Abstract
IntroductionIt is well acknowledged that the price of orphan drugs is normally higher than that resulting from the value-based pricing. A correlation between the cost of therapy for orphan drugs and the epidemiology (prevalence and incidence) of the related rare disease can be hypothesized.MethodsThis analysis includes all approved orphan drugs by European Medicines Agency whose reimbursement was granted for the first therapeutic indication in the years 2014–2019 in Italy. Regression and correlation analyses were performed to analyze the possible correlations between the logarithm of the annual therapy cost and the epidemiology of the rare diseases, between orphan drugs consumption and epidemiology of related rare disease and between therapy cost and the consumption.ResultsThe regression analysis between the annual cost of therapy estimated on the published ex-factory price and the prevalence showed a slightly decreasing, not statistically significant, trend (coefficient: −0.10, p-value: 0.41). The results were similar when using the price resulting from the application of Managed Entry Agreements (coefficient: −0.11, p-value: 0.40). The regression analysis between sales volume and prevalence showed a positive slope without an acceptable level of significance (p-value: 0.04). The correlation analysis between the therapy cost and the sales volume highlighted again an absence of significant association, similarly if considering only ATC L orphan drugs, or the incidence.DiscussionThe definition of the price of an orphan drug seems not to depend on the rarity of the disease, and sales volumes do not correlate with the epidemiology of the rare disease and with the annual cost of therapy.
Highlights
It is well acknowledged that the price of orphan drugs is normally higher than that resulting from the value-based pricing
In Europe, to receive an orphan designation by the European Medicines Agency (EMA), a drug must be intended for use in the treatment of a life-threatening or chronically debilitating disease with a EU prevalence being no more than 5 in 10,000 inhabitants (
Out of 89 orphan drugs which have applied for pricing and reimbursement (P&R) in Italy during the 2014–2019 period, the analysis included 58 drugs which were granted the reimbursability (A/H class; Figure 1)
Summary
It is well acknowledged that the price of orphan drugs is normally higher than that resulting from the value-based pricing. The current European legislation requires orphan medicinal products to have access to the centralized marketing authorization procedure in order to ensure early market access [with accelerated assessment programs [2]], as well as several economic incentives to compensate for the potentially low profitability of a medicinal product intended for a limited population of patients [3] With such a designation, manufacturers receive market exclusivity for 10 years (plus two more years if the orphan indication is extended to the pediatric population) in all EU countries and, in addition, financial incentives for research, development, scientific advice, marketing authorization application, inspections, post-authorization activities as well as market access in individual Member States. Manufacturers can submit their dossier immediately (instead of 3 months later, as usual, when regulatory approval is ratified by the European Commission), after a positive Committee for Medicinal Products for Human Use (CHMP) opinion, and be granted priority in the procedure for P&R decisions by the Italian Medicines Agency (AIFA)
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