Policymaker, please consider your needs carefully: Does outcomes research in relapsed or refractory multiple myeloma reduce policymaker uncertainty regarding value for money of bortezomib?

Value in Health

Volumn 17, Issue 2, 2014, Pages 245-253

  Franken, Margreet G ^a   Gaultney, Jennifer G ^a   Blommestein, Hedwig M ^a   Huijgens, Peter C ^b   Sonneveld, Pieter ^c   Redekop, William K ^a   Uyl De Groot, Carin A ^a  

^a ERASMUS UNIVERSITY ROTTERDAM   (Netherlands)

^b VU UNIVERSITY MEDICAL CENTER   (Netherlands)

^c ERASMUS MEDICAL CENTER   (Netherlands)

Author keywords

bortezomib; cost effectiveness; multiple myeloma; outcomes research; policy making; uncertainty

Indexed keywords

BORTEZOMIB; CYCLOPHOSPHAMIDE; DEXAMETHASONE; DOXORUBICIN; LENALIDOMIDE; MELPHALAN; PREDNISONE; THALIDOMIDE; VINCRISTINE;

ADULT; AGED; ARTICLE; COST EFFECTIVENESS ANALYSIS; DONOR LYMPHOCYTE INFUSION; DRUG COST; FEASIBILITY STUDY; FEMALE; FUNDING; HEALTH CARE COST; HEALTH CARE POLICY; HUMAN; MAJOR CLINICAL STUDY; MALE; MIDDLE AGED; MULTIPLE MYELOMA; NETHERLANDS; OUTCOMES RESEARCH; OVERALL SURVIVAL; PRIORITY JOURNAL; STEM CELL TRANSPLANTATION;

BORTEZOMIB; COST-EFFECTIVENESS; MULTIPLE MYELOMA; OUTCOMES RESEARCH; POLICY MAKING; UNCERTAINTY;

ADULT; AGED; ANTINEOPLASTIC AGENTS; BORONIC ACIDS; COHORT STUDIES; COST-BENEFIT ANALYSIS; FEASIBILITY STUDIES; FOLLOW-UP STUDIES; HEALTH POLICY; HUMANS; KAPLAN-MEIER ESTIMATE; MALE; MIDDLE AGED; MULTIPLE MYELOMA; NETHERLANDS; OUTCOME ASSESSMENT (HEALTH CARE); POLICY MAKING; PYRAZINES; REIMBURSEMENT MECHANISMS; RETROSPECTIVE STUDIES; STATISTICS, NONPARAMETRIC; SURVIVAL RATE; UNCERTAINTY;

EID: 84896337471     PISSN: 10983015     EISSN: 15244733     Source Type: Journal    
DOI: 10.1016/j.jval.2013.12.009     Document Type: Article

References (55)

