Adaptive trials move toward the mainstream

Adaptive trials move toward the mainstream

My friends at Cytel (CEO Ranganath Nayak and I used to work together at BCG) made the cover of the current Bio-IT World, where they are written up in Real-Time Trials. Cytel provides tools to help pharmaceutical and device companies with adaptive trial designs, which have the potential to substantially reduce the number of patients and/or time needed to conduct a clinical trial. That could be a major contributor to reducing the costs of drug development and to increasing the number of drugs in clinical development.

A problem with traditional (non-adaptive) clinical trials is that they require the designers to specify sample size and duration upfront, when there are still many uncertainties. As a result some trials are larger and longer than necessary to achieve their objectives. Other trials –especially by cash-strapped smaller companies– end up being too short or too small. The idea of adaptive trials is to analyze early trial data and use it to modify trial design –in a statistically valid way– while the trial is still in process.

Naturally, FDA is nervous about allowing such trials to proceed. It’s happening, though in the medical device arena, where about 10 percent of trials are already adaptive, and large companies like Pfizer and Novartis are dipping their toe in the water on the pharmaceutical side, even in the absence of clear guidance from FDA. (Ironically, the only example of a drug approved with an adaptive process is the extremely non-innovative Pravigard Pac, an attempt by BMS to extend the commercial life of Pravachol by co-packing it with baby aspirin.)

Companies like Cytel provide the sophisticated tools and expertise needed to pull off adaptive trials. There’s some debate in the article about whether companies can do the work internally or whether they need to rely on outside parties. It’s pretty clear to me that there’s some need for outside help: the math is complex and the FDA is more likely to trust a third-party to avoid the temptation of too much inappropriate peaking at the data.

I got a kick out of the following paragraph in the article:

“The value of an adaptive trial is really significant,”” predicts Gary Lubin, the hype-averse cofounder of Merck Capital Ventures. Lubin says adaptive designs could accelerate go/no-go decisions by five or six months. “The use of modeling will become an important part of designing these protocols. A lot of money will be spent in these [modeling and adaptive] areas.”

“Hype-averse” Gary may be, but the journalist probably should have mentioned that Merck Capital Ventures is an investor in Cytel.

June 29, 2006

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