ALS FDA Trials Guidance Opinion Questionnaire

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rknt50a

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This questionnaire was written by individuals with ALS. The results will be used to relay the needs and opinions of People with ALS to the ALS Association and FDA in regards to the recently issued FDA Guidance Document.

Please make your voice heard. It will only be open for a few days since the results will be useful for the ALSA workshop on Jul 12.

Thanks.

https://raleighwood.wufoo.com/forms/als-fda-trials-guidance-opinion-questionnaire/
 

rknt50a

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lgelb

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I'm all for rigorous patient-reported outcomes research and have published it, refereed it and reviewed federal proposals. This is a classic "ask what you want to hear" study, eschewing context and nuance, so you got virtually unanimous results on the order of "do you want world peace."

I'm saying this publicly because PALS who did participate should understand why their response cannot bear fruit. Apart from design issues and meaningless results, you cannot really expect the FDA to take this as a prime directive, if only because adequate trial populations are not the issue. The FDA is not supposed to care who is disappointed by exclusion criteria, but to help safe, effective treatments get to market and monitor them once they are there.

As for the ALSA, it grants money to researchers who naturally follow the FDA's dictates. And consider that lack of late-stage compounds and a categorical scale as the primary outcome measure are the real constraints on the efficacious treatments we all want. You could say "lack of money," but that is not the whole story.

Sorry for the lack of love (I did not pipe up earlier so as not to be accused of affecting the response rate of someone else's study), but this kind of "research" does not help the community to be taken seriously. LMK if your collective ever wants to field something that advances the plot.

Best,
Laurie
 

Nikki J

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My impression from conversations with one of the PALS who was involved in this was that it was more a protest than anything else. Of course PALS want expanded access, help with travel, less chance of placebo who would not.

The issues raised here are things that frustrate PALS who generally only get one shot at a trial if that.
 

lgelb

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Yes, I get that it's a protest -- just that if you're going to present it, that affects the "brand" going forward and this is not the first installment. It would have been better to present it as frustration than research!

As a reminder to all, these forums have detailed many approaches to improving quality and even length of life/avoiding complications like falls and subluxated joints, etc. These range from devices at the right time to pressure boots to passive range of motion exercise.

So if you want to protest a lack of rx and/or trial eligibility criteria, fine, but based on the present reality, consider balancing that out with well, helping yourself in ways that others can't. We are all happy to report the approaches that have worked for us.

Best,
Laurie
 
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rknt50a

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No doubt that the questions said as much as the answers.

A few things that might be of interest --

- The questions were all written by people with ALS.

- The workshop attendees included not only FDA "listeners" but also panelists who included investigators and clinicians and biopharm companies.

- There was a lot that many outside the FDA could pick up in the discussions to improve clinical trials themselves and their enrollments.

- The gentleman with ALS who coordinated the survey sent in the results documentation to the assigned ALSA email address prior to the workshop. He could not participate in the workshop itself online since he was having a lumbar puncture related to a clinical trial that day.

- ALSA did not mention the survey at all during the workshop. Not a peep.
 
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