Come along to a Muscular Dystrophy UK ‘Fast Track’ MP drop-in and Q&A discussion event on Wednesday 24 January, 12.30pm – 2.30pm, Macmillan Room, Portcullis House, House of Commons, hosted by Mary Glindon MP, Chair of the All Party Parliamentary Group for Muscular Dystrophy.
Aim of the event
With clinical trials in development and a number of potential treatments on the horizon, we want to ensure that Government, NICE and NHS England policy does not delay or restrict access to emerging treatments for rare diseases.
The purpose of the event is to:
- introduce people with muscle-wasting conditions to their local MP to share their views on the urgency of access to new treatments as quickly as possible and to enlist MPs’ support
- discuss how to remove the barriers to fast access to treatments which are set out in our Fast Track campaign
Concerns to be addressed
We are keen to use the event and panel discussion to highlight key areas of concerns, including:
- impact of the £20m per year cost limit which was introduced by NICE and NHS England in April 2017 and could restrict access to some costly rare disease drugs
- making sure that discussions between pharmaceutical companies and NHS England on treatment costs take place as soon as possible
- NICE’s capacity and criteria for undertaking assessments for rare disease treatments, which is restricted to two or three each year under the Highly Specialised Technologies process
- implementation of early access schemes without delays
- risk that the Government’s Accelerated Access Pathway for breakthrough treatments would favour treatments for more common conditions as there is only capacity for four or five treatments to be assessed each year
The Q&A discussion, including Sheela Upadhyaya from NICE and Prof Francesco Muntoni, will be an opportunity to address these issues. We also hope to be joined by an NHS England representative for the event.