Assess and address the lack of good quality clinical and health economic evidence using state of the art evidence generation methodologies
Provide a clear, engaging and strategic approach to communications with a wide range of stakeholders to address the poor awareness and understanding of rare conditions in the wider community
Effectively communicate the value of orphan and ultra orphan products to address the higher degree of uncertainty inherent to their evidence bases
The National Institute for Health and Care Excellence (NICE) is revising its criteria for the routing of technologies to the Highly Specialised Technologies (HST) Programme. In this article, we explore NICE’s proposed changes to the criteria and provide our thoughts on what the revisions may mean for manufacturers of ultra-rare products.
The Rare Diseases team at Costello Medical is undertaking a research project in collaboration with Sheela Upadhyaya, exploring how expert judgement could be best used to support the development of and patient access to rare disease therapies. If you would like to be kept up to date with the progress of this research project, please register your interest below.
We recently developed a short guide, which introduces expert judgement, and provides an overview of its current use in rare diseases. This guide can be accessed here.
Our Rare Diseases team are committed to actively contributing research to overcome the challenges faced by industry and the patient community. Our research contributions include peer-reviewed manuscripts, panels and posters, to high impact journals and international conferences such as ISPOR and ISMPP.
