Three years ago, we produced a whitepaper setting out key challenges that manufacturers face when demonstrating the value of rare disease products to decision-makers, particularly in the context of health technology assessment (HTA), and suggested approaches to overcoming these challenges. Recently, one of the authors of the original paper, Lumanity’s Executive Chair of HEOR Ron Akehurst, has come off a 10-year membership at a key decision-making committee at the National Institute for Health and Care Excellence (NICE) in England: the Highly Specialised Technologies (HST) committee, which is responsible for determining which new medicines should be reimbursed in very rare diseases. Thus, it was an appropriate time to update our earlier advice to reflect the recent developments and experience gained, both from the committee’s recent decisions and from elsewhere in the HEOR sector.
Fill out the form on the right to download the paper.
Download the pdf
"*" indicates required fields
