Title: An early stage research proposal to explore diagnostic pathways in a rare inflammatory disease.
Advisor: GR (Bham)
The applicant approached the RDS in October 2019 with a proposal to explore the use of MRI in the management of a particularly rare paediatric inflammatory disease. The research hypothesis is concerned with how monitoring with MRI rather than usual care can result in reductions in both under and over treatment using steroids and optimise weaning time to facilitate remission.
The research proposal being developed by the client, with the assistance of the RDS, is a highly complex one. The rarity of the disease concerned means that the use of trial methodology is not appropriate in this case and even the development of a prognostic model has significant barriers.
However the exploration of the research question could yield benefits for a group of patients which, whilst small, face the challenges of living with a rare and debilitating disease.
Following advice from the RDS and a number of meetings and iterations of a research proposal, the applicant was encouraged to build an international collaboration to increase the available data to support a research concept. They have recently (March 2021) engaged with a pan-European clinical network with a view to exploring possible multi-national approaches to the research question and specifically to explore the possibility of developing and observational study.