FDA, CMS target gene therapy barriers

FDA and CMS initiatives aim to simplify gene therapy approvals and expand access to treatments for rare diseases.

1

The FDA proposed a framework allowing one biologics license application to cover multiple patient-specific genome-editing therapies for rare genetic diseases.
2

Under the proposed framework, developers could add variants within predefined individualized feature space boundaries using laboratory data without additional clinical trials.
3

The draft guidance addresses manufacturing, safety testing, unintended genetic changes, naming, and labeling, but does not approve a therapy or report product-specific safety findings.
4

CMS plans to expand its voluntary Cell and Gene Therapy Access Model beyond sickle cell disease to include treatments for other conditions.
5

CMS said centralized outcomes-based negotiations reduced state administrative burdens and strengthened negotiating leverage; the application process for the expanded model has no announced release dat

1