Évaluation des médicaments anticancéreux par la commission de la transparence : choix des comparateurs et conséquences sur l'accès au marché

Abstract : Given the increase of costly innovations and because of the need to control health expenditures, the goal of regulators is to allow early access to drugs while paying the fair price and ensuring their financial sustainability. Within the framework of a value-based pricing system, reimbursement and pricing decisions are based on Health Technology Assessment bodies’ appraisals which determined the relative efficacy of new medicines as compared to existing alternatives. In France, the clinical assessment performed by the French National Authority for Health (HAS)’s Transparency Committee (TC) is one of the key step to regulate health expenditures. As the TC principles are grounded on evidence-based medicine, this assessment should be ideally supported by randomised controlled trials (RCT) versus clinically relevant comparators (CCP) used in clinical practice at the same place in the treatment pathway. The comparator choice is a major and strategic component of medicines market access because it directly impacts evidence quality, the relevance of data and finally pricing. An observational study was conducted at HAS to estimate the place of comparators in the assessment of oncology treatment by the TC. The study suggests that the relevance of comparators has an impact on the clinical added value score (ASMR) of medicines which could impact pricing and market access considerations. At this time, in absence of a new integrative methodology, RCT versus CCP seems to remain the best design to establish the relative effectiveness of medicines.
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Master Thesis
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Henri Marfin. Évaluation des médicaments anticancéreux par la commission de la transparence : choix des comparateurs et conséquences sur l'accès au marché. Sciences pharmaceutiques. 2018. ⟨dumas-01963033⟩

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