Health Informatics and Evidence-based Practice

The Efficacy - Effectiveness Gap

• The lecture discusses the gap between efficacy and effectiveness in medical treatments.

• Efficacy: The power or ability to produce an effect, best tested using randomized controlled trials (RCTs).

• Effectiveness: The degree to which something successfully produces a desired result, best tested in 'real-world' trials.

• Efficacy-Effectiveness Gap: Differences in outcomes between patients treated in RCTs (ideal conditions) and those in real-world settings (messy conditions).

Limits of Randomized Controlled Trials

• RCTs are experiments designed to reduce bias when testing treatment efficacy.

• They involve:

  • Random allocation of subjects to groups.
  • Different treatments, with comparison of responses.
  • An experimental group receiving the intervention (e.g., a drug).
  • A control group receiving an alternative (e.g., a placebo).
  • Monitoring the groups to determine the efficacy of the experiment.

• Strengths of RCTs:

  • Accurate, unbiased measurement of treatment effect.
  • High degree of internal validity.
  • Strict eligibility criteria leading to a homogenous study population.
  • Reduced inter-patient variability.

• Limits of RCTs:

  • Suboptimal representation of real-world patients, especially those with advanced age or greater comorbidities.

Real World Trials

• Alternative to RCTs.

• Employ less restrictive recruitment criteria to ensure representation of individuals with previously ineligible characteristics.

Achieving Implementation of Evidence

• Implementation Science: The study of methods for integrating research and evidence-based interventions into routine clinical practice.

• Research funders are increasingly interested in seeing successful treatments implemented without delay.

• Key aspects for successful implementation:

  • Being prepared.
  • Communicating effectively.
  • Ensuring required resources are available.
  • Enlisting needed help.
  • Using resources efficiently.

Antibiotics and Implementation Challenges

• Context: Prescribing of antibiotics, particularly for respiratory infections.

• Issues:

  • Questionable effectiveness in respiratory infections.
  • Association with increasing bacterial resistance.
  • Associated with preventable deaths in hospital, can be costly, and has side effects.

• Example Study: Amoxicillin for acute lower-respiratory-tract infection in primary care.

  • A 12-country, randomised, placebo-controlled trial published in The Lancet (2013).
  • DOI: 10.1016/S1473-3099(12)70300-6
  • Conclusion: Amoxicillin provides little benefit for acute lower-respiratory-tract infection in primary care when pneumonia is not suspected clinically and may cause slight harms.

• BMJ Open Study: Serious bacterial infections and antibiotic prescribing in primary care.

  • Conclusion: No population-level evidence that family practices with lower total antibiotic prescribing might have more frequent occurrence of serious bacterial infections overall.
  • Improving the recording of infection episodes could inform better antimicrobial stewardship in primary care.

Areas of Implementation

• Individual clinician (investigation, prescribing, non-drug interventions).

• Local groups (hospital teams, general practices, community teams).

• Professional groups (paediatricians, infectious disease clinicians, public health clinicians).

• Organisation.

• Region.

• Society.

Facilitators and Obstacles to Implementation

• Facilitators:

  • Experienced leadership.
  • Detailed planning.
  • Team engagement.
  • Prioritisation.
  • Provision of required resources.

• Obstacles:

  • Lack of commitment by management.
  • Disorganised work culture.
  • Poor readiness to change.
  • Poor team structure.

Implementation Challenges

• Implementing research evidence to improve health outcomes.

• Preventing harm from discredited treatments by implementing research evidence against them.