Among the group of 16

A few days ago the Federal Register had the announcement of the disease areas FDA will hold meeting about: “Prescription Drug User Fee Act Patient-Focused Drug Development; Announcement of Disease Areas for Meetings Conducted in Fiscal Years 2013-2015”

“…. FDA has selected the following diseases to be addressed in FY 2013-2015:

  • Alpha-1 antitrypsin deficiency;
  • breast cancer;
  • chronic Chagas disease;
  • female sexual dysfunction;
  • fibromyalgia;
  • hemophilia A, hemophilia B, von Willebrand disease, and other heritable bleeding disorders;
  • HIV;
  • idiopathic pulmonary fibrosis;
  • irritable bowel syndrome, gastroparesis, and gastroesophageal reflux disease with persistent regurgitation symptoms on proton-pump inhibitors;
  • lung cancer;
  • myalgic encephalomyelitis/chronic fatigue syndrome;
  • narcolepsy;
  • neurological manifestations of inborn errors of metabolism;
  • Parkinson’s disease and Huntington’s disease;
  • pulmonary arterial hypertension; and
  • sickle cell disease.  ….”

Remember that the FDA meeting on April 25/26 is the first of the meetings in this initiative. This is an impressive list of diseases and we are right in there!

We are setting the stage for the disease groups that come after us. Let’s shine like never before!

FDA has a Web site on Patient-Focused Drug Development: This Web site contains the general schedule of upcoming meetings for FY 2013-2015, information on how stakeholders can prepare for upcoming meetings, and information on how stakeholders may leverage Patient-Focused Drug Development to generate input on disease areas not addressed through the Patient-Focused Drug Development PDUFA V commitment. The Web site will be updated as new information becomes available.

Web page for scheduled meetings:

PDUFA 2012 slides includes info PFDD meetings

More info from FDA about PFDD meetings:

Enhancing Benefit-Risk Assessment in Regulatory Decision-Making

Ensuring the safety, effectiveness and quality of human drugs is an increasingly complicated regulatory task, requiring FDA’s expert consideration of a multitude of complex factors. Over the past several years, FDA has developed an enhanced structured approach to benefit-risk assessment in regulatory decision-making for human drug and biologic products.

The Benefit-Risk Assessment Framework was developed through extensive review and analysis of previous and ongoing regulatory decisions. PDUFA V commitments include further development and implementation of the Framework into FDA’s human drug and biologic review process. Section 905 of the FDA Safety and Innovation Act also requires FDA to implement a structured benefit-risk framework in the new drug approval process.

In PDUFA V, FDA also committed to a new initiative called Patient-Focused Drug Development with the goal of obtaining the patient perspective on certain disease areas during the five year period of PDUFA V. Assessment of a product’s benefits and risks involves an analysis of the severity of the condition treated and the current treatment options available for the given disease. This information is a critical aspect of FDA’s decision-making as it establishes the context in which the regulatory decision is made. FDA believes that drug development and FDA’s review process could benefit from a more systematic and expansive approach to obtaining the patient perspective on disease severity and current available options in a therapeutic area.

This page contains information related to FDA’s implementation of the PDUFA V commitments regarding enhancing benefit-risk assessment in regulatory decision-making.


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