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REG Cost-Effectiveness Workshop


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REG Cost-Effectiveness Workshop presented on May 2015 in Denver, Colorado

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REG Cost-Effectiveness Workshop

  1. 1. REG Cost-Effectiveness Workshop (Part II) Jonathan D. Campbell; Piyameth Dilokthornsakul
  2. 2. Workshop Objectives • Introductions • Cystic fibrosis model example • What can we do with CEA models? What can’t we do? • Unveil the cost-effectiveness model black box. • Review current evidence gaps in respiratory health technology assessment • Collectively propose study designs and other solutions toward HTA respiratory evidence • Who would like to participate in a REG working group? • Research priorities of a cost-effectiveness REG working group and action items for initiating a working group?
  3. 3. Forecasting the Lifetime Outcomes and Cost of Ivacaftor in Patients with Cystic Fibrosis in the United States Piyameth Dilokthornsakul; Ryan N. Hansen; Jonathan D. Campbell
  4. 4. CF Model Objectives • To forecast lifetime outcomes and cost to compare ivacaftor plus usual care versus usual care alone • To indirectly compared the long-run projected impact of ivacaftor to the non- CF U.S. population. 4
  5. 5. Model and assumptions • Lifetime Markov model • CF patients aged 6 or more • U.S. payer perspective with 3% discount for cost and outcomes • Incorporated exacerbation in each health state • Assumption: • Progressive approach in usual care alone • Efficacy of ivacaftor after 2 years • Inputs from literatures outside USA • Cost of ivacaftor after patent expired 5 Scenarios Description (Efficacy after 2 years) Treatment duration Base-case scenario 50% efficacy Lifetime Optimistic scenario Full efficacy Lifetime Intermediate scenario 66% efficacy Lifetime Conservative scenario Patients stop the treatment Two years Health state 1: Mild lung disease %FEV1≥ 70 Health state 2: Moderate lung disease 40 ≤ %FEV1<70 Health state 3: Severe lung disease %FEV1<40 Health state 4: Lung transplantation Health state 5: Death
  6. 6. Inputs 6 Inputs Data sources References Clinical Efficacy of ivacaftor Two landmark randomized controlled trials Ramsey BW. N Engl J Med. 2011;365(18):1663- 1672 Davies JC. Am J Respir Crit Care Med. 2013;187(11):1219-1225 Clinical Transition probabilities Australian study3 (Usual care) van Gool K. Value Health. Mar-Apr 2013;16(2):345- 355 Clinical Mortality U.S. life tables A previous study for relative risk of death in CF patients with certain FEV1 Karem E. N Engl J Med. 1992; 326:1187-91 Economics Cost U.S. studies for treatment cost REDBOOK for cost of medication Lieu TA. Pediatrics. Jun 1999;103(6):e72 Bentley TS. U.S. organ and tissue transplant cost estimates and discussion. Brookfield, WI: Miilliman;2011 Patient-centered Utility A U.K. study Whiting P. Health Technol Assess. Mar 2014;18(18):1-106
  7. 7. Analysis • One-way sensitivity analysis • Probabilistic sensitivity analysis • Simple budget impact analysis • For the first 3, 5, and 10 years of ivacaftor use 7
  8. 8. Model example
  9. 9. Results: Base-case and scenario Findings (Ivacaftor +UC Vs. UC) Base-case scenario (50% efficacy) Optimistic scenario (100% efficacy) Intermediate scenario (66% efficacy) Conservative scenario (Stop treatment) Incremental estimated life expectancy 5.31 (4.45 to 6.08) 8.61 (8.19 to 8.99) 5.74 (5.05 to 6.35) 0.14 (0.08 to 0.23) Incremental QALYs 4.52 (3.69 – 5.40) 7.45 (6.49 to 8.41) 4.89 (4.12 to 5.60) 0.12 (0.06 to 0.19) Incremental costs ($) $3,740,480 ($2,199,261 to $4,915,629) $3,837,481 ($1,627,340 to $5,481,593) $3,751,831 ($2,0418,971 to $4,978,556) $507,043 ($-43,931to $737,964) 9 Abbreviations: QALY; quality-adjusted life years, UC; usual care
  10. 10. Results: One-way Baseline %FEV1 predicted for moderate lung disease (Incremental QALY) Cost of ivacaftor (Incremental cost) 10
