ALS Advocacy

ALS Advocacy
Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Saturday, September 7, 2013

Aren't There Ever Any Scouts At The Table During Trial Design?

Envision a person with ALS who has volunteered for a clinical trial -- a trial designed in much the same way that researchers have traditionally designed trials for rashes or cholesterol control or male pattern baldness.  There is trial period with the drug or placebo, and when that's over, everyone waits while results will be evaluated and the drug company will decide if and how to proceed to another trial phase.

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.




4 comments:

  1. 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.

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  2. Keep 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

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  3. Ah, 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.

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  4. Have done phase ll cytk als research had marked improvements Turned down for future based on 2 year rule

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