Ferrario, Alessandra
(2016)
Access to cancer medicines in Europe: an analysis of existing challenges and countries’ responses.
PhD thesis, London School of Economics and Political Science.
Abstract
Access to new cancer medicines, particularly their coverage and affordability, is a matter of great concern to payers, patients and the pharmaceutical industry. Yet, little comparative evidence on actual use and its determinants is available. This thesis aimed to analyse the extent to which access to cancer medicines varies across a sample of European countries, what determines these differences, whether they matter, and what are countries doing to improve access. These objectives were achieved through the use of quantitative and qualitative methods including multilevel mixed effects models, survival analysis and the complementary log-log transformation of the Cox proportional hazard model, literature reviews and interviews to inform the development of conceptual frameworks, and a comparative longitudinal analysis of the implementation of MEAs. Results show that there are wide cross-country differences in access to cancer medicines in Belgium, Estonia, Scotland, and Sweden. These differences were determined by time to entry of new medicines, medical need (i.e. incidence of the disease) and factors affecting treatment decisions (e.g. coverage, prices, and financing mechanisms). The added clinical value of medicines explained the shorter time to launch in Estonia and higher consumption in Scotland but not in the other countries. This emphasises the importance of health technology assessment processes in identifying and making cost-effective medicines available and limiting the utilisation of non-cost-effective ones. The latter is particularly important given the opportunity cost of funding new cancer medicines. Managed entry agreements have increasingly been implemented to address issues of uncertainty and high prices when making coverage decisions. Through their action on price, effectiveness and use, they are able to influence cost-effectiveness and budget impact, two key variables guiding reimbursement decisions. Some of the most important contributions from this thesis include: (1) the development of a blueprint to analyse access to cancer medicines in Europe quantitatively and qualitatively; (2) the identification of likely determinants of differences in access to cancer medicines which can guide policy-makers efforts in managing access to new therapies; and (3) the development of a conceptual framework for managed entry agreements which can support their design and evaluation.
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