Cell and Gene Collective calls for more political leadership to ensure patient access to gene therapies

Written by Jeff Courtney, May 10, 2022

The Haemophilia Society is part of a group of rare disease charities called the Cell and Gene collective that work together to increase political awareness of cell and gene therapies and to help ensure patient access.

The group met recently to discuss the key challenges in the cell and gene therapy policy environment. We contributed to the debate to share our insight into how the process for clinical trials, licensing and consideration by NICE is taking place for gene therapies in haemophilia.

We will be taking these challenges to parliament and policy makers in the coming months to ensure that the NHS is ready for these potentially transformative new treatment options.

A key issue is patient knowledge and decision-making. People with haemophilia need to understand how gene therapies work and both the benefits and risks of choosing them as their treatment option. There may be different stages of life when gene therapy is right for people. Some people may wish to be involved with a trial or have the first licensed gene therapies while other will want to wait until second or third generation treatments are available.

Another challenge is how gene therapies integrate with current care. It will be important for peoplethat they still have access to their haemophilia centre for access to physio, psychological support, liver health care and specialist nursing.

Last year the Cell and Gene Collective published a report which included input from the Haemophilia Society. The report called for a common language when discussing gene therapy, good quality information, improved clinical trial design and innovative methods for assessing value of gene therapies.