Abstract
BackgroundSince the implementation of the Regulation on Patient Integration (2003), the Act on the Reorganization of the Pharmaceutical Market (2011), and the Patient Rights Law (2013), the inclusion of patient perspectives has been further anchored in the German early benefit assessment process. During the assessment of rare disease interventions, patient perspectives are particularly important, as clinical studies are often designed acknowledging small samples and patients suffering from severe symptoms and the chronic course of the disease. Therefore, our research question is whether patient perspectives are considered as part of early benefit assessments for rare diseases. We also strive to examine how patient perspectives are methodologically elicited and presented.MethodsOur empirical evidence comes from a systematic review of orphan drug value dossiers submitted to the German Federal Joint Committee as well as the corresponding evaluations conducted between January 1, 2011 and March 1, 2019 (n = 81). Data on patient perspective integration were extracted using the following patient-reported outcome subcategories: clinical patient-reported outcomes, health-related quality of life, patient preferences, and patient satisfaction.ResultsThe analysis demonstrates the specific relevance of patient-reported outcomes raised as part of the medical data set and presented during the early benefit assessment process. They are predominantly presented in the form of health-related quality of life data (n = 75%) and clinical outcomes (n = 49%). Preferences (n = 2%) and satisfaction (n = 1%) are still rarely presented, although the heated methodological discussion in Germany would suggest otherwise. While various methodologies for the integration of clinical outcomes and quality of life data were found, presenting data on satisfaction and preferences still lacks methodological rigor. The German Federal Joint Committee has not yet integrated these data in their decision text. Clinical outcomes and quality of life have been included in 46% and 73% of the cases, respectively.ConclusionsThe underlying analysis demonstrates that there is still a relative high potential for the regular and systematic inclusion of patient perspectives within the early benefit assessment process for rare diseases. In particular, patient preferences and patient satisfaction are still rarely included suggesting the need for a clear-cut methodological foundation and incentives.
Highlights
The relevance of patient perspective integration in health care In Germany, the 2003 Patient Participation Regulation [1] as well as the 2013 Patient Rights Law [2] form the most important basis for the integration of patient perspectives within the health care system
We examine the development of a methodologically grounded and direct approach to patient perspective integration, using the concept of patient reported outcomes, and analyzing the data submitted during early benefit assessments for rare diseases in Germany
It should be noted that, whereas all agents cross passing the 50 million euro limit were commissioned to the IQWiG, applications for additional application areas were assessed by the Gemeinsamer Bundesauschuss – Federal Joint Committee (GBA) itself
Summary
The relevance of patient perspective integration in health care In Germany, the 2003 Patient Participation Regulation [1] as well as the 2013 Patient Rights Law [2] form the most important basis for the integration of patient perspectives within the health care system. Important attributes of patient-oriented health care systems are identified as: high-quality information generation and transparency, patient perspective integration through customization and collaboration, as well as the integration of patient choice and responsibility. Since the implementation of the Regulation on Patient Integration (2003), the Act on the Reorganization of the Pharmaceutical Market (2011), and the Patient Rights Law (2013), the inclusion of patient perspectives has been further anchored in the German early benefit assessment process. We strive to examine how patient perspectives are methodologically elicited and presented
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