Artikel
The impact of German benefit assessment on endpoint selection in clinical research and drug development in Germany – lessons (to be) learned
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Veröffentlicht: | 25. September 2014 |
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Since 2011, the German social insurance code requires the submission of a dossier on product benefit for newly licensed medicinal products. Based on these dossiers, additional benefit is acknowledged by the Federal Joint Committee (G-BA), the highest decision-making body in the German healthcare system. Following the rules of evidence based medicine, the additional benefit of the novel product over a so called appropriate comparator has to be evaluated for endpoints which are relevant for the patients. The additional benefit shall be demonstrated on valid endpoints reflecting mortality, morbidity, quality of life and/or patient safety. However, discrepancies between regulatory requirements and acceptance of endpoints by the G-BA have led to heated debates, disappointments and even market withdrawals. A common example is progression-free survival within oncological indications – an efficacy endpoint commonly accepted by regulatory authorities, however often disregarded by the G-BA due to lack of surrogate validity and relevance for the patients. Similarly, other common endpoints reflecting disease severity or activity based on laboratory or imaging findings are often considered not relevant for the patients. On the other hand, assessments of quality of life are now not only mandatory, but could indeed generate an additional benefit, if certain quality requirements (e.g. instrument validity) are considered. Therefore, careful consideration of patient-relevance of study endpoints and their operationalization are crucial prerequisites for achieving an additional benefit.