Should the governance of individual treatment attempts (″Individuelle Heilversuche ″) include praxis evaluation? Results from qualitative stakeholder interviews.

Abstract

Introduction: Individual treatment attempts (ITAs) are a German concept for the treatment of individual patients by physicians with nonstandard therapeutic approaches. ITAs span from nonstandard off-label drug uses to first–in–human uses of newly developed drugs/ interventions. Due to the lack of evidence, ITAs come with a high amount of uncertainty regarding the risk-benefit ratio. At present, no prospective review and no systematic retrospective evaluation of ITAs are required in Germany; therefore, no opportunity exists for the basic evaluation of the frequency, type, or outcomes of ITAs. Our objective was to explore stakeholders′ attitudes toward the retrospective evaluation (monitoring) or prospective evaluation (review) of ITAs. Methods: We conducted a qualitative interview study among relevant stakeholder groups. We used the SWOT framework to represent the stakeholders′ attitudes. We applied content analysis to the recorded and transcribed interviews in MAXQDA. Results: Twenty interviewees participated in the study. The interviewees pointed to several arguments in favor of the retrospective evaluation of ITAs, such as the knowledge gain about and setting and circumstances of ITAs. At the same time, the interviewees expressed concerns regarding the validity and practical relevance of the evaluation results. The viewpoints on review addressed several contextual factors (e.g., different medical disciplines) that should be acknowledged when judging the necessity of a review. Conclusion: One main conclusion is that the current situation with a complete lack of evaluation insufficiently reflects safety concerns. German health policy decision makers should be more explicit about where and why some sort of evaluation is needed or not needed. Another conclusion is that there is no one-size-fits-all model for evaluating ITAs. Both prospective and retrospective evaluations should be piloted in areas of ITAs with particularly high risks and conflicts of interest, such as in the application of very experimental therapies (e.g., bench-to-bedside applications) outside clinical trials.