Biotech Values

[biomaven0]

>>disease modifying

There's a good recent discussion of this in the NEJM in the context of Parkinson's. Here's what that particular trial needed to show in order to be considered "disease modifying":

In this double-blind trial, we examined the possibility that rasagiline has disease-modifying effects in Parkinson's disease. A total of 1176 subjects with untreated Parkinson's disease were randomly assigned to receive rasagiline (at a dose of either 1 mg or 2 mg per day) for 72 weeks (the early-start group) or placebo for 36 weeks followed by rasagiline (at a dose of either 1 mg or 2 mg per day) for 36 weeks (the delayed-start group). To determine a positive result with either dose, the early-start treatment group had to meet each of three hierarchical end points of the primary analysis based on the Unified Parkinson's Disease Rating Scale (UPDRS, a 176-point scale, with higher numbers indicating more severe disease): superiority to placebo in the rate of change in the UPDRS score between weeks 12 and 36, superiority to delayed-start treatment in the change in the score between baseline and week 72, and noninferiority to delayed-start treatment in the rate of change in the score between weeks 48 and 72.

http://content.nejm.org/cgi/content/short/361/13/1268

It's really a statistical nightmare to meet these conditions (or even to design a trial to demonstrate disease modification that will keep everyone from grumbling about the design and the interpretation of the results), and probably kind of moot. If the drug works, it's going to be used whether or not they can ever demonstrate disease modification. The only practical implication would be whether you need to treat early or not, and in a drug with a good SE profile that isn't too significant a question.

Peter