西班牙高级治疗性研究药物临床试验（2004-2022）：未来的经验与挑战。

Clinical Trials on Advanced Therapy Investigational Medicinal Products in Spain (2004-2022): Experience and Challenges for the Future.

机构信息

Clinical Trials Division, Department for Human Medicinal Products, Spanish Agency for Medicines and Medical Devices (AEMPS), Madrid, Spain.

Division for Biological Products, Advanced Therapies and Biotechnology, Department for Human Medicinal Products, Spanish Agency for Medicines and Medical Devices (AEMPS), Madrid, Spain.

出版信息

Adv Exp Med Biol. 2023;1430:23-39. doi: 10.1007/978-3-031-34567-8_2.

DOI:10.1007/978-3-031-34567-8_2

PMID:37526840

Abstract

Clinical investigation is the basis for establishing how useful advanced therapy investigational medicinal products (ATiMP) are for the treatment of serious diseases.In Spain, clinical trials (CT) on ATiMP need to follow the general European legislation on CT with medicinal products plus some specific legislation and guidance depending on the type of ATiMP.This chapter describes the characteristics of CT on ATiMP authorized in Spain in the period 2004-2022 and the legislation applicable along this period. There are clear differences in the clinical trials conducted by non commercial and commercial sponsors: the first have been more involved in CT on somatic cell therapy medicinal products (sCTMP) and tissue-engineered products (TEP), while the second drive more the CT on gene therapy medicinal products (GTMP) in the last years. Difficulties of budget and resources especially by non-commercial sponsors to meet the regulatory requirements are highlighted. The importance of complying with transparency rules with respect to CT on ATiMP is also discussed.

摘要

临床研究是确定先进治疗性医药产品（ATiMP）在治疗严重疾病方面有效性的基础。在西班牙，ATiMP 的临床试验（CT）需要遵循欧洲关于 CT 和医药产品的一般立法，再加上一些特定的立法和指导方针，具体取决于 ATiMP 的类型。本章描述了 2004 年至 2022 年期间在西班牙获得授权的 ATiMP 临床试验的特点，以及这期间适用的立法。非商业和商业赞助商进行的临床试验存在明显差异：前者更多地参与体细胞治疗医药产品（sCTMP）和组织工程产品（TEP）的 CT，而后者在最近几年更多地推动基因治疗医药产品（GTMP）的 CT。非商业赞助商在满足监管要求方面面临预算和资源方面的困难尤为突出。本文还讨论了遵守 ATiMP CT 透明度规则的重要性。