We must act now to unlock the potential that gene therapies could offer people with rare diseases.
Gene therapies and other advanced therapy medicinal products
(ATMPs) have the potential to make a real difference to people living with rare diseases. While a small number of gene therapies have been launched in the UK to date, there are many more in the treatment pipeline. With the National Institute for Health and Care Excellence (NICE) scheduled to assess around 30 gene therapies and other ATMPs by 2023,1 the reality is that many of these gene therapies may struggle to reach patients through our current medicines assessment and reimbursement systems.
But, we have an opportunity now to evolve our systems to ensure these therapies are accessible in the future and the UK can cement its position as a leader in this area.
To achieve this, Pfizer proposes the formation of a Gene Therapy Taskforce. Bringing together the UK Government, NHS, National Institute for Health and Care Excellence (NICE), Medicines and Healthcare products Regulatory Agency (MHRA), the pharmaceutical
and biotech industries, patient groups, and other stakeholders, the taskforce would work towards a shared goal of ensuring that these new advances can ultimately reach patients living with genetic rare diseases.
Rare diseases are, in fact, not so rare. While individually uncommon, collectively rare diseases impact over 400 million patients worldwide.2 Of the approximately 6,000 to 8,000 rare diseases known to exist, 80 per cent are genetic in origin, weaved into a person’s DNA.3 For some patients, gene therapy can target underlying causes of diseases and address them in a single treatment, eliminating the need for continuous, often life-long medication.
This is an exciting prospect, but our healthcare system must rapidly adapt to ensure that when these treatments arrive, they can be fully embraced by the system and be made accessible for the patients who need them. A sense of urgency and collaboration, within a missionled response could unlock change at pace and scale, just as we saw with the COVID-19 response.