Abstract
BackgroundThe introduction of the orphan drug legislation led to the increase in the number of available orphan drugs, but the access to them is often limited due to the high price. Social preferences regarding funding orphan drugs as well as the criteria taken into consideration while setting the price remain unclear. The study aimed at identifying the determinant of orphan drug prices in France using a regression analysis.MethodsAll drugs with a valid orphan designation at the moment of launch for which the price was available in France were included in the analysis. The selection of covariates was based on a literature review and included drug characteristics (Anatomical Therapeutic Chemical (ATC) class, treatment line, age of target population), diseases characteristics (severity, prevalence, availability of alternative therapeutic options), health technology assessment (HTA) details (actual benefit (AB) and improvement in actual benefit (IAB) scores, delay between the HTA and commercialisation), and study characteristics (type of study, comparator, type of endpoint). The main data sources were European public assessment reports, HTA reports, summaries of opinion on orphan designation of the European Medicines Agency, and the French insurance database of drugs and tariffs. A generalized regression model was developed to test the association between the annual treatment cost and selected covariates.ResultsA total of 68 drugs were included. The mean annual treatment cost was €96,518. In the univariate analysis, the ATC class (p = 0.01), availability of alternative treatment options (p = 0.02) and the prevalence (p = 0.02) showed a significant correlation with the annual cost. The multivariate analysis demonstrated significant association between the annual cost and availability of alternative treatment options, ATC class, IAB score, type of comparator in the pivotal clinical trial, as well as commercialisation date and delay between the HTA and commercialisation.ConclusionThe orphan drug pricing is a multivariate phenomenon. The complex association between drug prices and the studied attributes and shows that payers integrate multiple variables in decision making when setting orphan drug prices. The interpretation of the study results is limited by the small sample size and the complex data structure.
Highlights
The introduction of the orphan drug legislation led to the increase in the number of available orphan drugs, but the access to them is often limited due to the high price
Almost 100 orphan molecules holding a marketing authorization (MA) were identified through the European Medicines Agency (EMA) database of orphan drug designations
The results demonstrated that repurposed orphan drugs, oral formulation and availability of alternative treatments were associated with lower annual treatment costs, while multiple orphan indications, drugs for chronic treatments and improvement in overall survival or QoL were associated with higher annual treatment costs
Summary
The introduction of the orphan drug legislation led to the increase in the number of available orphan drugs, but the access to them is often limited due to the high price. Small number of potential patients and, low profitability of drugs for rare conditions made them unattractive for investments. In order to guarantee access to appropriate treatments for patients suffering from rare diseases, governments of several countries introduced a special orphan legislation [1]. Granting an orphan designation allows a drug. Introducing the orphan legislation led to the fast growth of the number of authorized orphan products. More than 100 orphan drugs have been granted a MA by the European Medicines Agency (EMA) since the establishment of orphan drug designation in 2000 [2].
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