Review of regulatory recommendations for orphan drug submissions in the netherlands and scotland: focus on the underlying pharmacoeconomic evaluations

Commonalities and differences in HTA outcomes: A comparative analysis of five countries and implications for coverage decisions

“…The latter may put more emphasis on other considerations such as the severity of the condition or the need for treatment alternatives; this is well illustrated in the case of orphan drugs, characterized by high and uncertain cost-effectiveness, which in the majority of cases have been rejected in Scotland but are all listed in Sweden. Several studies have identified factors driving access to orphan drugs in different countries, where cost-effectiveness in some countries was complemented by other considerations such as disease severity, or the availability of other treatment alternatives [31,32,49,50,60]. The analysis has also demonstrated that there are different perspectives in the assessment of me-too drugs.…”

“…small patient population often associated with severe disability), it is more difficult to collect sufficient data to demonstrate efficacy and safety in very small patient populations, and as such, orphan drugs are often more prone to significant uncertainty in cost-effectiveness. A number of studies focus specifically on orphan drug characteristics and emphasize their clinical uncertainty and high cost-effectiveness [31,32,49,50], making HTA processes more difficult and subject to interpretation in each setting.…”

Current Problems in Provision of Orphan Drugs and Ways to Solve Them

“…Соответственно, процесс регистрации орфанных лекар-ственных средств проходит по-разному, в зависимости от национального законодательства [15]. Так, в Евросоюзе этот вопрос находится в юрисдикции Комитета по орфан-ным медицинским продуктам Европейского медицинского агентства (European Medicines Agency, EMA) [16]. В стра-нах EC признание продукта орфанным дает фармацевти-ческим компаниям различные преференции, такие как 10-летний статус эксклюзивного производителя, содей-ствие в составлении протокола клинического исследова-ния, снижение взносов для проведения исследований под эгидой EMA и предоставление грантов на клинические исследования [17].…”

Access to orphan drugs in western Europe: can more systematic policymaking really help to avoid different decisions about the same drug?

“…Examples of ERTs are laronidase for mucopolysaccharidosis I, idursulfase for mucopolysaccharidosis II, and agalsidase alfa and beta for Fabry disease. These therapies are associated with high costs, with annual costs ranging from e200,000 to 600,000 per patient [2].…”