This content has been developed and funded by Pfizer UK.
Gene therapies and other advanced therapy medicinal products offer the potential to transform lives – but a “mission- led” approach is needed to ensure treatments can reach eligible patients
Individual rare diseases might affect a minority of patients, but their collective impact is significant. In the UK alone, approximately 3.5 million people navigate the daily challenges of living with a rare disease and patients can often require lifelong medical support and care. With 95 per cent of rare diseases having no approved treatments,  there is a considerable burden on health systems as those living with conditions face multiple hospital appointments and complex management.
However, hope is on the horizon. The theoretical promise of gene and cell therapies is now starting to become a reality and, alongside other advanced therapy medicinal products, these innovative treatments could potentially transform the lives of people living with rare diseases and help to alleviate the long-term burden on the health and care system.,
This is because gene and cell therapies are different to traditional medicines. They are highly personalised, typically “one-off” medical treatments, that may deliver benefits over the course of a patient’s lifetime.
Systems must evolve to keep pace with the speed of scientific innovation
Gene and cell therapies represent an exciting change in how we treat some diseases, but with science progressing at pace and a number of potential ATMPs being launched over the coming years, we need to act now to ensure the right mechanisms are in place for eligible patients to access them.
The reality is that gene and cell therapies present challenges to the traditional approach of medicine adoption meaning many may struggle to be approved by the National Institute of Health and Care Excellence as it stands. This is because the current model of assessment and reimbursement was not designed with these new treatments in mind. Challenges include uncertainties over long-term patient outcomes at the point of appraisal, short-term affordability and ongoing data collection.
Prioritising collaboration and championing evolution across the health system is vital to keeping pace with scientific innovation and ensuring equitable access to treatments
To enable timely access to new treatments, there is a need for a specific focus on the adoption of gene and cell therapies, reflecting the complexities involved in bringing them to patients and the need for concerted action across the system to deliver much needed changes.
The time for collective action is now. The creation of a government-led gene therapy taskforce would provide an opportunity for healthcare professionals, key government departments and agencies, academia and industry to come together with a “mission- led” approach to tackle the barriers ahead.
The covid-19 pandemic showed us the true power of collaboration to transform outcomes, and we hope a similar approach can now be applied to bring together stakeholders from across the system to drive the reform needed to unlock the potential benefits of ATMPs.
All healthcare stakeholders are united by a common goal – we want to provide access to potentially life-changing treatments for the people who need them the most. Prioritising collaboration and championing evolution across the health system is vital to keeping pace with scientific innovation and ensuring equitable access to treatments.
Date of preparation: January 2024