Lawmakers Ask FDA to Improve Approval Processes for Orphan Drugs

A Tuesday letter spearheaded by Sen. Edward Markey (D.-Mass.) and signed by 38 members of Congress calls on FDA Commissioner Margaret Hamburg to commit to improving the consistency of the agency’s approval processes for drugs that treat rare diseases and/or meet unmet medical needs.

The lawmakers ask the FDA to ensure that its standards for orphan drug approval contain clearly defined and consistent terminology, such as the “preliminary clinical evidence” and “surrogate endpoints” laid out in two of the expedited approval pathways passed in the 2012 FDA Safety and Innovation Act.

“Consistency in use of these concepts and predictable engagement on these standards across the agency serves as a way to encourage drug sponsors to pursue expedited approval designations,” the legislators say.

They also requested that the agency uniformly implement a provision of FDASIA that encourages reviewers to ask for input from patients, researchers and doctors.

The letter occurs at the same time that the FDA is moving to create greater specialization within the agency that aims to organize regulatory and compliance programs around drugs, devices and food. The move is part of a larger restructuring effort aimed at heightening the technical expertise throughout the agency, increasing and improving communications internally and making it easier for stakeholders to interact with the FDA, Howard Sklamberg, director of CDER’s Office of Compliance, said recently.

FDA spokeswoman Erica Jefferson told Drug Daily Bulletin that the agency plans to respond directly to members of Congress that signed the letter. Read it at www.fdanews.com/ext/resources/files/03/03-06-14-MarkeyLetter.pdf. — Lena Freund

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