Rare Diseases Medicines

The NIHR Innovation Observatory (IO) aims to identify innovative medicines that may be launched in the United Kingdom within 5 years of receiving a product licence (Marketing Authorisation). We identify such technologies by systematically scanning primary and secondary information sources, including clinical trial registries {ClinicalTrials.gov, European Union Regulating Authorities Clinical Trails Database (EudraCT)}, Pharmaprojects and Trialtrove (Citeline), European Union published designations, press releases, news sources, and direct engagement with companies.

Once identified, information about the innovative medicine is added to the IO’s horizon scanning database (Medicines Innovation Database) as a ‘Technology record’ which includes details about the intervention, indication, clinical trials (phase I/II, II/III, III or pivotal phase I), company information, and regulatory information. The information contained in this Rare Disease Medicines Dashboard relates to new and repurposed medicines currently in clinical trials which are commercially sponsored or linked to an industry. The clinical trial locations include the UK, Europe, USA, Canada, Australia and more recently, Japan and Singapore.