President Donald Trump has signed into law the “Right to Try Act”, a measure aimed at helping terminally ill patients by giving them expanded access to drug treatments not yet fully approved by the US Food and Drug Administration (FDA). Let’s break down what that means.
After a drug is developed, researchers begin pre-clinical testing of its safety and effectiveness on animals. This information is then passed along to the FDA in an application, which includes the drug’s composition, manufacturing, and plans for testing the drug on humans.
The drug is then moved through three phases at the agency level: a safety review with 20-80 people, followed by an efficacy review with several hundred. The last phase studies both the drug’s safety and efficacy in thousands of people by analyzing its effect on different populations, as well as varying dosages and potential conflicts when used in combination with other drugs.
Under the Right to Try law, patients are given the right to access drug treatments that have passed the first phase and are still in clinical trials. By given the right, Trump says “hundreds of thousands” could be saved.
"With the passage of this bill, Americans will be able to seek cures," he said in the White House surrounded by patients and families affected by the signing of the bill.
Supporters of the bill agree. It provides an alternative treatment to terminally ill patients who have otherwise exhausted all other treatment options and are too ill to be in the clinical trials themselves. By bypassing FDA permission, proponents further say it could give these patients more time and, in essence, provide for further information to be gathered about how treatments affect humans.
Others say it deregulates the FDA process and the agency’s ability to safeguard public health. Opponents further argue that putting a patient on a drug without knowing enough information about its administration, side effects, or how it affects a patient’s already fragile health could do more harm than good.
A paper published last month in the New England Journal of Medicine notes the FDA has expanded compassionate use policies in the use of drugs outside of clinical trial to give terminally ill patients access to investigational medicines. As it stands, states that have right-to-try laws allow patients to work with their doctors in approaching companies with drugs currently in clinical trials to apply for its use outside of current testing.
Over a 10-year period, around 99 percent of applications to access nearly 9,000 drugs were approved between January 2005 and December 2014, according to a 2016 study published in Therapeutic Innovation & Regulatory Science.
The bill expands on that access by not only introducing legislation across state borders but by eliminating the application for access. In the past, the FDA would give recommendations about how to use the drug and what side effects to look for – information that will now be readily available directly to patients.