最近美国仿制药行业将有一位新成员进入的消息引起了很大反响，但却并没有很多关于这一新成员计划如何运营的信息。据 Drug Store 新闻网报道“不久将有一家新的仿制药制造商出现，一家拥有 450 家医院的基地。四大卫生系统 — 位于盐湖城的 Intermountain 医疗保健与 Ascension、SSM 医疗和 Trinity 医疗，正在与退伍军人事务部协商，共同建立一个非营利的仿制药公司。”
据报道，这个新成员是非营利性的，也许这就是其计划如何保证较低药价，但这是假设他们也可以从规模经济中获益，因此这一因素也应被考虑在计划之内。问题是，它将如何运转？如果新公司拥有自己的 FDA 批准的生产设施，则公司必须要么购买现有的设施，要么建造新的生产场地。后者可能是一个漫长的过程。如果他们计划在这个新场地生产仿制药，那么他们需要建立一个 CGMP 合规设施，并构建监管、质量和合规基础设施来实现他们的目标。然后需要有必要的 ANDA 监管申报以及必须获得的州和联邦许可。
如果他们使用合同生产场地，在商业化之前仍必须通过 FDA 监管申报程序。生产、相关的质量和合规工作成本，以及监管提交流程仍是固定的。
另一方面，如果他们计划作为 503B 外包设施运营，制造目前 FDA 短缺清单之外的药品，那么 FDA 关于与已获批产品实质一致的新指南可能会影响其最终目标。【FDA 2018 配药政策重点计划 2018/01/24】 他们是否计划集中在短缺清单上的非肠道药品？他们是否计划作为 503B 设施生产固体口服制剂，这就会引发生物等效性问题，尤其是对于关键或窄治疗指数的药品，以及与在上述指南中表达的当前 FDA 观点相关的问题。
这个想法非常有意思，但是关于实施、采购和批准等大量没有得到解决的问题，使得这种方法的可行性存在一定程度的不确定性。记得 Hatch-Waxman 法案实施早期的时候，那时我还在 FDA 工作，我们担心创新者会全力涌入仿制药市场，或者使用降价来匹配仿制药价格以保留市场份额。后一种方法并没有真正实现，只有少数公司实际上获得了自己的仿制 ANDA 的独立批准，但是许多公司使用授权仿制药（品牌产品标为仿制药）作为保留市场份额的策略。
Drug Price Competition and Patent Term Restoration Act of 1984 (Hatch-Waxman Act)
Mandatory Reading:
Regulatory Affairs (Reg)
Intellectual Property (IP)
Quality Assurance (QA)
Legal Department
Work Suggestions:
Reg: Ensure the company's drug applications comply with the new drug application procedures and bioequivalence standards.
IP: Monitor patent term extensions and the impact on the company's patent strategy.
QA: Verify that manufacturing processes meet the identity, strength, quality, and purity requirements.
Legal Department: Advise on patent infringement issues and the legal implications of abbreviated new drug applications.
Scope of Application: The Drug Price Competition and Patent Term Restoration Act of 1984 applies to chemical drugs, including new molecular entities and generic drugs, in the United States. It is intended for regulatory bodies, pharmaceutical companies, and legal entities involved in drug development and approval processes.
Key Points Summary:
Abbreviated New Drug Applications (ANDAs): The Act allows for the streamlined approval of generic drugs by submitting abbreviated applications showing bioequivalence to the listed drug, without repeating costly and time-consuming clinical trials.
Patent Term Restoration: Offers a mechanism to extend the effective patent life of a drug to partially compensate for the time lost during the regulatory review process, up to a maximum of five years.
Data Exclusivity: Provides a period of data exclusivity, during which the FDA cannot approve ANDAs for other companies that rely on the innovator's safety and efficacy data.
Patent Certification: Requires ANDA applicants to certify about the listed drug's patents or periods of exclusivity, which can trigger a patent infringement lawsuit.
Regulatory Review Period: Defines the regulatory review period for calculating patent term extensions and sets rules for due diligence during the application process.
Conclusion: The Drug Price Competition and Patent Term Restoration Act of 1984 is a landmark legislation that balances the need for accessible, affordable medications with the incentive for innovation. It has significantly impacted the pharmaceutical industry by fostering competition and ensuring that both innovator and generic drug companies have clear pathways to market. The above points are not exhaustive; for comprehensive understanding, the full text of the Act should be consulted.
