Getting a Drug from Bench to Bedside: The MHRA and NICE Processes
Getting a Drug Approved for Use on the NHS
These are exciting times for those living with FOP and what we have all been waiting for. We now have two potential treatments for FOP which have received FDA approval. This means that pharmaceutical companies will soon be applying to the UK’s MHRA and NICE for licensing and access approval, which will make them available for prescription on the NHS.
We have broken down the process, including how the two bodies work and their different roles, so you can clearly understand where new treatments stand and what lies ahead for our community.
The role of the patient voice in this process is critical; without it, getting treatments approved and accessible simply cannot happen. Here, we outline the vital role you, our patients and families, can play to give us all the best chance of securing available treatments for FOP.
Across the UK, each of our four nations has its own system for making new treatments available through the NHS. While the MHRA licenses medicines nationwide and NICE evaluates treatments for England, Scotland, Wales, and Northern Ireland follow their own distinct pathways. Navigating these interconnected systems can feel complex, but ensuring equal access everywhere remains vital.
FOP Friends is committed to working towards approval and access to new treatments in all four nations of the UK. We will continue to work with patients, families, healthcare professionals, and decision-makers to ensure that the needs and experiences of our community are heard throughout this process.
Our aim is to give everyone living with FOP across the UK the best possible chance of accessing new treatments through the NHS.
For information on the devolved nations, visit: Devolved NHS Access Pathways
The National Institute for Health and Care Excellence (NICE) is the public body responsible for evaluating new medicines for both NHS England and NHS Wales.
One of NICE’s primary roles is to assess how well new drugs work and decide whether the NHS should fund them, ensuring patients gain access to effective treatments.
For more information
NICE Homepage: www.nice.org.uk
NICE Explainer Video: Watch
Technology Appraisal Processes & Timelines: NICE Technology Appraisal Processes Guide
Health Technology & Highly Specialised Technologies Guidance Manual: NICE Health Technology Evaluation Manual
About Highly Specialised Technologies (HST): NICE Highly Specialised Technologies Guidance
