20 Sep The Food & Drug Administration & Compassionate Use
The U.S. Food & Drug Administration (FDA) is the federal agency that is responsible for protecting the public health by assuring the safety, efficacy and security of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation.
The FDA recently implemented new developments in their compassionate use (“expanded access”) program in hopes of solving problems that have been created by state efforts to pass legislation making it easier for patients to access drugs that are still in clinical trials. These state laws are often called “Right to Try” laws.
The FDA’s expanded access program essentially allows terminal patients with no other medical alternatives to access investigational drugs (i.e. medical products that have not yet been approved by the FDA).
Many in the patient advocacy community have argued that the expanded access program isn’t effective enough and advocated for state legislation that would permit terminally ill patients the “right to try” investigational drugs.
There are three main problems with the “right to try” laws, however:
- The FDA still has complete control of programs more extensive than its expanded access program. In other words, no matter how many state laws are passed, nothing happens unless the FDA approves it.
- Manufacturers are constantly threatened with liability, meaning that if someone is harmed by a drug the manufacturer is going to be held liable, even though the drug is still being tested.
- The process to provide investigational drugs is simply too precarious for manufacturers. If a very ill patient dies after taking an investigational drug (which maybe more likely given the patient’s health), the adverse event is then reported to the FDA. These adverse events make it harder for manufacturers to get approval from the FDA.
The FDA has attempted to alleviate some of these issues by adopting two new guidelines:
- “Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers”
- “Charging for Investigational Drugs Under an IND – Questions and Answers”
These guidelines include “expanded access protocol,” “new treatment IND,” and the use of institutional review board review. In addition, only a year’s worth of the drug can be charged without the FDA reevaluating and in the case where a manufacturer does not have an active IND, a physician can submit an expanded access IND instead.
The FDA’s new program is also voluntary, and thus cannot compel a manufacturer to supply investigational drugs. More importantly, the FDA’s guidelines still do not grant immunity for participants, as the FDA can still use adverse events against manufacturers in determining whether or not to grant approval of the drug.
There is legislative pending in Congress, the Trickett Wendler Right to Try Act of 2016, which if passed, would prohibit the use of adverse events from negatively affecting possible FDA approval and would not hold a manufacturer liable if there is an adverse event.
It is still unclear how much effect the states can have if the FDA is not in complete accordance with them. Nevertheless, only time will tell how successful these recent developments will be.
For more information about the expanded access program, visit: www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm.