Protocol for an eDelphi study to identify consensus on policy metrics which should be included in future assessments of the England Rare Diseases Action Plans
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Introduction
Governments create policies to address societal needs and then assess their effectiveness through policy metrics. The UK Rare Diseases Framework and its England Rare Diseases Action Plans aim to improve the lives of people with lived experience of rare diseases and their carers. Reaching these goals requires more effective engagement with those least likely to interact with NHS services. This paper outlines a protocol to identify relevant high-level policy metrics while engaging underserved and underrepresented groups.
Methods and Analysis
A long list of candidate metrics for the 36 Actions linked to the four Priority Areas of the Framework was compiled from government logic models. The list was discussed in three workshops, and irrelevant, ambiguous, or time-irrelevant metrics were removed. Additional metrics were identified through an evidence review and expert opinion.
In the first round of a two-round eDelphi study, shortlisted metrics will be grouped into the four Priority Areas: Faster Diagnosis, Increased Healthcare Professional Awareness, Better Care Coordination, and Improved Access to Specialist Care, Treatment, and drugs. An eDelphi panel of 50 people living with rare disease or their carers, as well as 50 professional stakeholders, will rate each metric on a 1-9 scale. The sample size allows for 20% attrition. Consensus for inclusion or exclusion will be based on 70% agreement (7-9 for ‘very valuable’, 1-3 for ‘not valuable’), with fewer than 15% rating the metric at the opposite end of the scale.
Metrics will be included or excluded if consensus is reached in both groups; otherwise, they will remain under consideration in Round 2.
In Round 1, eDelphi participants may propose additional metrics, which will be included in Round 2. After two rounds, a final set of policy metrics will be selected. Any discrepancies in Round 2 ratings will be explored through further research.
Ethics and Dissemination
The Coventry University Group Research Ethics Committee has approved the study (P183377). The results will be disseminated to people with lived experience of rare disease and professional stakeholders through conferences, events, and newsletters and submitted to a peer-reviewed journal. If adopted, the identified metrics will inform decision-making on rare disease policies in England and beyond.
Registration Details
ISRCTN41639707
ARTICLE SUMMARY
Strengths and limitations of this study
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The study’s strength lies in its equal representation of individuals with lived experience of rare diseases and professional stakeholders, ensuring contextually relevant and widely accepted policy metrics.
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Efforts are made to include underserved groups, ensuring the meaningful representation of underrepresented sub-populations.
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The findings will primarily apply to the England Rare Diseases Action Plans, limiting their generalisability to other countries or healthcare systems.
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The heterogeneity of the study population may pose challenges in reaching consensus, potentially impacting the robustness of the agreed-policy metrics.
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Despite targeted recruitment efforts, challenges in including underserved groups may result in a less representative sample, affecting the study’s generalisability.
