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Pharmacoeconomics Research: Its Role in Clinical Trials

Pharmacoeconomics Research: Its Role in Clinical Trials. Kent H Summers, PhD Associate Professor Purdue University, School of Pharmacy May 14, 2008. Pharmacoeconomics Goal & Objectives.

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Pharmacoeconomics Research: Its Role in Clinical Trials

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  1. Pharmacoeconomics Research:Its Role in Clinical Trials Kent H Summers, PhD Associate Professor Purdue University, School of Pharmacy May 14, 2008

  2. PharmacoeconomicsGoal & Objectives Goal:To increase knowledge of pharmacoeconomics research to better understand the significance of this data collected in clinical trials Objectives • Identify the role of economic and humanistic studies in drug product development • Identify the types of health outcomes studies used in drug product development • Identify where health outcomes studies are performed in the drug development process

  3. Constrained ? Economic Environment Growing

  4. Why Clinical Trials ? • Registration • Safety • Efficacy • Inform prescribing decisions • Inform drug policy decisions ???

  5. Clinical Pharmaceutical Research Phase 1 Phase 2 Phase 3 Phase 4 Safety Safety & Efficacy Treatment decisions Policy decisions

  6. Can you assume that RCT results will be reflected in actual practice? EFFECTIVENESS “Outcomes” studies EFFICACY RCTs

  7. Effectiveness The extent to which medical interventions achieve health improvements in real-life settings (usually differs from efficacy, as determined in controlled clinical trials). Efficacy Effectiveness Efficiency Does it contribute to a more efficient use of resources? Does it work in real life? Can it work in clinical practice? (proven in RCTs  ideal conditions)

  8. Managed care’s interest in economic evaluations Formulary management may involve (and economic evaluations can inform decisions regarding): • requirement of prior authorization for payment of certain drugs or unusual dosages • limits on the quantity of product dispensed • tiered copayments, in which patients are required to pay less for cheaper medications (e.g., generic medications) and more for expensive brands. (How to select products for tier levels?) Also, the Food and Drug Administration Modernization Act of 1997 (FDAMA): promotion, AMCP-format formulary submission

  9. Pharmacoeconomics research informationVALUE • Is a pharmaceutical product worth its price? • In which patients does it produce the optimal benefit (both cost and quality) ?

  10. Safety Efficacy Effective- ness??? Reporting Potential for Bias AMCP-format Formulary Submission Disease and Product Description Clinical Studies Submission Document Economic Modeling Overall Product Cost and Value Plan Data Resource Utilization Published Economic Evaluations Costs Source: Mather et al, AJMC 1999.

  11. ECHO Model of Outcomes Assessment Economic (Cost offsets, Productivity) Clinical (BP, FEV HbA1c) Humanistic (QoL, Satisfaction) Source: Kozma C, et al.Clin Ther 1993

  12. Pharmacoeconomics CLINICAL ECONOMIC HUMANISTIC Efficacy Side effects Satisfaction Quality of Life Bothersomeness, tolerability Safety Work productivity Resources consumed Direct Medical Costs

  13. Selection of costs (and outcomes) in an analysis depends on perspective Source: Lyles A, “Formulary Decision-Maker Perspectives: Responding to Changing Environments” (Chapter 7) in, Economic Evaluation in U.S. Health Care: Principles and Applications (Pizzi, Lofland, eds.), 2006

  14. Economic Analysis “A comparative analysis of alternative courses of action in terms of both their costs and consequences. ” Drummond et al., Oxford University Press, 1987:8.

  15. Types of Economic Analyses

  16. Cost Effectiveness Ratio NumeratorCosts included depends on perspective Patients’ Perspective Deductible costs Co-insurance costs Co-pay costs Out-of-pocket costs Transportation costs Payer’s Perspective Hospital costs Physician costs Lab costs Rx costs (if covered) Employers’ Perspective Hospital costs Physician costs Lab costs Rx costs (if covered) Indirect (productivity) costs Life-year Gained

  17. CUA 1.0 Quality-adjusted Life Years QaLYs gained QaLYs - patient 1 QaLYs - patient 2 0 Death Death Denominator: CEA v. CUA Time Life-year Gained = CEA

  18. CUA ratio denominator Healthy Quality-adjusted Life Year (QaLY) Gained Morbidity Mortality

  19. CUA: Cost/QALY "League Table"

  20. Pharmacoeconomics CLINICAL ECONOMIC HUMANISTIC Efficacy Side effects Satisfaction Quality of Life Bothersomeness, tolerability Safety Work productivity Resources consumed Direct Medical Costs

  21. Humanistic (“Intangible”) Outcomes "Not everything that counts can be counted, and not everything that can be counted counts."

  22. Source: MC Glossary

  23. What are PrOs? • PrOs = patient’s reports of a health condition and its treatment • Health-related Quality of Life (HrQoL) = patient’s evaluation of the impact of a health condition and its treatment on daily life • Therefore, validation of data collection instruments vitally important! Source: Acquadro, et al, ViH, 2003

  24. The value of patient-reported outcomes (PROs): • The patient’s perspective is a key element in medical diagnosis and treatment. • PRO data are unique and complementary indicators of disease activity. • PROs in clinical trials provide important data for evaluating the effectiveness of new treatment. • • Consistent with the definition of a scientific instrument, PRO measures provide precise, reliable, valid, and reproducible data. • • PRO data are essential for evidence-based practice. • • For new pharmaceuticals, PRO data from clinical trials support evidence-based practice. • • Professional organizations recognize the key role PRO data play in diagnosis and treatment, as evidenced by their clinical practice guidelines. • The inclusion of PROs in clinical trials is sanctioned by professional organizations, as evidenced by their trial guidelines, including FDA.

