The US Food and Drug Administration (FDA) has announced a second batch of meetings intended to facilitate the development of drugs for neglected conditions by taking into account the perspective of the patients suffering from the conditions.
In recent years, some patient groups—and in particular groups representing patients suffering from serious diseases with few or no suitable treatments—have begun to express their concerns that FDA is too stringent when it comes to approvals, denying patients the opportunity to widely access potentially life-saving or life-changing therapies. Patients' tolerance for risk, some groups (and patients) say, is far higher than regulators realize.
In light of those concerns, legislators included a provision (Section X) in the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) instructing FDA to assess its risk-benefit decision making as it relates to disease areas where treatment options are less than optimal, and to facilitate a program that assesses tolerance for risk on a disease-wide level, instead of doing so on a piecemeal basis for individual products.
"The human drug and biologic review process could benefit from a more systematic and expansive approach to obtaining input from patients who experience a particular disease or condition," FDA conceded in a September 2012 Federal Register posting announcing the Patient-Focused Drug Development program.
That same notice explained that initially FDA would seek out opinions from patients in 17 different disease areas over the next five years, with each disease area meriting its own public meeting. Topics are set to include the severity of the disease, the spectrum of severity, the impact of the disease on patients, what patients perceive to be beneficial outcomes and the benefit of existing treatment regimens.
As of October 2014, FDA has already held meetings for 11 disease areas under the program, and plans to hold an additional six meetings by the end of 2015.
[For more, please see Regulatory Focus' extensive Patient-Focused Drug Development Tracker, which includes an extensive breakdown of all questions asked of patients at these meetings.]
16 New Disease Areas Proposed
Now FDA has announced another group of diseases it plans to hold meetings on under the Patient-Focused Drug Development Program.
In a 7 October 2014 Federal Register notice, FDA said it plans to hold meetings on the following 16 diseases:
- alopecia areata
- autoimmune disorders treated with immune globulins
- diabetic foot infection
- hereditary angiodema
- melanoma, specifically unresectable loco-regional disease
- neurologic disorders treated with immune globulins
- nontuberculous mycobacterial infections
- ovarian cancer
- patients who have received an organ transplant
- primary humoral immune deficiencies
- thrombotic disorders
Notable among the list are autism—thought to affect approximately 1 in 68 children—and depression—thought to affect one in 10 adults. While both conditions are widely known, FDA says it's interested in assessing conditions which have few therapies, therapies which do not adequately address patient needs, or affect patients in ways which might not be captured in clinical trials.
The list is still "preliminary," meaning the agency will accept nominations for additional disease areas if it believes they are warranted.
Comments on the draft list or suggestions on which disease areas to add should be submitted to FDA by 5 December 2014.
Federal Register Notice
Patient-Focused Drug Development Tracker