About Rare Insights

Rare diseases affect around 3.5 million people in the UK (that’s 1 in 17) and impact more people globally than cancer and AIDS combined.

Despite their collective prevalence, patients often face misdiagnosis, delayed treatment, inadequate care coordination, and a lack of support.

That’s where patient groups come in.

Why are patient groups so vital?

They provide essential support to patients and families and play a critical role in the wider ecosystem.

They inform research, shape policy, and support life science professionals through lived-experience insight and patient engagement, often in the absence of any alternative provision.

They often provide the first support for newly diagnosed patients and families, demonstrating how vital their work is.

Find out more about patient groups

Patient groups: vital yet undervalued, underfunded and overburdened

Despite being that lifeline for the rare disease community, many patient groups are frequently undervalued and operate with limited resources, relying heavily on volunteers whilst facing growing expectations from across the rare disease ecosystem.

While researchers and industry partners often pay professional consultants for their expertise, patient groups are routinely expected to contribute their time, insight, and networks without appropriate reimbursement.

Without sustained and appropriate support from external stakeholders (such as funders, policymakers, researchers, and industry), many patient groups risk burnout, stagnation, or closure, limiting their ability to continue supporting their communities.

The Rare Insights Study aims to address this.

In 2016 Genetic Alliance UK published The Rare Reality Repo rt (2016) which focused on the patient experience and the challenges those with rare diseases face.

We want to build on this by focusing on another vital, yet often overlooked part of the rare disease community: patient groups.

Our study will help to amplify the voice of patient groups and ensure their expertise is more widely recognised, strengthening collaboration across the rare disease community and wider ecosystem.

Rare Insights: tapping into the potential of patient groups

Our study will gather evidence from:

A patient group survey

Focus groups

1:1 interviews

This will inform our report’s recommendations on how best to support patient groups and champion their vital work within the rare disease community.

We are aiming to capture the experiences of 100 patient groups through the Rare Insights Study.

Our study report will inform future investment, training, and capacity-building opportunities for patient groups across the country.

Keep an eye out around Rare Disease Day 2027 for our official report publication!

Did you know?

The Rare Insights Study is endorsed by our 22-member Patient Group Engagement Committee (PGEC), which emphasised the need for rare disease patient group-specific data.

They raised patient groups’ reality of being volunteer-led by those personally connected to the condition, as well as many patient group leaders having to balance caring responsibilities and work, alongside community support.

Get involved in the Rare Insights study by sharing your experiences now!

Take the patient group survey today!

Thank you to our funders

Beacon’s work is only possible thanks to our wonderful funders and their generous contributions.

BioMarin

Novartis

UCB

Our sponsors and supporters have provided financial support for this study. This study has been organised by Beacon and neither our sponsors nor supporters have determined the content or organisation. All funds received by pharmaceutical companies have been used in accordance with the ABPI code.