STAT+: ALS patients support bill that could help patients with other rare diseases
ALS and rare disease advocates spoke in support of a bill that would create a new provisional approval path for drugs.
WASHINGTON — A month after advisers to the Food and Drug Administration overwhelmingly voted against the experimental ALS drug NurOwn, a Senate committee is considering a bill that would allow provisional approvals of the drug and other investigational treatments for rare, incurable diseases.
Balancing high approval standards and fast access to promising drugs has always been a wobbly act. FDA approvals can take longer than a decade, so Congress has given the FDA the power to speed approvals and allow compassionate use of unapproved drugs, and given patients the “right to try” investigational drugs without going through the FDA.
Some believe those measures suffice, but a bipartisan group of six senators believes more is needed. They’re backing a bill by Sen. Mike Braun (Ind.), the ranking Republican on the Senate Aging Committee, that would create a provisional approval path for drugs intended for serious or life-threatening diseases when there is early evidence that a drug works. The provisional status would run two years and could be renewed for a total of eight years. During that time, drug companies would be required to use registries that track patient use of drugs.
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