Evaluation of Anti-cancer Therapies with Reimbursement Limited to Comprehensive Cancer Centres Using the European Society for Medical Oncology Magnitude of Clinical Benefit Scale
Authors:
T. Büchler 1; B. Melichar 2; D. Vrána 2; R. Lemstrová 2; J. Fínek 3; L. Dušek 4,5; K. Petrakova 6; J. Prausová 7
Authors‘ workplace:
Onkologická klinika 1. LF UK a Thomayerova nemocnice, Praha
1; Onkologická klinika LF UP a FN Olomouc
2; Onkologická a radioterapeutická klinika LF UK a FN Plzeň
3; Ústav zdravotnických informací a statistiky, Praha
4; Institut biostatistiky a analýz, LF a PřF MU, Brno
5; Klinika komplexní onkologické péče LF MU a Masarykův onkologický ústav, Brno
6; Onkologická klinika 2. LF UK a FN Motol, Praha
7
Published in:
Klin Onkol 2017; 30(5): 349-360
Category:
Original Articles
doi:
https://doi.org/10.14735/amko2017349
Overview
Background:
The costs of oncology treatments are increasing, due to the rising prevalence of malignant diseases and the introduction of expensive novel anti-cancer agents. The new European Society for Clinical Oncology (ESMO) has recently developed a new parametric system to evaluate the clinical benefit of drugs. The Magnitude of Clinical Benefit Scale (ESMO-MCBS) compares the contribution of a novel drug based on overall and progression-free survival and quality of life with those of current treatment options.
Material and Methods:
An expert group of the Czech Oncological Society conducted an assessment based on published data and an ESMO-MCBS methodology for antineoplastic agents used for the treatment of solid tumors with limited reimbursement to Comprehensive Cancer Centers. We evaluated drugs categorized as “S” that were eligible for public health insurance as of January 1, 2017.
Results and Conclusion:
The ESMO-MCBS score is a promising new parameter for the evaluation of new anticancer drugs. The ESMO-MCBS method for assessing the clinical benefit of drugs is simple, robust, and reproducible. The advantage of the assessment is that it is not based on a single index but rather combines several dimensions of drug performance. This parameter will be gradually added to Czech cancer guidelines. Scores obtained in the majority of cases correspond to the observed benefit of a drug in routine clinical practice.
Key words:
tumors – farmacotherapy – assesment study as a subject – survival – protocols of anti-cancer therapy
The authors declare they have no potential conflicts of interest concerning drugs, products, or services used in the study.
The Editorial Board declares that the manuscript met the ICMJE recommendation for biomedical papers.
Submitted:
3. 5. 2017
Accepted:
20. 6. 2017
Sources
1. Cherny NI, Sullivan R, Dafni U et al. A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Ann Oncol 2015; 26 (8): 1547–1573. doi: 10.1093/annonc/mdv249.
2. Cherny NI, Sullivan R, Dafni U et al. ESMO – Magnitude of Clinical Benefit Scale V.1.0 questions and answers. ESMO Open 2016; 1 (5): e000100. doi: 10.1136/esmoopen-2016-000100.
3. Kiesewetter B, Raderer M, Steger GG et al. The European Society for Medical Oncology Magnitude of Clinical Benefit Scale in daily practice: a single institution, real-life experience at the Medical University of Vienna. ESMO Open 2016; 1 (4): e000066. doi: 10.1136/esmoopen-2016-000066.
4. Cherny NI, Sullivan R, Dafni U et al. A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Ann Oncol 2016; pii: mdw258. doi: 10.1093/annonc/mdw258.
5. Hurwitz H, Fehrenbacher L, Novotny W et al. Bevacizumab plus irinotecan, fluorouracil, and leucovorin for metastatic colorectal cancer. N Engl J Med 2004; 350 (23): 2335–2342. doi: 10.1056/NEJMoa032691.
6. Saltz LB, Clarke S, Díaz-Rubio E et al. Bevacizumab in combination with oxaliplatin-based chemotherapy as first-line therapy in metastatic colorectal cancer: a randomized phase III study. J Clin Oncol 2008; 26 (12): 2013–2019. doi: 10.1200/JCO.2007.14.9930.
