Gold Ralf, Toumi Mondher, Meesen Bianca, Fogarty Emer
Department of Neurology, St. Josef-Hospital, Ruhr-University Bochum, Bochum, Germany
Department of Complex Decision Sciences and Health Policies, University of Lyon, Lyon, France.
Mult Scler. 2016 Aug;22(2 Suppl):60-70. doi: 10.1177/1352458516650743.
In Europe, there exists considerable variability in access to care and treatment for multiple sclerosis (MS).
To improve this situation, we identified key issues payers should take into account when making decisions on access to care and treatment for MS. We also give an overview of the different dimensions determining total MS burden and discuss why it is key to integrate the patient's perspective in estimating this burden.
The total burden of MS relates to three dimensions: clinical, humanistic and economic. Although the clinical burden is extensively studied, crucial information is still missing about MS pathophysiology, how MS-related symptoms will develop during the disease course and which patients will progress more rapidly. With regard to the humanistic burden, information on patient-reported quality of life systematically collected in clinical trials for registration purposes is still scarce. Early engagement between pharmaceutical companies, the European Medicines Agency and health technology agencies to prospectively identify key evidence needs for the regulatory and reimbursement processes is required as a first step towards more equal access to care and treatment in MS in Europe. Patients' expectations regarding treatment outcomes should be better researched and integrated into decision-making and patients should be counselled in this process.
在欧洲，多发性硬化症（MS）的护理和治疗可及性存在很大差异。
为改善这种情况，我们确定了支付方在就MS的护理和治疗可及性做出决策时应考虑的关键问题。我们还概述了决定MS总负担的不同维度，并讨论了为何在估计这一负担时将患者的观点纳入其中至关重要。
MS的总负担涉及三个维度：临床、人文和经济。尽管对临床负担进行了广泛研究，但关于MS病理生理学、MS相关症状在疾病过程中将如何发展以及哪些患者病情进展更快等关键信息仍然缺失。关于人文负担，在临床试验中为注册目的而系统收集的患者报告的生活质量信息仍然很少。制药公司、欧洲药品管理局和卫生技术机构之间应尽早开展合作，前瞻性地确定监管和报销流程所需的关键证据，这是欧洲实现MS护理和治疗更平等可及性的第一步。应更好地研究患者对治疗结果的期望并将其纳入决策过程，在此过程中应为患者提供咨询。
