Regulatory Focus™ > News Articles > FDA Plans to Evaluate Risk-Benefit Paradigms for 20 Disease Areas

FDA Plans to Evaluate Risk-Benefit Paradigms for 20 Disease Areas

Posted 21 September 2012 | By Alexander Gaffney, RAC 

The US Food and Drug Administration (FDA) is planning a meeting to discuss the agency's patent-focused drug development initiative, an attempt to identify neglected or under-served diseases with an eye on generating a more nuanced understanding of patients' tolerance for risk.

The initiative was included in the recent reauthorization of the Prescription Drug User Fee Act (PDUFA), contained within the FDA Safety and Innovation Act (FDASIA). Section X of PDUFA instructs FDA to assess its risk-benefit decision making as it relates to disease areas where treatment options are less than optimal. Patients may be willing to accept more risk if the alternative to treatment is worse, proponents of the section hypothesized, and FDA was ordered to facilitate a program that assesses that 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 noted in its Federal Register posting announcing the program.

According to FDA, the program will reportedly seek out opinions from patients in 20 difference disease areas over the next five years, with each disease 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.

FDA said it is also interested in additional disease areas not covered among its list of 20, and said it will accept proposals for additional disease areas for consideration so long as they can prove the disease is severe, affects functioning or activities of daily living, disproportionately affects certain sub-populations, lacks adequate therapies or affects a broad spectrum of patients with different needs.

FDA explained that these new areas will be included in a second wave of meetings to take place in the 2016-2017 time period.

FDA's meeting on the subject will take place on 25 October 2012 at FDA's White Oak, Silver Spring, MD campus.

Read more:

FDA - Meetings: Prescription Drug User Fee Act Patient-Focused Drug Development

FDA - Meetings: Request for Notification of Patient Stakeholder Intention to Participate; Prescription Drug User Fee Act V Patient-Focused Drug Development

Regulatory Focus newsletters

All the biggest regulatory news and happenings.