Cost Effectiveness Working Group Meeting ERS 2017 Milan, 9th September 2017

Agenda Attendees and core members Working group goals Priorities for future research Review potential studies Other research ideas Funding opportunities Setting priorities Additional items

Attendees J van Boven (chair) A Azuma A Cruz U Seppala G Crater P Rivera Ortega

WG core members Lead: Associate prof. dr. Jonathan D Campbell (Denver, USA) Assistant prof. dr. Job FM van Boven (Groningen, Netherlands) Assistant prof. dr. Mohsen Sadatsafavi (Vancouver, Canada) Assistant prof. dr. R Brett McQueen (Denver, USA)

Goals of cost-effectiveness working group Perform real-life and modelling studies on the cost-effectiveness of respiratory treatments Raise awareness on the importance of cost-effectiveness and identify gaps in currect research Provide input for clinical studies with an economic component Provide workshops/education on health economic issues in respiratory medicine inside and outside REG Job – for all of the above, our primary next step is to secure funding through an REG collaborative project in order to continue to run this working group.

Core Member Selected Recent Publications Asthma CEA Whittington MD, McQueen RB, Ollendorf DA, Tice JA, Chapman RH, Pearson SD, Campbell JD. Assessing the value of mepolizumab for severe eosinophilic asthma: a cost-effectiveness analysis. Annals of Allergy, Asthma & Immunology. 2016 Dec 3. S1081-1206(16):31267-4. Dilokthornsakul P, Chaiyakunapruk N, Campbell JD. Does the use of efficacy or effectiveness evidence in cost-effectiveness analysis matter? Journal of Asthma. 2017 Jan 2. 54(1):17-23. Price DB, van Boven JF, Law LM, Cifra A, McQueen RB. Cost comparison of asthma treatment in 12-week study: caution about matching and short follow-up. Multidiscip Respir Med 2016; 11:39 Kim CH, Dilokthornsakul P, Campbell JD, van Boven JF. Asthma cost-effectiveness analyses: Are we using the recommended outcomes in estimating value? JACI In Practice 2017 in-press. COPD CEA Van Boven JF, McQueen RB, Price DB. COPD case finding: effective, but also cost-effective? Lancet Respir Med 2016; 4(10):e49 Van Boven JF, Kocks JW, Postma MJ. Cost-effectiveness and budget impact of the fixed dose dual bronchodilator combination tiotropium-olodaterol for patients with COPD in The Netherlands. Int J COPD 2016; 11:2191-2201 JF van Boven. Costly comorbidities of COPD: the ignored side of the coin? European Respiratory Journal 2017; 49(7):1700917

Gaps identified through review van der Schans S, Goossens LM, Boland MR, Kocks JW, Postma MJ, van Boven JF, Rutten-van Mölken MP. Systematic Review and Quality Appraisal of Cost-Effectiveness Analyses of Pharmacologic Maintenance Treatment for Chronic Obstructive Pulmonary Disease: Methodological Considerations and Recommendations. Pharmacoeconomics. 2017 Jan;35(1):43-63 CH Kim, P Dilokthornsakul, JD Campbell, JF van Boven. Asthma cost-effectiveness analyses: Are we using the recommended outcomes in estimating value? Journal of Allergy & Clinical Immunology: In Practice 2017 Both reviews identified the need for: Incorporation of side-effects (local/systemic), comorbidities and adherence Standardization of costs and (respiratory) outcomes Better reflection of treatment patterns in real-life

Asthma Gaps A lack of information on the impact of adherence on effectiveness and cost-effectiveness. A lack of sufficient and sensitive health-related quality-of-life preference scores in pediatrics, mild severity, or persistent asthma 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 A lack of indirect cost estimation, especially for pediatric populations Gaps in Knowledge

COPD Gaps Efficacy versus Effectiveness and how this translates to Cost-Effectiveness Exacerbations: should be standardized; severe: hospitalization, non-severe: ED or GP visit. Adherence Populations Comorbidities Long-term side effects and their clinical and economic consequences Utility: standardize estimates for GOLD stages and exacerbations. Outcomes: Ideally both the EQ-5D and a COPD specific HRQoL should be used. Perspective: payer versus societal Indirect costs

Prioritizing Research   Topics/research areas for consideration 1 Indirect Costs of Asthma, COPD, and Overlap Syndrome (ACOS) 2 Quality of life measurement and preference scores in real-life asthma and COPD health states 3 Long-run clinical and economic consequences of oral steroids 4 Comparison of effectiveness vs. efficacy in asthma/COPD models 5 Validating long-term outcomes predicted by models vs. real-life data 6 Rare disease modelling 7 Cost-effectiveness of new technologies in asthma/COPD 8 Development of respiratory disease specific CEA guidelines Other ideas?

Solving the Puzzle Marrying high-impact research with funding and expertise Respiratory CEA gaps identified Respiratory CEA expertise identified Funding welcomed CEA working group is seeking pilot funding routed through REG

Active research seeking funding PharmacoEconomics journal has requested a review of the value of approved and emerging asthma biologics (Dr. McQueen is leading this effort) Aim of paper is to systematically review economic data on biologic treatments for asthma and to provide critical insight into key drivers of cost-effectiveness This effort could be organized through the REG CEA Working group We are seeking a funding collaborator for this research Robert.McQueen@ucdenver.edu

Any other business? Contact us for new REG studies: USA: Jon D Campbell (jon.campbell@ucdenver.edu) Europe: Job FM van Boven (j.f.m.van.boven@rug.nl)