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Advising researchers, ensuring research is relevant: How our work with patients is bearing fruit

Justine Hill, PPI Lead, NIHR Rare Diseases Translational Research Collaboration discusses the importance of working with patients and its new patient advisory group. 

By 2025, any research without evidence of public involvement will be seen as ‘flawed’.That’s the vision of national advisory group INVOLVE – and it’s one that the Rare Disease Translational Research Collaboration shares. 

Who we are

The Rare Disease Translational Research Collaboration was established to support research that will benefit patients by improving the diagnoses and treatment of rare diseases.  

The UK is already a world-leader in this area of research, and our collaboration brings together centres of excellence across England, harnessing the strength of the NHS and the resources in the National Institute for Health Research (NIHR). 

We support a range of research projects into rare diseases across England, which are covered by fourteen thematic areas: Cancer; Cardiovascular; Central Nervous System; Eye Diseases; Gastrointestinal; Immunological Disorders; Paediatrics; Metabolic & Endocrine Diseases; Musculoskeletal Disorders; Muscle and Nerve Diseases; Non-malignant Haematology; Renal; Respiratory Diseases and Skin Diseases. Find out who is leading each theme and some of the research that is being carried out.

Working with patients

Since inception we have been working with patients and carers on participation and engagement activities – and towards the end of 2015 we took this to another level, with the first meeting of our new patient advisory group. 

Seven patients representing five of the themes we support attended, including one patient via Skype. Three more patients representing two further themes have expressed an interest in coming to future meetings.

The group, both collectively and individually, has a lot of experience and different skill sets, including using social media tools to connect with other patients / researchers. We believe they’ll be invaluable to us, contributing ideas and views from a patient’s perspective, to help improve treatments and services for patients with rare diseases.

As part of our commitment to draw on the group’s expertise to guide our own research, we will host twice-yearly meetings with them, following the Strategic Oversight Group (SOG) meetings, which also meets every six months and which helps identify topics for rare disease research and makes sure the best and most relevant research is funded.

Communications between the two groups will be strengthened by the fact that the patient members will take it in turns to also attend the SOG when it meets.

Why research into rare diseases is so important

Rare diseases are defined as those affecting fewer than 5 in 10,000 of the population. Although individually rare, there are more than 7,000 rare diseases and together they affect 7% of the population, or 3 million people in the UK. 

Rare disease research can be difficult because diagnosis is often challenging and patients are distributed over a wide geographical area, limiting the number of patients available for local research studies.

We want to translate the outcomes of research into the development of new, more effective therapies and real-life patient benefits.

If you are interested in finding out more visit our website rd.trc.nihr.ac.uk for more information on our research projects and the researchers leading them.

Who can join the patient advisory group?

We still have vacancies for patients and carers who’d like to join our group. Ideally you’ll be involved in our existing research studies, but you don’t have to have a scientific, research or clinical background; you simply need to be able to listen, discuss issues and give your views from the patient’s perspective.

Your participation could:

•    Add value to the rare disease research that we fund;

•    Identify and raise concerns on the behalf of the public and patients; and

•    Help provide clear communications from us to the patient community.

Want to find out more?

Work is underway to recruit more patient members from the themes that aren’t yet represented. So if you feel that being a member of the group is something that interests you, please contact me, Justine Hill, tel 01223 254804, for an informal chat. I’ll be able to discuss this in more detail and can answer any questions you may have.

Tell us your story

If you are a patient or carer who has been affected by rare disease, with a story to tell, we would love to hear from you.  Maybe you or a family member has benefited from taking part in a research study, or perhaps you’d like to highlight your rare disease and support network in our newsletter or website. 

We also have opportunities for patients and carers who are taking part in research that we are supporting to share their views and experiences with researchers.

Photo: Bardet-Biedl and Alström Syndromes are rare genetic diseases found at birth or in childhood that are chronically debilitating and complex. At present there is no specific treatment. This is one of our paediatric studies which is being led by Great Ormond Street Hospital. Photo: author’s own.