FDA finalizes rules on faster approval for some drugs
The Food and Drug Administration has published final guidance to help manufacturers of drug and biologics bring products for treatment of unmet needs to the market faster than they can through the usual approval pathways.
“For those drugs that qualify, participating in one of these expedited programs can reduce the time and possibly the cost of developing new therapies that can save lives,” Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, said in a written statement. “That’s a win for drug innovation and for patients.”
The guidance describes four expe- dited programs and the criteria that qualify products for these faster pathways. These include fast-track designation, breakthrough therapy designation, accelerated approval and priority review designation. A draft version of the guidance, which received comments from major companies including Merck & Co., Novartis and Pfizer, was released last summer.
The regulations are intended to speed the availability of new therapies to patients with serious conditions, especially when there are no satisfactory alternative therapies, while preserving appropriate standards for safety and effectiveness. The FDA said the regulations recognize that “patients and physicians are generally willing to accept greater risks and side effects from treatment of life-threatening and severely debilitating diseases than they would for other diseases.” The agency also recognized that certain aspects of drug development that are feasible for common diseases might not be feasible for rare diseases and that development challenges are often greater with increasing rarity of the disease.
Nearly half of the 27 new drugs the FDA approved last year took advantage of at least one of these expedited drug development and review approaches, with review times as short as 4½ months. That compares with the year or more review period for other drugs, according to the agency.
The release of the final guidance comes nearly three months after the FDA alerted drugmakers that they have to change labeling on drugs approved through the accelerated approval pathway so doctors and patients know such approvals could subsequently be revoked.
Approval granted through one of these programs can be revoked in cases where a trial required to verify the clinical benefit of the product fails to document that benefit. Other criteria for revocation include when evidence comes to light the product is not safe or effective, and when a manufacturer does not conduct the required postapproval trial of the drug or disseminates false or misleading promotional materials relating to the product.