7. Linkos. Klinický registr CORECT. [online]. Institut biostatistiky a analýz Masarykovy univerzity, Brno. [citováno 1. ledna 2017]. Dostupné z: http: //www.linkos.cz/prehled-registru/klinicky-registr-corect/.
8. Douillard JY, Tabernero J, Salvatore S et al. The PRIME study: survival outcomes in patients with KRAS/NRAS wild-type metastatic colorectal cancer and non-liver-limited disease. Ann Oncol 2014; 25 (Suppl 2): abstr. 0004. doi: 10.1093/annonc/mdu164.4.
9. Peeters M, Oliner KS, Price TJ et al. Analysis of KRAS/NRAS Mutations in a Phase III Study of Panitumumab with FOLFIRI Compared with FOLFIRI Alone as Second-line Treatment for Metastatic Colorectal Cancer. Clin Cancer Res 2015; 21 (24): 5469–5479. doi: 10.1158/1078-0432.CCR-15-0526.
10. Kim TW, Elme A, Kusic Z et al. A phase 3 trial evaluating panitumumab plus best supportive care vs best supportive care in chemorefractory wild-type KRAS or RAS metastatic colorectal cancer. Br J Cancer 2016; 115 (10): 1206–1214. doi: 10.1038/bjc.2016.309.
11. Amado RG, Wolf M, Peeters M et al. Wild-type KRAS is required for panitumumab efficacy in patients with metastatic colorectal cancer. J Clin Oncol 2008; 26 (10): 1626–1634. doi: 10.1200/JCO.2007.14.7116.
12. Van Cutsem E, Lenz HJ, Köhne CH et al. Fluorouracil, leucovorin, andirinotecan plus cetuximab treatment and RAS mutations in colorectal cancer. J Clin Oncol 2015; 33 (7): 692–700. doi: 10.1200/JCO.2014.59.4812.
13. Giantonio BJ, Catalano PJ, Meropol NJ et al. Bevacizumab in combination withoxaliplatin, fluorouracil, and leucovorin (FOLFOX4) for previously treatedmetastatic colorectal cancer: results from the Eastern Cooperative Oncology Group Study E3200. J Clin Oncol 2007; 25 (12): 1539–1544. doi: 10.1200/JCO.2006.09.6305.
14. Van Cutsem E, Tabernero J, Lakomy R et al. Addition of aflibercept tofluorouracil, leucovorin, and irinotecan improves survival in a phase III randomized trial in patients with metastatic colorectal cancer previously treated with an oxaliplatin-based regimen. J Clin Oncol 2012; 30 (28): 3499–3506. doi: 10.1200/JCO.2012.42.8201.
15. Peeters M, Price TJ, Cervantes A et al. Randomized phase III study of panitumumab with fluorouracil, leucovorin, and irinotecan (FOLFIRI) compared with FOLFIRI alone as second-line treatment in patients with metastatic colorectal cancer. J Clin Oncol 2010; 28 (31): 4706–4713. doi: 10.1200/JCO.2009.27.6055.
16. Bennouna J, Sastre J, Arnold D et al. Continuation of bevacizumab after first progression in metastatic colorectal cancer (ML18147): a randomised phase 3 trial. Lancet Oncol 2013; 14 (1): 29–37. doi: 10.1016/S1470-2045 (12) 70477-1.
17. Jonker DJ, O’Callaghan CJ, Karapetis CS et al. Cetuximab for the treatment of colorectal cancer. N Engl J Med 2007; 357 (20): 2040–2048. doi: 10.1056/NEJMoa071834.
18. Grothey A, Cutsem EV, Sobrero A et al. Regorafenib monotherapy for previously treated metastatic colorectal cancer (CORRECT): an international, multicentre, randomised, placebo-controlled, phase 3 trial. Lancet 2013; 381 (9863): 303–312. doi: 10.1016/S0140-6736 (12) 61900-X.
19. Romond EH, Perez EA, Bryant J et al. Trastuzumab plus adjuvant chemotherapy for operable HER2-positive breast cancer. N Engl J Med 2005; 353 (16): 1673–1684. doi: 10.1056/NEJMoa052122.
