Will right-to-try improve patient access to investigational drugs?
The Trickett Wendler, Frank Mongiello, and Jordan McLinn Right to Try Act of 2017 unanimously passed the Senate on August 3, 2017. The law “authorizes the use of unapproved medical products by
patients diagnosed with a terminal illness in accordance with State law, and for other purposes.” However, opinions are mixed on whether the law will further patient access to drugs under development, even after it passes the House and is approved by the president.
The legislative template for the right-to-try, which is currently approved in 37 states, was developed by the Goldwater Institute, a think tank based out of Arizona. According to the institute’s website, right-to-try measures “would allow terminal patients access to investigational drugs that have completed basic safety testing, thereby dramatically reducing paperwork, wait times and bureaucracy, and, most importantly, potentially saving lives.”
However, if bypassing bureaucracy and wait times within the FDA is the underlying objective, the regulatory body already has an Expanded Access program in place, and, according to a report by the Regulatory Affairs Professional Society, the FDA’s Center for Drug Evaluation and Research approved 99.4% of the 1554 expanded access requests it received.
The stumbling block to patient access to these investigational treatments, according to a Peter Lurie, MD, MPH, associate commissioner for public health strategy and analysis, Office of Policy, Planning, Legislation, and Analysis, FDA, is the high rate at which pharmaceutical companies turn down physician applications for these drugs.
In her opinion piece, Lisa Kearns, senior research associate at the NYU School of Medicine’s Division of Medical Ethics wrote that it’s a myth that the right to try legislation will help patients get experimental drugs faster. Instead, she feels that FDA involvement, via the expanded access program, “can help patients determine their best options, and [the FDA’s] review and ability to require changes to treatment plans help physicians design plans most likely to help, and not hurt, patients.”
Physician organizations, such as the American Society of Clinical Oncology (ASCO), have also voiced their concerns on this issue. In a position statement released earlier this year, ASCO’s chief medical officer Richard L. Schilsky, MD, FACP, FASCO, said, “We don't support right to try legislation, however, because these laws ignore key patient protections without actually improving patient access to investigational drugs outside of clinical trials.”
In her article, Kearns proposes several solutions to overcoming this problem from the drug developer’s perspective, specifically addressing their concerns with adverse events affecting product approval, financial challenges that smaller developers might face, and the need to encourage transparency between the regulatory body and drug developers.