Evidence-based Medicine: Losing the Patient’s Voice?
Although it was some time ago, I remember sitting in on a number of addiction medicine boards and committees when the concept of “evidenced-based treatment” came to the forefront. It was very exciting because this concept offered the prospect of great progress and comfort. All of a sudden we could treat addiction, which is fundamentally incurable, and use a simple outcome measure, such as negative urine or breath tests, absence of emergency room (ER) visits, or the attainment of a new job, as “evidence” that our treatment has merit. For years, the concept of simple outcome evidence for each patient worked as every specialty picked its own guideposts and bettered patient care.
Pain treatment clinicians started using a reduction in pain score, a lack of ER visits, and a decrease in bed-bound days as evidence of effective treatment. Currently, however, there is an increasing trend toward claiming that the only evidence of effective treatment is that derived from a double-blind, randomized controlled study. While such studies have great merit, the danger arises when the value of individual patient response regarding the efficacy of a treatment is dismissed.
Although a little tangential to our pursuit, the current bevacizumab (Avastin, Genentech/Roche) controversy is instructive. In December 2010, the FDA recommended removing the breast cancer indication from bevacizumab’s label because the drug has not been shown to be safe and effective for that use. The agency made its recommendation after reviewing the results of four clinical studies of bevacizumab in women with breast cancer and determining that “the data indicate that the drug does not prolong overall survival in breast cancer patients or provide a sufficient benefit in slowing disease progression to outweigh the significant risk to patients.”1 This action followed an independent advisory committee review, composed primarily of oncologists, which voted 12 to 1 to remove the breast cancer indication from bevacizumab’s label.
By contrast, the National Comprehensive Cancer Network (NCCN), a consortium of 21 leading oncology programs, affirmed its existing recommendation of bevacizumab in combination with paclitaxel (Taxol, Bristol-Myers Squibb) as an appropriate therapeutic option for metastatic breast cancer “with the evidence designation 2A, meaning that it is based on lower-level evidence and uniform agreement of the panel as to its appropriateness.”2 The group based its recommendation on randomized clinical trials, which “document that the addition of bevacizumab to some first- or second-line chemotherapy agents modestly improves time to progression and response rates but does not improve overall survival.”
Included in the NCCN are practicing oncologists from such prestigious institutions as Dana-Farber/Brigham and Women’s Cancer Center in Boston, the University of Texas MD Anderson Cancer Center in Houston, and Memorial Sloan-Kettering Cancer Center in New York City. The vote of this group was 24 in favor, 1 abstention, and none opposed.
Here’s the problem. How do two respected organizations come to such divergent recommendations? First it must be said that any double-blind, randomized trial has the intrinsic problem that a subgroup of patients in a trial may really benefit, but be declared “insignificant” if the statistical end point isn’t reached. Let’s say that 30% of patients have their lives saved by the treatment but 70% get no effect. The statistical evidence will label the 30% as irrelevant placebo responders and reject the treatment as unworthy and recommend it not be used. Unfortunately, this blinkered adherence to statistics is starting to deprive suffering pain patients of needed treatments. We even have a call for placebo-controlled pain studies. Yes, it’s true. Some researchers want to give placebo to patients in pain.
I’ve recently come across several evidence-based claims that, if acted upon, may eliminate a pain treatment agent or procedure that can be beneficial for many patients.3-10 Examples include:
- Epidural corticoid injections should not be reimbursed by insurance companies because there is no evidence that they work.
- Patients with lumbar spine disease should never have surgery.
- Opioids given in doses of 120 or 200 mg per day of morphine equivalence don’t work.
- The only “evidence-based” drug to be effective in diabetic neuropathy is pregabalin.
- Electromagnetic energy devices should not be used because they haven’t been subjected to double-blind studies.
- Only anti-inflammatory agents and never opioids should be prescribed to the elderly.
- Propoxyphene recently was pulled from the market on the most flimsy evidence of side effects.
I’ll bet you, as do I, have patients in your practice who have benefited from every one of the aforementioned. While many evidence-based studies have great merit, the pain practitioner is encouraged to carefully review statistical evidence with “eyes wide open.” Author bias (even at an almost subconscious level), omission of seminal studies on the basis that they were done too long ago, and decisions regarding inclusion/exclusion criteria can combine with a myriad of other factors to make the “evidence” suspect.
The blatant fact is that pain treatment is never “one-way.” Severe cases, particularly those tragic patients with central pain, may simultaneously require multiple pain medications on the same day. Pain treatment is temporal and sequential. Treatment may be lifelong and change rather dramatically in the course of a single year. In my practice, no two patients have the same treatment regimen, and some regimens are pretty strange.
The bottom line is that physicians need to be advocates for their pain patients. Pain treatment has lots of evidence of effectiveness at every clinic visit. Every patient and their family are good statistical controls in that they can report pain relief, improved function, and enhanced well-being. Yes, evidence-based medicine definitely has its place and value. But, let us never allow it to diminish the value of the “real” evidence presented by patients and their families.
—Forest Tennant, MD, DrPH
Editor in Chief