used to be a years-long process to review and approve new therapies. Critical funding for FDA infrastructure is supplemented through the collection of user fees from pharmaceutical companies to supplement federal allocations. These funds support the commitments negotiated through the user fee agreements and have resulted in the reduction of regulatory bottlenecks and created faster processes to review potentially life-saving therapies and bring them to market. In the years since the original PDUFA was passed, additional user fee bills for medical devices, biosimilars and generic drugs have been created, with a new category added to the Senate bill this year for diagnostic test user fees. The bill must be reauthorized every five years. The seventh iteration of PDUFA, known as PDUFA VII, must be reauthorized by September 30, 2022.

Both houses of Congress take the reauthorization process as an opportunity to augment the legislation with policies to create specific regulatory efficiencies and to address present day needs in the public health arena. For the EveryLife Foundation and the community of rare disease patient advocates, the reauthorization process is an opportunity to introduce reforms that can further advance rare disease therapy development for the 93-95 percent of rare diseases for which there are no FDA-approved treatments. Former PDUFA cycles have yielded the establishment of the accelerated approval pathway, the patient focused drug development movement, the incorporation of patient experience data into regulatory decision making, and more.

The House Energy & Commerce Committee completed work on its user fee reauthorization bill including PDUFA and the reauthorization for medical device, generic drug, and biosimilar user fees. Once the Senate HELP Committee completes its work, lawmakers from each chamber will work to resolve the legislative differences between the two versions. A final compromise package must be approved by both chambers and then sent to the White House for consideration by the President. Upon the President’s approval, the reauthorization legislation is signed and becomes law.

To learn more about PDUFA, visit RDLA’s Policy Primer. You can register today for a PDUFA recap and status update at the June 23, 2022 RDLA webinar.