Right-to-try movement gains momentum at state, federal level

By Kathryn Kundrod
Graduate Intern, Center for Health and Biosciences

The Right-to-Try (RTT) movement toward medical deregulation has seen a legislative push on the state and federal level in the United States. In 2015, Texas passed an RTT law (known as the Andrea Sloan Right to Try Act, named after an advocate who died of ovarian cancer in 2014) to establish RTT for terminally ill patients. The federal RTT (know as the the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act) was signed into law in 2018 to capture the remaining states that did not pass one. In the 86th Texas legislative session (from January to May 2019), a new effort was made to include severe chronically ill patients in the RTT law — H.B. 805. This bill would have expanded access to a large and largely undefined population. It would have caused considerable harm to a vulnerable patient population, hopeful to try investigational treatments without adequate oversight or evidence of efficacy. Though H.B. 805 did not make it to a House floor vote this legislative session, it reflects efforts to further expand RTT in Texas. Policymakers should instead work to improve the existing compassionate investigational new drug use pathway through the U.S. Food and Drug Administration (FDA).

RTT aims to expand access to experimental drugs and devices outside of the Expanded Access Program (EAP) through the FDA. RTT advocates cite bureaucratic holdups in the EAP process, including laborious applications and long approval times. The FDA responded to these frustrations by reducing the requirements so that the application takes approximately 45 minutes to complete. Further, the FDA reduced turnaround times for non-emergency requests to four days and emergency requests to less than one day, on average. Further, the FDA points out that over 99% of applications are approved. Critics of RTT believe that these laws created a second and unnecessary system that lacks adequate oversight, therefore generating confusion and introducing new barriers (e.g. patients needing to get drugs directly from a manufacturer) and increasing the risk of “fraud and abuse” of vulnerable patients. RTT requires investigational drugs to have passed Phase 1 clinical trials in the FDA, leading RTT advocates to claim that RTT drugs are safe. However, 90% of Phase 1 drugs do not make it through Phase 2 and 3 clinical trials due to safety and efficacy concerns. Additionally, investigational drug use under RTT occurs outside of the clinical trial process, reducing public knowledge of the drug’s safety and efficacy, and ultimately setting back the FDA in its mission to monitor investigational drugs for safety and efficacy and to improve public health. Therefore, RTT does not always provide an easier route to access and can be harmful to hopeful patients in vulnerable situations.

In addition, not all drug manufacturers are interested or able to participate in the RTT program. For example, a high-profile RTT advocate and ALS patient, Matthew Belina, gained access to an experimental drug from BrainStorm Cell Therapeutics. He has self-reported improvement in his condition, but the company said the costs are too high to provide any other patients access under RTT. Another prominent advocate for whom the national RTT bill is named, Frank Mongiello, was denied access to the same drug given to Bellina on the basis of cost. In Texas, a Houston physician, Ebrahim Delpassand, treated 144 patients under the FDA EAP program, and later treated 178 patients under the Texas RTT law. Unfortunately, in order to get his patients access to the investigational drug, Delpassand had to cover the costs of treatments personally — since RTT drugs are still experimental and therefore not covered by insurance.

Despite these concerns, State Representative Tan Parker proposed H.B. 805 to expand RTT to severe chronically ill patients. In statements regarding the bill, Rep. Parker claimed that access to investigational drugs remains too difficult for patients with chronic, non-terminal illnesses, and asserts that “patients with a severe chronic disease have a fundamental right to attempt to pursue the preservation of their state of life by accessing available investigational drugs, biological products, and devices.” The designation of “severe chronic disease” was defined in the bill as “a condition, injury, or illness that may be treated, may not be cured or eliminated, and entails significant functional impairment or severe pain.” In addition, H.B. 805 explicitly cited the “tremendous success” of Texas’ RTT law in “saving the lives of many patients with a terminal illness” as rationale for expanding access to severe chronically ill patients. However, due to a lack of oversight and transparency, the total number of people accessing investigational drugs under RTT is unknown.

Ultimately, H.B. 805 did not make it through the 86th Texas legislative session. However, it caused concerns for many patient safety advocates. Expanding RTT to chronically ill patients would have increased the number of patients who are subjected to potential false hope at best, and fraud and dangerous side effects at worst. Collaboratively improving the FDA EAP program with input from patient advocates and pharmaceutical companies to facilitate higher rates of access would lead to safer use of investigational treatments and better public health reporting, and is therefore a better approach than medical deregulation through RTT expansions moving forward.

This entry originally appeared on medium.com on June 3, 2019.