The Right to Try bill signed into law by President Trump on May 30th this year allows terminally ill patients residing in the U.S. to gain access to medicines that have passed Phase 1 of the FDA approval process but have not established efficacy in post Phase 1 clinical trial testing. Prior to Trump’s signing of the legislation 38 states had already enacted Right to Try laws.
Critics of Right to Try have focused on the regulatory and safety aspects. Here, however, the emphasis is on logistics and equity concerns.
Crucially, in order for Right to Try to be realized a pharmaceutical firm must be willing to provide experimental drugs to the patient. Nothing in the legislation makes it mandatory for drug companies to provide these medications. Furthermore, even if offered to patients many will not have access to medical facilities capable of administering investigational medications, and the vast majority of payers will not reimburse the medicines or required ancillary services. Nor can most patients obtain the necessary resources for reimbursement through industry-sponsored patient assistance programs or charitable organizations.
Expanded Access, also called Compassionate Use, is the name of a program that already does practically the same thing as Right to Try. It allows patients with serious and life-threatening diseases to obtain experimental treatments. The difference is that Compassionate Use operates through the Food and Drug Administration (FDA), which may deny requests for use of unapproved drugs. FDA’s oversight duties are not limited to assessing requests. The agency also leverages its knowledge of investigational medications to help physicians and other healthcare providers with proposed treatment plans, including dosing adjustments.
The FDA regulatory process exists for many reasons, not least of which is to enforce the do no harm principle, but also to establish when companies may charge for experimental treatments.
If made available by pharmaceutical companies, unapproved drugs – whether in the Compassionate Use program or under Right to Try arrangements - tend to be provided to those who know about the possibility, have access to hospitals with the appropriate infrastructure to deliver investigational medicines to patients, can afford the full array of treatment costs, or are the beneficiaries of charitable donations.
The Right to Try law is well-intended, but hollow. A rigorous regulatory framework, such as the one that undergirds the Compassionate Use program, is needed to prevent undue harms to patients. At the same time, and equally important in my view, policymakers need to find ways to address systemic inequalities in access to investigational drugs. Without a means of defraying costs for patients who cannot afford an investigational treatment offered by a drug company or don’t have access to a nearby medical center with the appropriate infrastructure, the bill will exacerbate the inequality that already exists in the U.S. healthcare system.
