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We must act now to unlock the potential that gene therapies could offer people with rare diseases

We must act now to unlock the potential that gene therapies could offer people with rare diseases

Credit: Adobe

Owen Marks, Head of Rare Disease

Owen Marks, Head of Rare Disease | Pfizer UK

4 min read Partner content

The formation of a Gene Therapy Taskforce could help ensure people with genetic rare diseases can access the latest scientific advances.

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, [i] 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.[ii] 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. [iii]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 mission-led response could unlock change at pace and scale, just as we saw with the COVID-19 response.

In our new report ‘From Promise to Reality: a blueprint for building a mission-led partnership approach to make the UK a world-leader in the adoption of cell and gene therapies’ we highlight three focus areas for the proposed gene therapy taskforce:

  • new ways of assessing new treatments that better capture the value offered to patients, the NHS and wider society such as gene therapies.
  • rethinking the way medicines are funded, for example through outcomes-based agreements which are based on how well the medicines work for patients.
  • strengthening data infrastructure so we can understand long-term patient outcomes.

The UK is already making progress. This year marked England’s first Rare Diseases Action Plan[iv], which aims to reach the goals of the Rare Diseases Framework[v] over the next five years. Meanwhile, the Life Sciences Vision aims to build on the collaborative innovative effort triggered by the pandemic, mentioning rare diseases as a focus.

Now is the time to act to ensure these treatments become a reality for patients in the future and the UK achieves its vision of levelling up world class health outcomes. And, through the formation of a Gene Therapy taskforce, I believe we have a strong chance of delivering that.

You can find Pfizer's blueprint for the UK adoption of cell and gene therapies here

‘Developed and funded by Pfizer UK Ltd’

PP-UNP-GBR-0446 April 2022.


[i]Accelerated Access Collaborative. Advanced Therapy Medicinal Products. 2020. Available at: https://www.england.nhs.uk/aac/wp-content/uploads/sites/50/2020/01/aac005-atmps.pdf [Accessed: March 2022].

[ii] Global Genes. Rare Disease facts. Available at: https://globalgenes.org/rare-disease-facts/ [Accessed: March 2022].

[iii] Rare Disease UK. Illuminating the Rare Reality. 2019. Available at: https://www.raredisease.org.uk/wp-content/uploads/sites/7/2019/02/Illuminating-the-rare-reality-2019.pdf [Accessed: March 2022].

[iv] Rare Diseases Action Plan. 2022. Available at: https://www.gov.uk/government/publications/england-rare-diseases-action-plan-2022 [Accessed: March 2022]

[v] Rare Disease Framework.2021. Available at: https://www.gov.uk/government/publications/uk-rare-diseases-framework [Accessed: March 2022]

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