A lack of knowledge and understanding is resulting in inequalities surrounding the prescription of drugs for rare diseases, reports Michael Sobanja
Orphan drugs are medicines that treat rare diseases and 434 products fall into this category. However, with a lack of knowledge in the NHS and inconsistency in funding are patients being treated fairly?
It has been estimated that there are about 6,000-8,000 of these rare conditions in the EU requiring treatment by orphan drugs, out of an approximate total of 30,000 worldwide.
There is considerable variation in the attitude to orphan drugs across Europe and during February the NHS carried out a survey to examine the UK position in more detail. The results demonstrated just how far there is to go if we are to create a level playing field for those needing orphan drugs.
Although 95 per cent of respondents had heard of the term orphan drugs only 55 per cent were aware that they were used in rare conditions, hardly any were aware of the requirements for granting of orphan drug status and nobody was aware of assistance offered by the EU.
Most respondents said there was no local specific process for managing orphan drugs. Funding mechanisms also appeared to be unclear.
There is now an opportunity for a better approach for patients with most rare conditions, with the review of specialist commissioning. Services for rare conditions should be appropriately commissioned and the funding for medicines considered at strategic health authority level. This could help avoid unjustified postcode prescribing.
It is time we took a more rational approach to the treatment of rare conditions and the National Institute for Health and Clinical Excellence, the Scottish Medicines Consortium and the All Wales Medicines Strategy Group could take the lead in providing credible early reviews of these products to ensure national consistency.