ABPI response to England's 2023 Rare Disease Action Plan

The Department of Health and Social Care has published the 2023 England Rare Diseases Action Plan, setting out the actions that will be delivered this year to support people with rare diseases in England. 

In response to the Plan, Paul Catchpole, ABPI’s Director of Value and Access Policy, said:

“This Action Plan demonstrates good progress in supporting patients with rare diseases particularly investment in Biomedical Research Centres, support for NHS research data infrastructure, and the use of genomics for diagnosis.

“We welcome the commitment to review the effectiveness of the UK’s early access mechanisms, which have the potential to help patients living with rare diseases get faster access to the latest innovative medicines. We look forward to taking part in the review and supporting improvements which can be made."

Note: In Action 25 of the England Rare Diseases Action Plan 2023 NHS England, MHRA and NICE will review the impact of the Innovative Licensing and Access Pathway (ILAP), the Early Access to Medicines Scheme (EAMS) and the Innovative Medicines Fund (IMF), including their effectiveness in improving access to treatments for people living with rare diseases. This will aid in transparency, provide an understanding of how the schemes are working for rare diseases medicines, and identify any potential gaps or improvements.