PRIORITIZATION OF FUTURE OUTCOMES RESEARCH STUDIES IN CHRONIC MYELOID LEUKEMIA- VALUE OF INFORMATION ANALYSIS

Author(s)

Rochau U1, Kühne F2, Jahn B2, Kurzthaler C2, Corro Ramos I3, Chhatwal J4, Stollenwerk B5, Goldhaber-Fiebert JD6, Siebert U7
1UMIT - University for Health Sciences, Medical Informatics and Technology/ ONCOTYROL - Center for Personalized Cancer Medicine, Hall in Tyrol/ Innsbruck, Austria, 2UMIT - University for Health Sciences, Medical Informatics and Technology, Hall in Tyrol, Austria, 3Erasmus University Rotterdam, Rotterdam, The Netherlands, 4MD Anderson Cancer Center, Houston, TX, USA, 5Helmholtz Center Munich, Neuherberg, Germany, 6Western University, London, WA, USA, 7Medical Informatics and Technology, and Director of the Division for Health Technology Assessment and Bioinformatics, Oncotyrol, Hall i. T, Austria

OBJECTIVES Value-of-Information analysis can help to guide decision about future research priorities: If and what further research is needed? Our aim was to guide decision regarding future outcomes research on parameters related to different regimens for chronic myeloid leukemia (CML). METHODS We updated a previously developed state-transition Markov model of CML, which evaluates seven treatment regimens including tyrosine kinase inhibitors, chemotherapy and stem cell transplantation (SCT). We derived model parameters from published trials data, Austrian clinical, epidemiological, and economic data. We performed a cohort simulation over a lifetime horizon, adopted a societal perspective, and discounted costs and benefits at 3% annually. We calculated the expected value of perfect information (EVPI), partial perfect information (EVPPI), and the population EVPI (PEVPI). Additionally, we examined the expected value of sample information (EVSI) for different trial sizes. RESULTS Three strategies are on the efficiency frontier: imatinibàchemotherapy/SCT, nilotinibàchemotherapy/SCT (140,000 €/QALY) and nilotinibàdasatinibàchemotherapy/SCT (176,000 €/QALY). The EVPI for eliminating all uncertainty resulted in a curve with two peaks. One peak is around a WTP threshold of 150,000 €/QALY (EVPI 4,600 €) and another peak is at 180,000 €/QALY ( EVPI 7,700 €). The PEVPI for Austria assuming a 10-year technology horizon was 2.5 million € (WTP 150,000 €/QALY) and 4.5 million € (WTP 180,000 €/QALY). EVPPI identified four parameters most responsible for decision uncertainty: duration of first-line therapy, probability of progressing from chronic phase to accelerated phase, probability of receiving a SCT, and the health-utility after SCT. The EVSI commented on the optimal study size for these parameters given the cost of obtaining information. CONCLUSIONS Acquiring additional evidence could prove valuable for determining optimal treatment regimens for chronic myeloid leukemia. If further research were funded, studies should examine a combination of natural history, treatment, and quality of life parameters, especially the effectiveness of first-line TKI treatment.

Conference/Value in Health Info

2014-11, ISPOR Europe 2014, Amsterdam, The Netherlands

Value in Health, Vol. 17, No. 7 (November 2014)

Code

PCN143

Topic

Economic Evaluation

Topic Subcategory

Cost-comparison, Effectiveness, Utility, Benefit Analysis

Disease

Oncology, Systemic Disorders/Conditions

Explore Related HEOR by Topic


Your browser is out-of-date

ISPOR recommends that you update your browser for more security, speed and the best experience on ispor.org. Update my browser now

×