Abstract
Objective: To assess shares of reimbursed orphan drugs and agreement in reimbursement decision-making in different European Union member states as well as to define odds for reimbursement influenced by the presence of conditional approval or exceptional circumstances granted by the European Medicines Agency (EMA) or by type of the disease.Methods: The list of authorized drugs with current orphan designations was collected from the website of the EMA. For each drug, the information regarding conditional approval or approval under exceptional circumstances was collected. The reimbursement statuses were available on national reimbursement or HTA agencies websites. The agreement for reimbursement decisions between selected countries was assessed using the κ coefficient for the measurement of agreement. The impact of the EMA's conditional approval as well as approval under exceptional circumstances was assessed using the logistic regression and presented as odds ratio.Results: The percentage of reimbursed orphan drugs varied significantly from 27% in Poland to 88% in Denmark, with an average value of 51% (p < 0.0001). Regarding the reimbursement status, the highest, substantial agreement was observed between Spain and Italy, and the lowest agreement was observed between Germany and England, with κ of 0.64 and 0.01, respectively. Conditional approval status significantly decreased the chance for reimbursement in France, Italy, and Spain by 77–80%; however, approval granted under exceptional circumstances had significant impact only in Germany with 85% decrease in chances for reimbursement. The type of the disease (oncology or metabolic) was significantly associated with both conditional approval (p of 0.03—oncology drugs were more likely to be conditionally approved then the rest of analyzed drugs) and exceptional circumstances (p of 0.02—drugs for metabolic diseases were more likely to be approved under exceptional circumstances).Conclusions: Access to reimbursed orphan drugs varies significantly across EU countries. The highest, substantial agreement in reimbursement decisions was observed between Italy and Spain and the lowest between Germany and England. Conditional approval and approval under exceptional circumstances were significant negative predictors of reimbursement in some countries and they were significantly associated with the type of the disease (oncology or metabolic).
Highlights
There is no common definition of an orphan drug, which is the reason for discrepancies among the definitions implemented by different countries in their drug reimbursement decisionmaking process
The highest, substantial agreement was observed between Spain and Italy, and the lowest agreement was detected between Germany and England, with κ of 0.64 and 0.01, respectively (Table 4)
Approval under exceptional circumstances was associated with the reimbursement status only in Germany, where the odds for reimbursement were 85% (OR, 0.15; confidence intervals (CIs) 95%, 0.04–0.53; p = 0.0034) lower for drugs approved under exceptional circumstances when compared with other drugs (Table 6)
Summary
There is no common definition of an orphan drug, which is the reason for discrepancies among the definitions implemented by different countries in their drug reimbursement decisionmaking process. There is general acceptance that the definition should be based on the prevalence of rare diseases treated by orphan drugs. According to the current definition provided by the European Union (EU), rare diseases mostly include inherited, life-threatening, or chronically debilitating diseases that affect fewer than 5 out of 10,000 people (EMEA, 2017). According to the definition by the European Medicines Agency’s (EMA), the prevalence is 5 persons per 10,000, which translates into around 246,000 people affected by rare diseases, considering 27 EU member states (Winstone et al, 2015; EMEA, 2017). Different types of rare diseases can be defined and the broadest categories include oncologic diseases (around 32.5% of all orphan drugs; Gammie et al, 2015) and metabolic conditions.
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