Musketeers' Memorandum

 

 

There are over 7000 rare diseases, which collectively affect 1 in 17 people with most being the result of a genetic disorder.

When viewed as a group, rare diseases are very common. The national approach made possible by the NIHR Clinical Research Network Portfolio, means we are able to collect together enough people with a particular condition to make a research project feasible. This leads to improved care and the development of new treatments. A prime example of this work is the Musketeers’ Memorandum.

The NIHR Musketeers’ Memorandum allows a single lead UK genetics unit to rapidly enable set-up for a rare disease non-CTIMP (Clinical Trial of an Investigational Medicinal Product) project, which can then be delivered across all regional genetics centres.

At a meeting of NIHR Senior Investigators, Professor Sir John Burn and his team were tasked with working with NOCRI (NIHR Office for Clinical Research Infrastructure) to develop a national agreement. When a regional genetics centre, supported by their NHS Trust, developed a rare disease non-CTIMP project, the R&D teams responsible for all the other regional genetics centres in the UK would sign off without a full review of protocol and the need for individual negotiations.

The cost to trusts is minimal and each will benefit from portfolio recruitment funding. The benefits to people with rare disease will be significant too. It is hoped that every centre in the UK joins the Musketeers' Memorandum - all for one and one for all!

Under this agreement residual material from the blood, saliva and previously collected tissue collected in studies and stored in biobanks, can be shared for the benefit of research into rare genetic disease. The local processes of each biobank with stored material would need to be applied. If you think your study might benefit from the Musketeers’ Memorandum, please read the eligibility criteria.

For further information please contact musketeers@nihr.ac.uk

Professor Sir John Burn describes the impact of the Musketeers' Memorandum on rare disease research