gms | German Medical Science

Background: In Germany newly authorized pharmaceuticals have to undergo the procedure of the early benefit assessment since 2011. The assessment focuses on patient benefits and the findings of the early assessment to form the basis for subsequent price negotiations. The assessment is solely based on data from clinical trials. Against this backdrop we will examine which data could enrich the assessment. We aim to better include patient relevant outcomes, which may be achievable in real-life health care settings.

Materials and Methods: We base the analysis on statutory and sub-statutory regulations together with the conducted assessments and appraisals. The evaluation of relevant clinical and non-interventional studies is included in the analysis.

Results: Early benefit assessment leaves decisive questions unanswered, since the additional benefit resulting from random controlled trials (RCT) is not necessarily identical with the benefit in real life settings. However, patient relevant benefits become apparent only in real life settings. Contrary to RCT, non-interventional studies (NIS) are based on everyday care, i. e. non interference by the treating physician in regard to indication or method and implementation of the treatment. Nevertheless, NIS are not accepted as proof of benefit. The high standard set for health care studies by G-BA make it difficult to evaluate which benefits the patient can profit from in the end.

Therefore the term “patient relevance” for the purpose of benefit assessment is to be questioned: Following a paternalistic attitude IQWiG and G-BA are currently entitled to define standards despite well known methods which support the patient’s involvement when it comes to the essential question of what is relevant to him.

Conclusion: Notwithstanding the limitations inherent in NIS, they art a valuable contribution towards benefit assessment of new treatment options. If patient relevance is taken seriously, greater attention must be paid to “Patient Reported Outcomes” and patient’s preferences.