Linked to EAHP Statements:
Section 1 – Introductory statements and governance
Section 2 - Selection, Procurement and Distribution
ACPE UAN: 0475-0000-16-021-L04-P. An application based activity.
Rare diseases are life threatening or chronically debilitating, with a prevalence lower than 5 in 10 000 Europeans. For patients who suffer from rare diseases it isn't uncommon to go for diagnosis and treatment in different EU-states due to the rarity of the condition.
Today more than 115 orphan drugs are authorised within all EU Member States for the diagnosis, prevention and treatment of rare diseases. Besides dispensing through the supply chain all pharmacy services for these patients need to be harmonized that focus on various aspects of the use of orphan drugs such as cost and reimbursement, adherence to (oral) medication, off-label and unlicensed use, compounding procedures, pharmacovigilance, companion diagnostics, adaptive pathways and home treatment.
New partnerships with patient organisations, small/medium sized enterprises and academia in the diagnosis, prevention and treatment of these patients will be discussed as well as new technologies such as advanced therapy medicinal products, hospital exemption, direct-to-consumer genetic testing, full genome newborn screening, public private partnership in drug development and cross border healthcare.
• To define the responsibilities of the hospital pharmacists in the production and dispensing of new technologies such as advanced therapy medicinal products;
• To highlight the opportunities of the new partnerships for patients with (ultra)rare diseases;
• To demonstrate the necessity of a European platform for pharmacists with special interest in rare diseases.