The Orphan Medicines Industry Group (OMIG) is a group of pharmaceutical companies that are members of the Association of the British Pharmaceutical Industry (ABPI) working together for the benefit of patients with rare diseases in the UK.
OMIG works with a range of stakeholders to ensure that people in the UK with rare diseases get access to the medicines they need.
What are orphan medicines?
Orphan medicines are medicines used to treat rare conditions. They are defined as innovative treatments in an area of unmet need for rare but serious diseases with a typical prevalence of less than five per 10,000 population.
In order to be granted orphan medicine status by the European Union (EU), companies must demonstrate that:
The disease or conditions for which the medicinal product would be administered affects not more than five in 10,000 persons in the community at the time at which the application for designation is submitted1
The disease or condition should be of a life-threatening or chronically debilitating nature1
Without incentives it is unlikely that the marketing of the medicinal product in the community would generate sufficient return to justify the necessary investment1
There exists no satisfactory method of diagnosis, prevention or treatment of the condition in question, or if such method exists, the medicinal product will be of significant benefit to those affected by that condition.1
Challenges in accessing treatment
In the UK around 5.7% of the population is affected by a rare disease at any one time.2 Challenges continue to exist in accessing treatments that can help. One of the main reasons why patients do not have access to orphan medicines is that current health technology appraisal (HTA) processes are not appropriate for these medicines.
Even though European marketing authorisation has been granted for 50 orphan drugs, only three have been recommended by NICE.
In Scotland, the Scottish Medicines Consortium (SMC) uses HTA to determine whether orphan medicines should receive funding. As of May 2010, the SMC had appraised 46 orphan medicines and issued the following guidance:
Despite the addition of modifiers to the SMC process, which give special consideration to rare or terminal illness treatments, there has been minimal impact on the positive appraisal of orphan medicines.
Commissioning processes at local level are not appropriate for funding orphan medicines. OMIG welcomes the National Framework4 being developed by the Department of Health and looks forward to working with stakeholders to ensure patients receive the treatments they need.
Data on file
Department of Health, Strengthening national commissioning: a consultation document (2009)