Background: Despite international initiatives on collaboration within the field of rare
diseases, patient access to orphan medicinal products (OMPs) and healthcare services …

Objectives To review recent studies reporting health care expenditures (budgetary impact)
for orphan medicinal products (OMPs) in Europe and to contribute to our understanding of …

Background Successful development of new treatments for rare diseases (RDs) and their
sustainable patient access require overcoming a series of challenges related to research …

Background The number of authorized orphan and non-orphan medicines for rare diseases
has increased in Europe. Patient access to these medicines is affected by high costs, weak …

Background Personalized medicine and orphan drugs share many characteristics—both
target small patient populations, have uncertainties regarding efficacy and safety at payer …

Background Conducting clinical studies in small populations may be very challenging;
therefore quality of clinical evidence may differ between rare and non-rare disease …

Objectives: This research aims to evaluate the methodological quality of budget impact
analyses for orphan drugs and to provide suggestions for future analyses. Methods …

Background and objective Little is known about how much public payers spend on orphan
medicines. This study aimed at identifying information on orphan medicine expenditure …

Purpose Over the last few years, the share of public spending for orphan drugs (ODs) has
increased in several western countries, raising concern on the exemptions granted to this …

Objectives: The aim of the study was to compare the access of patients with rare diseases
(RDs) to biotechnological drugs in several Central and Eastern European countries …