2 月 14 日的普享药协会（AAM）年会上，美国 FDA 药品审评与研究中心（CDER）主任 Janet Woodcock 就有关在不远的将来开展的仿制药申请新评估方式给了仿制药申请人一些希望。评估新体系的目标是改善审评过程的效率和有效性。为什么这种改变是必要的？Woodcock 承认，随着递交给 FDA 的提交数量不断增加，目前的审评范式不可持续。
Woodcock 表示，虽然首轮批准率在 GDUFA I 期间有所改善，从 GDUFA 实施之前不到 1% 到 2015 财年 10.7%，2016 财年 14.3% 以及 2017 财年 12.8%，但是“我们需要更多的首轮批准”。不提高审评过程的效率，就没有希望首轮批准率达到新药审评计划近 90% 的水平。Woodcock 指出，从 PDUFA I 开始用了 25 年的时间在新药领域取得了这一成就，但希望 GDUFA 的努力能让 ANDA 首轮批准朝着正确的方向发展。
“我们需要更好地理解我们的审评过程的稳定状态，以更好地了解我们将需要多少人员和资源”，以保持投入和产出的平衡。她指出，2017 财年有 2666 件 ANDA 和对 ANDA 的增补提交到 OGD，这比预期的要多得多。Woodcock 承认，FDA 必须明确申请中所需的内容，并明确清楚地向行业提供该信息，以确保提交过程中具有更高的确定性。
新审评范式被称为 KASA（Knowledge Aided Assessment and Structured Application，知识辅助评估和结构化申请），KASA 应使审评过程更多地依赖审查清单决策制定，并限制目前削弱审评人员满足当前工作量能力的冗长叙述。
FDA 在实现这一目标方面还有多远？Woodcock 表示，“我们还没有达到这一目标，可能需要几年的时间才行”，但这是 CDER 高层管理人员首次承认，目前的审评过程不具有可持续性。目前正在开展一个试点项目，涉及在这一新模式中的制剂和原料药。正在开发、完善并将在未来几年内实施审评模块。审评模块将在内部实施完成后推出，希望有助于减少拒绝接收行动的数量，并减少审评周期数。
在 ANDA 评估中寻求的变化会使 FDA 和行业都受益。对于 FDA 来说，可以通过减少审评负担来加快产品上市批准速度；对于行业来说，可以提供更大的审评确定性并避免让审评人员询问了解也好（nice to know）的问题而不是有必要了解（need to know）的问题，这是自 Hatch-Waxman 法案实施以来行业的普遍抱怨。随着新审评范式的形成，需要提交多少文件以及 FDA 审评必需的复杂性和程度如何将有望成为关注焦点。
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.
