On ‘Right to Try,’ the FDA Should Proceed With Caution More access to unapproved drugs could be good policy, but there are risks even to terminal patients. By Henry I. Miller See note please
https://www.wsj.com/articles/on-right-to-try-the-fda-should-proceed-with-caution-1489445804
Again, unless there is real tort reform the FDA will have a permanent problem, and so will the pharmaceutical companies…..rsk
The Food and Drug Administration is the nation’s most ubiquitous regulatory agency, overseeing everything from syringes and CT scanners to drugs, vaccines and most foods. These products account for more than $1 trillion annually, or about a quarter of U.S. consumer spending. This slow, dysfunctional agency needs drastic reform of its requirements, procedures and attitudes.
One reform Scott Gottlieb, President Trump’s nominee to lead the agency, will likely embrace is “right to try”—that is, giving terminally ill patients access to unapproved medicines. He could remove the FDA from judgments about “compassionate use” of unapproved drugs. There is already a trend in this direction: Thirty-three states have passed laws aimed at providing easier access to experimental treatments that are still in the earliest stages of human testing.
The right to try unapproved drugs has the potential to be compassionate and sound public policy—but there are dangers. The concept must be implemented in a way that takes into consideration the realities of drug testing.
According to the libertarian Goldwater Institute, right-to-try legislation would allow “terminally ill Americans to try medicines that have passed Phase I of the FDA approval process and remain in clinical trials but are not yet on pharmacy shelves.” It would also expand usage of “potentially life-saving treatments years before patients would normally be able to access them.”
But here’s the rub: About three-quarters of drugs that pass Phase I will never be accessible. They ultimately won’t be approved, because of either safety concerns or lack of efficacy. Most legislative proposals, including the one recently introduced by Sen. Ron Johnson (R., Wis.), would enable patients to request the drugs after only the most meager safety testing.
Phase I testing, often the first time a new drug has been administered to humans, provides extremely limited information. These trials are performed on between 20 and 100 patients and last only a short time. They’re usually administered to paid, healthy volunteers, who may not provide a good representation of how the drug will affect terminal patients. Such trials essentially exist to determine what doses of the drug are tolerated without causing gross safety problems such as seizures, organ failure or death.
The determination of efficacy starts in Phase II, when the drug is administered to volunteers who suffer from the disease or symptom for which the drug is intended. If the results of Phase II are promising, the drug moves into still larger Phase III trials—the most extensive and expensive part of drug development.
A physician at a large health insurer, who spoke to me on the condition of anonymity, recently raised concerns about right to try. He wonders “where liability will ultimately lie when and if something goes wrong.” Even trickier: “Who is the deep pocket if and when the treatment fails and the patient’s family is looking for someone to blame?” He warns that the right to try could become an “unfunded mandate” and raises questions about who will pay for the drugs and how their prices will be determined. Medical insurance as we know it was never designed or intended to cover unproven treatments of last resort. CONTINUE AT SITE
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