CONGRESS gave its final approval on Tuesday to a bill that would allow patients with serious illnesses to try unproven experimental treatments, sending the measure to President Donald Trump, a strong supporter of the legislation.
By a vote of 250-169, the House passed the “Right To Try” bill that was unanimously approved last August in the Senate. The bill would, in effect, allow dying patients to bypass the Food & Drug Administration (FDA) and obtain an “investigational drug” with the approval of their doctors, if the drug manufacturer agrees to supply it.
On at least three occasions in the past four months, Mr Trump has urged Congress to pass the bill. Patients with terminal conditions, and terminal illness, should have access to experimental treatment immediately and should not have to go from country to country to seek a cure, he said in January.
In a Twitter post on Tuesday, FDA commissioner Scott Gottlieb said: “When the House passes the #RightToTry legislation, I stand ready to implement it in a way that achieves Congress’ intent to promote access and protect patients.”
The chief sponsor of the bill, Republican senator Ron Johnson said it “expands the scope of individual liberty” and provides “hope for terminally ill patients who have nowhere else to turn”.
Critics said the bill is not as innocuous as it sounds. It was opposed by many Democrats and by dozens of organisations that represent patients, including the National Organisation for Rare Disorders and the lobbying arm of the American Cancer Society.
The right-to-try bill “could greatly increase the likelihood of our patients being harmed by unsafe and ineffective experimental therapies,” the health groups said in a letter to House leaders. Friends of Cancer Research chairman Ellen V. Sigal said: “This bill endangers patients and will offer them no value, just false hope.”
The main lobbies for the drug industry, the Pharmaceutical Research and Manufacturers of America and the Biotechnology Innovation Organization, said they have not taken a position on the bill.
Under the bill, a patient is eligible to receive an unapproved drug if he or she has “a life-threatening disease or condition” and has “exhausted approved treatment options and is unable to participate in a clinical trial” of the drug. Sponsors of the bill said it could help people with cancer, muscular dystrophy and Lou Gehrig’s disease, among other conditions.
Patients will have to provide informed consent in writing before obtaining an unapproved drug under the bill. Manufacturers will have to provide the government with an annual summary of use of the drug, including “any known serious adverse events”.
Nothing in the bill requires pharmaceutical companies to provide experimental drugs to patients who request them. Drug-makers sometimes turn down requests because they have only a limited supply or because they are concerned about legal and medical risks.
To address such concerns, the legislation would shield drug companies, doctors and hospitals from some of the legal risks of providing unapproved drugs to patients. Doctors and hospitals would generally be protected unless they engaged in “reckless or willful misconduct” or “gross negligence”.
The House passed a similar bill with more protections for patients in March, but the Senate never took it up. So the House decided on Tuesday to pass the Senate bill.
Several former commissioners of the FDA and some clinical experts at the agency said the bill was unnecessary because patients with a serious illness could already obtain experimental medicines through a programme known as “expanded access or compassionate use”.
The agency says it approves 99 per cent of the requests it receives under this programme, but in some cases, it recommends changes to protect patients before allowing the treatment to proceed.
Representative Frank Pallone Jr said reputable drug companies would not provide drugs to patients without the blessing of the FDA, but the bill opens the door for fly-by-night outfits to promote the equivalent of snake oil.
He added that the bill’s definition of “life-threatening disease” could be stretched to allow access to experimental drugs for people with manageable conditions, like severe diabetes or chronic heart failure.
Scientists must complete the first phase of clinical research on a drug before it can be made available under the bill. Phase 1 trials typically assess the safety and toxicity of a drug in a small number of volunteers.
The drive for right-to-try legislation was spearheaded by the Goldwater Institute, a public policy organisation that advocates free markets and limited government. Its spokesman Starlee Coleman said 40 states had adopted right-to-try laws in the past four years. “‘Right To Try’ is the opposite of ‘Right To Die’. It is for people who are not ready to stop fighting.”