Challenges and Solutions for the Benefit Assessment of Tumor-Agnostic Therapies in Germany

Precision medicine is increasingly important in cancer treatment. Tumor-agnostic therapies are used regardless of tumor entity because they target specific biomarkers in tumors. In Germany, the benefit assessment of oncological pharmaceuticals has traditionally been entity specific. Thus, the assessment of tumor-agnostic therapies leaves stakeholders with various challenges. Our aim was to systematically identify challenges and possible solutions for the benefit assessment of therapies in tumor-agnostic indications using a 2-step sequential qualitative approach. To identify relevant challenges, we conducted qualitative interviews with different stakeholders who were involved in previous benefit assessments of tumor-agnostic therapies in Germany. To identify possible solutions for these challenges, we systematically searched MEDLINE, Embase, and the websites of European health technology assessment bodies for relevant literature. We identified 9 categories of challenges of which the following were deemed particularly relevant: the absence of direct comparative studies, challenges regarding the use of basket studies and indirect comparisons, challenges in determining the appropriate comparative therapy in a tumor-agnostic indication, and challenges on the system side. Seven categories of solutions were identified, including an increased use of real-world evidence, making conditional decisions in the context of systematic reassessments, splitting the field of application, and finding (new) ways to design and analyze basket studies. A range of possible solutions, which can help to meet the identified challenges in Germany, have been found. Future research should investigate the acceptance and feasibility of these solutions. •The benefit assessment of tumor-agnostic therapies leaves stakeholders with various challenges.•The article systemized these challenges and identified possible solutions for the benefit assessment of tumor-agnostic therapies in Germany. Categories of challenges included the determination of the appropriate comparator, no comparative studies, and basket studies. Solutions proposed in the literature are manifold and include, for example, the increased use of real-world evidence.•We identified a range of possible solutions, which could guide future reforms, in Germany and beyond, toward more value in health. Future research should investigate the acceptance and feasibility of these solutions.