Department of Health and Human Services

Patient Network

Bringing Your Voice to Drug and Device Approval and Safety

Patient-Focused Drug Development Meetings

FDA performs an important public health task by making sure human drug and biologic products are safe and effective. Over the past several years, FDA has developed a better approach to making benefit-risk assessments in regulatory decision-making. The Benefit-Risk Assessment Framework was developed through the review of previous and ongoing regulatory decisions. The fifth reauthorization of the Prescription Drug User Fee Act (PDUFA V) committed the FDA to further develop the Benefit-Risk Framework into the review process.

The assessment of a medical product’s benefits and risks includes an:

  • analysis of the severity of the condition, and
  • analysis of the available treatment options.

This information is a critical part of FDA’s decision-making, since it sets the framework in which regulatory decisions are made. In PDUFA V, FDA committed to a new program called Patient-Focused Drug Development. FDA believes that drug development and FDA’s review process could benefit from a more organized and expansive approach to gathering patient perspectives on their disease and available options.

During the five year period of PDUFA V, FDA will hold a public meeting on each selected disease area to collect patient perspectives. On April 11, 2013, FDA announced the disease area meetings planned for Fiscal Years 2013-2015. The meetings are listed below. More information on the selection of disease areas can be found in the Federal Register Notice published on April 11, 2013.

Additional information on the Patient-Focused Drug Development initiative can be found here: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm.

Meetings Planned for FY 2014 and FY 2015

  • Hemophilia A, Hemophilia B, von Willebrand disease, and other heritable bleeding disorders: September 22, 2014
    • Meeting Information
  • Idiopathic Pulmonary Fibrosis: September 26, 2014
  • Alpha-1 antitrypsin deficiency
  • Breast cancer
  • Chronic Chagas disease
  • Female sexual dysfunction
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  • Irritable bowel syndrome, gastroparesis, and gastroesophageal reflux disease with persistent regurgitation symptoms on proton-pump inhibitors
  • Parkinson’s disease and Huntington’s disease

Meetings Completed for FY 2014

Meetings Completed for FY 2013