Silver Spring, MD (Public Statement) – For patients with serious or im­medi­ately life-threatening diseases, the FDA remains committed to enhancing access to promising inves­ti­ga­tional medicines for those unable to access prod­ucts through clin­i­cal trials. This is the mission of our expanded access pro­gram. The agency is dedicated to these pur­poses, and it has been for more than three decades.

We’ve taken many steps to im­prove our process through which patients can access promising inves­ti­ga­tional drugs. We under­stand that treat­ment de­ci­sions for those facing terminal illnesses are best made by patients and families, with the sup­port of their treating physicians. When appro­pri­ate, those suffering from a terminal illness who’ve exhausted avail­able options should be able to access promising treat­ments being studied in clin­i­cal trials, or prod­ucts under active review by the FDA. The agency is faithfully committed to these goals, to protecting patients, and to making sure they’re able to make informed de­ci­sions.

Today, the Pres­i­dent signed into law the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 (Right to Try Act). At the FDA, we stand ready to implement this legislation in a way that achieves Congress’ intent to promote access and protect patients. The FDA is dedicated to achieving the goals that Congress set forth in this legislation, so that patients facing terminal con­di­tions have an addi­tional avenue to access promising inves­ti­ga­tional medicines.

This new law amends the Federal Food, Drug, and Cosmetic Act to estab­lish­ a new path­way aimed at in­creas­ing access to unapproved, inves­ti­ga­tional treat­ments for patients diag­nosed with life-threatening diseases or con­di­tions who have exhausted approved treat­ment options and who are unable to par­tic­i­pate in a clin­i­cal trial. Our implementation of the Right to Try Act will build on our long-standing efforts to help patients and families who are facing life-threatening diseases or con­di­tions, in a way that seeks to protect their autonomy, their safety, and the safety of others fol­low­ing in their paths.

The de­ci­sions we reach related to prod­ucts that can serve as an effective treat­ment for a terminal illness, or that can arrest a dev­as­tat­ing and debilitating con­di­tion, are among the most im­por­tant and carefully con­sidered judgments that we make. We recog­nize the im­por­tant bal­ance be­tween making sure patients have the assurances Congress intends, while enabling timely access to promising treat­ments in these dev­as­tat­ing cir­cum­stances. And we’ll implement this new law con­sis­tent with these longstanding values.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, security of human and veterinary drugs, vaccines and other biological prod­ucts for human use, and medical devices. The agency is also responsible for the safety and security of our nation’s food supply, cosmetics, dietary supple­ments, prod­ucts that give off electronic radiation, and for regulating tobacco prod­ucts.

Source: Food and Drug Admin­istra­tion.