FDA Asked To Improve Approval Processes For Orphan Drugs

Lawmakers appealed to FDA Commissioner Margaret Hamburg to improve consistency of approval processes for orphan drugs, in a letter spearheaded by Senator Edward Markey.

“We write in recognition of the Food and Drug Administration (FDA's) efforts to ensure public access to safe, innovative and novel therapeutics, particularly for rare diseases and where there are unmet medical needs, and to ask that you continue to commit to ensuring that potential new medicines are guided and reviewed consistently across the agency,” the letter stated, which was signed by 38 members of Congress. The lawmakers said that many families continue to struggle with limited options for rare diseases and development of new, more effective medical treatments often comes too slow. “Innovation of new and safe drugs is especially urgent for rare diseases, for which either no approved therapeutics or no cures currently exist,” the letter stated.

Specifically, the letter asked that the FDA ensure its standards for orphan drug approval are clearly defined and use consistent terminology such as “preliminary clinical evidence” and “surrogate endpoints” cited in 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. This in turn spurs the development of new drugs for rare diseases and for those with unmet clinical needs, an often risky and expensive prospect for industry. Increased utilization of new pathways will move treatments more efficiently to the patients that need them most,” the lawmakers said.

The legislators also requested uniform implementation of a FDASIA provision which encourages reviewers to ask input from patients, doctors, and researchers.

Erica Jefferson, spokeswoman of the FDA, said in an interview that the agency plans to issue a direct response to the members of the Congress who signed the letter.