On January 5th, the FDA released a summary of performance metrics for novel drugs introduced in 2016 and it is important to recognize that the data shows the current process for novel drug review is working very well. Novel drugs are innovative in that they advance clinical care, especially by offering therapy for previously untreatable diseases.
According to FDA’s annoucment,“[W]e also focus on the medical value of many of these new drugs, their contributions to enhanced patient care, and the various regulatory tools [the] CDER [Center for Drug Evaluation and Research] used to help ensure their safe and efficient development and approval. In 2016, novel drug approvals included the first treatment for patients with spinal muscular atrophy, the first drug approved to treat Duchenne muscular dystrophy, a new drug to treat hallucinations and delusions in some people with Parkinson’s disease, a new drug to treat patients with a rare chronic liver disease known as primary biliary cirrhosis, and two new treatments for patients with hepatitis C. The field also includes new treatments for patients with ovarian cancer, bladder cancer, soft tissue sarcoma, and chronic lymphocytic leukemia—as well as two new diagnostic agents for detecting certain forms of cancer.”1
These approvals offer afflicted patients hope for improved outcomes.
New drug approvals
In 2016, the FDA approved 22 novel drugs, including New Drug Applications (NDAs) or therapeutic biologics under the Biologics License Applications (BLAs). (Omitted from this group are new drugs that do not offer unique clinical advantages over previously approved drugs, for example, generics.) From 2007 to 2015, the approval rate for novel drugs averaged about 30. While approvals are down, the number of applications filed has remained stable, at about 40.
FDA stratified-approval scenarios
CDER used at least one or more of the following scenarios to expedite the development and regulatory approval of 16 novel drugs in 2016: fast track (eight approvals), breakthrough therapy (seven approvals), priority review (15) and accelerated approval (six).
PDFUFA performance
The Prescription Drug User Fee Act (PDUFA) allows the FDA to charge fees to drug developers to meet performance goals. FDA met the mandated performance measures for 21 of the 22 drug approvals.
First country of approval
Decades ago, it was common for firms to first seek marketing approval for therapeutics in countries outside the U.S.A. In 2016, however, 19 of the 22 novel drugs were approved in the U.S.A. first.
FDA’s report concludes: “More important than the quantity of novel drugs approved by CDER in 2016 is their medical value and the important new roles they are serving to advance patient care.”1
All involved in drug research and production should be proud of the fine work that went into adding 22 new products to the therapy arsenal.
Reference
- http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ ucm534863.htm
Robert L. Stevenson, Ph.D., is Editor Emeritus, American Laboratory/Labcompare; e-mail: [email protected].