循证建立我国FSMP综合评价指标体系及其转化研究

Chronic diseases has became the first global burden of diseases. The prevalence of malnutrition in patients with chronic diseases is too high to influence the clinical outcomes. The Food for Special Medical Purpose(FSMP) has played an increasingly important role in the treatment of chronic diseases with the aging of population, and has huge market potential in China. Recently, the Food Safety Law clearly stipulates CFDA to establish the strict registration and management for FSMP in China. Although the “general rule” and “good manufacturing practice” for FSMP had published in 2014, no technical specifications of clinical trial and evaluation criterion for the registration of FSMP till now, which will surely affect the implement of the new regulations for the registration and management of FSMP in the future, and hinder the clinical application of FSMP. Therefore, this project aims to take diabetes mellitus as example, to build evaluation index system and decision-making model for FSMP from clinical efficacy, safety, economics, and applicability using evidence-based methods and Delphi methods, and conduct retrospective and prospective studies to increase the transformation of the aboving evaluation index system and model; explore and establish monitoring systme to reassess FSMP in the post-market; coutinuously improve the evaluation index system, decision-making model and monitoring systme via demonstration expansion from Sichuan province to southwestern region, and finally the whole country. The project aims to provide scientific basis for the government to standardize management of FSMP in the future, and promote the construction of clinical nutrition disciplines and the healthy development of FSMP industry.

慢性病已上升为全球疾病负担首位，患者营养不良发生率高，严重影响临床结局。随着我国人口日益老龄化，特殊医学用途食品（FSMP）已在慢病治疗中扮演越来越重要的角色，市场潜力巨大。新《食品安全法》明确要求食药局对FSMP实施严格注册管理。尽管我国2014年已出台FSMP通则及良好生产规范，但至今仍缺指导FSMP注册的临床试验技术规范及评价标准，这将极大限制FSMP注册管理办法实施，难以有效保障FSMP的临床应用。本项目拟用循证方法结合Delphi专家咨询法，以糖尿病为例，从FSMP安全性/有效性/经济性/适用性四个维度，构建我国FSMP评价指标体系/决策模式，采用回顾性/前瞻性人群试验验证其可转化性；首次探索建立FSMP应用监测系统，进行上市后再评价；将评价指标体系和监测系统在省/地区/全国逐级推广转化并加以完善。旨在为政府规范FSMP注册管理提供科学依据，推动临床营养学科及FSMP产业健康发展

特殊医学用途食品（FSMP）在慢病营养治疗中扮演越来越重要的角色，为保障临床治疗质量，国家市场监督管理总局要求对特定全营养配方食品进行上市前临床试验，但缺指导临床试验的技术和评价标准。产品上市后规范化管理是保障临床营养质量的重要环节，而目前我国医疗机构针对FSMP临床应用的后效评价研究几乎为空白。本项目通过循证+Delphi专家咨询的方法，结合原始研究构建了糖尿病FSMP上市前临床评价指标体系，经循证+问卷调查的方式全面了解了我国医疗机构FSMP临床供给、应用及管理现状，基于上述指标体系和调查情况，设计开发了医疗机构FSMP临床应用及不良反应监测系统，经多轮培训后在多家医院推广应用。经过系统评价及三轮的专家咨询，最终获得了包括安全性、营养性、有效性、经济性等4个一级指标在内的27个末级评价指标，并制定了临床试验结果的判断标准；通过问卷星调查全国20多个省份592家医疗机构，发现我国医院中FSMP供应无法满足临床需求，FSMP临床应用监测技术、方法、流程不统一，没有实现电子化和数据化，无统一规范的监管模式。项目组就FSMP不良反应监测系统开展了2次全省培训，目前系统内注册单位达174家，已有45家医院报告161例FSMP不良反应，与四川省特殊食品处联合发布《四川省不良反应监测管理办法》指导全省医疗机构FSMP不良反应监测工作。基于第一部分建立的评价指标体系，结合临床及营养专家意见初步开发了医疗机构FSMP临床应用信息化系统，目前已完成试运行，收集数据达2万余条。本项目成果将为我国FSMP上市前产品评审，上市后临床应用监测、后效评价提供科学依据和平台支撑，促进FSMP产业健康发展，提升临床营养治疗质量。