In a Washington Post article, Advisers blast FDA over Alzheimer’s drug, Laurie McGinley reports that Biogen’s promising experimental Alzheimer's drug has run into strong opposition from an FDA advisory committee.
An experimental drug designed to slow cognitive decline in those with Alzheimer’s disease ran into a buzz saw of skepticism Friday as government advisers said there was not enough evidence to show that the drug was effective. The reaction raised questions about whether the Food and Drug Administration would approve the drug, which would be first new Alzheimer’s treatment in almost two decades. [sic]
But the advisory committee ran into its own buzz saw:
The advisory committee, whose members repeatedly criticized the FDA for what they said was an overly positive view of the drug, met in an all-day session that included emotional testimony from desperate patients and families who pleaded for approval of the medication.
One family member, Jim Taylor, said his wife, Geri, showed “extremely slow” progression of the disease while she was in a clinical trial for the drug, called aducanumab, for four years. But when the trial was halted, he said, her cognitive decline rapidly accelerated.
But the committee stood firm:
Nearly all the members of the advisory panel, however, while expressing sympathy for Alzheimer’s patients and their families, disputed, sometimes angrily, an assessment by the FDA review division that the effectiveness data for the drug was “extraordinarily persuasive.”
FDA skeptics included Scott Emerson, a biostatistician from the University of Washington and others, who “cast doubt on [the drug’s] efficacy.” These skeptics want to keep potentially helpful medicines from patients who willingly want to use the drug. Keep in mind, patients will be denied not because a drug has proven unsafe, but because it is deemed to not be effective.
But efficacy and safety are two very different things. That is why:
The drug has strong support from many Alzheimer’s advocates. Joanne Pike, the chief strategy officer for the Alzheimer’s Association, said at the meeting that requiring another late-stage trial would be a mistake, because four more years would go by before the drug was potentially available.
This controversy shows why "Right-to-Try" laws, while a good start, should be expanded to cover all experimental drugs. Efficaciousness should be eliminated as a criteria for FDA approval. Safety should be the only criteria, as it was before the 1960s when efficacy was added to FDA standards. No patient should ever be denied medicine simply because government bureaucrats and "expert" statisticians say it doesn't "work" good enough.
Experts can continue to monitor for drugs’ efficaciousness (as well as safety) while in wide use. Who really knows, based on small trials, how well the drug really works? Maybe they work better, maybe worse. For example
Using these methods on clinical trial data help us gauge the effectiveness of medicines, but they don’t take into account the differences among the patients taking them. Through genetic variation, human bodies vary considerably in the way they interact with drugs, potentially making them more effective, less effective, or something else entirely.
Why should couples like Jim and Geri Taylor be denied their right to use the drug (subject to the drugmaker’s agreement, of course)? The answer is, they shouldn’t. In collaboration with their doctors, they should decide for themselves based on known facts and new facts that come along with usage. It is their moral right, and should be a legally recognized right. It is, in fact, every person’s right.
41 states and the Federal Government now have Right-to-Try laws on the books. These laws are very restrictive. The angst of these Alzheimer's patients shows that Right-to-Try should be expanded to more drugs and more patients.
Related Reading:
The Right to Try, to Hope, to Live by Christina Sandefur for TOS
The Cure in the Code: How 20th Century Law is Undermining 21st Century Medicine by Peter W Huber
Huber on the Personalized Medicine Revolution—and the Government Roadblocks
Close the FDA's "Loopholes" of Statism, not Freedom
Pharmaphobia: How the Conflict of Interest Myth Undermines American Medical Innovation by Thomas P. Stossel
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