Update on Impact of The Additional Benefit Extent of Orphan Drugs on Price Negotiations In The German Outpatient Sector

Abstract

For orphan drugs an additional benefit is granted by market authorization of the EMA .[1] However, companies have to demonstrate the extent of this additional benefit as basis for subsequent reimbursement price negotiations by submitting a simplified dossier to the Federal Joint Committee.[2]The objective of this analysis was to assess whether the additional benefit extent of orphan drugs does impact the rebate size of the price negotiations. The hypothesis indicated an inversely proportional correlation between additional benefit extend and negotiated rebate size. In a first step orphan substances affected by an early benefit assessment were identified within the German healthcare market. Correlation between additional benefit extent and rebate amount after negotiations was analyzed by Spearman correlation analysis. Data were collected from publicly available information of the Federal Joint Committee as well as price information from the German pharmacy pricing data base LAUER-TAXE[3]. By May 2017, 99[4] orphan drugs with active orphan drug designation were identified in the European Union. 40[5] were currently distributed in the outpatient German healthcare market and underwent additional benefit extent assessment as well as price negotiations. Correlation between additional benefit extent and rebate size could only be identified if the category “not quantifiable additional benefit” was excluded. There is only limited correlation between the extent of additional benefit and rebate size for orphan drugs. This is mainly caused by a large rebate range for orphan drugs for which the additional benefit extend was not quantifiable. Further factors impacting the price negotiation were identified as: European comparison prices, treatment area, negotiation management and prevalence of indication. [1] European Medicines Agency, European Community, Regulation (EC) No 726/2004. [2] German courtesy translation: Gemeinsamer Bundesausschuss, Nutzenbewertung nach § 35a SGB V, 19.05.2017: https://www.gba.de/informationen/nutzenbewertung/. [3] LAUER-TAXE®, 19.05.2017. [4] EuropeanMedicines Agency, 19.05.2017 [5] Database Quintiles Commercial Germany GmbH 19.05.2017