Major determinants of delayed access to innovative medicines for metastatic melanoma: The results of Melanoma World Society and European Association of Dermato-Oncology survey
2018-10-01

Background: According to data from the recent studies in Europe and worldwide, a large percentage of patients have restricted access to innovative medicines for metastatic melanoma. In the current study, major determinants of restricted and delayed access to the first-line recommended treatments were analyzed. Methods: Web-based survey was conducted among melanoma experts from 27 European countries, USA, China, Australia, Argentina, Brazil, Chile and Mexico from September 1st to January 1st, 2018. Data on reimbursement of medicines for metastatic melanoma were correlated with: data on health expenditure per capita (HEPC), Mackenbach score of health policy performance, health technology assessment (HTA), ASCO and ESMO scores of clinical benefit market price of medicines and type of reimbursement. Regression analysis was done using SPSS software (significant correlation if p < 0.05). Results: The recommended BRAFi+MEKi combination and anti-PD1 immunotherapy were registered and fully reimbursed in 17/34 (50%) countries, and combination anti-CTLA4+anti-PD1 in 9/34 (26.4%) countries. In only 14/34 (41.1%) countries majority of patients (>90%) were treated according to the recommended guidelines, and estimated number of patients without access in surveyed countries was 10131 patients. Median delay in reimbursement was 871 days (0-1274 days). It was in correlation with ASCO (rho=0.819, p = 0.004), ESMO scores of clinical benefit (rho=0.933, p < 0.01), median market price (rho=0.694, p = 0.026), as well as with HEPC, health policy performance scores and HTA assessment implementation (p < 0.05). In majority of countries (64.2%) price negotiations or managed entry agreements (MEA) with national authorities were necessary for reimbursement. Conclusions: HTA implementation, price negotiations and MEAs were associated with more timely reimbursement. Adding ASCO and ESMO scales of clinical benefit to HTA could better prioritize medicines for reimbursement at international level. Development of patient-oriented research, development, market access and reimbursement mechanisms would be necessary in the future. Legal entity responsible for the study: Melanoma World Society and European Association of Dermato-Oncology. Funding: Has not received any funding. Disclosure: L.O. Kandolf Sekulovic: Speakers' fee: Roche, Novartis, BMS, MSD outside the submitted work. S.S. Agarwala: Travel, accommodations, expenses: MSD, Bristol-Myers Squibb. A. Hauschild: Consulting or advisory role, Travel, accommodations, expenses, Honoraria: Amgen, Bristol-Myers Squibb, MedImmune, Merck Serono, Merck Sharp & Dohme, Novartis, OncoSec, Roche, Nektar, Philogen, Provectus, Regeneron; Research Funding: Amgen, Bristol-Myers Squibb, Celgene, Eisai, GlaxoSmithKline, Merck Serono, Merck Sharp & Dohme, Novartis, Roche. J. Guo: Consulting or advisory role, Travel, accommodations, expenses: Roche, MSD, Bayer, Pfizer. G.A. McArthur: Research funding: Celgene, Genentech/Roche, Bristol-Myers Squibb, Amgen, Array BioPharma, Novartis/Pfizer. P. Lorigan: Consulting or advisory role: Merck Sharp & Dohme, Bristol-Myers Squibb, Novartis, Amgen, GlaxoSmithKline, NeraCare GmbH, Pierre Fabre; Speakers' bureau: Merck Sharp & Dohme, Novartis, Bristol-Myers Squibb, Roche; Travel, accommodations, expenses: Merck Sharp & Dohme, Novartis, Bristol-Myers Squibb. G. Cinat: Consulting or advisory role: MSD, Roche, Novartis, Bristol-Myers Squibb; Research funding: Genentech, Novartis, GlaxoSmithKline, MSD, Bristol-Myers Squibb. C. Caglevich: Consulting or advisory role: MSD, Bayer, AstraZeneca, Bristol-Myers Squibb; Speakers' bureau: MSD, Bayer, Lilly, Bristol-Myers Squibb; Travel, accommodations, expenses: Boehringer Ingelheim, Bristol-Myers Squibb; Research funding: MSD, Bayer, AstraZeneca, Bristol-Myers Squibb, Medivation, Boehringer Ingelheim, GlaxoSmithKline, Roche, Astellas Pharma. C. Lebbe: Honoraria, Consulting or advisory role, Speakers' bureau: Roche, Novartis, MSD, Amgen, Bristol-Myers Squibbs; Research funding: Roche, Bristol-Myers Squibbs. P.L. Rutkowski: Honoraria: MSD, Novartis, Roche, Pfizer, Bristol-Myers Squibbs; Consulting or advisory role: MSD, Novartis, Amgen, Blueprint Medicines. H. Gogas: Consultant or advisory role: BMS, MSD, Amgen, Novartis, Roche, Pierre Fabre; Honoraria: BMS, MSD, Amgen, Novartis, Roche; Travel expenses: Roche, BMS; Research funding: BMS, MSD, Roche, Novartis. R. Dummer: Outside the submitted work: Research funding: Novartis, MSD, BMS, Roche, GSK; Consultant or advisory board: Novartis, MSD, BMS, Roche, GSK, Amgen, Takeda, Pierre Fabre. B. Neyns: Outside the submitted work: Consulting or advisory role: BMS, MSD, Roche, Novartis; Speakers' fees and advisory board meetings: Novartis; Travel, Accommodations, Expenses: BMS, MSD, Roche, Novartis, Amgen; Research funding: Pfizer, Novartis, Merck KGaA; Research funding to the institution: Pfizer, Novartis. L. Bastholt: Advisory boards: Roche, BMS, Merck MSD, Novartis, Eisai; Travel, accommodations, expenses: BMS, Merck MSD, Novartis. A. Wainstein: Travel, accomodation: MSD, BMS, Roche. D. Herceg: Speakers' fees: MSD, Novartis; Consulting or advisory role: BMS; Travel, accommodations, expenses: Roche. C. Garbe: Outside the submitted work: Speakers' fees: Amgen, BMS, MSD, LEO, Roche, Philogen; Research funding: BMS, Novartis, Roche. All other authors have declared no conflicts of interest.