Generic Substitution of Orphan Drugs for the Treatment of Rare Diseases: Exploring the Potential Challenges
Generic Substitution of Orphan Drugs for the Treatment of Rare Diseases: Exploring the Potential Challenges
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DOI：
10.1007/s40265-018-0882-x
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发表时间：
2018-03-01
期刊：
影响因子：
11.5
通讯作者：
Arrigoni, Elena
中科院分区：
文献类型：
--
作者：
Di Paolo, Antonello;Arrigoni, Elena
仿制药是医疗监管部门为降低治疗成本而推出的措施的重要组成部分。在大多数患者和条件下，从品牌药物转换到其仿制药的过程中没有出现重大并发症。然而，来自复杂疾病的证据表明，仿制药替代在某些情况下需要仔细评估，目前的生物等效性标准可能并不总是足以确定品牌产品和仿制药的互换性。罕见疾病，也称为孤儿疾病，是一组具有重要特征的异质性疾病：除了稀缺性外，大多数都是严重的、慢性的、非常虚弱的，通常出现在幼儿时期。找到一种罕见疾病的治疗方法是具有挑战性的。由于鼓励罕见疾病研究和开发计划的激励措施，目前有几种孤儿药物可用。孤儿药物价格的上涨是一个备受争议的问题，也是许多患者获得治疗机会有限的原因之一。由于几种孤儿药的专利保护和专营期即将到期，孤儿药的仿制药应该很快就会上市，人们对其对罕见病经济负担的影响寄予厚望。然而，与其他复杂的疾病一样，仿制药的替代可能需要深思熟虑，在一些罕见的情况下甚至可能是禁忌。这篇文章概述了罕见疾病的特征，回顾了有问题的仿制药替代的报道，并讨论了为什么孤儿药物的仿制药替代可能具有挑战性，应该在罕见疾病患者中谨慎地进行。
Generic drugs are important components of measures introduced by healthcare regulatory authorities to reduce treatment costs. In most patients and conditions the switch from a branded drug to its generic counterpart is performed with no major complications. However, evidence from complex diseases suggests that generic substitution requires careful evaluation in some settings and that current bioequivalence criteria may not always be adequate for establishing the interchangeability of branded and generic products. Rare diseases, also called orphan diseases, are a group of heterogeneous diseases that share important characteristics: in addition to their scarcity, most are severe, chronic, highly debilitating, and often present in early childhood. Finding a treatment for a rare disease is challenging. Thanks to incentives that encourage research and development programs in rare diseases, several orphan drugs are currently available. The elevated cost of orphan drugs is a highly debated issue and a cause of limited access to treatment for many patients. As patent protection and the exclusivity period of several orphan drugs will expire soon, generic versions of orphan drugs should reach the market shortly, with great expectations about their impact on the economic burden of rare diseases. However, consistent with other complex diseases, generic substitution may require thoughtful considerations and may be even contraindicated in some rare conditions. This article provides an overview of rare disease characteristics, reviews reports of problematic generic substitution, and discusses why generic substitution of orphan drugs may be challenging and should be undertaken carefully in rare disease patients.