Artikel
Further Research Needed for Chronic Myeloid Leukemia? A Value-of-Information Analysis
Suche in Medline nach
Autoren
-
Ursula Rochau
- Institute of Public Health, Medical Decision Making & Health Technology Assessment, Dept. of Public Health & Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Hall in Tirol, Österreich; Area 4: Health Technology Assessment and Bioinformatics, Oncotyrol - Center for Personalized Cancer Medicine, Innsbruck, Österreich
-
Felicitas Kühne
- Institute of Public Health, Medical Decision Making & Health Technology Assessment, Dept. of Public Health & Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Hall in Tirol, Österreich
-
Beate Jahn
- Institute of Public Health, Medical Decision Making & Health Technology Assessment, Dept. of Public Health & Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Hall in Tirol, Österreich
-
Christina Kurzthaler
- Institute of Public Health, Medical Decision Making & Health Technology Assessment, Dept. of Public Health & Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Hall in Tirol, Österreich
-
Isaac Corro-Ramos
- Institute for Medical Technology Assessment, Rotterdam, The Netherlands, The Netherlands
-
Jagpreet Chhatwal
- Department of Health Services Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA, USA
-
Björn Stollenwerk
- Institute of Health Economics and Health Care Management, Helmholtz Zentrum München (GmbH), German Research Center for Environmental Health, Germany, Deutschland
-
Jeremy Goldhaber-Fiebert
- Stanford Health Policy, Centers for Health Policy and Primary Care and Outcomes Research, Department of Medicine, Stanford University, Stanford, CA, USA, USA
-
Uwe Siebert
- Institute of Public Health, Medical Decision Making & Health Technology Assessment, Dept. of Public Health & Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Hall in Tirol, Österreich; Area 4: Health Technology Assessment and Bioinformatics, Oncotyrol - Center for Personalized Cancer Medicine, Innsbruck, Österreich; Center for Health Decision Science, Department of Health Policy and Management, Harvard T. H. Chan School of Public Health, Boston, USA; Institute for Technology Assessment and Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, USA
| Veröffentlicht: | 27. August 2015 |
|---|
Gliederung
Text
Introduction: Value-of-Information analysis extends traditional decision analysis and can help to guide decisions about future research priorities: If and what further research is needed? Our aim was to guide decisions regarding prioritization of future outcomes research on parameters related to different treatment 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 (imatinib, dasatinib, and nilotinib), chemotherapy and stem cell transplantation (SCT). We derived model parameters from published clinical studies; and 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) and expected net benefit of sampling (ENBS) for different trial sizes.
Results: Three strategies are on the cost-effectiveness frontier: imatinib, after failure followed by (-->) chemotherapy/SCT, nilotinib-->chemotherapy/SCT (141,500 €/QALY) and nilotinib-->dasatinib-->chemotherapy/SCT (178,600 €/QALY). The EVPI for eliminating all uncertainty resulted in a curve with two peaks. One peak is around a WTP threshold of 142,000 €/QALY (EVPI 4900 €) and another peak is at 179,000 €/QALY (EVPI 8200 €). The PEVPI for Austria assuming a 10-year technology horizon was 4.8 million € (WTP 179,000 €/QALY) and 297 million € for the European Union. We identified 23 parameters/groups of parameters that had a non-zero EVPPI at the WTP of 179,000 €/QALY. Four parameters had an EVPPI greater than 1000 €. The EVPPI for the utility of living with chronic graft-versus-host disease after SCT was 1218 €, for the probability of progressing from chronic phase to accelerated phase 2324 €, and for the probability of receiving a SCT after TKI failure 2418 €. The highest EVPPI was reached by the parameters characterizing the duration of first-line TKI therapy. A future study collecting information on the duration of first-line TKI therapy with imatinib, nilotinib and dasatinib would have an EVPPI of 5787€. For these for parameters, the expected value of new research was compared to the costs of conducting new research. In addition, the ENBS and the optimal sample size of future trials were determined (e.g., survey 120 Austrian physicians for the probability of receiving a stem cell transplantation after previous treatment failure, perform a quality-of life study with 1020 participants).
Conclusions: We performed a comprehensive value-of-information analysis and identified parameters with high EVPPIs. Subsequent EVSI and ENBS analyses identified the optimal sample size for these different parameters and respective studies. Our findings can help to guide future research priorities and the scale of relevant studies in Austria with regard to CML.
This work was supported by the COMET Center ONCOTYROL, which is funded by the Austrian Federal Ministries BMVIT/BMWFJ (via FFG) and the Tiroler Zukunftsstiftung/Standortagentur Tirol (SAT).
References
- 1.
- Rochau U, Kuehne F, Jahn B, Kurzthaler C, Corro-Ramos I, Chhatwal J, Stollenwerk B, Goldhaber-Fiebert JD, Siebert U. Prioritizing the Focus and Scale of Future Research for Chronic Myeloid Leukemia: A Value-of-Information Analysis. 7. Jahrestagung der Deutschen Gesellschaft für Gesundheitsökonomie; March 16-17, 2015; Bielefeld, Germany.
- 2.
- Rochau U, Kuehne F, Jahn B, Kurzthaler C, Muka A, Corro-Ramos I, Stollenwerk B, Goldhaber-Fiebert JD, Siebert U. When is enough evidence enough? Value-of-Information Analysis for Prioritizing Additional Outcomes Research on the Treatment of Chronic Myeloid Leukemia. The 36th Annual Meeting of the Society for Medical Decision Making (SMDM); October 18-22, 2014; Miami, USA.
- 3.
- Rochau U, Kuehne F, Jahn B, Kurzthaler C, Corro-Ramos I, Chhatwal J, Stollenwerk B, Goldhaber-Fiebert JD, Siebert U. Prioritization of Future Outcomes Research Studies in Chronic Myeloid Leukemia: Value of Information Analysis. ISPOR 17th Annual European Congress; November 8-12, 2014; Amsterdam, The Netherlands.
