Funding rare disease treatments in the UK – navigating the labyrinth
With the appraisal process for highly specialised treatments in the UK described by one NHS insider as 'impenetrable', and The MPS Society's (The Society for Mucopolysaccharide Diseases) recent launch of a petition calling for the reinstatement of the procedure which was scrapped as part of the Health Social Care Act (HSC) 2012, this article investigates the path to market for such drugs and the effects on patients.
Patients with rare diseases and the companies developing specific treatments for them both need to understand the process by which a treatment becomes accessible in the UK.
The first orphan-designated treatments were launched in 2001, for a complex condition called Fabry disease (Replagal, TKT Europe [now Shire] and Fabrazyme, Genzyme [now Sanofi]).
How far have we come since then in developing a clear pathway to bringing a licensed treatment to the patients who need them? At a recent meeting discussing the evaluation of licensed rare disease treatments (so-called Highly Specialised Technologies, HSTs) one NHS insider used the term 'impenetrable' to describe the commissioning arrangements for HSTs.
To describe all of the arrangements for commissioning rare disease treatments in the UK would require a manual, such is their complexity, but some of the main pathways and the issues shaping their development will be covered here.
full article at http://www.pharmaphorum.com/articles/funding-rare-disease-treatments-in-the-uk-navigating-the-labyrinth