FDA invites involvement in guidance plans

The Food and Drug Administration wants to become more transparent and informative in its drug regulatory decision making, Dr. Jonathan Wilkin said at the Caribbean Dermatology Symposium.

“Part of this approach is the structured benefit-risk assessment tool,” he said in an interview.

The Prescription Drug User Fee Act (PDUFA) is reauthorized every 5 years, most recently in 2013, Dr. Wilkin said at the meeting, provided by Global Academy for Medical Education.

Two notable changes to PDUFA V (the fifth authorization of PDUFA) from previous versions are a focus on getting input from patients and their families on what constitutes a clinically meaningful improvement, and the publication of the FDA’s draft document on Structured Benefit-Risk Assessment, which is written for FDA staff and regulated drug manufacturers, explained Dr. Wilkin, a dermatologist in the Cayman Islands.

These changes are playing out with specific steps to increase patient participation and informing into the FDA’s decision-making process, said Dr. Wilkin, a former director of the Food and Drug Administration’s Division of Dermatologic and Dental Drug Products.

“Patients, their families, and patient advocacy groups are being invited to attend meetings with FDA to discuss patient needs in specific diseases, such as psoriasis and alopecia areata. This is expected to lead to perspectives on the benefit-risk assessments by the patients, for example, what risks they are willing to accept for what level of direct benefit, and their relationship to patient-reported outcome measures in clinical trials,” he said. “In the future, these patient reported outcome measures may be in package inserts and could be useful in physicians' discussions of therapeutic options with patients.”

The FDA’s guidance documents are designed to represent the current thinking of the FDA on a particular topic, said Dr. Wilkin. The guidance documents are written to be generally applicable, and are not binding. “Other approaches may be used as long as they comply with governing statutes and regulations,” he said at the meeting.

In addition, it may even be possible that under some circumstances FDA guidance documents may be deemed inappropriate, and the FDA reserves the right to adopt a different approach if the circumstances require it, he added.

Clinicians need not stay on the sidelines when it comes to guidance documents, said Dr. Wilkin, who highlighted several ways dermatologists can get involved in the process:

• Offer input on works in progress: Clinicians can provide input on FDA guidance documents under development.

• Propose something new: Clinicians can make a case for why a guidance document is needed in a particular area.

• Draft your own: Clinicians can even submit drafts of their proposed guidance documents for consideration by the FDA. To submit a draft for the FDA’s consideration, mark the document “Guidance Document Submission,” and send it to: Division of Dockets Management (HFA-305), 5630 Fishers Lane, Room 1061, Rockville, MD, 20852.

• Suggest a revision: Clinicians can, at any time, suggest that the FDA revise or withdraw an already existing guidance document. The suggestion should address why the revision or withdrawal is needed, and how it should be revised, if applicable.

• Leave a comment: Once a year, the FDA publishes a list of potential topics for guidance document development or revision in the next year. The list is published in the Federal Register and online. Clinicians can comment on the list by proposing alternative topics or making recommendations related to the topics being considered.

“To participate in the development and issuance of guidance documents through one of the mechanisms described, contact the center or office that is responsible for the regulatory activity covered by the guidance document,” Dr. Wilkin said.

Global Academy and this news organization are owned by the same parent company. Dr. Wilkin had no relevant financial conflicts to disclose.

This article was updated 3/21/16.