This year, 2020, will bring many NICE things. In memory of the foresight of the founder of the MPS Society, Christine Lavery, we would like to talk about something unique.
Christine was the first signatory to the very first Highly Specialised Technology (HST) Managed Access Agreement (MAA) for Vimizim, an innovative new enzyme replacement therapy used to treat patients with MPS IVA better known as Morquio. MAAs have been used routinely in Single Technology Appraisals (STAs), particularly oncology, but not for rare treatments reviewed under HSTs.
The MAA was created to address some of the uncertainties NICE and NHS England had around long term clinical outcomes in small patient populations and for the first time it included disease specific quality of life (Q of L) measures. Its primary aim was to collect robust clinical and Q of L data against a set of eligibility criteria which was routinely reviewed by Pharma, NHS England, NICE, clinicians and patient groups over the past four years to consider the longer term effectiveness of the treatment.
This first MAA for HSTs has given rise to many MAAs both in the UK and around the world to help address uncertainty in small patient populations with limited historical data. We look forward to NICE bringing to bear it first class evaluation of the clinical effectiveness, and we know the world will also be looking on.