随着多发性硬化症(MS)疗法的专利到期,后续疾病改善治疗(FO-DMT)以更低的成本获得。人们担心更便宜的FO-DMT仅仅用于降低医疗保健成本。然而,MS患者的福祉应优先考虑。
通过评估已发表的证据,就原则和共识声明达成共识,确定FO-DMT开发和使用的最佳实践。
经过系统审查,我们制定了五项总体原则和13项共识声明。来自17个欧洲国家的多学科小组对原则和声明进行了表决,阿根廷,加拿大和美国。
所有原则和陈述都得到了80%以上的小组成员的认可。简而言之,在高度监管区域内批准的FO-DMT可以被认为是有效和安全的参考产品;FO-DMT可以逐案评估,并不总是需要III期试验;需要长期的药物警戒和透明度;缺乏FO-DMT之间的多重和交叉转换的证据;需要教育来解决剩余的问题。
发布的数据支持在MS中使用FO-DMT。共识可能有助于共同决策。虽然我们的共识集中在欧洲,结果可能有助于提高其他地方使用FO-DMT的质量标准。
As patents for multiple sclerosis (MS) therapies expire, follow-on disease-modifying treatments (FO-DMTs) become available at reduced cost. Concerns exist that cheaper FO-DMTs are used simply to reduce healthcare costs. However, the well-being of people with MS should take priority.
To identify best practices for FO-DMT development and use by agreeing on principles and consensus statements through appraisal of published evidence.
Following a systematic review, we formulated five overarching principles and 13 consensus statements. Principles and statements were voted on by a multidisciplinary panel from 17 European countries, Argentina, Canada and the United States.
All principles and statements were endorsed by >80% of panellists. In brief, FO-DMTs approved within highly regulated areas can be considered effective and safe as their reference products; FO-DMTs can be evaluated case by case and do not always require Phase III trials; long-term pharmacovigilance and transparency are needed; there is lack of evidence for multiple- and cross-switching among FO-DMTs; and education is needed to address remaining concerns.
Published data support the use of FO-DMTs in MS. The consensus may aid shared decision-making. While our consensus focused on Europe, the results may contribute to enhanced quality standards for FO-DMTs use elsewhere.