  11. 11. 11 0 0.1 0.2 0.3 0.4 0.5 0.6 0.7 0.8 0.9 1 $- $1,000,000 $2,000,000 $3,000,000 $4,000,000 Cost Effectiveness Acceptibility Curve Ivacaftor Usual Care Willingness-to-pay ($) Probabilityofbeingcost-effective Results: Probabilistic $(4,000,000) $(3,000,000) $(2,000,000) $(1,000,000) $- $1,000,000 $2,000,000 -0.5000 -0.4000 -0.3000 -0.2000 -0.1000 0.0000 0.1000 0.2000 0.3000 Incremental cost and QALYs for Ivacaftor vs Usual Care in patients with cystic fibrosis QALY Cost($)
  12. 12. Results • Budget impact analysis Time horizon Budget impact (per member per month) 3 years $0.091 ($0.069 to $0.113) 5 years $0.088 ($0.067 to 0.109) 10 years $0.081 ($0.061 to $0.100) 12
  13. 13. Gaps of knowledge in COPD CEA studies • 24 published studies in last 5 years identified the following gaps: • Most of studies use clinical trials as efficacy data, they might not be representative of the real-world population (Efficacy and Effectiveness issue) • A lack of quality of life evidence in exacerbation state. • A limitation of Markov assumption which transition probabilities are assumed to be constant overtime. It might not be similar to real-world. • Several models use lung function as a proxy of COPD severity. However, other factors could be predictors of disease severity but are not captured in the model (i.e. GOLD A-D vs. GOLD 1-4). • A lack of information on the long-term effect of interventions when the time horizon of the model was longer than that of clinical trials
  14. 14. Gaps of knowledge in asthma CEA studies • 25 published studies in last 5 years identified the following gaps: • A lack of information on the impact of adherence on effectiveness and cost-effectiveness for evidence used in the CEA model • A lack of sufficient and sensitive health-related quality-of-life preference scores (utility) data in pediatrics, during exacerbation, mild severity, or uncontrolled asthma populations • A lack of indirect cost estimation, especially for pediatric populations • A lack of information on the long-term effect of interventions when the time horizon of the model was longer than that of clinical trials • Other gaps of knowledge • A lack of CEAs on asthma patients who smoke, who have exercise-induced bronchoconstriction • A lack of CEAs related to the minimal effective dose of inhaled corticosteroid
  15. 15. REG Research Priorities for CEA • Collectively propose study designs and other solutions toward HTA respiratory evidence gaps. • Should REG develop a working group and identify funding to address this line of research? • Do we also have interest in acting as internal consultants to other REG working groups within cost-effectiveness applications? • Should REG develop and validate global asthma and chronic obstructive pulmonary disease policy models that could be tailored for use by HTA stakeholders in their real-world value assessment of existing and emerging interventions?
  16. 16. Policy Models • Well-designed Health Care Policy Models with robust inputs are powerful tools for analyzing health care policy and clinical trial investment decisions. • Such models are frequently used by the National Cancer Institute, the Congressional Budget Office, and other policy analysts when short- run trial-based or observational evidence is not enough to address all of the relevant policy issues.
  17. 17. Cost-Effectiveness Working Group Members and Action Items • Members • Action Items
  18. 18. Jonathan D Campbell, PhD Assistant Professor Director, Pharmaceutical Outcomes Research Graduate Program Center for Pharmaceutical Outcomes Research University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences | Anschutz Medical Campus Department of Clinical Pharmacy p: 303.724.2886 | f: 303.724.0979 | Mail Stop C238 12850 E. Montview Blvd, V20-1205 Aurora, CO 80045 Piyameth Dilokthornsakul, PharmD Lecturer Center of Pharmaceutical Outcomes Research Faculty of Pharmaceutical Sciences, Naresuan University Muang, Phitsanulok, Thailand Tel: 66-86-7354746 E-mail: