The Cell and Gene Therapy Catapult (CGT Catapult), along with the Duchenne Muscular Dystrophy (DMD) Hub and the Northern Alliance Advanced Therapies Treatment Centre (ATTC), has published a white paper that shares recommendations on how to improve patient referral pathways for gene therapies.
Duchenne muscular dystrophy (DMD) is a genetic disorder caused by errors in the DMD gene. DMD leads to progressive muscle weakness and wasting which eventually affects all the muscles in the body, including the heart and lungs.
Earlier this year, the US was the first country in the world to approve a gene therapy for DMD. While this treatment is not yet approved in the UK, there are DMD gene therapy clinical trials taking place that give eligible patients the opportunity to access these potentially transformative treatments.
However, the current referral processes for both gene therapy clinical trials and licensed gene therapies varies across the UK. The processes often rely upon locally held registries and specialist knowledge to connect patients with the appropriate trials and treatment.
An opportunity to provide recommendations on how to improve the patient referrals pathway process for gene therapies, including for DMD, was identified by the Advanced Therapies Treatment Centre (ATTC) Network, which is coordinated by the CGT Catapult.
Following discussion with Pfizer, a delivery team of the CGT Catapult, the DMD Hub, Newcastle University and the Northern Alliance Advanced Therapies Treatment Centre was established. The team analysed patient referral pathways for clinical trials and for licensed gene therapies in the UK to identify best practice and draft recommendations which could be implemented by the healthcare system.
Following their analysis, the collaborators have published a white paper, Evaluation of the Gene Therapy Patient Referral Pathways in the UK, which shares their full findings.
The recommendations include:
- Ensuring that adequate and sustainable infrastructures are in place for effective patient referrals to gene therapy clinical trials
- A centralised national database which includes potentially eligible DMD patients for gene therapy
- Implementation of national referral pathways for clinical trials and licensed gene therapy treatments supported by a referral porta
- Evaluating where additional clinical trial and commissioning sites for DMD gene therapy should be located geographically to improve access to these therapies and invest in developing infrastructures to deliver these trials
“There are many advanced therapies currently in development or being tested in clinical trials which may soon become available. We hope the recommendations we have developed with our collaborators will help provide guidance on how the healthcare system can build upon its current processes, to streamline how eligible patients are identified and to improve their access to potentially life-changing therapies.”
Matthew Durdy, Chief Executive at the Cell and Gene Therapy Catapult
The DMD Hub is a collaboration between the John Walton Muscular Dystrophy Research Centre at Newcastle University, one of the UK neuromuscular centres of excellence, and Duchenne UK, the leading UK medical research charity for DMD. The DMD Hub is ideally placed to support improvements in patient referrals for clinical trial and licensed gene therapies based on its previous and ongoing work, leveraging the expertise and experience of DMD Hub sites. This work of the DMD Hub is funded by Duchenne UK.
Pfizer Ltd provided financial support for this project to Cell Therapy Catapult Ltd in the form of a grant. Pfizer Ltd 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.