The prospect of Accelerated Approval

Novel Drug Approvals 2016

I found it interesting to review the 2016 stats for Novel Drug approvals.

Quote:
Drugs for Rare Diseases
Nine of the 22 novel drugs approved in 2016 (41%) were approved to treat rare or “orphan” diseases that affect 200,000 or fewer Americans. This is significant because patients with rare diseases often have few or no drugs available to treat their conditions.


Then I read the section on Accelerated Approval and thought I'd bring to the HC Forum any reflections on this statement in relation to what we know about Trofinetide's progress and the fairly consistent reference to running a P3 on the back of efficacy from the current P2b.


Quote:
The Accelerated Approval program allows for early approval of a drug for serious or life threatening illness that offers a benefit over current treatments.....This approval is based on a “surrogate endpoint” (e.g., a laboratory measure) or other clinical measure that we consider reasonably likely to predict a clinical benefit of the drug. Once Accelerated Approval is granted, the drug must undergo additional testing to confirm that benefit. This speeds the availability of the drug to patients who need it.


Does Accelerated Approval rank as a prospect in your evaluation of Trofinetide's likely pathway?