FDA to Focus on Autism, Psoriasis Under New Patient-Focused Program

Regulatory NewsRegulatory News | 01 July 2015 |  By 

The US Food and Drug Administration (FDA) has released a pared-down list of diseases it plans to assess over the coming two years as part of its Patient-Focused Drug Development (PFDD) program—an integral part of the most recent authorization of the Prescription Drug User Fee Act (PDUFA).

The PFDD was created in an attempt to bring patients into the regulatory process in a more systematic way. Instead of asking for patient feedback on a drug-by-drug basis, the PFDD asks patients with a specific disease to describe the burden of their disease, their assessment of current treatment options, their ideal treatment and the risks they are willing to assume in return for more advanced treatment options.

The hope of regulators is that these preferences can then be incorporated into the regulatory review process. Companies, too, might stand to benefit by obtaining data from patients during the development process to support more lenient drug approval standards (i.e. desperate patients are more willing to assume some risk in return for the chance of treating symptoms of their disease).

For more, please read our Patient-Focused Drug Development Tracker

Current State of Affairs

To date, FDA has held 15 patient-focused drug development meetings, with another planned for the end of 2015.

But those 16 diseases aren't the only ones set to receive attention from FDA. In October 2014, it also announced its intention to continue its PFDD program for several more years, focusing on a batch of potential new diseases and conditions.

At the time, FDA said it was considering 16 new conditions for possible inclusion in the PFDD program:

AchondroplasiaAlopecia areata
AutismAutoimmune disorders treated with immune globulins
DepressionDiabetic foot infection
Hereditary angiodemaMelanoma, specifically unresectable loco-regional disease
Neurologic disorders treated with immune globulinsNontuberculous mycobacterial infections
Ovarian cancerPatients who have received an organ transplant
Primary humoral immune deficienciesPruritis
SarcopeniaThrombotic disorders

New Disease Areas

On 1 July 2015, FDA announced that just eight conditions will receive attention from FDA under its PFDD program between Fiscal Years 2016-2017:

  1. Alopecia areata
  2. Autism
  3. Hereditary angioedema
  4. Non-tuberculous mycobacterial infections
  5. Patients who have received an organ transplant
  6. Psoriasis
  7. Neuropathic pain associated with peripheral neuropathy
  8. Sarcopenia

Notably, two of these conditions—psoriasis and neuropathic pain associated with peripheral neuropathy—were not among FDA's initial list of potential disease candidates.

The selected group of diseases and conditions will bring FDA through the end of the current authorization period for PDUFA, which is set to expire in September 2017. FDA has previously said it is committed to holding at least 20 PFDD meetings, and is on pace to hold 24 meetings.

The current popularity of the programs all but ensures the PFDD program will continue into the next reauthorization of PDUFA, which is currently being negotiated.

FDA Federal Register Notice


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