ACPE UPN: 0475-0000-14-023-L01-P. An application based activity.
Abstract
Rare diseases are life threatening or chronically debilitating diseases with prevalence lower than 5 in 10 000 Europeans. For patients who suffer from rare diseases it is not uncommon to go for diagnosis and treatment in different EU-states because of the rarity of the condition.
Today more than 65 orphan drugs are authorized within all EU Member States for the diagnosis, prevention and treatment of rare diseases. Besides dispensing all pharmacy services for these patients need to be harmonized that focus on several aspects of the use of orphan drugs such as cost and reimbursement, adherence to (oral) medication, off-label and unlicensed use, compounding procedures, pharmacovigilance and home treatment.
New innovative aspects in the diagnosis and treatment of these patients will be discussed such as advanced therapy medicinal products, hospital exemption, direct-to-consumer genetic testing, public private partnership in drug development, cross border healthcare and European tender operations.
Teaching Goals:
- to illustrate the innovative aspects of orphan drugs in the diagnosis, prevention and treatment of patients with rare diseases,
- to define the responsibilities of the hospital pharmacists in the production and dispensing of innovative products such as advanced therapy medicinal products,
- to demonstrate the necessity of a European platform for pharmacists with special interest in rare diseases.
Learning Objectives:
After participating in the workshop the participant should be able:
- to create best practices in the management of orphan drugs,
- to identify the clinical added value of orphan drugs in the diagnosis, prevention and treatment of rare diseases,
- to install clinical pharmacy services to patients with rare diseases.