Ending Medical Reversal

1. Medical reversal is common and harmful: Many accepted practices are later proven ineffective

Doctors often pursue treatments that improve survival while making patients miserable in the process. Patients usually have precisely the opposite goals—they value feeling better much more than living longer.

Prevalence of reversal. Studies suggest that 40-50% of current medical practices may be ineffective or harmful when rigorously tested. Examples include:

• Stents for stable heart disease
• Vertebroplasty for osteoporotic fractures
• Hormone replacement therapy for postmenopausal women

Harms of reversal:

• Patients exposed to ineffective or harmful treatments
• Wasted healthcare resources
• Erosion of trust in medical system
• Delayed adoption of truly effective therapies

Medical reversals occur because practices are often adopted based on limited evidence like:

• Mechanistic reasoning
• Observational studies
• Expert opinion
• Financial incentives

2. Evidence-based medicine is crucial: Randomized controlled trials are the gold standard

The randomized controlled trial is arguably the most important medical technology of the 20th century.

Hierarchy of evidence. Randomized controlled trials (RCTs) provide the strongest evidence for determining if a treatment works. Key aspects:

• Randomly assign patients to treatment or control group
• Control for known and unknown confounding factors
• Can prove causation, not just correlation

Other study types have limitations:

• Observational studies: Subject to confounding
• Case-control studies: Prone to bias
• Surrogate endpoints: May not translate to clinical benefit

Importance of replication. Even well-designed RCTs can produce false positives. Replication in multiple trials provides greater confidence in results.

3. Surrogate outcomes can be misleading: Focus on meaningful clinical endpoints

Remember, we say sometimes. They are not always wrong. If they were always wrong, it would be easy—always do the opposite. Research says observational studies are wrong somewhere between 15 and 50 percent of the time.

Definition. Surrogate outcomes are measurable markers used as substitutes for clinically meaningful endpoints. Examples:

• Blood pressure for heart disease
• Tumor shrinkage for cancer survival
• Bone density for fracture risk

Limitations. Relying on surrogate outcomes can lead to adopting ineffective or harmful treatments:

• Lowering blood sugar doesn't always improve diabetic outcomes
• Cholesterol-lowering drugs may not reduce heart attacks
• Tumor shrinkage doesn't necessarily mean longer survival

Focus on patient-centered outcomes:

• Mortality
• Quality of life
• Functional status
• Symptom relief

4. Screening tests require careful evaluation: Benefits must outweigh potential harms

Overdiagnosis occurs when some of the cancers that are found through screening are insignificant. They are cancers that have no potential to make people sick or die.

Criteria for effective screening:

1. Finds disease early
2. Reduces disease-specific mortality
3. Improves overall survival

Potential harms of screening:

• False positives: Anxiety and unnecessary procedures
• Overdiagnosis: Treatment of disease that would never cause symptoms
• Opportunity costs: Resources diverted from more effective interventions

Examples of controversial screening:

• PSA testing for prostate cancer
• Mammography for women under 50
• Whole-body CT scans

Carefully weigh benefits and harms, considering factors like:

• Age and risk factors of target population
• Accuracy of screening test
• Effectiveness of available treatments
• Psychological impact of screening

5. Industry influence can bias medical research and practice

Trials are also paid for by agencies that do not have a vested interest in one outcome or another—agencies such as the U.S. National Institutes of Health, the Veterans Administration, and the Department of Defense.

Sources of bias:

• Industry-funded clinical trials
• Conflicts of interest in guideline development
• Marketing to physicians and patients

Consequences:

• Selective reporting of positive results
• Exaggeration of treatment benefits
• Downplaying of side effects
• Promotion of marginally effective treatments

Strategies to mitigate bias:

• Independent funding of clinical trials
• Strict conflict of interest policies
• Critical appraisal of evidence
• Transparency in research and guideline development

6. Medical education needs reform: Emphasize empiricism over reductionism

In our quest to decrease medical reversal, time would be spent in small groups learning to critically evaluate the evidence behind every decision made during an actual patient encounter.

Current limitations:

• Overemphasis on basic sciences
• Insufficient training in evidence appraisal
• Lack of focus on patient-centered outcomes

Proposed reforms:

• Earlier clinical exposure
• Integration of evidence-based medicine throughout curriculum
• Training in critical appraisal of research
• Focus on patient-centered care and shared decision-making

Benefits:

• Improved clinical reasoning skills
• Better understanding of evidence hierarchy
• Increased skepticism of unproven practices

7. Burden of proof should be on new treatments: Higher standards for adoption needed

The burden of proof, in cases of newly discovered species, must be on the discoverer.

Current problem: Many treatments are adopted before rigorous evidence of efficacy exists.

Proposed solution: Require strong evidence of clinical benefit before widespread adoption.

Key principles:

• Randomized controlled trials as gold standard
• Focus on patient-centered outcomes
• Replication of results in multiple studies
• Consideration of cost-effectiveness

Challenges to implementation:

• Pressure for rapid adoption of new technologies
• Financial incentives for industry and providers
• Patient demand for latest treatments

8. Patients can protect themselves by asking key questions about treatments

If you have been offered a treatment that is proved to affect end points you care about, you can decide whether you want it. You have considered the "number needed to treat." Now ask those questions that you wanted to ask from the start. How does it work? What are the side effects? Does my insurance cover it?

Key questions for patients:

1. What outcomes matter most to me?
2. What is the evidence supporting this treatment?
3. How much will this treatment help?
4. What are the potential harms?
5. Are there alternative options?

Strategies for shared decision-making:

• Ask about "number needed to treat" (NNT)
• Request to see relevant research studies
• Discuss natural history of condition if untreated
• Consider participating in clinical trials

Empowering patients:

• Recognize limitations of medical knowledge
• Understand that "doing something" isn't always best
• Be comfortable with uncertainty in medicine

Last updated: November 19, 2024