Rare Disease Day 2022 – ‘Patient & Public Involvement in Era of ATMPs’

In celebration of Rare Disease Day 2022, we have partnered with the Association of the British Pharmaceutical Industry (ABPI) to raise awareness of the importance of patient and public involvement (PPI) to achieve greater access to advanced therapy medicinal products (ATMPs) for rare diseases.

ATMPs that aim to repair, replace, regenerate and re-engineer genes, cells and tissues offer new horizons for people affected by some rare, lifelong conditions, with the potential to redefine current care pathways. The UK is leading the charge of this rapidly moving field; as of 2020-2021, over a quarter (29%) of Europe’s ATMP developers were headquartered here and 154 ATMP clinical trials were ongoing, representing a 12% global share.1

Interest in the development and use of ATMPs is growing and with 12 ATMPs licensed by the European Medicines Agency available in the UK,¹ more are expected to gain market authorisation and eventual reimbursement. As such, the ATMP PPI working group, co-chaired by Genetic Alliance UK and the Accelerated Access Collaborative (part of NHS England and NHS Improvement), was created to ensure that patient priorities are embedded in ATMP research and development, together with raising the public profile of these pioneering treatments and to produce clear patient-facing information.

Dr Nicola Allen, Value & Access Policy Manager at the ABPI, who supported the collaboration, said:

“Inclusion of appropriate patient and public involvement from research to patient access is essential for developing medicines that can have a meaningful impact on quality of life.”

To learn more about our participation in the ATMP PPI working group, read our article titled, “Moving beyond box-ticking and lip service – why patient involvement matters in a new era of ATMPs for rare disease” – available on the ABPI website here.


About Rare Disease Day

Rare Disease Day is organised by EURORDIS and takes place on the last day of February each year. The main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers across the world about rare diseases and their impact on patients’ lives.

For further information, visit the official campaign website.


About the ABPI

The ABPI is the trade association for the research-based pharmaceutical industry in the UK. They work closely with government, the NHS and the life science community to improve access to new medicines and vaccines, so everyone in the UK can get the latest treatments. Their member companies develop, research, manufacture and supply more than 80% of the medicines prescribed through the NHS.

The ABPI is responsible for the ABPI Code of Practice, which is the industry’s commitment to operate in a professional, ethical and transparent manner, for the benefit of patients and the public.


Contact us

Have any questions or would like to know more? Please get in touch via our contact page (you can refer to our privacy policy statement here).

You can also connect with us on TwitterInstagram and LinkedIn.

On The Pulse


  1. Cell and Gene Therapy Catapult. The Cell and Gene Therapy Catapult UK clinical trials database. 2020. [Online]. Available at: https://ct.catapult.org.uk/clinical-trials-database [Accessed 27 February 2022]