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

Background: Decision-makers have implemented a variety of value assessment frameworks
(VAFs) for orphan drugs in European jurisdictions, which has contributed to variations in …

Background The reimbursement of orphan drugs (OD) is an increasingly important for
country policymakers, and still insufficiently understood, especially in Central and Eastern …

Patients with chronic lymphocytic leukemia (CLL) have a high risk of poor outcomes related
to coronavirus disease 2019 (COVID-19). This multicenter cohort study evaluated the impact …

Background There are about 7000 rare diseases worldwide, of which only 5% of the
diseases can be treated with medicines, showing that it's important to improve patient …

Objectives: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators,
Kalydeco®(ivacaftor), Orkambi®(lumacaftor/ivacaftor) and Symkevi®(tezacaftor/ivacaftor) …

Introduction Ensuring both financial and physical access to medicines is a challenge for the
reimbursement system. How countries are currently tackling this challenge is an issue worth …

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 …

This study aimed to analyze four current pathways affecting the listing and post-listing prices
of new orphan drugs (ODs) in South Korea. These mechanisms were:(1) essential OD,(2) …

Highly efficacious, potentially curative gene therapies holds immense clinical promise, but
also present complex challenges. At the time of regulatory approval and health technology …