When we say that we are embarking on a new era in clinical trials, what do we mean? After all, we have seen previous FSHD trials, including those by Wyeth, aTyr, Acceleron, and Fulcrum’s ReDUX4. What makes the current era different is that, for the first time, we have multiple trials running in parallel, including one in Phase 3, which is the furthest stage of development any therapeutic trial in FSHD has ever gotten!
Amongst the ongoing trials, REACH (Fulcrum’s Phase 3 trial) will be recruiting 230 individuals with FSHD this year, while the MOVE and MOVE+ natural history studies for FSHD are seeking 450 volunteers. In addition to these ongoing studies, both Roche Pharmaceuticals and Avidity Biosciences are planning to launch their own Phase 1/2 studies in 2023, each requiring about 50 participants.
Finding more than 700 people to volunteer may not seem like a tall order until you consider this sobering statistic: Across all 20+ Clinical Trial Research Network (CTRN) sites in the US, Canada, UK, and Europe, there are roughly 2,500 patients. But only about 10 percent – or 250 – of these patients might meet the inclusion criteria for a clinical trial. As a community, we will have to work hard to fill the need for just this year. This is our immediate challenge.
If a clinical trial fails to show efficacy, that’s a huge disappointment, but it can provide valuable insights about whether the selected outcomes were sensitive or whether the trial duration was sufficient. This type of information can further encourage other companies to improve their trial designs to increase their chances of showing an effective outcome.
But if a trial cannot be completed at all because it was unable to recruit enough volunteers, that is the worst kind of failure, because it discourages other companies from taking the risk to develop a drug.
This is the crux of the problem – not having enough engaged individuals in the FSHD community to quickly fill up the clinical trials that are coming. If we do not solve it, the longer-term consequences could be dire.
