The Power of Real-World Data: Why Patients’ Experiences Prove the Case for SMA Drugs
When new drugs for rare conditions like SMA are approved, they are primarily judged on data from strict clinical trials. But those trials are limited, they often involve small groups of people and only run for a set period.
That’s why SMA UK along with our partners Treat SMA and MDUK are stressing the Power of Real-World Evidence (RWE).
Since risdiplam and Spinraza have been made available through a Managed Access Agreement (MAA), people across the UK with SMA have been using them in their daily lives. The data collected during this period isn’t theoretical; it’s a record of what actually works for real patients.
Why Real World Evidence is Essential
- It Represents Everyone: Unlike trials, RWE includes people of all ages and all types of SMA, including those who may have been too complex for the original studies. This gives a much clearer, truer picture of the drug’s impact on the entire SMA community.
- It Shows Long-Term Success: RWE shows how well the treatments hold up over years, not just months. It shows the impact on a family’s day-to-day life, the reduction in caregiver strain, and sustained improvements in function and well-being.
- It Guides Doctors: Because every patient is different, RWE helps specialist clinicians understand who responds better to which drug.
We are asking the NICE committee to give significant weight to this real-world evidence. These are the verified, lived experiences of patients and families showing the profound, positive differences these therapies make. In rare diseases, the experience of those currently on treatment is the most compelling proof.
Technology Appraisal Committee Meeting
The registration facility for the committee meeting for the above appraisal, to be held on Tuesday 11 November 2025, is now open on the NICE website. The meeting will be held virtually, using Zoom, a secure web conferencing tool. The decision on access to both treatments will be made after the 11th November. We will update as and when we are able.
You can find the links to register directly on Zoom on this page.
Registration will close at 4.00pm on Tuesday 4 November 2025.
As a public observer, you will be able to listen to the business of the meeting, except where confidential information is being discussed, however, you will not be able to:
- participate in committee discussions
- ask questions, take part in voting or put your views to members of the committee
For further information about meetings in public please refer to the following document: Common questions and answers about virtual committee meetings.
