Drugs

FDA Urged to Clarify Rare Disease Approval Pathways

FDA’s new commissioner, once confirmed and sworn in, should provide greater clarity on regulatory pathways for rare disease therapies, especially the use of single-arm clinical trials, a rare disease advocate argued in a new Washington Examiner commentary.

David Gusick said uncertainty over FDA’s expectations could delay development of treatments for small patient populations and rapidly progressing diseases. He pointed to FDA commissioner nominee Heidi Overton’s recent confirmation hearing, where she identified rare disease treatments as a “key priority,” as well as ongoing negotiations over the PDUFA VIII user fee agreement.

Gusick said developers remain uncertain about FDA’s position on single-arm trials following comments attributed last year to CBER officials that raised questions about whether such evidence would continue to support rare disease approvals (see earlier story). FDA leadership subsequently said those comments did not represent agency policy, according to the commentary.

Single-arm studies can be particularly important in rare diseases because small patient populations can make randomized controlled trials difficult to enroll and, when no approved therapy exists, use of placebo or an inferior treatment may be impractical or ethically problematic, Gusick wrote.

“The goal is not to lower standards,” he said, arguing that FDA should establish clearer expectations for evidence while accommodating small patient populations and rapidly progressing diseases.

Gusick also called for greater incorporation of patient experience into regulatory discussions, including consideration of quality of life, hospitalization and mortality. Patient perspectives should provide context for clinical evidence rather than replace it, he said.

He urged FDA’s next leadership team to expand opportunities for patients, physicians and researchers to inform regulatory decisions while maintaining existing safety and efficacy standards.

Gusick is the founder of patient support/education organization Somebody To Talk To.

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