20. Slamon DJ, Leyland-Jones B, Shak S et al. Use of chemotherapy plus a monoclonal antibody against HER2 for metastatic breast cancer that overexpresses HER2. N Engl J Med 2001; 344 (11): 783–792. doi: 10.1056/NEJM200103153441101.
21. Kaufman B, Mackey JR, Clemens MR et al. Trastuzumab plus anastrozole versus anastrozole alone for the treatment of postmenopausal women with human epidermal growth factor receptor 2-positive, hormone receptor-positive metastatic breast cancer: results from the randomized phase III TAnDEM study. J Clin Oncol 2009; 27 (33): 5529–5537. doi: 10.1200/JCO.2008.20.6847.
22. Geyer CE, Forster J, Lindquist D et al. Lapatinib plus capecitabine for HER2-positive advanced breast cancer. N Engl J Med 2006; 355 (26): 2733–2743. doi: 10.1056/NEJMoa064320.
23. Miller K, Wang M, Gralow J et al. Paclitaxel plus bevacizumab versus paclitaxel alone for metastatic breast cancer. N Engl J Med 2007; 357 (26): 2666–2676. doi: 10.1056/NEJMoa072113.
24. Swain SM, Baselga J, Kim SB et al. Pertuzumab, trastuzumab, and docetaxel in HER2-positive metastatic breast cancer. N Engl J Med 2015; 372 (8): 724–734. doi: 10.1056/NEJMoa1413513.
25. Verma S, Miles D, Gianni L et al. Trastuzumab emtansine for HER2-positive advanced breast cancer. N Engl J Med 2012; 367 (19): 1783–1791. doi: 10.1056/NEJMoa1209124.
26. Cortes J, O’Shaughnessy J, Loesch D et al. Eribulin monotherapy versus treatment of physician’s choice in patients with metastatic breast cancer (EMBRACE): a phase 3 open-label randomised study. Lancet 2011; 377 (9769): 914–923. doi: 10.1016/S0140-6736 (11) 60070-6.
27. Baselga J, Campone M, Piccart M et al. Everolimus in postmenopausal hormone receptor-positive advanced breast cancer. N Engl J Med 2012; 366 (6): 520–529. doi: 10.1056/NEJMoa1109653.
28. Zhou C, Wu YL, Chen G et al. Erlotinib versus chemotherapy as first-line treatment for patients with advanced EGFR mutation-positive non small cell lung cancer (OPTIMAL, CTONG-0802): a multicentre, open-label, randomised, phase 3 study. Lancet Oncol 2011; 12 (8): 735–742. doi: 10.1016/S1470-2045 (11) 70184-X.
29. Fukuoka M, Wu Y-L, Thongprasert S et al. Biomarker analyses and final overall survival results from a phase III, randomized, open-label, first-line study of gefitinib versus carboplatin/paclitaxel in clinically selected patients with advanced non-small-cell lung cancer in Asia (IPASS). J Clin Oncol 2011; 29 (21): 2866–2874. doi: 10.1200/JCO.2010.33.4235.
30. Sequist LV, Yang JC, Yamamoto N et al. Phase III study of afatinib or cisplatin plus pemetrexed in patients with metastatic lung adenocarcinoma with EGFR mutations. J Clin Oncol 2013; 31 (21): 3327–3334. doi: 10.1200/JCO.2012.44.2806.
31. Solomon BJ, Mok T, Kim DW et al. First-line crizotinib versus chemotherapy in ALK-positive lung cancer. N Engl J Med 2014; 371 (23): 2167–2177. doi: 10.1056/NEJMoa1408440.
32. Scagliotti GV, Parikh P, von Pawel J et al. Phase III study comparing cisplatin plus gemcitabine with cisplatin plus pemetrexed in chemotherapy-naive patients with advanced-stage non-small-cell lung cancer. J Clin Oncol 2008; 26 (21): 3543–3551. doi: 10.1200/JCO.2007.15.0375.
33. Ciuleanu T, Brodowicz T, Zielinski C et al. Maintenance pemetrexed plus best supportive care versus placebo plus best supportive care for non-small-cell lung cancer: a randomised, double-blind, phase 3 study. Lancet 2009; 374 (9699): 1432–1440. doi: 10.1016/S0140-6736 (09) 61497-5.
