President Donald Trump has signed a controversial “right-to-try” bill that will give terminally ill patients the chance to try drug treatment programmes that are still in clinical trials, but have passed the Food and Drug Administration’s (FDA) Phase 1 testing.
Supporters of the bill, which essentially expands right-to-try laws that already exist in 38 states, argue that the law will open up the possibility for people with terminal illnesses to seek potentially life-saving treatments that would not normally be available to patients.
But, not everyone is on board with the legislation, which Mr Trump signed Wednesday: Opponents say the legislation will leave fragile patients potentially more vulnerable, while undercutting the FDA testing system.
Here’s what you need to know.
Who can pursue these experimental treatments?
The expressed goal of this bill and others like it is to give patients who have exhausted all other FDA-approved treatment options the chance to try some other, potentially life saving, drug. These types of patients are typically too sick to enrol in a clinical trial, so getting the treatment through other options is necessary.
Extremely sick patients in 38 countries already have this “right”, which allows them to work with their doctors to approach drug companies and ask for the option to try the drug outside of the clinical trials set up to comply with the FDA.
Still, detractors of the federal law say that it won’t change much, and will adversely impact FDA safeguards for public health.
Were very sick patients unable to get experimental drugs in other states before?
Steven Joffe, a professor in medical ethics and health policy at the University of Pennsylvania’s Perelman School of Medicine, expressed those concerns above in a paper published in the New England Journal of Medicine last month, and argued in the paper that the FDA has systems to expand access for very sick patients, which are known as expanded-access or compassionate use programmes.
“Emergency requests for individual patients are usually granted immediately over the phone and non-emergency requests are generally processed within a few days,” Dr Scott Gottlieb, the FDA commissioner, said last year.
A study by FDA researchers concluded that expanded access requests for 9,000 experimental drugs were approved 99 per cent of the time between 2005 and 2014.
What do supporters say?
Supporters say that the new bill gets rid of the application requirements of the FDA’s expanded access program, and that doing so will save patients time.
And the opponents?
Mr Joffe told CNN that there often is not much public data on these treatments, so it can be difficult for outside doctors to know things like how much of the drug to give, or what side effects the drugs might have.
He also argued that cutting the FDA out of the picture could put patients at risk, and that “shady actors” could pop up and start offering experimental therapies to at-risk patients.
“We've already seen some of that in the stem cell clinics popping up around the country that try to provide stem cell treatments for a host of problems: eye problems, heart problems,” he told that cable network. “While there are legitimate efforts to develop effective stem cell therapies, a lot of these clinics that are popping up around the country that are poorly regulated are taking people's money and they are hurting people.”
On the other hand, supports have noted that the treatments will be in some phase of testing with the FDA, and that nobody is going to be forced to take experimental drugs.
