According to Bloomberg News, North Carolina Democratic Senator Kay Hagan is set to introduce a bill that would create new “progressive” and “exceptional” approval processes for new drugs to treat unmet needs. These would be similar to the FDA’s existing “accelerated approval” process, open primarily to AIDS and cancer drugs, which permits the agency to grant a conditional approval once intermediate clinical trials demonstrate improvement in a so-called “surrogate end-point” such as tumor shrinkage. Once granted accelerated approval, these drugs must still complete more rigorous Phase III clinical trials to demonstrate improvement in the ultimate clinical end-point of patient survival, or the agency may revoke the conditional approval.
Details of the bill are not yet available, but I understand that “progressive approval” would function essentially like accelerated approval does now, but that the pathway would be open to any drug that would be the first approved treatment for a given disease or condition, or for an identifiable sub-population with the disease or condition. “Exceptional approval” would be available in cases where standard controlled clinical trials would be unfeasible to conduct, such as when the patient population is so small that enrolling a large clinical trial of thousands of patients would be nearly impossible. It would also be open to cases in which it would be unethical to conduct a standard placebo-controlled trials because that entails some patients getting no active treatment.
The accelerated approval process has been somewhat controversial, inasmuch as it can result in the withdrawal of an approved drug that has benefited some, but not all, patients taking it. That’s what happened with the breast cancer indication for the drug Avastin, which I wrote about earlier this year. It also becomes difficult for manufacturers to enroll clinical trials once a product may be legally sold to any patient whose doctor prescribes it. There’s a critical discussion of accelerated approval here.
The process is by no means perfect. But to the extent that it helps get needed drugs to market sooner, accelerated approval has been a tremendous boon for patient health. So, I look forward to seeing Sen. Hagan’s bill, and I applaud her efforts to help expedite the availability of new treatment options.