Major Determinants of Delayed Access to Innovative Medicines for Metastatic Melanoma: The Results of the Melanoma World Society (MWS) and European Association of Dermato-Oncology (EADO) Survey - European Medical Journal

Major Determinants of Delayed Access to Innovative Medicines for Metastatic Melanoma: The Results of the Melanoma World Society (MWS) and European Association of Dermato-Oncology (EADO) Survey

3 Mins
*Lidija Kandolf Sekulovic,1 Sanjiv Agarwala,2 Gabriela Cinat,3 Axel Hauschild,4 Claus Garbe5

Dr Kandolf Sekulovic has no conflict of interest to declare regarding this article but has received relevant financial funding for activities outside the submitted work, which are speaker’s fees from Roche, Novartis, Bristol-Myers Squibb, and MSD. Dr Agarwala has received travel and accommodations expenses from MSD and Bristol-Myers Squibb. Dr Hauschild has received clinical trial support, speaker´s honoraria, or consultancy fees from Amgen, Bristol-Myers Squibb, Merck Serono, MSD, Novartis, Oncosec, Philogen, Pierre Fabre, Provectus, Regeneron, and Roche. Dr Cinat has received speaker’s honoraria or consultancy fees from Novartis, MSD, Bristol-Myers Squibb, Merck Serono, and Roche. Dr Garbe has declared no conflicts of interest.


The authors would like to acknowledge the support of their coauthors involved in the survey. Dr Kandolf Sekulovic was responsible for preparation of this ESMO abstract summary.

EMJ Oncol. ;6[1]:76-78. Abstract Review No. AR5.

Each article is made available under the terms of the Creative Commons Attribution-Non Commercial 4.0 License.

At the 2018 European Society for Medical Oncology (ESMO) Congress in Munich, Germany, the Melanoma World Society (MWS) and European Association of Dermato-Oncology (EADO) presented the results of a survey assessing worldwide access to first-line recommended treatments for metastatic melanoma and the major determinants of access.

Metastatic melanoma is a chemotherapy-resistant cancer with a median survival of 6–9 months prior to 2010.1 In recent years, a major breakthrough was achieved with targeted therapy and immunotherapy, leading, for the first time, to significantly prolonged survival for this group of patients, with nearly 50% of patients in good prognostic groups surviving up to 5 years based on recent trials.2-5 However, despite the high efficacy of targeted therapy and immunotherapy, they have high costs, which has led to restricted access to these treatments in parts of Europe; in 2016, >5,000 patients did not have access to these treatments.6 Significant delays in reimbursement and different insurance coverage are some of the challenges healthcare systems face when trying to adapt to the rising costs of cancer care.6 In this setting, there is a clear need for an objective measurement of clinical benefit of every treatment and development of value-based pricing. The American Society of Clinical Oncology (ASCO) framework net clinical benefit 16 score and the ESMO magnitude of clinical benefit score have both been developed with the intention of being used for developing pricing and prioritisation of medicines for reimbursement and/or insurance coverage.7,8

The degree of inequality and major determinants of access to innovative treatments for metastatic melanoma have been largely unexplored. The MWS and EADO conducted a web-based survey on access to first-line recommended treatments for metastatic melanoma by current guidelines (National Comprehensive Cancer Network [NCCN], ESMO, and European Organisation for Research and Treatment of Cancer [EORTC]/EADO/European Dermatology Forum [EDF]) among melanoma experts from 1st September 2017–1st December 2018 from 34 countries: the USA, China, Australia, Argentina, Brazil, Chile, Mexico, and 27 European countries. Data on licensing and reimbursement of medicines and the number of patients treated were correlated with the data on health expenditure per capita, Mackenbach score of health policy performance, health technology assessment, and the ASCO and ESMO magnitude of clinical benefit scores of clinical benefit and market price of medicines.7-10 Regression analysis for evaluation of correlation between the parameters was carried out using SPSS software.

In this study, the estimated number of patients without access in surveyed countries worldwide was 10,131 (Table 1). The recommended BRAFi+ MEKi combination and anti-PD1 immunotherapy were registered and fully reimbursed in 17 (50.0%) of the countries, and anti-CTLA4+ anti-PD1 combination in 9 (26.4%) countries. In 14 (41.1%) countries, the majority of patients were treated according to the recommended guidelines. Median delay in reimbursement was 871 days (range: 0–1,274 days). These results were in correlation with ASCO (rho=0.819; p=0.004), and ESMO scores of clinical benefit (rho=0.933, p<0.01) and median market price (rho=0.694, p=0.026), as well as with health expenditure per capita, health policy performance scores, and health technology assessment implementation (p<0.05). The medicines with the highest scores of clinical benefits were the ones with the longest delay in access. In the majority of countries (64.2%) price negotiations or managed entry agreements with national authorities were necessary for reimbursement.10

Table 1: Estimated number of patients without access to innovative medicines in surveyed countries.
NA: data not available.

In conclusion, great discrepancy exists in metastatic melanoma treatment globally. Access to innovative medicines correlated with economic parameters as well as with healthcare system performance parameters. Patient-orientated drug development, market access, and reimbursement pathways must be urgently found.

Eigentler TK et al. Palliative therapy of disseminated malignant melanoma: A systematic review of 41 randomised clinical trials. Lancet Oncol. 2003;4(12): 748-59. Ugurel S et al. Survival of patients with advanced metastatic melanoma: The impact of novel therapies-update 2017. Eur J Cancer. 2017;83:247-57. Long GV et al. Long-term outcomes in patients with BRAF V600-mutant metastatic melanoma who received dabrafenib combined with trametinib. J Clin Oncol. 2018;36(7):667-73. Callahan MK et al. Nivolumab plus ipilimumab in patients with advanced melanoma: Updated survival, response, and safety data in a Phase I dose-escalation study. J Clin Oncol. 2018;36(4):391-5. Hodi SF et al. Durable, long-term survival in previously treated patients with advanced melanoma (MEL) who received nivolumab (NIVO) monotherapy in a Phase I trial. Abstract CT001. AACR Annual Meeting, 16-20 April, 2016. Kandolf Sekulovic L et al. More than 5000 patients with metastatic melanoma in Europe per year do not have access to recommended first-line innovative treatments. Eur J Cancer. 2017;75:313-22. Schnipper LE et al. Updating the American Society of Clinical Oncology Value Framework: Revisions and reflections in response to comments received. J Clin Oncol. 2016;34(24):2925-34. Cherny NI et al. ESMO-Magnitude of Clinical Benefit Scale version 1.1. Ann Oncol. 2017;28(10):2340-66. Mackenbach JP, McKee M. A comparative analysis of health policy performance in 43 European countries. Eur J Publ Health. 2013;23(2):195-201. Kandolf Sekulovic L et al. Access to innovative medicines for metastatic melanoma worldwide: Melanoma World Society and European Association of Dermato-oncology survey in 34 countries. Eur J Cancer. 2018;104:201-9.

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