It has also led to a plethora of high-priced drugs to treat diseases like cancers, multiple sclerosis and Type 2 diabetes that are all more effective than a placebo but have often not been tested very much against one another to determine which are most effective.
In today’s complex world, clarification is needed to determine just what kind of effectiveness the FDA should demand. And should that be the job of the FDA alone?
For example, should drugmakers prove a drug is significantly more effective than products already on the market? Or demonstrate cost-effectiveness — the health value of a product relative to its price — a metric used by Britain’s health system? And in which cases is effectiveness against a surrogate marker — like an antibody level — a good enough stand-in for whether a drug will have a significant impact on a patient’s health?
In most industrialized countries, broad access to the national market is a two-step process, said Aaron Kesselheim, a professor of medicine at Harvard Medical School who studies drug development, marketing and law and recently served on an FDA advisory committee. The first part certifies that a drug is sufficiently safe and effective. That is immediately followed by an independent health technology assessment to see where it fits in the treatment armamentarium, including, in some countries, whether it is useful enough to be sold at all at the stated price. But there’s no such automatic process in the U.S.
When Pfizer applies for authorization, the FDA may well clear a booster for the U.S. market. The Centers for Disease Control and Prevention, likely with advice from National Institutes of Health experts, will then have to decide whether to recommend it and for whom. This judgment call usually determines whether insurers will cover it. Pfizer is likely to profit handsomely from a government authorization, and the company will gain some revenue even if only the worried well, who can pay out-of-pocket, decide to get the shot.
To make any recommendation on a booster, government experts say they need more data. They could, for example, as Dr. Anthony Fauci has suggested, eventually green-light the additional vaccine shot only for a small group of patients at high risk for a deadly infection, such as the very old or transplant recipients who take immunosuppressant drugs, as some other countries have done.
But until the United States refines the FDA’s “safe and effective” standard or adds a second layer of vetting, when new products hit the market and manufacturers promote them, Americans will be left to decipher whose version of effective and necessary matters to them.
(KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.)©2021 Kaiser Health News. Visit khn.org. Distributed by Tribune Content Agency, LLC.