The Federal Right to Try Act of 2017—A Wrong Turn for Access to Investigational Drugs and the Path Forward

Abstract

In 2017, President Trump said that "one thing that's always disturbed" 1 him is that the US Food and Drug Administration (FDA) denies access to experimental drugs even "for a patient who's terminal… [who] is not going to live more than four weeks [anyway.]" 1 Fueled by emotionally charged anecdotes recirculated by libertarian political activists, 38 states have passed Right to Try laws. In 2017, the US Senate approved a bill that would create a national law (Box). 2 As of December 2017, the US House of Representatives was considering the bill.Although the FDA has an expanded access option for utilizing experimental drugs outside of clinical trials, Right to Try laws create an alternative pathway that bypasses the agency. Moreover, the term "Right to Try" is a misnomer: the legislation creates a right for a patient to ask a company to provide a product, the same right that currently exists. 3 As is the case with the FDA program, companies are not obligated to provide access.The proposed federal Right to Try legislation 2 sets the threshold for patients to access investigational drugs at the completion of phase I (dosage-determination) trials, grants companies and physicians broad immunity from liability, and largely blinds the FDA to safety or efficacy data from these therapeutic attempts. Such changes would upend the agency's expanded access program, which has worked successfully for decades to provide patients who are seriously ill and without therapeutic options access to investigational drugs and still assure the safety and efficacy of new drugs before these products gain marketing approval. The legislation does not solve actual concerns, such as lack of knowledge that the program exists and the very limited supply of many investigational products.A 2016 review 4 of 10 years of FDA records found that the Center for Drug Evaluation and Research receives over 1000 expanded access applications per year. The FDA reviews such requests in days-or hours when an emergency so requires-and approves over 99%. 5 This is no mere rubber stamp of proposals: unlike individual physicians, the FDA has access to proprietary data about the risks and benefits of the investigational product or others in the same class. In some instances, the agency can improve the proposed treatment via modifications of drug dosage, dosing schedule, or other aspects. 6 Eliminating the FDA's review of expanded access requests is perilous, because most of the drugs that succeed in phase 1 trials turn out to be too unsafe or ineffective for clinical use. Phase 1 trials are intended to find a reasonable dosage of a drug in a small number of subjects, who may not even have the disease in question.

Availability of Investigational Medicines Through the US Food and Drug Administration’s Expanded Access and Compassionate Use Programs

“…The Right to Try Act, on the other hand, encourages sponsors to make investigational medicines available earlier in the clinical development period, in part because it shields them from any liability associated with patient injury due to expanded access. 4 Nevertheless, under both the FDA’s expanded access program and the proposed Right to Try Act, sponsors may still refuse access to their investigational medicines. Therefore, legislative efforts aiming to safely ensure drug availability should work to involve both investigational medicine manufacturers and the FDA.…”

“…3,4 Thus, the FDA is balancing 2 competing priorities: investigational new drug access and protection of patients from therapies without established safety. To our knowledge, no studies have examined the timing and duration of drugs made available through expanded access programs to determine whether the program was serving its original purpose.…”

Ethics codes and use of new and innovative drugs

“…These laws have received extensive coverage in both biomedical and lay journals and have resulted in very lively discussions about patients' access to investigational drugs. Notably, they have been heavily criticized by experts for offering false hope to patients without providing any actual improvements in access to investigational drugs .…”

Value in Oncology: It Is in the Eyes of the Beholder

“…Right to try legislation might be seen as a consequence of these realities despite the well-known limitations of and opposition to this law. 19,20 Although the influences are many, the core principle in the patient perspective of "the right drug at the right time" appears heavily weighted to unrestricted access.…”