Miscellaneous Statistics, Multilevel Modeling, Public Health

Orphan drugs and forking paths: I’d prefer a multilevel model but to be honest I’ve never fit such a model for this sort of problem

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Amos Elberg writes: I’m writing to let you know about a drug trial you may find interesting from a statistical perspective. 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 (CMT) 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…
Original Post: Orphan drugs and forking paths: I’d prefer a multilevel model but to be honest I’ve never fit such a model for this sort of problem