PRO82 How Do RARE Disease Patient Numbers in Germany Affect the Price of NON-Oncology Orphan Drugs?

Abstract

HTA bodies and payers face uncertainties when evaluating rare diseases including the ability to quantify the magnitude of clinical benefit, sizing patient populations, and anticipating budget impact. Having previously qualified the relationship between price and prevalence in rare diseases across EU5 and Japan, how does this look when the spotlight is on Germany? This study analyses 22 non-oncology orphan drugs that have received EMA approval since July 2012 and are chronic, non-curative treatments. Decision (Bechluss) reports from the G-BA (Gemeinsamer Bundesausschuss – Federal Joint Committee) and assessment reports from IQWiG (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen – Institute for Quality and Efficiency in Healthcare) were used to identify the number of patients eligible for treatment in Germany as an indicator of prevalence, calculate the annual treatment cost (ATC) using APU / HAP (Herstellerabgabepreis – manufacturer selling price) in the first indication, and extract the added benefit rating and trial design. This study shows that there is a relationship between price (ATC) and prevalence in which ATC tends to decrease as patient numbers increase. Although the trend appears to be consistent within ranges of patient numbers, it is not linear, and there are some notable deviations. Our research indicates that the non-linearity can be in part explained by exploring other factors beyond patient number that may influence payers’ willingness to pay, namely the G-BA added benefit rating and clinical comparator. In some cases, recognition of added benefit appears to influence price and cause trendline deviations. However, the trial comparator does not seem to impact the ability to demonstrate an added benefit, which indicates payer willingness to value disease rarity and innovation. This study provides evidence that payers in Germany assign some value to disease rarity, though this is only part of the equation during price negotiations.