GAO: FDA Improved Expanded Access but More Clarity Necessary

Posted 11 July 2017 By Zachary Brennan

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With the rise of Right-to-Try legislation across states in the US, it might seem like patients with serious or life-threatening ailments and no other comparable medical options might have difficulties in obtaining access to investigational drugs outside of a clinical trial because of the US Food and Drug Administration (FDA).

But a new Government Accountability Office report released Tuesday lends credence to many who have voiced concerns with the Right-to-Try laws, especially as the laws do not compel manufacturers to provide access to experimental drugs and as FDA approves more than 99% of all single-patient expanded access requests in emergency (often in less than a day) and non-emergency situations (within the 30 days allotted to the agency).

"From fiscal years 2012 through 2015, of the more than 2,300 emergency expanded access IND requests that were submitted, FDA’s median response time was within less than a day," the report says. And according to data released in March, FDA again granted more than 99% of all requests.

Manufacturer Communications

Last year, FDA also simplified its expanded access process and offered more guidance on expanded access.

And though GAO says the agency now offers "clear guidance on the expanded access data that must be submitted by physicians and manufacturers and recently took steps to communicate how it will use these data, the agency’s communication lacks clarity and specificity. In addition, this information is not consistently communicated in other documents that FDA uses to communicate with manufacturers about the program."   

GAO also notes the rare but concerning aspect of expanded access for manufacturers, which is that companies "may be more likely to deny expanded access requests for fear that any adverse events associated with these often terminally ill patients may delay the development of the drug should FDA place a clinical hold due to the adverse event."

Such a situation has occurred at least once in the recent past.

"Such delays in clinical trials can have a significant impact on a manufacturer, especially on small companies trying to make a drug available to a larger patient population. Without clearly communicated information from FDA on how adverse event data from expanded access is used, manufacturers do not have the information they need to make informed decisions about expanded access," GAO says.

GAO also recommends that FDA Commissioner Scott Gottlieb "clearly communicate how the agency will use adverse event data from expanded access use when reviewing drugs and biologics for approval for marketing and sale in the United States."

GAO Report: FDA Has Taken Steps to Improve the Expanded Access Program but Should Further Clarify How Adverse Events Data Are Used

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Categories: Drugs, Regulatory strategy, Regulatory intelligence, News, US, FDA

Tags: expanded access, compassionate use, GAO reports, adverse events

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