According to a story from Medscape, the House of Representatives recently voted to pass a “right to try” bill. The bill they voted on was previously approved by the Senate last year and will now head to the desk of the President. The stated aim of the bill is to allow patients who are facing life threatening diseases to have easier access to experimental drugs outside of clinical trials. This could have major implications for rare disease patients, who often have limited treatment options.
While this sounds like a win for patients, many former FDA commissioners and other officials, bioethicists, and patient advocacy groups have come out in opposition to the legislation. These groups include the National Organization for Rare Disorders. These groups oppose right to try because it tosses aside regulatory measures that are meant to protect patients from being harmed or killed by untested treatments. The law also undermines the regulatory authority of the FDA.
Support for right to try from organizations such as Americans for Prosperity, which has consistently opposed economic regulation of any kind, suggests that the primary goal of the law is to further deregulate the drug industry via bypassing the FDA. Michael Carome, MD, who directs the Health Research Group at Public Citizen, says that patients could be exposed to drugs that could be entirely ineffective, or even inflict pain or greater suffering on patients that are already desperate for relief.
The main argument from supporters of right to try is that patients are being held back from getting access to the treatments that they need. The reality is that it is already possible for patients to get access to investigational drugs through the FDA’s Expanded Access program. Around 99 percent of applicants to the program ultimately get approval for access to these treatments. In addition, the program incorporates rules that protect patients from potential harm.
