ClinPage has drunk the Kool-Aid about adaptive designs. We think they could forever alter the timelines and budgets of entire portfolios of clinical trials. But there is hardly a technology we don’t like. Cable TV, perhaps.

On the other hand, there is skepticism about adaptive trial designs. It boils down to three big factors.

1) Few companies can readily implement adaptive trials due to the antiquated, wretched technology silos in the life sciences.
2) There is more discussion of the adaptive approach than real usage of it.
3) The FDA position on the technique remains ambiguous at a time when the agency awaits both research (from academia) and dollars (from Congress).

Heavenly Herb

Could all that be changing? Yes. Yesterday, we noted a press release that would seem to contradict those objections to adaptive designs. South San Francisco’s Napo Pharmaceuticals announced it had won FDA fast-track designation for a trial of an anti-diarrhea medication for patients suffering HIV. The press release contains a capsule description of how the adaptive aspects of the design will unfold. The molecule in question is an herbal extract from South America that can be harvested in a sustainable way.

Will other sponsors use the anticipation surrounding adaptive trials to attract attention from investors? It’s hard to say. We know we’ll be at an inflection point when the technique is so routine that no one bothers to mention it any more. That’s probably a decade away, of course, with the first wavelets of adaptive trials just on the drawing boards.

Here’s a quotation from the Napo news release: “The Advent trial (“Anti-Diarrhea therapy in HIV disease-Emerging treatmeNT concepts) is a randomized, double-blind, parallel-group, placebo-controlled, two-stage, adaptive design study to assess the efficacy and safety of crofelemer 125 mg, 250 mg, and 500 mg oral doses twice daily for the treatment of HIV-Associated Diarrhea. Napo previously met with the United States Food and Drug Administration (FDA) on January 16, 2007, under the Special Protocol Assessment Process on this adaptive design of its Advent trial. The study will be carried out under an FDA Fast Track designation.”

PDUFA Angle

And another quote: “The IRB approval of our protocol with the adaptive design is a very important milestone for Napo”, said Lisa A. Conte, chief executive officer of Napo. “This represents the ability to proceed efficiently in our Phase 3 study, while gaining the commercial benefits of testing lower doses in the final study prior to filing our NDA for the AIDS diarrhea indication. While these efforts have prolonged the initiation of dosing in the trial, Napo is still aiming to submit an NDA for this indication in the next year, and targeting approval under fast-track designation by mid-2008.”

For reasons that can only be speculated about, the release noted that the trial’s special status was in part an outgrowth of PDUFA, the controversial legislation that diverts user fees from industry to augment an FDA budget that the U.S. Congress is unable to allocate funds for. Critics point to PDUFA as an example of unhealthy alignment between U.S. officials and the companies they are supposed to regulate. But the legislation is expected to be renewed after the usual posturing and pontificating by politicians in both parties.