34. Sandler A, Gray R, Perry MC et al. Paclitaxel-carboplatin alone or with bevacizumab for non-small-cell lung cancer. N Engl J Med 2006; 355 (24): 2542–2550. doi: 10.1056/NEJMoa061884.
35. Rini B, Escudier B, Tomczak P et al. Comparative effectiveness of axitinib versus sorafenib in advanced renal cell carcinoma (AXIS): a randomised phase 3 trial. Lancet 2011; 378 (9807): 1931–1939. doi: 10.1016/S0140-6736 (11) 61613-9.
36. Motzer RJ, Hutson TE, Tomczak P et al. Overall survival and updated results for sunitinib compared with interferon alfa in patients with metastatic renal cell carcinoma. J Clin Oncol 2009; 27 (22): 3584–3590. doi: 10.1200/JCO.2008.20.1293.
37. Motzer RJ, Hutson TE, Cella D et al. Pazopanib versus sunitinib in metastatic renal-cell carcinoma. N Engl J Med 2013; 369 (8): 722–731. doi: 10.1056/NEJMoa1303989.
38. Motzer RJ, Escudier B, Oudard S et al. Efficacy of everolimus in advanced renal cell carcinoma: a double-blind, randomised, placebo-controlled phase III trial. Lancet 2008; 372 (9637): 449–456. doi: 10.1016/S0140-6736 (08) 61039-9.
39. Hudes G, Carducci M, Tomczak P et al. Temsirolimus, interferon alfa, or both for advanced renal-cell carcinoma. N Engl J Med 2007; 356 (22): 2271–2281. doi: 10.1056/NEJMoa066838.
40. Escudier B, Pluzanska A, Koralewski P et al. Bevacizumab plus interferon alfa-2a for treatment of metastatic renal cell carcinoma: a randomised, double-blind phase III trial. Lancet 2008; 370 (9605): 2103–2111. doi: 10.1016/S0140-6736 (07) 61904-7.
41. Escudier B, Eisen T, Stadler WM et al. Sorafenib in advanced clear-cell renal-cell carcinoma. N Engl J Med 2007; 356 (2): 125–134. doi: 10.1056/NEJMoa060655.
42. Long GV, Stroyakovskiy D, Gogas H et al. Dabrafenib and trametinib versus dabrafenib and placebo for Val600 BRAF-mutant melanoma: a multicentre, double-blind, phase 3 randomised controlled trial. Lancet 2015; 386 (9992): 444–451. doi: 10.1016/S0140-6736 (15) 60898-4.
43. Flaherty K, Davies MA, Grob JJ et al. Genomic analysis and 3-y efficacy and safety update of COMBI-d: A phase 3 study of dabrafenib (D) + trametinib (T) vs D monotherapy in patients (pts) with unresectable or metastatic BRAF V600E/K-mutant cutaneous melanoma. J Clin Oncol 2016; 34: abstr. 9502.
44. Robert C, Karaszewska B, Schachter J et al. Three-year estimate of overall survival in COMBI-v, a randomized phase 3 study evaluating first-line dabrafenib (D) + trametinib (T) in patients (pts) with unresectable or metastatic BRAF V600E/K–mutant cutaneous melanoma. Ann Oncol 2016; 27 (Suppl 6): LBA40. doi: 10.1093/annonc/mdw435.37.
45. Robert C, Long GV, Brady B et al. Nivolumab in previously untreated melanoma without BRAF mutation. N Engl J Med 2015; 372 (4): 320–330. doi: 10.1056/NEJMoa1412082.
46. McArthur GA, Chapman PB, Robert C et al. Safety and efficacy of vemurafenib in BRAF (V600E) and BRAF (V600K) mutation-positive melanoma (BRIM-3): extended follow-up of a phase 3, randomised, open-label study. Lancet Oncol 2014; 15 (3): 323–332. doi: 10.1016/S1470-2045 (14) 70012-9.
47. Hauschild A, Grob JJ, Demidov LV et al. Dabrafenib in BRAF-mutated metastatic melanoma: a multicentre, open-label, phase 3 randomised controlled trial. Lancet 2012; 380 (9839): 358–365. doi: 10.1016/S0140-6736 (12) 60868-X.
