The Right to Try (R2T) Act of 2017 was signed into federal law on May 30, 2018.¹ This legislation amends the U.S. Food, Drug, and Cosmetic Act to allow patients with life-threatening diseases who lack approved treatment options to access eligible investigational drugs without formal compassionate use protocols. For an investigational drug to be eligible, at least one phase I trial must have been completed, with additional clinical trials ongoing. R2T also exempts pharmaceutical manufacturers and health care providers from liability associated with treating patients with investigational drugs under R2T provisions.

Before passage of this legislation, individual patients could access investigational treatments either by enrolling in clinical trials or through the FDA’s expanded access (EA) program, also known as “compassionate use.”² The individual EA program requires the patient’s physician to initiate the request and the pharmaceutical manufacturer to agree to provide the medication; the required forms and documents are then submitted to the FDA for review and approval. Oversight by local human subjects committees or IRBs is also a component of EA, providing important protections for patients. The R2T act essentially eliminates FDA/IRB oversight and vendor/provider liability from the investigational treatment process, though physician oversight, pharmaceutical manufacturer approval, and patient consent are still required.

Proponents of R2T cite that only 3% of terminally ill patients enroll in clinical trials and that only about 1000 EA approvals are granted by the FDA each year, making this legislation a way to help the many patients with a terminal illness gain access to unapproved therapies.³ The Goldwater Institute supports R2T, suggesting that “many patients run out of time before they can qualify for the exemption or complete the process. Right To Try laws help patients get immediate access to the medical treatments they need before it’s too late.”³

However, few, if any, patient advocacy groups or professional societies support R2T, and 75 patient advocacy groups, including the American Cancer Society, have signed a letter to Congress urging Congress to reject the bill.⁴ Opponents of R2T suggest this legislation is unlikely to improve access because physicians are not required to place R2T requests on behalf of their patients, insurers are not required to pay for R2T treatment, and pharmaceutical manufacturers are not required to fulfill R2T requests for investigational agents they are studying. Nor does the FDA’s individual EA program appear to be a major barrier – during 2000–2015, the FDA denied only about 1% of EA submissions.⁵

Opponents of R2T also cite significant patient safety concerns. Phase I clinical trials typically enroll 10–20 participants with the objective of determining the recommended phase II dose, not to establish efficacy. After only the first phase I study, opponents argue, so few patients have been studied that little, if anything, is known about the efficacy of the investigational agents – with knowledge gained of only the agents’ common adverse effects. Health care providers, including clinical pharmacists caring for patients under R2T, will thus need to make clinical decisions with limited information regarding the efficacy, adverse effects, administration procedures, bioavailability, and drug interactions of these R2T agents. In addition, though not required to pay for R2T treatments, health systems will likely be forced to bear some or all of these costs or risk significant negative publicity if patients cannot pay the costs themselves.

The impact of R2T on clinical care remains unknown. Before passage of the federal statute, R2T legislation had been approved in 40 states with little uptake or impact (positive or negative). However, to comprehensively manage medication regimens, including investigational agents, clinical pharmacists will need to understand the associated risks and benefits of these agents and convey them to their patients, including the fact that the benefits of investigational therapies are unproven and the associated adverse effects may be worse than no treatment at all.

References:

1. Congress.gov. S.204: Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017. Available at https://www.congress.gov/bill/115th-congress/senate-bill/204/text. Accessed July 12, 2018.
2. S. Food and Drug Administration (FDA). Expanded Access Categories for Drugs (Including Biologics). Available at https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/ucm431774.htm. Accessed July 12, 2018.
3. Right to Try. FAQ. Available at http://righttotry.org/faq/. Accessed July 12, 2018.
4. Dyer O. Trump signs bill to give patients right to try drugs. BMJ 2018;361:k2429.
5. Lurie P. Exploring a Right to Try for Terminally Ill Patients. September 2016. Available at https://www.fda.gov/NewsEvents/Testimony/ucm522044.htm. Accessed July 12, 2018.