UK Confirms its Leadership Position in Advanced Therapies

Cell and gene therapies and tissue-engineered products have the potential to offer a durable, life-changing effect, possibly with single administration

New report from Alliance for Regenerative Medicine and UK BioIndustry Association highlights progress in financing and clinical trials

24% of Europe’s ATMP developers headquartered in the UK

WASHINGTON, D.C. and LONDON, UK – 18 July 2019

The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader advanced therapies sector, and the BioIndustry Association (BIA), the trade association for innovative enterprises involved in UK bioscience, have today published a report setting out the scope of the UK’s robust and growing Advanced Therapy Medicinal Products (ATMPs) sector which includes the development of potentially transformative cell and gene therapies.

Key highlights include:

• Financings for ATMP companies grew 66% from £473m to £785m between 2017 and 2018 and stand at £347m for 2019 year to date
• More than 70 ATMP developers are active in the UK, including 56 headquartered in the country
• Ninety-three ATMP clinical trials are ongoing in the UK, including 27 sponsored by UK companies

According to the report, the UK is a leading source of innovation and development of advanced therapy medicinal products (ATMPs) in Europe. These products, which include gene therapies, cell therapies, and tissue-engineered products, are intended to augment, repair, replace, or regenerate organs, tissues, cells, genes, and metabolic processes within the body. These therapies have the potential to provide profound and durable responses—often with just a single treatment—for patients with a diverse array of serious diseases and disorders.

However, despite the strength of the UK sector, the report highlights the need for policymakers and other stakeholders to promote a positive scientific, regulatory, and reimbursement environment to ensure that patients are able to access these products in a timely manner.

Janet Lambert, CEO of ARM, commented: “Advanced therapy products are already on the market in the UK for some haematological malignancies, as well as certain rare genetic disorders. Going forward, we expect to see cell and gene therapies approved for additional rare diseases and solid tumours, and for diseases with larger patient populations, including additional cancers, cardiovascular indications, and certain neurodegenerative disorders, such as Alzheimer’s and Parkinson’s.

“Our priority is driving the necessary financial and commercial innovation needed to ensure that patients are able to access these transformative therapies. We are committed to continuing our work with policymakers, industry, and other stakeholders to ensure this sector continues to push forward.”

Steve Bates OBE, CEO of the BIA, said: “This is an extraordinarily exciting time in medical research as advances in biology, technology, and data science open up new frontiers in the treatments of disease. The UK has already played a major role in the development of cell and gene therapies, establishing itself as a leader in this field, however, as with any innovation, the need for cell and gene therapies risks outpacing society’s and government’s capacity to adopt them. The UK needs the right infrastructure, talent, and regulation in place to continue to be a leader in this space.  We need the commitment from all parts of the ecosystem to address potential barriers to the growth of the industry and to ensure that these treatments reach patients who will benefit from them.”

A copy of the full report can be accessed here.

Recommendations to continue driving ATMP development in the UK

Support scientific research to develop and advance both ATMPs and ancillary processes, including manufacturing and scale up.

Foster economic development and the creation of a skilled workforce to promote the continued growth of this industry in the UK.

Cultivate a positive regulatory environment for the research and development of ATMPs, including fostering accelerated pathways to ensure that patients are able to access safe and effective therapies in a timely manner.

Develop the necessary infrastructures within NICE and its counterparts in Scotland, Wales, and Northern Ireland to ensure health technology assessments are able to address the long-term value provided by ATMPs.

Collaborate with the NHS and other public and private payers in the UK to develop innovative financing models to ensure patients can access approved therapies in an efficient manner.