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May 30, 2018 - Federal right-to-try legislation aimed at loosening oversight of access to unapproved drugs for "compassionate use" was signed by President Trump today. The law allows patients with cancer or other serious illnesses to circumvent the Food and Drug Administration (FDA) when requesting access to experimental therapies not yet approved for public use. Congress passed the final right-to-try bill on May 22 after months of negotiation and changes to the legislation.
FORCE supports the ability of terminally ill patients to access experimental treatments through approved pathways, but the new law raises ethical issues and could be detrimental to patients. The FDA serves to protect Americans, including critically ill patients and their families, from the potential harms of untested drugs.
Traditionally, patients access unapproved treatments by enrolling in a clinical trial. Patients who are ineligible for trials can utilize the FDA’s expanded access program, which allows the agency to authorize use of an unapproved therapy if it determines that the drug’s potential benefit justifies the treatment risk for an individual or small group of patients. The FDA revamped and streamlined the expanded access program in 2016 and continues to look for ways to improve it further. In fact, FORCE was invited to give feedback on ways to improve the system earlier this year.
FORCE and many other patient and medical organizations question the effectiveness of right-to-try initiatives (see letter) because the expanded access program approves over 99% of all requests for access to experimental therapies. In 2017, nearly 1,800 applications were approved.
The law permits patients who have exhausted other options to access investigational therapies without approval or oversight from the FDA. It would nullify the FDA’s authority if a patient is harmed or dies as the result of a treatment administered; agency officials would not be allowed to use that information to delay or block approval of the therapy. We are especially concerned that the FDA will not be permitted to consider safety data from right-to-try patients. If an experimental drug causes harm to a person using it off-label, it has implications for others who take the drug.
Right-to-try laws sound beneficial, but they may do little to help patients because many pharmaceutical companies are reluctant to offer medications outside of clinical trials. These laws allow—but don’t require—drug companies to give patients access to experimental treatments. Reportedly, 38 states have passed right-to-try laws but very few patients have benefited.
The new law includes liability protections for manufacturers, sponsors, physicians, clinical investigators and hospitals, "except in cases of willful misconduct or negligence." Whether this will be successful in facilitating more access is unknown.
The U.S. regulatory system for medical products and research was developed in response to serious patient harm. While obtaining therapies for compassionate use outside of a clinical trial is not about research, the drugs remain experimental and ultimately have not been proven safe and effective for humans. As a result, we believe the FDA expanded-access program should not be sidestepped or circumvented. It serves to protect critically ill patients and their families from harm and additional heartache. We will continue to monitor this issue and the law's impact on the patient community.