The guidance describes four expedited 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 post-approval trial of the drug or disseminates false or misleading promotional materials relating to the product.
Follow Virgil Dickson on Twitter: @MHVDickson
