When Jenn McNary’s son Austin was three and her son Max was just a newborn, both boys were diagnosed with Duchenne’s Muscular Dystrophy— an incurable, fatal, degenerative muscle disorder. By the time Jenn learned of a promising treatment undergoing testing in clinical trials, Austin had declined so badly that he was restricted to a wheelchair. Jenn immediately tried to enroll both boys in the trial—only to learn that the trial was limited to ambulatory patients. That meant Max was eligible, but Austin’s disease had progressed too far to qualify. Jenn was forced to watch while one son’s condition improved significantly under treatment, and her other son’s condition worsened until he could no longer dress or use the restroom without help. Thirteen-year-old Max became sixteen-year-old Austin’s caregiver.
The United States’ drug approval system is broken. It blocks Americans from potentially lifesaving medicines and treatments until those treatments receive final approval from the Food and Drug Administration (FDA). And it takes an average of fourteen years and $1.4 billion for a drug to make its way through the clinical trial process and obtain FDA approval. But this is time that dying patients do not have. Patients like Austin and Max—and people like Jenn, who love them—are forced to suffer in limbo, with no say in their own destinies. All of this occurs despite the fact that one of the bedrock principles of medical ethics is patient autonomy: decisions about health care are ultimately for the patient to make.
A new project, called Right to Try, aims to change this. Now law in thirty-seven states, Right to Try statutes protect the right of terminally ill patients to access medicine that has received basic safety approval from the FDA— and that is being given to patients in ongoing clinical trials—but that has not yet received final approval for sale. This Article looks at some of the legal and ethical implications of these laws, and of the cumbersome, “consistently overconservative,” and sometimes life-destroying bureaucratic process by which the FDA restricts access to treatments patients need.