Rare & Forgotten Conditions
Research
In Europe, a disease is considered rare when it affects fewer than one in 2,000 people. For some of these rare disease there are drugs approved for treatment. This means they offer more clinical benefits than harm, at an acceptable risk, as determined by the regulatory agency, the European Medicines Agency (EMA). Some are formally designated as ‘orphan’ drugs in the European Union (EU), treating less than 5 in 10,000 people in the EU. However, not all approved drugs will be paid for (reimbursed) by the National Health Service (NHS). That reflects the need to consider value for money. This can be a source of frustration for many: patients, carers, and treating clinicians. Evidence suggests that current access to orphan drugs in the UK is at best inconsistent: whether you look internationally, or speak to clinicians and patient organisations who help individual patients when access is blocked within the UK. This is not a fair situation for those who face serious illness.
Successfully bringing a medicine all the way from initial basic research to the patient is difficult. The pathway for access to orphan drugs includes a number of key decisions: those made by researchers and companies during the research and development (R&D) phase, decisions by regulators, payers and those that advise payers as well as prescribing clinicians who make decisions with their patients.