Access to treatments

New recommendations for fair and timely access to DMD gene therapy clinical trials and treatments

There is currently no cure for Duchenne muscular dystrophy (DMD), but new and promising treatments are being developed. One of them is gene therapy. Gene therapy involves delivering new genetic material to cells to overcome errors (or mutations) on the dystrophin gene.

Understanding the gene therapy patient referral pathways in the UK

A gene therapy was approved as a DMD treatment in the US in June. This was the first DMD gene therapy to be approved anywhere in the world.

While not yet available as an approved treatment in the UK, there are DMD gene therapy clinical trials taking place that give eligible DMD patient the opportunity to access these, potentially transformative, treatments.

With gene therapy now being a reality for DMD, it has never been more important for patients and families to understand the access pathways in the UK and how they could be improved.

The DMD Hub (our joint programme with Newcastle University) worked with Cell and Gene Therapy Catapult (CGT Catapult) and the Northern Alliance Advanced Therapy Treatment Centre (NA-ATTC) to identify and analyse gene therapy patient referral pathways and has made recommendations on how they can be improved and developed for licenced products in a white paper published today. The project work was funded by a grant from Pfizer Ltd.

Neuromuscular consultants with experience in either referring or accepting patients for gene therapy clinical trials or licenced gene therapy products were surveyed and spoken to. This helped capture a broad understand of experiences with gene therapies and to analyse the referral processes for both gene therapy clinical trials and licenced gene therapy products. It also allowed an identification of the requirements for high quality referrals for gene therapy in DMD.

The proposed DMD referral pathway for gene therapy clinical trials and licenced products in the UK

The white paper outlines the following recommendations in the context of NHS England, but potentially applicable nationally.

Now that these recommendations have been developed, we can use them to drive best practice in DMD through their adoption in the NHS.

We will update you with our work as it progresses.

The gene therapy patient referral pathways report

You can read the full findings and recommendations in the report Evaluation of the Gene Therapy Patient Referral Pathways in the UK, which can be downloaded here.

You can email [email protected] if you have any questions about it.


Published on 4 September 2023

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Support from Pfizer Ltd for report

Pfizer Ltd provided financial support for this project, to Cell Therapy Catapult Ltd, in the form of a grant. Pfizer Ltd have had no influence in the initiation, development or delivery of this project, nor have they influenced the development or content of any materials produced as an output of this project.