Using Evidence to Combat Overdiagnosis and Overtreatment — Part 1

by Richard Bukata, MD

W. Richard Bukata, MD

It bothers me when I agree too often with Jerry Hoffman. I don’t know why — it just does. Well, unfortunately, here is another area where we are in sync. Overdiagnosis and overtreatment. Hoffman has been a dog with a bone regarding this topic. He bays at the moon and writes articles and goes to conferences specifically on this topic.

But he is not alone. More and more people are coming on board. In fact, there is a prestigious organization whose sole purpose is to get physicians to be aware of this topic and to act upon the compelling evidence. Remember the renowned cardiologist, Bernard Lown? Along with others, he has formed the Lown Institute. Each year they hold an invitation-only conference in Boston focusing on developing strategies to attack the pervasive overdiagnosis and overtreatment culture that is rampant in the United States. Now the conferences are expanding across the country and all are invited — and the tuition is dirt cheap.

Check out the Lown Institute website (http://lowninstitute.org/) and sign up for their weekly alerts on information pertinent to their quest — and take the Right Care Declaration (http://www.rightcaredeclaration.org/). The pledge challenges physicians to recognize their ethical obligation to put patient care first and to acknowledge that performing unnecessary tests and treatments is unethical and unacceptable. If you go onto the website you’ll notice pictures of “featured signatories.” As of the writing of this essay, our colleague, David Newman, MD, is prominently pictured.

I personally believe that we will never decrease our spending on healthcare (17% of GNP and growing) unless we stop our irrational, needless use of resources. The Accountable Care Act is a good thing — I know many of you disagree. However, we remain the only Westernized society in which all citizens are not covered for healthcare — and we remain the only society in which patients are subjected to inordinately large bills that often precipitate personal bankruptcy. There are over one million people who did not have health insurance who now have it and many more will become insured over time. Sure there are features of the Act that are frustrating and aggravating, but the bottom line — it is better than what we had for millions of Americans.

And one last point about Obamacare. I wonder what the citizens would say in the states where their Republican governors have refused to accept the Federal offer to pay for expanded Medicaid and, as a result, don’t receive healthcare (and are still paying taxes to cover the program that has been rejected by their leaders). The Feds have offered to pay for the whole cost for the first two years and then 95% thereafter — and the governors of a bunch of states have said “no thank you.” As of May 28, 2014, 27 states and the District of Columbia have accepted expanded Medicaid, four are considering it and 20 have declined — amazing. Even Arizona, a poster child for a state with a screwed up leadership, has accepted — resulting in 350,000 more citizens getting Medicaid. Here’s a list of some of other states and their expanded Medicaid roles (no data in some states):

  1. Arkansas — 250,000
  2. California — 1, 400,000
  3. Colorado — 160,000
  4. Connecticut –
  5. Delaware –
  6. District of Columbia — 35,000
  7. Hawaii –
  8. Illinois — 100,000
  9. Iowa — 100,000
  10. Kentucky –
  11. Maryland –
  12. Massachusetts –
  13. Michigan –
  14. Minnesota — 35,000
  15. New York —
  16. Nevada — 78,000
  17. New Hampshire — 50,000
  18. New Jersey –
  19. New Mexico –
  20. North Dakota –
  21. Ohio –
  22. Oregon –
  23. Rhode Island –
  24. Vermont — 47,000
  25. Washington –
  26. West Virginia — 90,000

If the four states considering participation come in, then estimated increased numbers are:

  1. Indiana — 500,000
  2. Missouri –
  3. Pennsylvania — 520,000
  4. Utah —

Back to the matter at hand — overdiagnosis and overtreatment. To date, the Affordable Care Act has largely focused on insurance reform. That was hard enough. But the much harder part is yet to come and will require physicians to step up to the plate and address overdiagnosis and overtreatment, among other challenges directly relating to the provision of care.

The fact is, in all candor, we don’t know how to reduce overdiagnosis and overtreatment. We were never taught how to do this. All of our training has focused on doing more — don’t miss the diagnosis, leave no stone unturned, don’t get embarrassed at morbidity and mortality rounds, the patients want tests, tests will make the patients more satisfied, we will make more money if we do more tests and procedures, we are going to get sued if we don’t do lots of tests, and on and on and on.

But how can we possibly curtail our healthcare spending (13% of GNP in 1991, 16.4% in 2011 and projected to continue to exceed GDP growth for the coming decades) if physicians don’t take the lead. Even healthcare professional societies are acknowledging the problem. Although only a small initial step, approximately 60 medical societies have signed on to the Choosing Wisely program and major citizen organizations (e.g., AARP, Consumer Reports, SEIU, the Leapfrog Group, National Business Group on Health) have bought in to support the efforts of these societies.

So overdiagnosis and overtreatment matter — big time. It is not just about money but also about ethics. But many physicians don’t know the data and need help in getting their arms around this problem. For openers, here is an article that gets right to the heart of the matter. But, before we get to the article, one last point. This is an essay — it is an opinion piece supposedly supported by facts. I know not all will agree but we’re open to your comments.

