On October 25, 2012, the FDA will provide an overview of Patient-Focused Drug Development and the public is invited to comment and share their voice. This will be the first of many public meetings held by the FDA over the next five years with a focus on 20 different disease areas. Registration for the October 25, 2012, public meeting ends on October 18. Written comments into the public docket (FDA-2012-N-0967) are due by November 1, 2012.
The focus of the meetings include considerations in FDA’s regulatory decision-making process — ranging from the severity of the disease and the current available treatment options to the benefit risk framework surrounding PDUFA V.
Examples of potential process topics include the following:
- Given the limits of FDA staff resources and time available, how to prioritize and balance different disease areas identified by different patient stakeholders.
- How to approach issues when patient stakeholders for the same disease area have different and potentially conflicting views.
- How to balance access to FDA for patient stakeholders who are local to FDA headquarters versus those in other locations who have less physical access.
- How to support engagement of patients in disease areas for which no formal advocacy organizations exist. What role, if any, might organized groups already play?
FDA is currently nominating disease areas as potential candidates for the focus of one of the 20 future public meetings and invites public comment on this preliminary list. Here are some examples of the diseases currently nominated. Few are rare diseases.
- Pulmonary arterial hypertension, Heart failure, Primary glomerular diseases, Narcolepsy, Huntington’s Disease, Depression, Autism, Peripheral neuropathy, Fibromyalgia, Obesity, Nocturia, 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, HIV, Sickle cell disease. Chronic graft versus host disease, Amyloidosis, Aplastic anemia, Melanoma, Lung cancer, Cancer and young patients, Clotting disorders (e.g., hemophilia), von Willebrand disease, Alpha-1 antitrypsin deficiency.
A list of criteria, patients/advocates would like to propose for consideration, can be found here. For more information and to view the preliminary list of the disease areas that will be addressed in these meetings, the FDA has published a Federal Registry Notice.
FDA is also interested in public comment on disease areas that are not represented on the preliminary list. In order to have a disease make the final cut, patients/advocates will need to unite in their push for their respective disease to be included in the final list of 20 diseases.
As part of the PDUFA V performance commitment, the FDA will be holding consultation meetings with patient stakeholders to address the challenges and considerations required to establish processes both useful to the patient community and the FDA.
Participation at the consultation meetings require a notification of intent no later than October 3, 2012, as the first meeting will be held on October 10, 2012. See the Federal Registration Notice for further information.