National Center for Priority Setting in Health-Care, Linköping University, Sweden and Academy for Health, Welfare and Work-life, University of Borås, Sweden.
Swedish Agency for Health Technology Assessment and Assessment of Social Services and The Dental and Pharmaceutical Benefits Agency, Sweden.
Semin Cancer Biol. 2017 Aug;45:58-63. doi: 10.1016/j.semcancer.2017.05.009. Epub 2017 May 31.
There is a strong patient demand for early access to potentially beneficial cancer drugs. In line with this authorization agencies like the European Medicines Agency are providing drugs with conditional market authorisation based on positive interim analyses. This implies that drugs are used with insecure evidence of efficacy and adverse side-effects. Several authors have pointed to ethical problems with such a system but up to date no indepth ethical analysis of this system is found which is the aim of this article. Drawing of the four generally accepted principles of medical ethics: beneficence, nonmaleficence, respect for autonomy and justice the ethical pros and cons of conditional market authorisation are analysed. From the perspective of beneficence and non-maleficence it is found that the main problem is not risk of adverse side-effects to patients, but rather risk of less beneficial outcomes than what can be expected which could change incentives for patients' choice of treatment. This is also related to the extent to which patients might make an autonomous choice, especially taking into account problematic psychological attitudes and biases in medical decision-making. However, the main problem is related to justice and an equitable distribution of scarce health-care resources given the opportunity cost of drugs treatment. When using resources on cancer treatments which later might be found to be less efficacious than was first expected, other patients (in and outside the cancer field) are deprived of potentially more beneficial treatments even though their needs might be equally or more severe. At the same time, demanding more evidence has an ethical cost to patients in terms of depriving them of potential benefits in terms of reduced mortality and morbidity. In order to handle these ethical conflicts further research and analyses are required and it is suggested that pricing strategies and information requirements are alternatives to be further explored.
患者强烈要求尽早获得可能有益的癌症药物。为了满足这一需求，欧洲药品管理局等授权机构正在根据中期分析的积极结果，为有条件批准的药物提供市场授权。这意味着这些药物的疗效和不良反应证据并不确定。一些作者已经指出了这种制度存在的伦理问题，但截至目前，尚未发现对该制度进行深入的伦理分析，本文旨在对此进行探讨。本文从医学伦理的四个公认原则——有利、不伤害、尊重自主权和公正出发，分析了有条件市场授权的伦理利弊。从有利和不伤害的角度来看，主要问题不是患者不良反应的风险，而是治疗结果不如预期的可能性更大，这可能会改变患者对治疗选择的激励。这也与患者自主选择的程度有关，特别是要考虑到在医疗决策中存在的心理态度问题和偏见。然而，主要问题涉及公平和稀缺卫生保健资源的公平分配，因为药物治疗存在机会成本。当利用资源治疗癌症时，后来发现这些药物的疗效不如最初预期的那么有效，那么其他患者（癌症领域内外）就会被剥夺潜在更有效的治疗方法，即使他们的需求可能同样或更严重。同时，对更多证据的要求会给患者带来伦理成本，使他们失去降低死亡率和发病率的潜在益处。为了处理这些伦理冲突，需要进一步的研究和分析，建议进一步探讨定价策略和信息要求等替代方案。
