Intended for healthcare professionals


Trump signs bill to give patients right to try drugs

BMJ 2018; 361 doi: (Published 31 May 2018) Cite this as: BMJ 2018;361:k2429
  1. Owen Dyer
  1. Montreal

The US Congress has passed a controversial bill that effectively ends the Food and Drug Administration’s control over access to unapproved drugs sought by patients who believe that they have run out of other options.1

President Donald Trump signed the bill on 30 May. A few days earlier he said that it “gives people hope . . . They won’t have to fly to Africa, they won’t have to fly to South America, they won’t have to fly to Europe.”

Trump misrepresented the current situation, saying that though many “amazing drugs” were awaiting FDA approval “these people had no access, they had no access at all to getting into that pipeline.” In fact the FDA approves over 99% of expanded access requests for experimental drugs and has been doing so for at least 10 years.2

The real barrier to early access has been the drug manufacturers themselves, which are unaffected by the new law. They must agree to the use of their experimental drug before an FDA application can be filed. Appeals for expanded or compassionate use are dealt with by company employees, who often lack a process to ensure fairness.

Companies worry about perceptions that they are giving early access to rich people, given that neither public nor private insurers will cover such use and recipients must have the financial resources to cope with adverse outcomes. They also fear that any adverse events or outcomes arising from compassionate use could slow approval.

No overall figures are available, but Janssen Pharmaceuticals, which sought advice from New York University’s medical ethics department in devising a method to review requests for early access to its multiple myeloma treatment daratumumab, ultimately reported denying 98 of 160 requests.3

The new law, long sought by the Koch Family Foundations and the libertarian Goldwater Institute, does nothing to force companies to release more of their experimental drugs. But it does remove the process from FDA oversight, which experts have said could render drug makers even more skittish about supplying their unapproved products.

In an attempt to allay corporate fears, the law prevents the FDA from taking adverse events or outcomes during early access into account in its approval process, unless they are “critical to determining safety.”

Seventy five patients’ groups including the American Cancer Society, the American Lung Association, the National Organisation for Rare Disorders, and the Cystic Fibrosis Foundation, signed a letter to Congress urging them to reject the House’s version of a right to try bill, while conceding that it was better than the Senate’s version. It is the Senate’s version that has now passed into law.

Ellen Sigal, chair of Friends of Cancer Research, said, “This bill, at minimum, would create false hope for dying patients. At its worst, it would cause serious harm to the most vulnerable.”

Any drug that has passed phase I trials is eligible for early access under the new law, but the great majority of such drugs never obtain approval, especially in the area of cancer treatment.

Four former FDA commissioners, two appointed by Barack Obama and two by George W Bush, also wrote to Congress to urge the scrapping of the bill. “There is no evidence that either [House or Senate] bill would meaningfully improve access for patients, but both would remove the FDA from the process and create a dangerous precedent that would erode protections for vulnerable patients,” they wrote.

The current FDA commissioner, Scott Gottlieb, who himself has had cancer, also opposed right to try legislation before it passed Congress. “The vast majority of people who will use a drug through expanded access are using a drug that doesn’t work,” he told a House committee in October.

The Senate’s bill, by broadening the definition of eligible patients from those with “‘terminal disease” to all those with “life threatening illness,” risked “undermining the central purpose of this legislation,” said Gottlieb then.

But Gottlieb seemed resigned to the new reality this week, tweeting, “I stand ready to implement it in a way that achieves Congress’s intent to promote access and protect patients.”


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