‘From Promise to Reality: A blueprint for building a mission-led partnership approach to make the UK a world-leader in the adoption of gene therapies’ explores how the UK can tackle some of the challenges it currently faces which could prevent it from fully embracing gene therapy in the future.

The report calls for the formation of a Gene Therapy Taskforce, building on the mission led approach we saw with the Vaccine Taskforce created as a result of the COVID-19 pandemic and applying the same sense of urgency and collaboration to another area of great unmet need for patients. Stakeholders from across the rare disease community could work together towards a shared goal of ensuring gene therapies can reach the patients who need them.

In the report Pfizer UK suggests the taskforce could focus on the following priorities:

1. New ways of assessing new treatments: evolve how NICE assesses these new technologies to make sure it better captures the value offered to patients, the NHS and wider society.

2. Changing the way medicines are funded: to ensure patients can access approved treatments, the NHS must be open to looking at new ways of paying for medicines. For example, through outcomes-based agreements which are based on how well the medicines work for patients.

3. Strengthening data infrastructure: to understand long-term patient outcomes requires systems that support the collection and use of data on how and when medicines are used, and the outcomes they deliver.