Rare disease action plan

Scotland’s first action plan for rare disease sets out the actions that we, our NHS, partners and stakeholders will take to improve the lives of people in living with a rare disease. The plan supports the shared priorities of the 2021 UK Rare Diseases Framework.

3. Our approach to engagement

During the development of this Action Plan, we have carried out more engagement than ever before, and rightly so. We have worked closely with Genetic Alliance UK to facilitate a number of virtual events to enable the rare disease community to influence and shape our policies. Our engagement during this period has been conducted in virtual spaces by necessity due to the Covid-19 pandemic. Following the publication of our Action Plan, we will consider how best to safely hold further engagement events in ways that will increase the diversity of patient voices that we can reach.

Our engagement took a "What matters to you?" approach. This approach is about listening and understanding what matters to a patient within the larger context of their life. Fundamentally it means asking not "What's the matter with you?", but: "What matters to you?". This model supports NHS Scotland staff to understand what's important to the people they are caring for and supporting and establishing a caring compassionate connection. When patients are fully engaged with their health care decisions and the development of pathways, it can greatly improve their outcomes.

At the beginning of each of the following sections, a graphic sets out a snapshot of the feedback we have received from the rare disease community through this approach.

Engagement events with the Rare Disease Community

Our patient engagement programme in the development of this Action Plan was twofold. One strand of this was to create a formal advisory group, which became known as our Patient Voices Advisory Group. The membership was drawn from both representatives from patient advocacy groups, and from people with lived experience (and/or familial experience) of a rare disease. The group first met in December 2021. The meetings so far have been extremely helpful in identifying what matters to the rare disease community, what challenges they face and what their priorities are in their treatment and care. We recognise the difficulties and strain on individuals and small organisations in engaging on many platforms. As a result, our Voices Group hasn't perhaps had the turn out we would expect. However, we are reviewing our Patient Voices Advisory Group with Genetic Alliance UK to ensure it works and our methods for engagement do not create an unnecessary burden.

The other main strand of our engagement with the rare disease community has been through a more informal series of online events. Like the chairing of our Advisory Group, this has been led by led by Genetic Alliance UK. These events have provided an open platform for discussion and sharing of information and lived experience, as well as a chance for Scottish Government officials to share information about this Action Plan during its development. Patient feedback from these events, and from the meetings of the Advisory Group, has directly informed the structure and priorities of this Action Plan, and will continue to do so in future publications and the actions we take forward. At the beginning of each chapter of the plan, a summary of the feedback is represented in a graphic. We're keen to demonstrate the feedback we received and ensure our action plan is reflective of what we heard.

Timescales for action

The Rare Disease Framework has a lifespan of five years, and as we have learned from previous strategies, actions set out in year one can quickly expire or change due to innovation or the evolvement of other policies that replace the original action. With that in mind, this document sets out the specifics of our short term to medium term actions (2–3 years) and our ambitions for the long term (3+ years). Over the course of the next five years, we hope to have many iterations of this Action Plan building our medium- and long-term ambitions into actions.


Email: scott.thomson2@gov.scot

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