Revisiting "Right to Try" Legislation
In summer 2016, Pharmacy Times® published an article titled “‘Right to Try’ Legislation: A Developing Legal Issue Related to Medications” that discussed the basics of a popular health policy issue.
In summer 2016, Pharmacy Times® published an article titled “‘Right to Try’ Legislation: A Developing Legal Issue Related to Medications” that discussed the basics of a popular health policy issue.1
The article explored the nuances of different state legislation, as well as the ethical questions surrounding the laws. Since then, the number of states that have approved different right-to-try statutes has increased from 27 to 40, and Congress has enacted a similar federal bill.2,3 President Donald J. Trump signed the bill into law and, at the same time, altered the way terminal illness is treated in America. This discussion presents an update on this now federal issue and seeks to look at where FDA oversight ends and medical ethics begin.
Sen Ron Johnson (R-WI) introduced the Right to Try Act of 2017 in January 2017, after advocates ranging from the Goldwater Institute to Trump called for increased access to treatment options for terminally ill patients.4 This type of statute has become increasingly popular in state legislatures recently, with Colorado being the first state to enact such laws, in 2014.5 Multiple attempts to pass legislation have been made at the federal level, and until 2018, all had failed. The Right to Try Act of 2017 passed the Senate with unanimous consent and, after briefly being voted down in the House because of procedural issues, was approved by a vote of 250-169.3 It is the unfortunate reality that many bills in Washington have taken on a hyperpartisan identity in recent years, and this legislation was no exception.
Those in opposition to the bill say that it will not produce any benefit for patients because the stumbling block to access is unwillingness of the manufacturer to comply with requests, an issue that the bill fails to address. Indeed, in testimony before Congress in October 2017, FDA Commissioner Scott Gottlieb, MD, argued that very point.6 A little more than a year earlier, FDA Associate Commissioner for Public Health Strategy and Analysis Peter Laurie, MD, said that some companies had turned down “hundreds” of applications per year.7
The FDA receives more than 1000 requests per year from terminally ill patients for expanded access to unapproved medications and treatments, and the agency approves more than 99% of those requests.8
However, the FDA receives these applications only after both the manufacturer and the physician agree on enrolling the patient.9
Gottlieb’s less-than-ringing endorsement of the bill also provided recommendations to Congress for amendment language so that the patients in focus were those who had “a terminal illness” rather than a “life-threatening disease or condition.”6 The rationale behind this recommendation was that many chronic diseases are life-threatening but can be managed with behavioral and medical interventions that would not necessitate use of unapproved therapies, Gottlieb said.6
This FDA recommendation was not adopted in the final version enacted.
However, the ethical discussion of whether to allow patients with a life-threatening condition or disease greater access to a potentially lifesaving medication seems to be rather 1-sided. The more interesting discussion might be on the long-term effects of cutting FDA oversight out of the drug product approval process. The FDA provides the framework for compassionate-use protocols and acts as an advocate for patients who are vulnerable to exploitation by companies advertising false hope. Removing oversight, some argue, not only opens up patients to being taken advantage of but also removes a valuable source of information for the treating physician. When seeking to make an informed decision about the use of an investigational drug, a provider might encounter difficulty in searching primary literature because much of the information on the drug is still proprietary, being compiled for use in a new drug application submission. The FDA can make recommendations, such as administration and dosing, to providers while protecting the sensitive information from developers of pharmaceuticals. Such actions have been taken on 11% of expanded access requests.10
In conclusion, expanded access statistics show that the Right to Try Act of 2017 may have been passed more as a formality in response to the expansion of similar legislation being adopted at the state level. It may satisfy the political outcry inspired by anecdotal pathos and rapid state-level legislative action, but the evidence for its long-standing impact and necessity remains unclear.11,12
Eric M. Marr is a PharmD candidate at the College of Pharmacy and an MBA candidate at the Gatton College of Business and Economics, both at the University of Kentucky in Lexington.Joseph L. Fink III, BSPharm, JD, DSc (Hon), FAPhA, is a professor of pharmacy law and policy and the Kentucky Pharmacists Association Endowed Professorof Leadership at the University of Kentucky College of Pharmacy.