NHS in Yet Another Negotiated Discounted Deal for Game-Changing Libmeldy Drug

NHS England and Orchard have, however, agreed a discounted deal to enable the purchase to go ahead, after the National Institute for Health and Care Excellence (NICE) had earlier rejected its approval in 2021 on the grounds that it was too expensive, and the long-term effects were unproven.

This agreement follows hot on the heels of other exciting deals last year - such as for Inclisiran, an anti-cholesterol treatment that was predicted to save 30,000 lives in 10 years; and another for Zolgensma, Novartis's revolutionary cell and gene therapy treatment for babies with spinal muscular atrophy.

What is Libmeldy?

Libmedly treats metachromatic leukodystrophy (MLD) - an inherited neurodegenerative disorder which causes progressive disability in the patient's nervous system and organs, with seizures and difficulties with talking, swallowing and vision. Libmeldy treatment takes stem cells from the patient, modifies them to replace the defective gene, and then re-introduces them back into the patient through a single intravenous infusion. Children with the late infantile type of MLD quickly deteriorate and do not usually make it past the age of eight. Children with early juvenile MLD have a life expectancy of no more than 20 years.

NHS Chief Executive Amanda Pritchard said: "This revolutionary drug is a life-saver for the babies and young children who suffer from this devastating hereditary disorder and will spare their families untold heartache and grief."

The agreement coincides with NICE's publication of a positive final evaluation determination, which recognized the clinical impact and economic benefit of Libmeldy.

Professor Bobby Gaspar, CEO of Orchard Therapeutics, commented: "Today’s landmark agreement with NHS England follows a thoughtful and comprehensive value assessment by NICE and represents a major milestone for the MLD community. We are delighted that NICE and NHS England have recognized the transformational clinical impact and significant economic value of Libmeldy for eligible MLD patients."

The Importance of This Approval

The UK has often been criticised for not being able to pay for medicines that cost a lot of money, but there have been many good news stories recently which show the NHS and NICE will approve drugs that can make a huge difference to people's lives.

The 2021 PING Conference, which VWV held in collaboration with IQVIA, was entitled "UK Life Sciences Opportunities in a Changed World". The Conference heard many ways in which the UK is now innovating and bringing new treatments to patients, including from Claire Foreman, Director of Medicines Policy and Strategy at NHS England and Improvement, who spoke about the UK's passion for adopting innovative treatments.

PING Conference 2022

At the 2022 PING Conference - A Golden Age for Life Sciences Innovation - we will hear from the new Chief Executive of NICE, Dr Samantha Roberts, about NICE's five year strategy for adopting new processes and adapting to changing times to stay at the forefront of assessments of innovative treatments.

If you would be interested in hearing more about these issues and being invited to attend the 2022 PING Conference, please contact Paul Gershlick in our Pharmaceuticals and Life Sciences team on 07795 570072, or complete the form below.