Crowdsourcing Project Predicts Progression of ALS

sciencehabit writes Using data from old clinical trials, two groups of researchers have found a better way to predict how amyotrophic lateral sclerosis (ALS) progresses in different patients. The winning algorithms—designed by non-ALS experts—outperformed the judgments of a group of ALS clinicians given the same data. The advances could make it easier to test whether new drugs can slow the fatal neurodegenerative disease. For the competition, participants were given just a slice of this data set, collected over 3 months, and asked to design an algorithm to predict how patients would fare in the subsequent 9 months, according to a standard functional scale that measures their ability to move and care for themselves. When predictions from the two winning algorithms were combined, they outperformed estimates solicited from a dozen ALS clinicians who pored over the same data, the authors report. They estimate that using these algorithms to predict outcomes could allow a drug sponsor to reduce the size of the trial by at least 20% and save as much as $6 million in a large phase III trial.

Comments

Cost savings - so what?

[DoofusOfDeath]

Seems to me that drug makers will charge whatever maximizes their sales revenue, regardless of their development costs.

Lowering their development costs for them might affect whether or not they bring it to market. But if they do, they'll charge as much as they can get away with.

Re:Cost savings - so what?

[sconeu]

My late wife was in a clinical trial for ALS. Unfortunately, it didn't work and there was no Phase3 III. We also found out after the fact she was in the placebo group.

But you're absolutely right intoit, if you're ever unlucky enough to lose the ALS lottery (or have your SO do so), you will grasp at every fucking straw that comes your way. If that includes becoming a pin cushion or a guinea pig, you do it.

Re:Cost savings - so what?

[ihtoit]

an associate of mine was diagnosed with ALS in 1998 following a single incident of unexplained loss of motor control (which just so happened while he was meeting with a physician). He was permanently confined to a wheelchair within three weeks and a pine box 4 months later. It was terrifying how quickly it took him considering his lifestyle (cycled *everywhere*, his car actually expired in his garage through underuse). ALS is not fussy about how fit you are or how stressful your job is or pretty much any other external influence. If it wants you, it'll just take you - on its own timetable.

Re:Cost savings - so what?

[ihtoit]

the way they negotiate lower prices is to guarantee exclusivity in the supply chain. A treatment available on Medicaid from GSK won't be available through the same route from Pfizer, because Pfizer will be frozen out of the chain by virtue of a GSK-included clause in the Medicaid contract.

To borrow a car analogy, it's like buying a 2014 Rolls Royce Phantom (V12 Merlin, come on! Tease me some more!) and being told that your warranty will be null and void if you swap out the engine block for a 410 Ford V8.

Re:Too late for Lou Gehrig

[ihtoit]

I would say very much so, considering he's been dead for 73 years.

Re: Too late for Lou Gehrig

[superswede]

Everyone in the US, but in the rest of the world almost no one knows who Lou Gehrig was.

It's better to call deceases by their medical names and not based on some person's name.

Complete Misunderstanding of Epidemiology

[The_Laughing_God]

They can't validate a scale using unintervened progression or existing treatments, then pretend it says ANYTHING about a new/unknown treatment. The whole point of a new treatment is to alter the progression of the disease in a new/different way; the whole point of clinical trials is to determine the NEW course of the disease using the NEW treatment.

The claim made here is: a better tool to predict the time progression of headaches treated with aspirin (or beer or sex) can better predict the time progression of a headache treated with some yet-uninvented drug, so we needn't test the new treatment as thoroughly to characterize it. That's like saying "the more predictable sex with your partner is, the more you know about sex with a different partner"

And yes, I AM a physician and molecular biologist.