Expanding Access To Experimental Drugs

Drug and biologic developers have faced increasing pressure from patients and their advocates to make investigational drugs available for compassionate use prior to approval by the Food and Drug Administration (FDA). Over the past year social media campaigns have spotlighted patients seeking early access to potentially life-saving treatments, drawing attention to the growing debate between patient advocates promoting wider access to investigational drugs, and those urging more cautious approaches. Propelled by the plight of critically ill patients desperately seeking new treatments, several states have passed laws giving patients the "Right to Try" investigational drugs or biologics. FDA has also taken steps to make its "Expanded Access" process more user-friendly, and the Agency continues to solicit input from patient groups on risk-benefit analysis and available treatments as part of its Patient-Focused Drug Development Initiative.

Companies may want to monitor regulatory and legislative developments relating to early access to investigational treatments, and to adopt formal compassionate use policies.

Right-to-Try Laws

The Goldwater Institute in Phoenix, Arizona, with backing from patients and their advocates, drafted and successfully orchestrated introduction of "Right to Try" measures in 40 states, with 17 of the states enacting legislation and Arizona passing a ballot initiative. In theory, the laws provide terminally or seriously ill patients access to experimental treatments, while circumventing more rigorous requirements imposed by the FDA under its "expanded access" program. Proponents of the laws maintain that the "Right to Try" measures protect the "fundamental right of patients to save their own lives."

Those on the other side of the debate, including the American Society of Clinical Oncology (ASCO), have raised concerns that the laws give patients false hope of a cure, noting that the unproven treatments are potentially toxic and may exacerbate symptoms and/or hasten death. Detractors also worry that allowing patients to obtain experimental drugs outside of the traditional clinical process will jeopardize patient enrollment in clinical trials and prevent drug companies from fulfilling their ethical duty to obtain timely FDA approval, so that patient populations—not just single patients-can be treated. Others also fault state laws for exempting insurers from paying for the experimental treatments and leaving patients at financial risk for covering the high costs of the experimental treatments and at risk for withdrawal of hospice care eligibility.

Some critics also question whether states have the power to regulate access to investigational treatments, when drug regulation falls under the purview of the FDA and the federal government. Some have re-dubbed the measures "Right to Ask" laws, noting that the new laws foster a second illusory hope of direct access to experimental treatments, when states cannot require drug manufacturers to provide the experimental therapeutics to patients. In fact, drug/biologic manufacturers subject to FDA regulation may insist on adherence to FDA's stricter "expanded access" requirements before making investigational drugs available, if at all.

FDA's More Streamlined and User-Friendly Expanded Access Procedure

Although it is unclear whether state Right-to-Try laws have had any direct benefit...