Should dying patients have the right to try unapproved drugs without any oversight by the FDA? Thirty-eight states have “right-to-try” laws. But federal law trumps state law so patients still need FDA approval in order to get access to these unapproved drugs.
In his State of the Union address, President Trump called on Congress to pass “right-to-try” legislation. Vice-President Mike Pence endorsed the legislation. The Senate passed it unanimously. The Senate bill has three key provisions. Then, in March, the House of Representative failed to pass the bill. So what are the objections?
The bill’s critics raise three main concerns, one based on the possibility of harm to each patient and the other two more concerned with the potential implications of right-to-try policies on drug development and health disparities. First, there is concern that access to unapproved products may harm patients more than it helps them. That is because there is an extremely low probability of benefit for each new drug, but a high possibility of toxic side effects that may hasten death or lead to a worsened quality of life for frail patients or those with advanced disease. Second, such a law may undermine clinical trials and delay the generation of data to evaluate the safety and efficacy of new drugs because patients may choose to get access to the drugs outside of clinical trials rather than taking the risk of being randomized to standard care or placebo. Third, some policy makers and bioethicists worry that such laws will widen health disparities and make new drugs available only to those who can afford to pay for them out-of-pocket.
In a broader sense, critics are worried that the law will undermine the authority of the FDA and that, if the principle is established that patients can get drugs that have not been carefully evaluated for safety and efficacy, then the public will be worse off. After all, the FDA was created with the goal of protecting the public from inadequately studied drugs.
The current process for obtaining FDA approval for compassionate use is not particularly onerous. The FDA says its current application takes about 45 minutes to complete. The agency approves more than 99 percent of compassionate-use requests, usually within four days.
The American Medical Association said it “does not believe that the bill will substantially improve patient access to investigational therapies.” The American Society of Clinical Oncology, said the bill “could do more harm than good for patients with life-threatening illnesses” because it would remove the Food and Drug Administration from the evaluation of the risks and potential benefits of some treatments. Many patient advocacy groups also oppose the legislation.
One thing is clear. Such laws are not going away. House leaders vowed to try again to get the laws passed. If you have an opinion, write to your legislator.
