ALS Signal Clinical Research Dashboard

[rknt50a]

This was executed as a completely volunteer effort by an ALS caregiver and people w ALS and other caregivers.

They wanted to provide a dashboard with information on promising therapies that would save others, especially the newly diagnosed, hours of searching.

There is also a PaCTD clinical trials star rating system there. It indicates the humaneness of the trial from the patient perspective. There is an objective scoring system.

Enjoy.

[link to page that includes donation request deleted, but dashboard is available from I Am ALS site]

 

[rknt50a]

The PaCTD rating system explained --

https://iamals.org/wp-content/uploads/2020/07/PaCTD-Page-8.pdf

 

[lgelb]

Everyone here supports getting safe, efficacious therapies to market as quickly as possible. This "rating system" is not grounded in either science or reality, so I wouldn't use these ratings to inform any decisions.

Best,
Laurie

 

[affected]

I think it is a great document, thanks for sharing.

It clearly states:
PaCTD ratings do not measure or evaluate the treatment’s safety or efficacy. A high rating on this scale does not indicate promising science and a low rating on this scale does not mean the treatment is not effective -- it purely measures the design of the trial from the patient and caregiver perspective across the criteria outlined.

Everyone here is smart enough to read it and make their own decision - I like that this is designed by PALS and CALS so is very grounded in the reality of our situation.

 

[lgelb]

I understand the stated (but non-existent) distinction, thanks. "Safety and efficacy" are established by the very trials these criteria purport to help P/CALS select among. However, the "patient and caregiver perspective" to the extent that P/CALS would like to receive therapies that help more and harm less, are not well-represented by this document, and I'll stand by the statement that science and reality, ahead of one-size-fits-all, ill-informed wish lists, should inform a potentially life-changing decision.

I Am ALS is doing P/CALS no favors here.

You can blend all these wishes -- 100% safety, 100% tolerability, efficacy, unlimited eligibility, unlimited access, shorter trials, into a single "I want a pony" manifesto, sure. You can wave that manifesto around till the cows come home, but most P/CALS will grab with both hands for what they can get, as well they should.

Even once on the market, anything powerful enough to help also has the power to harm. We have run-in periods to have confidence in treatment effects. We have controls so efficacy can be established statistically. Etc. But every treatment is still a risk.

So even if these criteria made sense and all the trials were correctly classified by this matrix (they're not; for example, every trial has early stopping rules), what tradeoff are they informing? Every PALS must decide in terms of their own tolerance of risk, travel logistics, treatment burden, end of life plans, etc. This matrix is about advocacy on behalf of the organization, not decision support.

I've designed, conducted, reviewed, and published health services, drug, and device research for a few decades now. Happy to chat off line about any of the criteria or trials.

Best,
Laurie

 

[rknt50a]

There is no reason that a clinical trial can 't get 5 stars on the PaCTD scale and still deliver excellent science. In fact, if more sponsors paid attention to some of the items in the PaCTD standards, they would actually do better when it comes to delivering the scientific truth quickly and humanely.

I'm grateful that these people with ALS are working with sponsors to insure Open Label Extension in trials. It is outrageous when a participant in a trial has no option to continue (or even try for the placebo group) after fulfilling every trial requirement. Multiple lumbar punctures with no option for a try at the real experimental therapy is not right.

Too many sponsors blame the FDA for lack of EAPs when it is indeed the sponsors themselves who have not been building them into their plans. EAPs can provide an important adjunct to trial data in a broader population and provide humane access to experimental therapies to dying people who can't get into trials at the same time.

These are all options that are allowed today and are actually encouraged in the FDA ALS Guidance. I'm personally glad that some people with ALS are reasonably working with sponsors to make sure that they are delivering them.

The people in this IAA team are not whackadoodles. They have outstanding backgrounds in science and the pharmaceutical industry. I am grateful for their hard work as they are simultaneously dealing with caregiving and ALS itself.

 

[Anjum nazir]

Such a helpful thing...may God help you for helping the pals...

 

[lgelb]

Everything comes at a cost, humanity/expanded access included.

When I was in pharma, we had meetings to decide what we could afford to develop, and under what circumstances, and what we couldn't. When I was in managed care, we decided what we could reimburse, in devices we decided what we could build, likewise in health care we decided what we could buy in the way of equipment, etc. Every commitment is a roll of the dice, and a loss of the ability to commit to something else. So if too many fixed costs are part of a given option, it's less likely to go forward at all.

I have no problem with bringing a patient voice to industry, and have done so myself for some years, but I also know it would be foolhardy for P/CALS, from the standpoint of their own health, to decide among trials based on the criteria presented in this matrix. That, and only that, is my point.