FDA Approved Nearly All Expanded Access Requests in FY2016

The US Food and Drug Administration (FDA) granted more than 99% of requests for patients to access unapproved drugs and biologics in fiscal year 2016, according to data released last week.

Through FDA's expanded access program, the agency allows patients with serious or life-threatening diseases to access unapproved drugs.

Under the program, physicians can request access to a drug for individual patients or a group of patients under an expanded access investigational new drug application (IND) or via a new protocol under an existing IND.

Right to Try?

The figures come in the midst of a nationwide debate over access to unapproved drugs, with "right-to-try" proponents criticizing FDA for restricting access to unproven treatments.

Currently, more than 30 states have adopted right-to-try laws, which allow terminally ill patients to get unapproved drugs from manufacturers without FDA's involvement, and a bill before Congress would do so on a national level.

However, FDA's experience with its expanded access program paints a different picture.

Industry Declines Requests

In total, FDA's Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) received 1757 expanded access requests during FY2016, with CDER approving 99.4% of the 1554 requests it received, and CBER approving 96.6% of its 203 requests.

These figures reflect a consistent trend of FDA approving the vast majority of the 1,000 plus expanded access requests it receives each year. In fiscal years 2015, 2014 and 2013, FDA approved 99.0%, 99.3% and 98.9% of expanded access requests, respectively.

Recently, FDA has sought to further improve the expanded access program by releasing a streamlined application form and by finalizing two question and answer guidance documents related to the program.

However, despite FDA's high acceptance rate for expanded access requests and efforts to improve the program, many patients do not gain access to the unapproved treatments they seek.

Testifying before the Senate Committee on Homeland Security and Governmental Affairs last September, FDA's Associate Commissioner for Public Health Strategy and Analysis Peter Lurie explained that this is usually a result of industry declining requests.

"Based on information available, it appears that pharmaceutical companies turn down considerably more applications from physicians than does the agency. For example, one company indicated that it had turned down 98 of 160 applications for a single drug in a six-month period," Lurie said, citing a recent article in JAMA by bioethicist Arthur Caplan.

Lurie also addressed another major criticism of FDA's expanded access program. Many have suggested that companies are less willing to provide drugs via expanded access because FDA can consider any adverse events that occur under an expanded access protocol when reviewing a drug.

However, Lurie said that FDA's recent guidance makes clear that the agency recognizes there are differences between the patients seeking expanded access and those enrolled in clinical trials.

"This guidance makes clear that the agency understands that expanded access is a very particular context (sicker patients, multiple illnesses, concurrent medications, etc.) and that FDA takes that context into account when interpreting adverse events," Lurie said.

Right-to-try bills introduced at the federal level, in addition to side-stepping FDA's compassionate use program, seek to ensure that companies who allow compassionate use requests will not have to disclose information from such patients to FDA.

FDA