Freelance orphans: “33 comparisons, 4 are statistically significant: much more than the 1.65 that would be expected by chance alone, so what’s the problem??”

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From someone who would prefer to remain anonymous:

As you may know, the relatively recent “orphan drug” laws allow (basically) companies that can prove an off-patent drug treats an otherwise untreatable illness, to obtain intellectual property protection for otherwise generic or dead drugs. This has led to a new business of trying large numbers of combinations of otherwise-unused drugs against a large number of untreatable illnesses, with a large number of success criteria.

Charcot-Marie-Tooth is a moderately rare genetic degenerative peripheral nerve disease with no known treatment. CMT causes the Schwann cells, which surround the peripheral nerves, to weaken and eventually die, leading to demyelination of the nerves, a loss of nerve conduction velocity, and an eventual loss of nerve efficacy.

PXT3003 is a drug currently in Phase 2 clinical testing to treat CMT. PXT3003 consists of a mixture of low doses of baclofen (an off-patent muscle relaxant), naltrexone (an off-patent medication used to treat alcoholism and opiate dependency), and sorbitol (a sugar substitute.)

Pre-phase 2 results from PXT3003 are shown here.

I call your attention to Figure 2, and note that in Phase 2, efficacy will be measured exclusively by the ONLS score.

My reply: 33 comparisons, 4 are statistically significant: much more than the 1.65 that would be expected by chance alone, so what’s the problem??

I sent this exchange to a colleague, who wrote:

In a past life I did mutual fund research. One of the fun things that fund managers do is “incubate” dozens of funds with their own money. Some do very well, others do miserably. They liquidate the poorly performing funds and “open” the high-performing funds to public investment (of course, reporting the fantastic historical earnings to the fund databases). Then sit back and watch the inflows (and management fees) pour in.

The post Freelance orphans: “33 comparisons, 4 are statistically significant: much more than the 1.65 that would be expected by chance alone, so what’s the problem??” appeared first on Statistical Modeling, Causal Inference, and Social Science.

Data http://ift.tt/2xJ3Kea October 16, 2017 at 05:00PM

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