It’s called “comparative effectiveness research” or CER. The goal of CER is to quantify, as best as possible, the risks and rewards of various medical treatments for the “average” patient. Is a new drug or breakthrough medical procedure worth the cost to patients, insurance companies or the government? Under the health reform law, a new Patient-Centered Outcomes Research Institute would generate these findings and then share them with the public, including the FDA and health insurers.
The primary argument of CER supporters is that this research will better inform doctors and patients about what works and is cost-effective for the majority of patients. And, indeed, such research can prove very helpful to patients who want to make more informed decisions about their treatment options.
But this research can all too easily have dangerous consequences, if it’s used to dictate what kind of care patients can and can’t have access to. And even if the intention isn’t to influence the FDA in its drug approval process, or Medicare, Medicaid and private insurers as to what they will cover, the temptation will be there to do so.
The main problem with using CER to make coverage or approval decisions is that in the real world there’s no such thing as an average patient. Each responds differently to different treatments. And doctors need access to as many treatment options as possible to keep them healthy. If CER increasingly cuts off access to treatment options deemed un-economical for the “average” patient, the real-world kind will be hurt.
This isn’t fear mongering, it’s reality. In fact, we’ve had a foretaste of life under CER when the FDA decided to revoke its approval for the use of the drug Avastin as a stage IV breast cancer treatment.
The primary reason given by the FDA is that Avastin only prolonged these women’s lives by a few months, and at a high cost. The likely effect of this decision, however, will be to deny 17,500 women the chance to spend more time with their families, friends and children, as insurance companies follow the FDA’s lead and drop coverage.
Never mind the fact that thousands of women on Avastin have had far better than “average” results. Or that, as a result of losing its FDA approval, Avastin probably will only be available to those women able to afford the drug’s $90,000 a year price tag. All because Avastin’s data on paper didn’t make the FDA’s arbitrary cut.
This obsession with numbers has other consequences, too. CER will compound the cost and time required to bring a new drug to market.
Research shows that over the course of the decade, CER will require an extra $32 billion in R&D and result in fewer innovative drugs making it out of the lab and into your local pharmacy. And a recent study found that the use of CER in Europe delayed drug approvals there by an average of two years, compared with the U.S.[1]
And what about drugs developed for the treatment of rare diseases? These “orphan drugs” target maladies that affect a small percentage of people. They’re expensive to produce and typically don’t get as much funding or have as much priority as other, more common diseases. Under a CER system that focuses solely on the averages and medians of clinical trials, treatments for these patients — two thirds of whom are children — could easily get lost in the shuffle.
At least as far as orphan drugs go, Sen. Robert Casey (D-Penn.) has introduced legislation last March to support this research. The Creating Hope Act of 2011, will provide incentives “to develop treatments for rare diseases that are often less profitable than treatments for more common medical conditions.”
No matter how advanced our knowledge of the human body, there will always be some degree of unpredictability in medical science, making one-size-fits-all treatment impossible.
And to the vast majority of Americans, one-size-fits-most treatment isn’t much better.
Dr. Robert Goldberg is co-founder and vice president of the Center for Medicine in the Public Interest. He is also author of the book “Tabloid Medicine: How the Internet is Being Used To Hijack Medical Science For Fear and Profit.”
Note: [1] Fewer Drugs, Shorter Lives, Less Prosperity: The Impact of Comparative Effectiveness Research on Health and Health