Since the 2011 Act on the Reform of the Market for Medicinal Products, benefit dossiers are submitted by pharmaceutical companies to facilitate the Health Technology Assessment (HTA) appraisals in Germany. The Institute for Quality and Efficiency in Health Care conducts the added benefit assessment following their General Methods Paper, which was updated November 5, 2020. This White Paper is dedicated to patient-reported outcomes (PRO) to highlight their importance for the added benefit assessment. We focus on methodological aspects but consider also other relevant requirements and challenges, which are demanded by G-BA and IQWiG. The following topics will be presented and discussed: 1. Role of PRO in HTA decision making exemplary to benefit assessment in Germany 2. Guidances of PRO evaluations 3. PRO Estimand framework 4. Perception and requirements for PRO within the German benefit assessment 5. Validity of instrument 6. Response thresholds for assessing clinical relevance of PRO 7. PRO endpoints / outcome measures / operationalization 8. Missing PRO data 9. PRO after treatment discontinuation This White Paper aims to provide deeper insights about new requirements concerning PRO evaluations for HTA decision making in Germany, highlight points to consider that should inform global development in terms of study planning and frame the requirements also in the context of global recommendations and guidelines. We also aim to enhance the understanding of the complexity when preparing the benefit dossier and promote further scientific discussions where appropriate.
翻译:自2011年《医药产品市场改革法》以来,制药公司提交了福利档案,以便利德国的保健技术评估评估。保健质量和效率研究所在其总方法文件(该文件于2020年11月5日更新)之后,进行了额外福利评估。白皮书专门论述患者报告结果(PRO),以强调其对额外福利评估的重要性。我们侧重于方法方面,但也考虑GBA和IQWiG所要求的其他相关要求和挑战。将介绍和讨论以下专题:1. 项目在德国的保健技术评估决策中起到示范作用,对惠益评估起示范作用。2. 方案评价指南:Esimind框架:4. 德国福利评估中的PRO概念和要求。5. 文书的有效性。 评估PRO的临床相关性的应对阈值:PRO终点/结果措施/运行。8. 治疗结束后,PRO失踪数据。 PRO。本白皮书旨在更深入地了解德国HTA决定对项目评价的新要求。 将提出和讨论以下要点:在研究、规划和确定全球利益要求时,还应指导全球发展,同时促进科学评估的复杂程度。