Members of the U.S. House of Representatives overwhelmingly backed new user-fee legislation that includes a section on accelerating the review of new drugs that offer treatment for a serious or life-threatening condition--a broad mandate likely to draw close scrutiny from everyone engaged in drug development.
The passage of the House and Senate bills--which will now be reconciled--marks the culmination of months of intense lobbying by the biopharma industry: voices which have been insisting that the FDA needs to be more responsive to drug developers and more open to a quick-step review process.
"A new generation of modern, targeted medicines is under development to treat serious and life-threatening diseases, some applying drug development strategies based on biomarkers or pharmacogenomics, predictive toxicology, clinical trial enrichment techniques, and novel clinical trial designs, such as adaptive clinical trials," notes the legislation, laying the stage for the FDA to designate drugs that can be fast-tracked using surrogate trial endpoints or other tools to accelerate a review.
In place of a surrogate endpoint, developers will also be able to pursue an approval on an endpoint that can be "measured earlier than irreversible morbidity or mortality, that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit." This process, the bill continues, should allow for "fewer, smaller, or shorter clinical trials for the intended patient population or targeted subpopulation without compromising or altering the high standards of the FDA for the approval of drugs."
While the House and the Senate now have until October 1 to reconcile the two bills, both include language aimed at prodding the FDA to move more quickly on the approval process, mandating increased face time with developers and offering biopharma companies a chance of moving to the market sooner. But the House bill also includes a fast-track provision for disapprovals, allowing regulators a chance to yank a drug that hasn't been put through promised post-marketing studies or treatments that may have been improperly touted by the drugmaker.
Over the next 12 months we'll get a chance to see exactly how flexible the FDA will become. We'll also be able to see which therapies win the broader fast-track regulatory path as they blaze a trail for others to follow.