The NICE Process – work to change it and what happens in the rest of the UK

Last updated 7th May 2019

The appraisal process for nusinersen has clearly been shown to be ‘not fit for purpose’ for rare diseases treatments.

All Party Parliamentary Groups (APPGs) and meetings

We are taking every opportunity to attend these and to add our voice to the many who are saying at these meetings that  the system isn’t working.

Most recently, on 29th April 2019, we joined MPs, representatives of charities supporting patients affected by rare conditions, representatives from pharmaceutical companies and others for the launch of MAP Biopharma’s report ‘Access to Orphan Medicines: A Case for Change’. Cat Woodhead, CEO of MDUK, gave a powerful summary of the impact of the current system when she spoke about what has been happening with nusinersen and the ‘cruelty’ of this for families. All agreed that patient and clinical input into the current NICE methods review of the HST and STA systems is essential, and that change is needed as a matter of urgency. Dr. Jayne Spink, CEO of Genetic Alliance UK, spoke about their current work onResetting the Model’ as a way for rare condition patient groups to express their views (see also below). 

On 10th December 2018 and 4th April 2019, we were at the APPG on Access to Medicines and Medical Devices. 

On 3rd December 2018, we were at the APPG on Rare, Genetic and Undiagnosed Conditions which focused on access to rare diseases medicines in England. You can read the minutes from this meeting, here.

All these meetings are highlighting the same issues with a view to working together with MPs for change.

"Resetting the Model".

The All Party Parliamentary Group (APPG) on Rare, Genetic and Undiagnosed Conditions, has challenged Genetic Allicance UK to work with a range of stakeholder groups to propose a method of making decisions about rare disease medicines that is ambitious, effective, transparent and fair.

This is their 'Resetting the Model' project which they plan to launch in April 2019 and you can read about here.

Fasttrack Campaign

We are also supporting MDUK’s efforts to highlight nusinersen in national media as a strong example of what is wrong with the system. See their Fasttrack campaign.

How NICE works now

You can read how the NICE process works currently and what happens in the rest of the UK, here.