Access to information supporting availability of medicines for patients suffering from rare diseases looking for possible treatments: the EuOrphan Service

Abstract

Currently in Europe, approximately 30 million people suffer from rare diseases, and a major problem is that many patients do not have access to quality healthcare for their disorders. Moreover, there is also a lack of quality information and a networking system aimed at supporting interaction among patients, clinicians, researchers, pharmaceutical industries, and governmental bodies. The purpose of this article is to inform physicians, public health care professionals, and other health care providers about EuOrphan service, the aim of which is to ensure easier access to quality information on rare diseases and their treatment. A set of web-based services is available at www.euorphan.com where information for target-users on treatments and products available worldwide for rare disease care as well as indications about healthcare centers are provided. Moreover, the service aims at providing consultancies for pharmaceutical companies to ultimately support the European legislation in bringing new drugs of a high ethical standard to the market and to exert a positive impact on the large population of patients suffering from rare diseases in Europe. The services provided by EuOrphan can facilitate concrete networking among patients, patient associations, doctors, and companies and also support the organization of clinical trials. In this perspective, EuOrphan could become a very valuable tool for globalizing the information about the availability of treatment (authorized or under development) of orphan patients.

Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs

“…In particular, the rare conditions with the highest number of failures were in the oncology area and included: glioma (12), acute myelogenous leukaemia (11), pancreatic cancer (10) and chronic lymphocytic leukaemia (7). In the respiratory group, six failures were for cystic fibrosis.…”

Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen

“…EuOrphan is a database focused on drugs aimed to diagnose, prevent or treat a rare disease. It was created by Consorzio per Valutazioni Biologiche e Farmacologiche in the framework of a funded European IT-Technology project (eTen 510774 2003/C 118/19), as described by Stakišaitis and coll [15]. Since 2008 it is voluntary managed and regularly updated by Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus [16].…”

Reimbursed Orphan Medicines in Bulgaria and the Share of Biotechnology-Derived Products

“…diseases and orphan medicines was performed (27)(28)(29)(30)(31)(32)(33). the regulatory publications were reviewed and analysed from the perspective of their role for ensuring an access to adequate pharmacotherapy.…”