The NICE Process – Work To Change It

Last updated 10th September 2020

In July 2019, NICE confirmed details of its plans to review the methods it uses to develop guidance on drugs, medical devices and diagnostics. You can read about this and the work that is going on to ensure the patient voice is heard in this review.

For each area it looks at, NICE will explore the case for change, and will then present proposals for public consultation. The review will cover NICE’s technology appraisals (TA) and highly specialised technologies (HST).  Nusinersen went through a NICE technology appraisal that clearly showed that when it comes to rare diseases treatments, there are significant shortcomings in the current system. In the light of the COVID-19 epidemic, the timeline for this work has been changed - NICE now plan to hold a 6-week public consultation on the case for change starting in November 2020. This will be followed by a 6-week stakeholder consultation on the draft programme manual in February and March 2021. NICE anticipate publishing the final programme manual in September 2021. See the timelines in full on the NICE website

All Party Parliamentary Groups (APPGs) presents the case for change

In September 2019, the APPG on Access to Medicines and Medical Devices ’s summarised the views of a broadly-based group of stakeholders in its recommendations for the NICE Methods Review. SMA UK attended some of the meetings during when evidence was collected. You can read the report here. It will be taken to Government, the NHS and NICE in order to continue dialogue about what should change to support better patient access to innovative therapies.

MAP Biopharma’s work and Genetic Alliance UK's ‘Action for Access’

In April 2019, SMA UK 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. Biopharma continue to work with industry and patient groups (including SMA UK) on this.

In October 2019, we supported Genetic Alliance UK’s 'Action for Access' campaign to improve access to potentially life-changing treatments for people affected by rare diseases (including SMA) - read more

Fasttrack Campaign

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