Why are Clinical Trials Important to Health Care Importance des Essais Cliniques

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Why are Clinical Trials Important to Health
CareImportance des Essais Cliniques
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Why invest in RCTsCanadian Success stories!

• Canadian RCTs save the lives of Canadians
• Avoiding stroke with ASA and endarectomy
• Avoiding MIs with Heparin and ASA in unstable
  angina
• Decreased transfusions while saving lives in ICU
• Improving the quality of life of patients on
  dialysis with Erythropoeitin
• Decreasing relapses of patients who have an
  exacerbation of COPD

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Trials yet to be completed

• Many trials on alternative therapies including
  Chinese herbal remedies
• Major trial on peri-operative use of beta
  blockers (POISE trial)
• Comparison of two standard approaches to
  mechanical ventilation of in severe lung disease
• Comparative study of vasopressin in septic shock
• Comparative study of three antifibrinolytics in
  high risk cardiac surgery

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Benefits of CLINICAL TRIALS

• Enhances clinical care by bringing clinical
  research to the bedside
• Provides innovative care for patients
• Defines optimal care
• Decreases practice variation
• Offers mentorship and training for young clinical
  scientists
• Enables the Hospital and University to attract
  and retain best clinicians-scientists
• Helps establish programs that attract resources
• Major economic benefits through employment of
  highly skilled workforce

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Why Invest in Clinical Research?
Nature, 1998
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Why invest in RCTs?
Because we excel at the conduct of RCTs
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Why invest in RCTs?

• RCTs save lives of Canadians
• RCTs minimize harm to Canadians
• RCTs can promote new innovation

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Why are Clinical trials essential in medical care?

• Historically, magnitude of benefits was very
  large RCTs unnecessary
• More recently, magnitude of clinical benefit much
  smaller as a consequence, unbiased and more
  powerful methodology essential.RCT is gold
  standard.
• Multiple diagnostic and therapeutic
  choices.therefore choices not self-evident???

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What should the ideal RCT do?

• 1) Establish if a particular intervention
  works.
• 2) Determine the overall benefits and risks for
  given patients.
• 3) Minimizing the influence of chance, bias and
  confounding.
• 4) Accomplish these objectives with the fewest
  patients possible (efficiency).

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RCT design approaches

• Efficacy Does the therapy work?
• Effectiveness Does the therapy do
• more good than harm
• Both questions are clinically important and
  should be addressed sequentially.

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Points to remember about RCTs

• More than one RCT needed to test efficacy and
  effectiveness
• Fundamental methodology used in comparing health
  interventions
• RCTs linchpin in Evidence-Based Medicine
• Scientific method that might be used to test
  hypotheses
• In complex environments RCTs essential means of
  comparing therapies
• Results from RCTs essential for the adoption of
  findings into clinical practice

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Relationship between basic research and RCTs
Potential clinical benefits
Physiology, biochemistry, microbiology and
clinical immunology
Clinical Research
Knowledge Circle
Basic Sciences
Unexpected clinical benefits or harms
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Challenges

• New regulations.IND process
• Research ethics reviews for multicentre trials
• Importance of RCTs in health research agenda
• Leading trials rather then assisting companies
  with enrolment in studies
• Limited investment in clinical research
• Insurance for international trials
• Increasing accountability and conflict of
  interest concerns

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What do we need?

• Recognition of importance
• Leadership
• A core of well trained clinical epidemiologists
• Methodological and statistical expertise - George
  clones
• Versatile data and accurate management
• Excellent clinical care and health systems

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Conclusions

• Much more than the evaluation of DRUGS
• Academic leadership is essential
• Need much greater investment in clinical research
  and clinical researchers
• Investment should focus on mentorship, training
  of new health researchers and essential support
  staff, and clinical research networks
• Must have more of facilitation approach to health
  research not more rules
• Pharmaceutical trials beneficial but less
  important than development of academic leadership
  in research