48. Hauschild J, Grobb L, Demidov LV et al. An update on overall survival (OS) and follow-on therapies in BREAK-3. Ann Oncol 2014; 25 (Suppl 4): iv374–iv393. abstract 1092PD.
49. Hodi FS, O’Day SJ, McDermott DF et al. Improved survival with ipilimumab in patients with metastatic melanoma. N Engl J Med 2010; 363 (8): 711–723. doi: 10.1056/NEJMoa1003466.
50. Beer TM, Armstrong AJ, Rathkopf DE et al. Enzalutamide in metastatic prostate cancer before chemotherapy. N Engl J Med 2014; 371 (5): 424–433. doi: 10.1056/NEJMoa1405095.
51. Ryan CJ, Smith MR, de Bono JS et al. Abiraterone in metastatic prostate cancer without previous chemotherapy. N Engl J Med 2013; 368 (2): 138–148. doi: 10.1056/NEJMoa1209096.
52. Scher HI, Fizazi K, Saad F et al. Increased survival with enzalutamide in prostate cancer after chemotherapy. N Engl J Med 2012; 367 (13): 1187–1197. doi: 10.1056/NEJMoa1207506.
53. de Bono JS, Logothetis CJ, Molina A et al. Abiraterone and increased survival in metastatic prostate cancer. N Engl J Med 2011; 364 (21): 1995–2005. doi: 1056/NEJMoa1014618.
54. de Bono JS, Oudard S, Ozguroglu M et al. Prednisone plus cabazitaxel or mitoxantrone for metastatic castration-resistant prostate cancer progressing after docetaxel treatment: a randomised open-label trial. Lancet 2010; 376 (9747): 1147–1154. doi: 10.1016/S0140-6736 (10) 61389-X.
55. Parker C, Nilsson S, Heinrich D et al. Alpha emitter radium-223 and survival in metastatic prostate cancer. N Engl J Med 2013; 369 (3): 213–223. doi: 10.1056/NEJMoa1213755.
56. Llovet JM, Ricci S, Mazzaferro V et al. Sorafenib in advanced hepatocellular carcinoma. N Engl J Med 2008; 359 (4): 378–390. doi: 10.1056/NEJMoa0708857.
57. Lu LC, Shao YY, Chan SY el al. Clinical characteristics of advanced hepatocellular carcinoma patients with prolonged survival in the era of anti-angiogenic targeted-therapy. Anticancer Res 2014; 34 (2): 1047–1052.
58. Bang YJ, Van Cutsem E, Feyereislova A et al. Trastuzumab in combination with chemotherapy versus chemotherapy alone for treatment of HER2-positive advanced gastric or gastro-oesophageal junction cancer (ToGA): a phase 3, open-label, randomised controlled trial. Lancet 2010; 376 (9742): 687–697. doi: 10.1016/S0140-6736 (10) 61121-X.
59. Perren TJ, Swart AM, Pfisterer J et al. A phase 3 trial of bevacizumab in ovarian cancer. N Engl J Med 2011; 365 (26): 2484–2496. doi: 10.1056/NEJMoa1103799.
60. Bellmunt J, Théodore C, Demkov T et al. Phase III trial of vinflunine plus best supportive care compared with best supportive care alone after a platinum-containing regimen in patients with advanced transitional cell carcinoma of the urothelial tract. J Clin Oncol 2009; 27 (27): 4454–4461. doi: 10.1200/JCO.2008.20.5534.
Labels
Paediatric clinical oncology Surgery Clinical oncologyArticle was published in
Clinical Oncology
![Issue 5](https://www.prolekare.cz/media/cache/resolve/journal_issue/media/image_pdf/f3199336f587b851bd1c60af16642c6e.jpeg)
2017 Issue 5
Most read in this issue
- Controversy in the Postoperative Treatment of Low-grade Gliomas
- The Role of Chemotherapy in the Treatment of Low-grade Gliomas
- Isocitrate Dehydrogenase Mutations are Better Prognostic Marker than O6-methylguanine-DNA Methyltransferase Promoter Methylation in Glioblastomas – a Retrospective, Single-centre Molecular Genetics Study of Gliomas
- Treatment Refusal in Pediatric Oncology