Tuesday , February 13, 2018 - 4:30 AM1 comment
The idea that gravely ill patients should have access to experimental medicines is morally unassailable. A bill before Congress to establish a national "right to try," however, is the wrong way of going about it.
Passed by unanimous consent in the Senate last fall and endorsed with gusto by President Donald Trump in his State of the Union speech, the legislation would allow Americans to use yet-to-be-approved drugs that have passed only preliminary safety tests — without permission from the Food and Drug Administration. The bill is similar to right-to-try laws that have been passed by 38 states.
Though grounded in compassion, the bill would do little to actually improve the prospects of terminally ill patients — and may put some of them at greater risk. For one, its underlying assumption — that the FDA gets in the way — is plain wrong. The FDA already allows "compassionate use" of medicines still in development, approving almost every one of the applications it receives within days, if not hours. Any delays patients face usually come from the drugs' developers.
That stands to reason: Pharmaceutical companies have preciously small supplies of drugs that are still in clinical trials, and they don't want to take a chance on such drugs' causing painful, or even fatal, side effects in patients who may be too ill to be part of a trial. It's no wonder the pharmaceutical industry is not keen on bypassing the FDA: Taking the agency's expertise out of the picture increases the risk that patients may be harmed, and ensures that regulators learn nothing from the process.
And even as it cuts out the FDA, the law cannot require drug companies to dispense unapproved drugs. So it's left offering patients nothing beyond false hope.
Fortunately, discussions to improve the legislation in the House are underway. A smarter law would keep the FDA involved, and give the agency the added budget and personnel it needs to address compassionate-use applications and gather data on all such use. It would limit the program to people at risk of dying in a matter of months. It would help provide information to patients and doctors about drugs in development. And it would offer incentives for drug makers — for example, help from the FDA to make sure that granting a patient's request doesn't slow a company's drug-development timeline, or tax credits for supplying experimental medicines without charge.
Americans deserve to have access to powerful medicines, but also protection from any harm those drugs might do. Congress should not authorize an end run around the FDA that would complicate its vital mission to ensure safe access to effective drugs.
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