1. J.J. Carlson, S.D. Sullivan, and L.P. Garrison et al. Linking payment to health outcomes: a taxonomy and examination of performance-based reimbursement schemes between healthcare payers and manufacturers Health Pol 96 2010 179 190
2. J. Adamski, B. Godman, and G. Ofierska-Sujkowska et al. Risk sharing arrangements for pharmaceuticals: potential considerations and recommendations for European payers BMC Health Serv Res 10 2010 153
3. J.J. Carlson, L.P. Garrison Jr, and S.D. Sullivan Paying for outcomes: innovative coverage and reimbursement schemes for pharmaceuticals J Manag Care Pharm 15 2009 683 687
4. S.R. Tunis, and S.D. Pearson Coverage options for promising technologies: Medicare's 'coverage with evidence development' Health Aff 25 2006 1218 1230 (Pubitemid 44497620)
5. P.E. Mohr, and S.R. Tunis Access with evidence development: the US experience Pharmacoeconomics 28 2010 153 162
6. P. Trueman, D.L. Grainger, and K.E. Downs Coverage with evidence development: applications and issues Int J Technol Assess Health Care 26 2010 79 85
7. H.M. Krumholz Outcomes research: generating evidence for best practice and policies Circulation 118 2008 309
8. C.M. Clancy, and J.M. Eisenberg Outcomes research: measuring the end results of health care Science 282 1998 245
9. A. Towse, and L.P. Garrison Can't get no satisfaction? Will pay for performance help? Toward an economic framework for understanding performance-based risk-sharing agreements for innovative medical products Pharmacoeconomics 28 2010 93 102
10. M.G. Franken, C.W.M. van Gils, and J.G. Gaultney et al. Practical feasibility of outcomes research in oncology: lessons learned in assessing drug use and cost-effectiveness in The Netherlands Eur J Cancer 49 2013 8 16
11. K. Chalkidou, S. Tunis, and R. Lopert et al. Comparative effectiveness research and evidence based health policy: experience from four countries Milbank Q 87 2009 339 367
12. D.B. Rubin On the limitations of comparative effectiveness research Stat Med 29 2010 1991 1995
13. G.O. Delwel Guidance for Outcomes Research (Report No. 270) 2008 College voor Zorgverzekeringen Diemen, The Netherlands
14. M.R. Kuijpers, and W.G.M. Toenders Procedure beoordeling intramurale geneesmiddelen [Assessment Procedure for Inpatient Drugs] (Report No. 26022597) 2006 College voor Zorgverzekeringen Diemen, The Netherlands
15. P.G. Richardson, P. Sonneveld, and M.W. Schuster et al. Bortezomib or high-dose dexamethasone for relapsed multiple myeloma N Engl J Med 352 2005 2487 2498 (Pubitemid 41007864)
16. Commissie Farmaceutische Hulp Farmacotherapeutisch rapport bortezomib (Velcade®) (Report No. 25060777) 2005 College voor Zorgverzekeringen Diemen, The Netherlands
17. H.M. Lokhorst, I. Schmidt-Wolf, and P. Sonneveld et al. Thalidomide in induction treatment increases the very good partial response rate before and after high-dose therapy in previously untreated multiple myeloma Haematologica 93 2008 124 127 (Pubitemid 351156161)
18. J.G. Gaultney, M.G. Franken, and S.S. Tan et al. Real-world healthcare costs of relapsed/refractory multiple myeloma during the era of novel cancer agents J Clin Pharm Ther 38 2013 41 47
19. P.G. Richardson, P. Sonneveld, and M. Schuster et al. Extended follow-up of a phase 3 trial in relapsed multiple myeloma: final time-to-event results of the APEX trial Blood 110 2007 3557 3560 (Pubitemid 350159621)
20. International Society for Pharmacoeconomics and Outcomes Research. Pharmacoeconomic guidelines around the world. 2013. Available from: http://www.ispor.org/peguidelines/index.asp. [Accessed May 21, 2013].
21. D.T. Vogl, E.A. Stadtmauer, and P.G. Richardson et al. Impact of prior therapies on the relative efficacy of bortezomib compared with dexamethasone in patients with relapsed/refractory multiple myeloma Br J Haematol 147 2009 531 534
22. H.M. Lokhorst, B. van der Holt, and S. Zweegman et al. A randomized phase III study on the effect of thalidomide combined with adriamycin, dexamethasone, and high-dose melphalan, followed by thalidomide maintenance in patients with multiple myeloma Blood 115 2010 1113 1120
23. L. Garrison, A. Towse, and A. Brigss et al. Performance-based risk-sharing arrangements - good practices for design, implementation, and evaluation: ISPOR good practices for performance-based risk-sharing arrangements task force report Value Health 16 2013 703 719
24. J.G. Gaultney, M.G. Franken, and W.K. Redekop et al. PRM3: turning the tables to address the real value of real-world observational studies of novel anti-cancer agents in multiple myeloma Value Health 15 2012 A460
25. E. Cox, B.C. Martin, and T. Van Staa et al. Good research practices for comparative effectiveness research: approaches to mitigate bias and confounding in the design of nonrandomized studies of treatment effects using secondary data sources: the International Society for Pharmacoeconomics and Outcomes Research Good Research Practices for Retrospective Database Analysis Task Force Report - part II Value Health 12 2009 1053 1061
26. J. Concato, E.V. Lawler, and R.A. Lew et al. Observational methods in comparative effectiveness research Am J Med 123 2010 e16 e23
27. M.L. Johnson, W. Crown, and B.C. Martin et al. Good research practices for comparative effectiveness research: analytic methods to improve causal inference from nonrandomized studies of treatment effects using secondary data sources: the ISPOR Good Research Practices for Retrospective Database Analysis Task Force Report - part III Value Health 12 2009 1062 1073
28. B.M. Psaty, and D.S. Siscovick Minimizing bias due to confounding by indication in comparative effectiveness research JAMA 304 2010 897 898
29. A. Connors, T. Speroff, and N.V. Dawson et al. The effectiveness of right heart catheterization in the initial care of critically ill patients. SUPPORT Investigators JAMA 276 11 1996 889 897 (Pubitemid 26301608)