  25. Why are PrOs important in drug development? • Clinical response endpoints, alone, can underestimate treatment benefit • PrOs represent logical extensions of clinical effects • To understand impact on quality, in addition to quantity of life (esp. Oncology)

  26. Why will PrOs become increasingly important? • IOM report (patient-centered care) • FDA acceptance its use in promotion (FDAMA) • FDA guidance for its measurement • Future drug development: from chronic care (subject to price controls)  “lifestyle” drugs (out-of-pocket, less price restrictions, purchase decisions based on desires of patients) ???

  27. Multiple dimensions of HrQoL Physical Function Quality of Life Social & Role Function General well-being Psychological Function FUNCTION AFFECT

  28. HrQoL Measurement Issues • Health status: • Medical Outcomes Studies Short Form, 36 questions (MOS SF-36) • Multi-dimensional measurement of treatment impact • Impact on function, affect • Cross-condition comparisons • Responsiveness? • Utilities (preference-based measures): • EQ-5D • Non-affected population’s understanding of disease impact? • Single number (1 – 100) • Cross-condition comparisons • Responsiveness? • Condition-specific instruments: • Clinically sensible • Responsiveness • Cross-condition comparisons? • Interpretation? Usefulness in medication utilization decisions?

  29. Generic Instrument Condition- specific Instrument Example: Selection of HrQoL data collection instruments in eye disease studies • SF-36 (health status), EQ-5D (utilities) • Validated and reliable • Widely used in quality of life studies • Not specific to ophthalmology ------------------------------------------------------------------------------- • National Eye Institute Visual Function Questionnaire (NEI-VFQ-25) • Confirmed reliability and validity for a number of common eye conditions • Validated in type 1 diabetes cohort of the Wisconsin Epidemiologic Study of Diabetic Retinopathy (WESDR) • Strongly associated with vision, independent of retinopathy severity and other diabetic complications Linder M, et al. Arch Ophthalmol. 1999;117:1611-1616. Klein R, et al. Arch Ophthalmol. 2001;119:733-740. Sharma S, et al. Br J Ophthalmol. 2002;86:493-496.

  30. EQ-5D By placing a checkmark in one box in each group below, please indicate which statements best describe your own health state today. Mobility I have no problems in walking about o I have some problems in walking about o I am confined to bed o Self-Care I have no problems with self-care o I have some problems washing or dressing myself o I am unable to wash or dress myself o Usual Activities (e.g. work, study, housework, family or leisure activities) I have no problems with performing my usual activities o I have some problems with performing my usual activities o I am unable to perform my usual activities o Pain/Discomfort I have no pain or discomfort o I have moderate pain or discomfort o I have extreme pain or discomfort o

  31. EQ-5D: Visual Analog Scale (VAS)

  32. Information available at product launch? Phase 1 Phase 2 Phase 3 Phase 4 Registration Safety & Efficacy  Effectiveness? Treatment decisions Policy decisions

  33. Efficacy (from RCTs) Effectiveness (CE projections) Economic Models • Inputs • Safety & Efficacy Data (RCTs) • Epidemiology • Cost of disease Outputs Cost / effectiveness

  34. Basic Decision-analytic Model

  35. PharmacoeconomicsGoal & Objectives Goal:To increase knowledge of pharmacoeconomics research to better understand the significance of this data collected in clinical trials Objectives • Identify the role of economic and humanistic studies in drug product development • Identify the types of health outcomes studies used in drug product development • Identify where health outcomes studies are performed in the drug development process

  36. Pharmacoeconomic Research: Product Examples

  37. Example: Prozac (fluoxetine) Phase 1 Phase 2 Phase 3 Phase 4 Registration Safety & Efficacy  Effectiveness? Retrospective studies Prospective studies

  38. Example: Prozac (fluoxetine) Kupfer Curve Kupfer DJ. 1991

  39. Example: Actos (pioglitazone) Phase 1 Phase 2 Phase 3 Phase 4 Registration Safety & Efficacy  Effectiveness? Economic Model

  40. Example: Yentreve (duloxetine)

  41. Example: Yentreve (duloxetine) Urinary incontinence treatment management decisions based on: • Number of incontinence episodes • Impact on quality of life

  42. Example: Yentreve (duloxetine) Phase 1 Phase 2 Phase 3 X Phase 4 Registration Safety & Efficacy  Effectiveness? Treatment decisions Policy decisions QoL instrument development QoL in RCTs

  43. SUMMARYClinical trials as a model of medication performance The following data collected in RCTs is very important: • Side effects (even minor SEs, only potentially bothersome SEs) • Event rates: unscheduled phone calls (e.g., to answer patient questions about potential SEs), physician office visits, ED visits, hospital admissions • Secondary study endpoints, such as those collected in PrO instruments

  44. PharmacoeconomicsGoal & Objectives Goal:To increase knowledge of pharmacoeconomics research to better understand the significance of this data collected in clinical trials Objectives • Identify the role of economic and humanistic studies in drug product development • Identify the types of health outcomes studies used in drug product development • Identify where health outcomes studies are performed in the drug development process

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