The National Institute for Health and Care Research (NIHR) and the Medical Research Council (MRC) have published a landscape review report, revealing that £1.1bn has been invested in UK research for rare diseases.

The report findings aim to help focus areas for future funding and better target the needs of people with rare diseases.

The review found that the NIHR and MRC accounted for almost £627m in funding across 698 studies between 2016 and 2021 for rare disease research.

Comprised of 7,000 different types, rare diseases affect around 3.5 million people in the UK.

The report revealed that motor neuron disease received the most funding in the portfolio, accounting for 8%, followed by Huntington’s disease (5%) and then preeclampsia (5%).

Other diseases that received significant funding included cystic fibrosis (4%), frontotemporal dementia (4%) and idiopathic pulmonary (3%).

Over five years, approximately £99.7m out of the nearly £627m of investment was used for MRC fellowships and NIHR career development awards.

Additionally, over 5,000 participants were recruited in rare disease studies through investment in a research delivery workforce and specialist facilities, including NIHR Biomedical Research Centres and NIHR Clinical Research Facilities.

As part of the Association of Medical Research Charities, 107 of 177 invested £580m in over 2,600 rare disease research studies.

The Association of the British Pharmaceutical Industry and the Bioindustry Association’s report also identified 254 rare disease research projects supported by industry, 11% of which accounted for cystic fibrosis.

Since 2021, the NIHR and MRC have invested £14m in the UK Rare Disease Research Platform and LifeArc has invested £40m in a rare disease translational challenge.

Professor Lucy Chappell, chief executive of NIHR, said: “This report is the first time anyone has captured a detailed picture of the rare disease research being funded across the UK. Our findings are another step in understanding the strength and diversity in rare disease research.”

Professor Patrick Chinnery, clinical director of MRC, added: “It is vital that we have a shared understanding of where we are now, so together we can improve coordination and support for rare disease research that can lead to better patient outcomes.”

https://www.pmlive.com/pharma_news/a_landscape_review_reveals_1.1bn_of_uk_funding_for_rare_diseases_1501045