Earlier this week the FDA Revitalization Act became law. Though the major thrust of this legislation addresses drug safety and conflict of interest in FDA scientific advisory committees, there are several changes that are of special interest for the HD community.
The new legislation requires a mandatory public listing at NIH of all phase II and III clinical trials in the U.S., both at initiation and completion. This means that the Huntington community will be able to find information in one place about every drug trial that is sponsored by a company or NIH federal funding. And just as important, the law mandates the reporting of trial results from phase II and III, wheter negative or positive (time frame to be determined).
In recent years, with the notable exception of Crest-X (the creatine dosing study), Huntington Study Group (HSG) investigators have rapidly communicated clinical trial information. They have announced clinical trials as soon as the first site for testing is available, and they have rapidly reported (phase III) trial results for Prestwick's tetrabenazine and (phase II and III) Amarin's Miraxion EPA. As more clinical trials begin for HD, it will be very important for timely reporting of phase II results, so that those who are eligible for phase III trials will have greater information as they choose among clinical trials.
In another positive step for those who are not eligible for clinical trials, the law requires posting of expanded access (compassionate use) programs. In the past, this information was not broadly available. This information can help the patient community in two ways. First it will simplify the first steps of access to available programs. And second it will give opportunity to influence decisions to start these programs when not available.