美国特朗普总统的新任 FDA 局长 Scott Gottleib 医师正在拉开解决诸如孤儿药认定积压、努力启动《药品质量与安全保障法案》要求的落实、阿片类药物流行以及最重要的减少仿制药竞争障碍的序幕。在 6 月 21 日的博客中，Gottlieb 局长以下面的话开篇：
“太多患者因药价太高而不能获得他们所需的药品。虽然 FDA 在药品定价方面无法直接发挥作用，但我们可以通过批准低成本的仿制药来促进处方药市场竞争提升，从而帮助解决这一问题。”
高昂的品牌药价格是美国当前的药品问题，许多较少的新产品以及私人保险支付系统看起来对药品成本不敏感（只要保费保持上涨）的结果。仿制药承担着美国医疗保健的负担，以最便宜的价格提供 90% 的药品处方供应。仿制药定价由等同仿制药产品之间的竞争驱动。对于给定的产品，缺乏仿制药等同产品或拥有太少的仿制药竞争者，可以通过解决这两个最为重要的因素进一步降低美国药价。Gottlieb 局长将这两个问题作为 FDA 工作重点。
Gottlieb 局长的这篇博客值得再次阅读，因为此文解决了围绕仿制药批准的若干问题。【FDA 正努力撤除仿制药竞争障碍 2017/06/22】 FDA 迄今已采取了三项行动。首先，他们宣布将于 7 月 18 日举行公开会议寻求有关仿制药批准障碍的意见，从而帮助 FDA 了解仿制药研发商面临的问题范围。其次，修订了《MaPP 5240.3 第3 版 原始ANDA、增补和补充审评的优先级》，以确保除其它标准之外，对于少于 3 件获批 ANDA 且没有专利或专营权限制的产品 ANDA 将获得优先审评。最后，FDA 发布了《无已获批仿制药的专利到期、专营期到期品牌药清单》。
该清单列出了属于这一类别的产品，但未给出缺少 ANDA 的原因。一些可能的原因是：一些产品可能仅仅是因为产量太小而不足以支持 ANDA；一些可能没有生物等效性方法或方法太贵而不值得开展；一些可能没有可用的参照标准品，或者是因为没有上市的参照标准品或者是因为限制分销的 REMS。对于这些看似广泛开放的品牌药产品缺乏仿制药竞争的原因的全面理解将等待对每个没有等同仿制药的产品分配原因。
作者：识林-Pepper
编译：识林-蓝杉
英文原文
FDA Commissioner Gottlieb tackles Barriers to Generic Drug Approval
President Trump’s new FDA Commissioner, Scott Gottleib MD is off to a flying start addressing issues such as the backlog of Orphan Drug Designations, working to kick start the Drug Quality & Security Act requirements, addressing the Opioid Epidemic, and most importantly working to reduce barriers to Generic Drug Competition. In a blog post dated June 21, 2017 Commissioner Gottlieb starts by saying:
“Too many patients are being priced out of the medicines they need. While FDA doesn’t have a direct role in drug pricing, we can take steps to help address this problem by facilitating increased competition in the market for prescription drugs through the approval of lower-cost, generic medicines.”
Sky high brand drug prices are the pharmaceutical issue of the moment in the US, a result of many fewer new products and the private insurance payment system seemingly insensitive to drug cost (so long as insurance premiums keep on rising). Generic drugs shoulder the health care burden in the US providing 90% of all drug prescriptions filled at some of the cheapest prices in the world. Generic drug pricing is driven by competition among equal generic drug products. The lack of a generic equivalent or having too few generic competitors for a given product are the two most important factors that can be addressed to further drive down drug prices in the US. Commissioner Gottlieb has identified both of these issues as a priority for FDA to address.
The blog posting (https://blogs.fda.gov/fdavoice/index.php/2017/06/fda-working-to-lift-barriers-to-generic-drug-competition/) is worth reading as it addresses a number of issues around generic drug approvals. FDA has taken three actions so far. They have announced a meeting on July 18th to seek input on barriers to generic drug approval and so help FDA understand the scope of the issues facing generic dug developers. They have revised the MAPP that addresses prioritization of ANDA review (MAPP 5240.3 Rev. 3 Prioritization of the Review of Original ANDAs, Amendments, and Supplements) to ensure that, among other criteria, ANDAs for products with fewer than 3 approved ANDAs and no patent or exclusivity block will receive priority review. Finally FDA released a list of all brand products that have no patent or exclusivity block to approval and no approved ANDAs.
This list of products with no approved ANDAs and no patent or exclusivity block to approval list the products in this category but gives no reason for the lack of ANDAs. There are a number of possible reasons, some product may simply be too small in volume to support and ANDA, some may not have bioequivalence methodology or have methodology which is too expensive to be worth doing. Some may not have a reference standard available, either because it is not marketed or due to a restricted distribution REMS. A full understanding of the reasons for the lack of generic competition for these seemingly wide open brand products will await the assignment of the reason(s) for each product not having a generic equivalent.