The following very relevant article was found in a recent issue of PLoS Medicine — the open access journal that allows you to use their articles as long as you cite the article properly.

Using Evidence to Combat Overdiagnosis and Overtreatment: Evaluating Treatments, Tests, and Disease Definitions in the Time of Too Much, Moynihan, R., et al, PLoS Medicine, 11(7), e1001655. Doi:10.1371/jounal.pmed.1001655, July 1, 2014

Summary Points

  • Overdiagnosis and related overtreatment are increasingly recognized as major problems.
  • “Positive” average results from trials of treatments can mask situations where many participants at low risk of disease may receive no benefit.
  • The evaluation of diagnostic tests usually involves assessing how well tests detect presence versus absence of a certain disease — rather than how well they detect clinically meaningful stages of disease.
  • Changes to disease definitions typically do not involve evaluation of potential harms of overdiagnosis, and are often conducted by heavily conflicted panels.
  • We offer suggestions for improving the way evidence is produced, analyzed, and interpreted, to help combat overdiagnosis and related overtreatment. These include routine consideration of overdiagnosis and related overtreatment in studies of tests and treatments, and clearer stratification by baseline risk to identify treatment thresholds where benefits are likely to outweigh harms.

While a large part of the world’s population faces the problems of underdiagnosis and undertreatment, it is apparent that a “modern epidemic” of overdiagnosis afflicts high-income countries [1](the article was written by Jerry Hoffman and Rochelle Cooper), with tangible human and financial costs of the unnecessary management of overdiagnosed diseases [2],[3]. While there is ongoing debate about how to best describe the problem, narrowly defined, overdiagnosis occurs when increasingly sensitive tests identify abnormalities that are indolent, non-progressive or regressive and that, if left untreated, will not cause symptoms or shorten an individual’s life. Such overdiagnosis leads to overtreatment when these “pseudo-diseases” are conventionally managed and treated as if they were real abnormalities; because these findings have a benign prognosis, treatment can only do harm. More broadly defined, overdiagnosis happens when a diagnostic label is applied to people with mild symptoms or at very low risk of future illness, for whom the label and subsequent treatment may do more harm than good [3].

Among the drivers of overdiagnosis are technological developments producing ever more sensitive imaging and biomarker tests, and changing disease and treatment thresholds that medicalize more people [4]. For example, detection of indolent breast lesions is now recognized as an established risk of mammography screening [5]; widened definitions of chronic kidney disease label many asymptomatic seniors as diseased [6]; lowered thresholds increase concerns about overdiagnosis of attention deficit hyperactivity disorder [7]; and more sensitive imaging methods are causing the treatment of large numbers of potentially benign pulmonary emboli [8].

It’s important to note there is a complex interrelationship between overdiagnosis and overtreatment — which can occur for many reasons other than overdiagnosis. If we consider the narrow definition of overdiagnosis — where someone is diagnosed with a “disease” that will not progress or harm them — overdiagnosis generally leads to overtreatment. Writing about overdiagnosis in 1998, Black described the cycle of increasingly sensitive tests causing more “pseudo-disease” to be diagnosed and conventionally treated [9]. Because prognosis of “pseudo-disease” is generally benign, there is a perception that patients do well on treatment, reinforcing belief in the value of treatment to the widened patient pool, and in turn fuelling further overtreatment [9]. In other situations, inappropriate overtreatment can occur where there is a legitimate clinical diagnosis, and in some circumstances a degree of overtreatment may be warranted, for instance, the early use of parenteral antibiotics in someone suspected of having bacterial meningitis.

Considering the broader definition of overdiagnosis — involving the medicalization of people with mild problems or at very low risk of disease — it becomes more difficult to define what constitutes subsequent overtreatment. Those judgments will depend on a complex mix of evidence about individual risk, prognosis, and treatment benefit–harm calculations, combined with the personal values and preferences inherent in any decision-making. Cognizant of this complex context, this essay explores how the production, analysis, and interpretation of evidence — whether from individual studies or systematic reviews — might be improved to better inform those judgments, and to better understand and combat the challenges of overdiagnosis and related overtreatment.

Average Therapeutic Trial Results Can Mislead

It’s widely recognized that average treatment effects estimated by systematic reviews of primary therapeutic trials don’t really apply to any single patient, and an average benefit can mask both positive and negative effects in different patient subgroups. This leads to treatment of patients who don’t benefit, and may suffer harms. Almost two decades ago, advocates of the then emerging evidence-based approach stressed the importance of a nuanced application of evidence from primary trials and systematic reviews for individuals, taking into account a person’s absolute risk of an outcome and the need to weigh up potential benefits and harms[10].

More recently Kent and colleagues cited examples in which positive clinical trial results masked a lack of meaningful benefit for those at lower risks of illness, including trials involving statins, anticoagulant therapies, and some common surgical procedures [11]. The authors argued that this problem of trials masking the “heterogeneity of treatment effects” can result in guidelines that promote overtreatment as well as undertreatment, and they recommended estimation of treatment effects after stratifying trial participants according to baseline risk.

W. Richard Bukata, MD
Medical Editor