By Surabhi Dangi-Garimella, PhD

The Prescription Drug User Fee Act (PDUFA) is critical to drug approval and patient access to new drugs and biologics. Congress passed PDUFA in 1992 to allow FDA to collect fees from drug manufacturers, which can cover the cost of staffing and other resources needed for an expedited drug approval process. Reauthorized every five years since 1992, the current renewal (PDUFA VI) is through September 2022. 

Drug application review timelines have been considerably shortened as a consequence of PDUFA. The FDA has created four tracks for pharmaceutical companies to submit their drugs for review:

  • Accelerated Approval: Designed to facilitate development and expedite the review of drugs to treat serious conditions that meet an unmet medical need based on a surrogate endpoint—markers that can predict clinical benefit. (e.g., A1c levels for diabetes or cholesterol levels for the risk of heart attack). The surrogate/intermediate endpoints can save time in the approval process and allow earlier access to the treatment for the general population outside of a clinical trial. The drug manufacturer has to confirm clinical benefit by conducting phase 4 confirmatory trials
  • Breakthrough Therapy: Designed to facilitate development and expedite the review of drugs to treat serious conditions that show improvement on a clinically significant endpoint(s) when compared to existing therapy. Such an endpoint should be able to measure impact on irreversible morbidity, mortality, or symptoms that indicate serious consequences of the disease. The drug may also show an effect on:
    • An established surrogate endpoint  
    • A pharmacodynamic biomarker that suggests it could have a clinically meaningful effect on the underlying disease
    • Reducing toxicity compared to the existing therapy while maintaining similar efficacy 
  • Fast Track: Designed to facilitate development and expedite the review of drugs to treat serious conditions and fill an unmet medical need, the Fast Track process aims to get important new drugs to patients earlier. The objective is to get drugs for serious conditions into patient hands that can impact a patient’s:
    • Survival
    • Day-to-day functioning
    • Progression from a less to a more serious condition

A drug that fulfills an unmet need would:

  • Treat or prevent a condition that currently has no treatment option
  • Better existing therapies in:
    • Improved efficacy
    • Avoiding serious side effects
    • Earlier diagnosis that results in an improved outcome
    • Decreasing toxicity that can lead to treatment discontinuation

Diseases include AIDS, Alzheimer’s, heart failure, cancer, epilepsy, depression, and diabetes.  

  • Priority Review: A drug submitted under Priority Review is guaranteed an action within six months, instead of ten months and would be appropriate for drugs that would significantly improve the safety or effectiveness of a treatment, diagnosis, or prevention of a serious condition.

The goals of PDUFA VII, which will reauthorize the Act for 2023-2027, include plans for:

  • Modernizing the FDA’s data management technology
  • Enhancing the patient and caregiver voice in drug development via collaboration with patient experience experts outside of the FDA
  • Inviting public input on the submission and evaluation of patient experience data in areas of interest or concern for the public, including benefit-risk assessment of a treatment
  • “Patient preference information” to influence regulatory decisions

PDUFA VII also recognizes the need to strengthen staff capacity within the FDA for the regulatory review of cell and gene therapy products.   


Surabhi Dangi-Garimella, Ph.D. is a biologist with academic research experience, who brings her skills and knowledge to the health care communications world. She provides writing and strategic support to non-profit groups via her consultancy, SDG AdvoHealth, LLC.