30. C.C. Earle, J.S. Tsai, and R.D. Gelber et al. Effectiveness of chemotherapy for advanced lung cancer in the elderly: instrumental variable and propensity analysis J Clin Oncol 19 2001 1064 1070 (Pubitemid 32176282)
31. J. Röttger, D. Scheller-Kreinsen, and R. Busse Patient-level hospital costs and length of stay after conventional versus minimally invasive total hip replacement: a propensity-matched analysis Value Health 15 2012 999 1004
32. J.D. Seeger, A.M. Walker, and P.L. Williams et al. A propensity score-matched cohort study of the effect of statins, mainly fluvastatin, on the occurrence of acute myocardial infarction Am J Cardiol 92 2003 1447 1451 (Pubitemid 37532570)
33. W.S. Weintraub, M.V. Grau-Sepulveda, and J.M. Weiss et al. Comparative effectiveness of revascularization strategies N Engl J Med 366 2012 1467 1476
34. S. Weitzen, K.L. Lapane, and A.Y. Toledano et al. Principles for modeling propensity scores in medical research: a systematic literature review Pharmacoepidemiol Drug Saf 13 2004 841 853 (Pubitemid 40003819)
35. A.H. Briggs, M.C. Weinstein, and E.A. Fenwick et al. Model parameter estimation and uncertainty analysis: a report of the ISPOR-SMDM Modeling Good Research Practices Task Force Working Group-6 Med Dec Making 32 5 2012 722 732
36. O. Ghatnekar, T. Alvegård, and N. Conradi et al. Direct hospital resource utilization and costs of treating patients with multiple myeloma in Southwest Sweden: a 5-year retrospective analysis Clin Ther 30 9 2008 1704 1713
37. X. Armoiry, F. Fagnani, and L. Benboubker et al. Management of relapsed or refractory multiple myeloma in French hospitals and estimation of associated direct costs: a multicentre retrospective cohort study J Clin Pharm Ther 36 2011 19 26
38. D. Koleva, S. Cortelazzo, and C. Toldo et al. Healthcare costs of multiple myeloma: an Italian study Eur J Cancer Care 20 2011 330 336
39. J. Mehta, S.B. Duff, and S. Gupta Cost effectiveness of bortezomib in the treatment of advanced multiple myeloma Manag Care Interface 17 2004 52 61 (Pubitemid 40022080)
40. J. Hornberger, J. Rickert, and R. Dhawan et al. The cost-effectiveness of bortezomib in relapsed/refractory multiple myeloma: Swedish perspective Eur J Haematol 85 2010 484 491
41. J. Möller, L. Nicklasson, and A. Murthy Cost-effectiveness of novel relapsed-refractory multiple myeloma therapies in Norway: lenalidomide plus dexamethasone vs bortezomib J Med Econ 14 2011 690 697
42. S. de Groot, H. Blommestein, and M. Franken et al. PCN154 Are population-based registries a suitable tool for outcomes research in cancer? Experiences from four registries Value Health 15 2012 A437 A438
43. H. Brenner, A. Gondos, and D. Pulte Recent major improvement in long-term survival of younger patients with multiple myeloma Blood 111 2008 2521 2526
44. S.K. Kumar, S.V. Rajkumar, and A. Dispenzieri et al. Improved survival in multiple myeloma and the impact of novel therapies Blood 111 2008 2516 2520
45. D.W. Light, and R. Warburton Demythologizing the high costs of pharmaceutical research BioSocieties 6 2011 34 50
46. H. Kantarjian The price of drugs for chronic myelogenous leukemia (CML) is a reflection of unsustainable prices of cancer drugs: from the perspective of a large group of CML experts Blood 121 2013 4439 4442
47. National Institute for Health and Clinical Excellence Bortezomib Monotherapy for Relapsed Multiple Myeloma (NICE Technology Appraisal Guidance 129) 2007 National Institute for Health and Clinical Excellence London
48. E.I. Schippers Voorstellen voor voorlopige en voorwaardelijke opname van nieuwe geneesmiddelen in het pakket [Proposals for provisional and conditional inclusion of new drugs in the drug reimbursement scheme] Ministerial Lett 2011 GMT-U-3065578. Available from: http://www.rijksoverheid.nl/documenten-en- publicaties/kamerstukken/2011/05/19/kamerbrief-opname-nieuwe-geneesmiddelen-in- pakket.html. [Accessed February 21, 2014]
49. C.J. McCabe, T. Stafinski, and R. Edlin et al. Access with evidence development schemes: a framework for description and evaluation Pharmacoeconomics 28 2010 143 152
50. T. Stafinski, C.J. McCabe, and D. Menon Funding the unfundable: mechanisms for managing uncertainty in decisions on the introduction of new and innovative technologies into healthcare systems Pharmacoeconomics 28 2010 113 142
51. S.A. Williamson Report into the Uptake of Patient Access Schemes in the NHS. Northumbria Healthcare NHS Trust 2009 Cancer Network Pharmacist Forum North Shields, United Kingdom
52. P.J. Neumann, J.D. Chambers, and F. Simon et al. Risk-sharing arrangements that link payment for drugs to health outcomes are proving hard to implement Health Aff 30 2011 2329 2337
53. C.L.M. Sudlow, and C.E. Counsell Problems with UK government's risk sharing scheme for assessing drugs for multiple sclerosis BMJ 326 2003 388 392 (Pubitemid 36241480)
54. College voor Zorgverzekeringen. Pay-for-Performance overeenkomst omalizumab (Xolair) voor de behandeling van ernstige astma [Pay-for-Performance Agreement for Omalizumab (Xolair) for Treatment of Severe Asthma] (Report No. ZA/2012056679). Diemen, The Netherlands: College voor Zorgverzekeringen, 2012. Available from: http://www.cvz.nl/binaries/content/documents/zinl-www/ documenten/publicaties/rapporten-en-standpunten/2012/1208-pay-for-performance- overeenkomst-omalizumab-xolair-voor-de-behandeling-van-ernstige-astma/ 1208-pay-for-performance-overeenkomst-omalizumab-xolai.
55. E.I. Schippers Standpunt Pompe en Fabry [Ministerial point of view Pompe and Fabry] Ministerial Lett 2013 GMT-155165-111008. Available from: http://www.rijksoverheid.nl/documenten-en-publicaties/kamerstukken/2013/10/03/ kamerbrief-over-standpunt-pompe-en-fabry.html. [Accessed February 21, 2014]