Many conditions that we work with in Fighting Blindness are classified as a rare disease. This means that the condition affects less than one in 2,000 individuals. Common issues facing people and families living with any rare diseases include delayed diagnosis, access to care and medication and feelings of isolation.
Orphan products are medicines intended for the diagnosis, prevention, and treatment of rare diseases. Pharmaceutical companies that are developing therapies for a rare disease can apply to the European Medicines Agency (EMA) for orphan designation. This means that the company can benefit from a number of incentives, including protocol assistance, and market exclusivity once the medicine is on the market. The medicine must fulfil certain criteria for designation as an orphan medicine. Orphan drugs are typically very expensive and it can be difficult to get them reimbursed for use in the public health system. Many of the therapies which will become available in the future for our conditions are likely to be expensive orphan drugs. It is vital that we in Fighting Blindness understand the procedures involved to effectively advocate for equitable access to therapies for all people affected.
ECRD 2016 took place in Edinburgh from May 26 to 28. The conference covers research, development of new treatments, healthcare, social care, information, public health and support at European, national and regional levels. Fighting Blindness presented a poster at the conference about the Target 5000 research project and attended workshops about research, diagnosis and treatments for rare diseases. For more information about the conference please visit www.rare-diseases.eu.