Lessons from Healey ALS on how to build platform trials quickly

The factors that helped get a new ALS platform trial launched quickly

The first ever platform trial in ALS provides a recipe for getting these collaborative studies off the ground quickly and with early buy-in from companies.

Platform trials take advantage of a master protocol to test multiple agents in a single trial with a shared placebo arm. Their advantages are numerous but chief among them is that their adaptive design gets to answers faster, while the shared control arm means more patients are able to receive therapy.

Platform trials got their start in breast cancer, with the 2010 launch of the I-SPY 2 study. Others in pancreatic cancer, glioblastoma and acute myelogenous leukemia (AML) have followed.

But all of these trials took several years to get off the ground, largely due to the reticence of biopharma companies to participate. Their perception was that a third party wouldn’t be capable of running as robust a trial as they would.

The Healey ALS platform trial launched on Sept. 18 by Massachusetts General Hospital did not have this problem. The adaptive Phase IIb study went from idea to launch in just over one year and will start with five clinical candidates to test.

Factors behind the study’s haste include the experienced and close-knit clinical trials network in amyotrophic lateral sclerosis (ALS), the availability of several programs from smaller resource-constrained biotechs, and the short timeframe from diagnosis to death necessitating more efficient trial designs than conventional approaches.

“We are motivated by the urgency and the quick and

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