Orphan Drugs
The SMC have adopted the definition of orphan medicines provided by the European Agency for the Evaluation of Medicinal Products (EMEA) – “an orphan medicine is one licensed for treating or preventing life-threatening rare diseases affecting fewer than 5 in 10,000 people in the European Union”.
SMC requires that all submissions are comprehensive and all sections of the product assessment form complete. This requirement also exists for orphan drugs, for which a meaningful attempt needs to be made to produce robust clinical and economic data. SMC recognises that orphan drugs may have a less well developed clinical trials programme and, therefore, that less information than usual may be available for some sections. On the other hand, more detail may require to be submitted in other areas, e.g. on the relevance of surrogate markers and the theoretical basis for their selection, which should then be related to quality of life data.
As with all products, the managed introduction and subsequent monitoring of orphan drugs needs to be a joint responsibility between the manufacturer and the NHS. If there is a significant lack of data on long-term outcome with an orphan drug, this monitoring may include specific clinical audit and, where relevant, a patient register.
The assessment process for orphan drug submissions is the same as for all other drug submissions. However, in addition to the usual assessment of clinical and cost-effectiveness, SMC may consider additional factors, such as whether the drug: treats a life threatening disease; substantially increases life expectancy and/or quality of life; can reverse, rather than stabilise, the condition; or bridges a gap to a “definitive” therapy.
David Webb, Chairman
December 2007