在运行早期发展研究时，赞助商必须考虑在随机，安慰剂对照试验结束时提供开放标签，长期治疗。过去，赞助商已经犹豫不决，以提供开放标签扩展。但随着我们行业越来越重视患者为中心的护理，赞助商正在称重他们的选择，以将更多患者投入纳入早期发展，特别是在稀有疾病研究中使用有前途的新疗法时。
考虑开放标签扩展的含义
生物技术s and pharmaceutical companies have understandably been reluctant for many reasons. Exposing patients to long-term risks with no extenuating benefit has ethical implications and legal issues may arise with any negative outcome. Financially, implementing a longer trial can translate into higher development and drug production costs.
从临床发展的角度来看，还有一些重要的考虑因素，如：
- 与患者和调查人员相关的偏见了解治疗任务亚博全站官网
- 对治疗成功的预期
- 符合资格患者数量的可能减少未来登记导向研究
- 与想要继续在药物的患者可能发生冲突 - 即使在盲目的试验中，也没有停止过度效力和发展。
Shifting toward a patient-focused approach in rare disease studies
While the implications of open-label extension can seem intimidating, these risks may be offset by the benefits gained from a patient-centric approach in rare disease drug development.
With limited populations to test, each and every patient in a rare disease study is very valuable. An open-label extension helps maximize the amount of information extracted from each patient and gather crucial data that better informs downstream development.
However, incorporating a patient-focused approach takes more than simply running an open-label extension. Sponsors must consider:
- Early engagement with the patient community
- 自然历史数据的设防
- Acceleration of preclinical safety studies
- Early vetting with regulatory agencies
- Robust informed consent development with external input
- Development of a solid long-term plan for data interpretation to provide the reassurance needed to acquire adequate financial support.
随着患者中心的概念和实施继续获得牵引力，提案国将需要保持在这种转变之前，并仔细平衡对罕见疾病的早期获得治疗的需求，同时保护患者的安全性以及有前途的新型治疗的可行性。
