For ALS trials, it seems like the trial designers never seriously consider what happens to their trial volunteers if this stuff really works. It's not like they'll just have to scratch an itch or watch their diets or comb over while they wait to see what's next. They decline quickly. They die.
People with ALS who have volunteered for trials and who perceive improvements during the trials are left hanging out to dry. This all could be avoided with some creative trial design that gives them options to continue on the drug (or the placebo... if it works, they really don't care) before they sign on the dotted line.
Instead we have traditional trial designs and the subsequent cry for Expanded Access Programs after the fact. Perhaps we should get in front of the problem and have trial designers, including patients, talk about what happens to trial volunteers if this stuff really works. Be prepared.
Well - as an FDA Patient Representative for ALS - if I get the chance to infuence a trial design - this will be my priority topic at the table.
ReplyDeleteKeep up the good work, Tom. This issue, however, is less of a regulatory matter and more about the economics of expanded access programs (EAPs) for past trial participants and for ALS sufferers who don't qualify for trials. The ALS Emergency Treatment Fund (ALS-ETF) is a 501c3 non-profit that is deep in the trenches to deliver an EAP for one of the treatments in clinical trials for ALS. We invite the whole community to get behind this work; it will take a collective effort. www.ALSETF.org
ReplyDeleteAh, but this was about preventing a problem... about providing protection for c.t. volunteers up front in the design of the trial. It's about being prepared in case this stuff really works for some.
ReplyDeleteHave done phase ll cytk als research had marked improvements Turned down for future based on 2 year rule
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