Trial participation has huge value

People sometimes think that because they’re invited to participate in a research trial, participation is free. In the sense that free means without cost, it’s not. Trials are enormously complex, time consuming, and expensive. Participants might not be required to pay anything, but someone is paying.

In addition, for participants, there are costs other than money. Participation requires commitment to responsibilities that if they aren’t delivered on, could damage or even invalidate the trial. The most important of these is discretion. It’s rare that participation would need to actively be kept secret but anyone making public that they’re in a trial is simply oversharing.

(Before anyone ever posts anything about their condition or possible treatments in any online forum, they should consider that their post is likely to be read by a newly diagnosed person who will probably misunderstand and/or misinterpret the context.)

The ultimate trial format is double-blind, where neither researcher nor participant knows who’s getting whatever’s being tested and who’s getting a placebo but for several reasons trials can’t always be run that way. In the case of FA there are so few of us that it’s often difficult to get a decent sample size at all. Some trials are for equipment rather than drugs so there is no control sample. As a result, participants often know that they’re getting the treatment being tested and validity of the trial depends on trial protocols being followed exactly. Some people consider this rigidity inconvenient so it could be considered a cost.

The reward stage for a researcher often comes well after the trial itself, when results have been documented, presented and ideally published. That’s what leads to promotion and more grant funding for researchers. But nobody will publish results that are already in public domain. So, another cost might be the requirement for a participant to wait a frustratingly long time before knowing if their own results were representative and meaningful.

The website https://clinicaltrials.gov/, hosted in the US, lists clinical trials throughout the world, planned, recruiting, underway and completed. It’s a searchable database, so this link brings a list of studies related to FA.

When I last checked, the site listed 56 studies. The simple fact that studies are listed is progress already. As learnings are gained from each of those studies, that progress continues towards finding treatments, and even a cure for FA. Any of us who gets to contribute to that progress should seize and appreciate that opportunity, recognise its value and treat it with the immense respect it deserves.

Terry O’Hanlon
fara Ambassador