Nunez M*, Gil B, Sacristan JA and Dilla T
Background: Since the adoption of the European law on orphan drugs in 2000, there has been a significant increase in the number of new therapies for the treatment of rare diseases. The present study aimed to analyse the evidence available on orphan drugs in Spain from 2010-2015 in epidemiological, clinical and economic terms.
Results: During this period, 61 orphan drugs were authorised by the European Commission (EC), of which 44 (72.2%) were authorised in Spain and 19 (31.2%) were finally marketed. The average time from the authorisation to commercialisation was 527 days. The clinical trials were mostly phase III (57.7%), randomised (79.1%), doubleblind (54.2%) and/or open label (43.7%), with half using a placebo (49.9%). Quality-of-life measures were included in 62.4% of the trials and the number of patients in the trials ranged from 14-781. Pharmacological costs were negatively correlated with the prevalence of the diseases. In the absence of systematised economic evaluations in the Spanish setting, the reports published by the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) were reviewed and these showed a mean incremental cost-effectiveness ratio (ICER) of £121,072/ QALY (quality-adjusted life years).
Conclusion: Orphan drugs marketed in Spain account for one-third of all drugs approved by the EC, with an average time from approval by the EC to commercialisation of approximately one and a half years. Clinical trials of orphan drugs have mostly been phase III, randomised, double-blind and/or open label, although in several cases, the number of patients has been limited. An inverse correlation can be observed between the number of patients affected and the monthly pharmacological cost per patient. For 63% of the drugs, the ICERs were above the efficiency threshold.