Jerry Avorn wrote the book, “Powerful Medicines: The Benefits, Risks, and Costs of Prescription Drugs,” one of many great books written on this general theme in the past decade or so.
This week, the Harvard prof – Chief of the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital – writes a warning in the Annals of Internal Medicine about FDA deliberations taking place this summer. He warns against easing the restrictions on drug companies marketing their drugs for uses for which they have not been approved – so-called off-label use. To help make his case, he points to the marketing of dietary supplements as an example of the kind of “ludicrous” claims that could be expected if off-label marketing is not restricted.Excerpts:
“During the summer of 2015 the FDA will hold a public meeting to discuss whether its restrictions on what drug manufacturers can claim about their products improperly infringes the liberty of those companies. This revision of current standards is advocated by the pharmaceutical, biotech, and device industries, as well as those with libertarian philosophies who contend that the government should regulate very little of anything. The marketplace, they argue, will sort out which drugs work and are safe. Outright fraudulent claims would still be prohibited (even in this brave new world a drugmaker couldn’t claim its product confers immortality), but short of that, advocates argue that most kinds of “scientific evidence” should be grounds to promote both new and old products, whether or not the FDA agrees.
In my decades of practice as an internist, I’ve taken comfort that drugs on the market were there because their manufacturers had provided the Food and Drug Administration with at least some evidence that they worked, and that their known risks were depicted in the product labeling. I did not need to review on my own all the available evidence about efficacy and safety for each drug I prescribed. Even if I had the time and acumen to do so – and what busy practitioner has hundreds of hours to assess each new medication? – I knew that the FDA had additional thousands of details about these drugs I could never see, because they were the private property of the companies that had paid for the clinical trials. …
The FDA’s power to require efficacy data dates back only to the 1960s; it took the thalidomide tragedy of that era to convince Congress to give the agency new authority to ensure that useless or minimally effective products, or those with unacceptable safety problems, could not come into use. The agency then assessed thousands of drugs then on the market to weed out the many that were ineffective, dangerous, or both. The summer of 2015 could mark the beginning of a step back to the pre-1960s era for prescription drugs.
The proposals under discussion fly in the face of much of what we know about rigorously evaluating clinical interventions. First, they would allow companies to short-circuit the need for a review of the totality of the available data (including those secret proprietary files), and permit them to present clinicians with cherry-picked studies that may look good in isolation but could be methodologically inadequate, or fail to present a balanced picture of a drug’s benefits and risks. A second problem is the unreliability of “clinical experience” measures such as “observational studies, registries, and therapeutic use” as well as guidelines to assess efficacy, as is advocated in pending legislation. Yet all these measures would become fair game for companies seeking to promote their products to us. Non-commercial services, such as those that provide academic detailing, could try to provide some balance for clinicians, but could well be swamped by the drug industry’s unchained promotional leviathan.”
On his Wall Street Journal Pharmalot blog, Ed Silverman provided some background when he wrote, “Drug Maker Sues FDA Over Free Speech Right to Promote Off-Label.”
Scientific American wrote, “FDA to Hold Public Meeting on Off-Label Use of Prescription Drugs,” with more details about the summertime FDA public meeting. The story also explains that this comes “as a bill known as 21st Century Cures, designed to speed new drugs to market, is moving through Congress. Language in the bill is adding pressure on the agency to relax its guidelines.”
Dr. Avorn also recently wrote in the New England Journal of Medicine, “The 21st Century Cures Act — Will It Take Us Back in Time?” The article concludes:
“Over the past 80 years, this country’s regulatory approach has embraced steadily improving criteria for accurately assessing therapeutic efficacy and risk. Patients and physicians would not benefit from legislation that instead of catapulting us into the future, could actually bring back some of the problems we thought we had left behind in the 20th century.”
We’ll have more about 21st Century Cures Act in the future. The following interactive graphic about the legislation was provided by the US House Energy & Commerce committee.