September 25, 2012

FDA Notice on PDUFA

Attached is a notice from the FDA on Prescription Drug User Fee Act. It seeks to set up public meetings for 20 different disease areas. The candidates are:

Pulmonary arterial hypertension. 
Heart failure.
Primary glomerular diseases.
Huntington’s Disease. 
Peripheral neuropathy. 
Chronic fatigue syndrome.
Irritable bowel syndrome.
Inflammatory bowel disease.
Alopecia areata.
Diabetic ulcers.
Female sexual dysfunction.
Interstitial cystitis/painful bladder syndrome.
Fracture healing.
Diabetic foot infections.
Hepatitis C.
Patients who have experienced an organ transplant.
Sickle cell disease.
Chronic graft versus host disease.
Aplastic anemia.
Lung cancer.
Cancer and young patients.
Cancer treatment in pregnancy.
Cancer and sexual dysfunction.
Cancer and depression.
Clotting disorders (e.g., hemophilia A (factor VIII deficiency) and von Willebrand disease).
Thrombotic disorders (e.g., antithrombin deficiency and protein C deficiency).
Primary humoral immune deficiencies (e.g., common variable immune deficiency).
Neurologic disorders treated with immune globulins (e.g., chronic inflammatory demyelinating polyneuropathy).
Hereditary angioedema.
Alpha-1 antitrypsin deficiency. 

The FDA notice reads as follows:

As part of the performance commitments of the fifth authorization of the (PDUFA V), FDA has published a notice in the Federal Register related to Patient-Focused Drug Development, an initiative that provides for a more systematic approach to obtaining the patient perspective on certain disease areas in PDUFA V.  

Over the next five years, FDA will conduct public meetings for 20 different disease areas. The meetings will focus on the patient perspective regarding two key considerations in FDA’s regulatory decision-making: the severity of the disease and the currently available treatment options. In determining the disease areas that will be addressed in these meetings, FDA has published a preliminary list of nominated diseases in a Federal Registry notice. The public is invited to comment at a public meeting on October 25, 2012, where FDA will provide an overview of Patient-Focused Drug Development. Registration to attend this meeting must be received by October 18, 2012. For more information about this meeting, including the list of nominated diseases, see the Federal Register notice ( The public is also invited to submit written comments into the public docket FDA-2012-N-0967 (!searchResults;rpp=25;po=0;s=pdufa). Written comments are due by November 1, 2012.  

In addition to the October 25th meeting to “kick off” planning for the 20 disease-related meetings under the PDUFA V performance commitment, FDA will also conduct periodic consultation meetings with patient stakeholders regarding process issues related to the agency’s implementation of Patient-Focused Drug Development. The consultation meetings will address important considerations and challenges in establishing and conducting a process that will be useful to both the patient community and FDA. The first of these consultation meetings will take place on October 10, 2012. Notification of intention to participate in this series of meetings must be submitted by October 3, 2012. For more information about these meetings, see the Federal Register notice  (

We look forward to your participation in this process. If you have any questions, please feel free to contact the Office of Special Health Issues at301-796-8460.

1 comment :

  1. Sad thing is... Type 1 is not even listed specifically!