How We Could Save Billions by Finding Which Medical Treatments Don't Work

From the original published here.

A Health and Human Services Initiative to Cut Wasteful Spending and Improve Patient Lives

Challenge and Opportunity

Many common medical practices do not have strong evidence behind them. In 2019, a group of prominent medical researchers—including Robert Califf, the former Food and Drug Administration (FDA) Commissioner—undertook the tedious task of looking into the level of evidence behind 2,930 recommendations in guidelines issued by the American Heart Association and the American College of Cardiology. They asked one simple question: how many recommendations were supported by multiple small randomized trials or at least one large trial? The answer: 8.5%. The rest were supported by only one small trial, by observational evidence, or just by “expert opinion only.”

For infectious diseases, a team of researchers looked at 1,042 recommendations in guidelines issued by the Infectious Diseases Society of America. They found that only 9.3% were supported by strong evidence. For 57% of the recommendations, the quality of evidence was “low” or “very low.” And to make matters worse, more than half of the recommendations considered low in quality of evidence were still issued as “strong” recommendations.

In oncology, a review of 1,023 recommendations from the National Comprehensive Cancer Network found that “…only 6% of the recommendations … are based on high-level evidence”, suggesting “a huge opportunity for research to fill the knowledge gap and further improve the scientific validity of the guidelines.”

Even worse, as shown in a great book co-authored by current FDA official Vinay Prasad, there are many cases where not only is a common medical treatment lacking the evidence to support it, but also one or more randomized trials have shown that the treatment is useless or even harmful!

One of the most notorious examples is that of the anti-arrhythmic drugs given to millions of cardiac patients in the 1980s. Cardiologists at the time had the perfectly logical belief that since arrhythmia (irregular heartbeat) leads to heart attacks and death, drugs that prevented arrhythmia would obviously prevent heart attacks and death. In 1987, the National Institutes of Health (NIH) funded the Cardiac Arrhythmia Suppression Trial (CAST) to test three such drugs.

One of the drugs had to be pulled after just a few weeks, because 17 patients had already died compared with only three in the placebo group. The other two drugs similarly turned out to be harmful, although it took several months to see that patients given those drugs were more than two times as likely to die. According to one JAMA article, “there are estimates that 20,000 to 75,000 lives were lost each year in the 1980s in the United States alone” due to these drugs. The CAST trial is a poignant reminder that doctors can be convinced they are doing the best for their patients, but they can be completely wrong if there is not strong evidence from randomized trials.

As Prasad has shown, there are hundreds of examples where medical practice was ultimately proven wrong. Given how few medical practices, even now, are actually supported by strong evidence, there are likely many more examples of treatments that either do not work or actively cause harm. This is not only wasted spending, but also puts patients at risk.

We can do better – both for patients and for the federal budget – if we reduce the use of medical practices that simply do not work.

Plan of Action

The Secretary of Health and Human Services should create a cross-division committee to develop an extensive and prioritized list of medical practices, products, and treatments that need evidence of effectiveness, and then roll out an ambitious agenda to run randomized clinical trials for the highest-impact medical issues.

That is, the CMS needs to work with the NIH, the FDA, and the Centers for Disease Control and Prevention (CDC), to develop a prioritized list of medical treatments, procedures, drugs, and devices with little evidence behind them and for which annual spending is large and the health impacts could be most harmful. Simultaneously, the FDA needs to work with its partner agencies to identify drugs, vaccines, and devices with widespread medical usage that need rigorous post-market evaluation. This includes drugs with off-label uses, oncology regimens that have never been tested against each other, surrogate outcomes that have not been validated against long-term outcomes, accelerated approvals without the needed follow-up studies, and more.

With priority lists available, the NIH could immediately launch trials to evaluate the effectiveness of those treatments and practices to ensure effective health and safety. The Department should report to Congress on a yearly basis as to the number and nature of clinical trials in progress, and eventually the results of those trials (which should also be made available on a public dashboard, with any resulting savings). The project should be ongoing for the indefinite future, and over time, HHS should explore ways to have artificial intelligence tools identify the key unstudied medical questions that deserve a high-value clinical trial.

The potential here? In 2016, randomized trials of back fusion surgery found that it does not work. But a recent analysis by the Lown Institute found that the Centers for Medicare & Medicaid Services (CMS) spent approximately $2 billion in the past 3 years on more than 200,000 of these surgeries. Imagine what we could learn from a broader systematic effort as to all medical treatments.

Expected opponents to any such effort will be pharmaceutical, biotechnology and device companies and their affiliated trade associations, whose products might come under further scrutiny, and professional medical associations who are firmly convinced that their practices should not be questioned. Their lobbying power might be considerable, but the intellectual case behind the need for rigorous and unbiased studies is unquestionable, particularly when billions of federal dollars and millions of patients’ lives and health are at stake.

Conclusion

Far too many medical practices and treatments have not been subjected to rigorous randomized trials, and the divisions of Health and Human Services should come together to fix this problem. Doing so will likely lead to billions of dollars in savings and huge improvements to patient health.

This memo produced as part of the Federation of American Scientists and Good Science Project sprint. Find more ideas at Good Science Project x FAS