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Regulatory Shortcuts to Approval

Fast-Track drugs

The FDA’s fast-track program is saving tens of thousands of lives by helping to “develop and expedite approval of drugs and biologics that address unmet medical needs for serious or life-threatening conditions,” according to the Tufts Center for the Study of Drug Development. Initially focused on products for treating HIV/AIDS and cancer, the fast track program now targets a growing number of diseases, including Parkinson’s, and “has on average shaved two to two and a half years off the time usually required to develop a new drug and win approval.”^[2]

Within the program’s first five years, some 200 drugs received fast track designations and two dozen of these were approved for marketing to consumers. “Fast track designations for products aimed at treating diseases other than cancer and HIV/AIDS grew from more than 30 in 2001 to more than 50 in 2003.”^[2]

“The fast track program has had a significant public health impact by speeding access to new drugs… that afflict millions of patients and result in the loss of tens of thousands of lives every year in the U.S.,” said Tufts Center Associate Director Christopher-Paul Milne.^[2]

Smaller emerging companies see the FDA’s fast track designation and accelerated approval process as a way of hastening a drug s time to market, and sometimes of legitimizing their science. Consideration under the accelerated approval program can even land a biotech a development partner and better terms.

Fast track designation differs from “expanded access,” which allows patients with serious and life-threatening conditions to access investigational new drugs (INDs) prior to approval.

Sponsors can request, fast-track designation at any time in the drug development process. If approved, benefits include:^[3]

More frequent meetings with the FDA to understand exactly what the agency wants and quickly address any problems that arise throughout the process.
Option to submit a New Drug Application in sections rather than all at once.
Approval for a study evaluation based on a substitute measurement for the usual clinical end point of prolongation of survival.

Fast-track designation may or may not lead to a Priority Review, which decreases by four months the time the FDA targets for reviewing a marketing claim and approving the completed application. It can, but does not necessarily guarantee accelerated approval.

Accelerated access

The FDA also may grant accelerated approval to speed patient access to promising drugs for serious life-threatening diseases that provide meaningful therapeutic benefit over existing therapies. Based on preliminary evidence, approval is provisional, with the sponsor required to conduct post-marketing studies to gather complete efficacy information, verifying and describing the clinical benefit of the particular drug. Drug companies do not always make good on their promise to complete these “catch up” trials. If the studies are not completed with due diligence, or if they fail to verify clinical benefit, the FDA can withdraw marketing approval or modify labeling claims. Lawmakers have proposed legislation that would fine companies that fail to perform post-marketing studies within a designated time frame following approval.

Post-marketing studies are essential in assessing optimal use of drugs and biologics. Additional information, while not essential for approval, is important in improving the prescribing and use of the product; product quality; or consistency in product manufacturing. Post-marketing studies may confirm existing data, raise or answer questions, or provide new data.^[5]

Previously approved drugs (e.g. Paxil, Vioxx, Bextra) may be withdrawn from production because they prove to be riskier, have dangerous side effects, or are found to be less effective than originally thought. Some drugs (Amgen’s GDNF) are withdrawn during clinical trials because of the drug company’s perceived unacceptable risk to trial participants, while others (Tysabri for MS) were not withdrawn until they’d already been approved and in use.

Sources

[1] Guidance for Industry Fast Track Drug Development Programs — Designation, Development, and Application Review; FDA July 2004

[2] FDA’s Fast Track Initiative Cut Total Drug Development Time by Three Years, According to Tufts Center for the Center of Drug Development; Press release 11/13/2003

[3] East Bay Business Times March 11, 2005, Fast track Speeds Drugs to Market; Susan L. Thomas

[4] FDA Center for Drug Evaluation and Research, FDA, Oncology Tools

[5] FDA Center for Biologics Evaluation and Research Report to Congress Reports on Postmarketing Studies [FDAMA 130]