NORD Issues Statement in Response to PDUFA VI Commitment Letter

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On July 15, the Food and Drug Administration released the performance goals letter for the reauthorization of the Prescription Drug User Fee Act, also known as PDUFA VI.  The PDUFA program is the agreement between the FDA and the pharmaceutical industry to pay user fees to facilitate the review of pharmaceutical products. PDUFA must be reauthorized every five years, and it is often accompanied by important regulatory reforms.

While this agreement must still be approved by Congress, the agreement described in the goals letter has the potential to greatly benefit the rare disease community.

First and foremost, the letter enumerates the expansion of the Rare Diseases program (RDP) by integrating rare disease specialists in each review division. This can be incredibly impactful for rare disease drug review. Orphan therapies often necessitate the use of innovative trial designs, smaller patient populations, the use of biomarkers and surrogate endpoints, fewer confirmatory studies, and greater review flexibility to reflect the inherent difficulties of rare disease therapeutic development. By placing Rare Diseases Program staff in each review division, orphan drug development and regulatory review will be accelerated and streamlined to meet the needs of the rare disease community.

There are several other important proposed reforms, including:

  • Continuation of the Patient-Focused Drug Development (PFDD) Program: The FDA will publish several guidances for industry on how to include patients and their perspectives in the drug development process. This brings patients one step closer to impacting drug development for their disease from the very start, rather than simply accepting what industry has developed for them.
  • Expand the Benefit/Risk Framework development process: The FDA will expand its efforts to fully integrate patient-driven benefit/risk frameworks within drug reviews.
  • Combination Product Review: Many rare diseases are treated by innovative combination products. However, these products face unnecessary regulatory uncertainty and opaqueness. The agreement would streamline the review of combination products by increasing FDA staff capabilities.
  • Public workshops on Biomarkers and Surrogate Endpoints, and Real World Evidence: The FDA will hold public workshops on best practices in the use of biomarkers, surrogate endpoints, and real world evidence.
  • Biomarker Qualification Process: The FDA will expand their biomarker qualification capabilities and expand industry and public education and outreach on the qualification of biomarkers.
  • Breakthrough Therapies: This critical expedited review pathway will now be funded through dedicated user fees.

Each of these proposed reforms has the potential to move us closer to rare disease patient-driven drug development. NORD is excited to support these proposals, and we hope you join us at the August 15th FDA public meeting. Please register here:

For more information on the PDUFA reauthorization process, please visit:…/public-meeting-on-pdufa-vi-reauthorization-registration-26380227972ry/UserFees/PrescriptionDrugUserFee/ucm446608.htm

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