An online consensus-building study to agree on key policy measures for assessing whether the England Rare Diseases Action Plans (developed by the Department of Health and Social Care) are effective

eDelphi study to identify consensus on policy metrics which should be included in future assessments of the England Rare Diseases Action Plans

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

ISRCTN

Registry ID

ISRCTN41639707

Enrollment

100

Registered

2025-03-21

Start date

2025-03-25

Completion date

Unknown

Last updated

2025-03-31

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Any rare disease Other

Interventions

A long list of candidate metrics for the 36 Actions linked to the four Priority Areas of the UK Rare Diseases Framework (i.e., faster diagnosis

increased healthcare professional awareness, better care coordination and

improved access to specialist care, treatment, and drugs) was compiled from government logic models. The list was discussed in three workshops, and metrics irrelevant to the objectives, unclear in the

otherwise, they will remain under consideration in Round 2. In Round 1, the eDelphi participants may propose additional metrics, which will be included in round 2. After two rounds, a final set of po

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Have experience with rare diseases based in England 2. Be 16 years or older 3. Be able to read and write in English 4. Have access to the internet and a device to complete the questionnaire

Exclusion criteria

Exclusion criteria: 1. Have self-declared financial conflicts of interest in assessing the impact of the England Rare Diseases Action Plans 2. Be a member of the RareCare project team, Research Advisory Group, or Patient Public Involvement and Engagement Group

Design outcomes

Primary

Measure	Time frame
The number and list of candidate metrics achieving consensus for inclusion in the future monitoring of the England Rare Diseases Action Plans, assessed at the end of Round 2. Consensus is pre-defined as at least 70% of participants in both groups (people living with rare disease and professionals) rating a metric as extremely valuable (score 7, 8, or 9 on a 1–9 Likert scale), with fewer than 15% rating it as not valuable (score 1, 2, or 3) in the questionnaires.	—

Measure

Time frame

The number and list of candidate metrics achieving consensus for inclusion in the future monitoring of the England Rare Diseases Action Plans, assessed at the end of Round 2. Consensus is pre-defined as at least 70% of participants in both groups (people living with rare disease and professionals) rating a metric as extremely valuable (score 7, 8, or 9 on a 1–9 Likert scale), with fewer than 15% rating it as not valuable (score 1, 2, or 3) in the questionnaires.

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Secondary

Measure	Time frame
1. The extent to which consensus levels differ between people living with rare diseases (or their carers) and professional stakeholders, assessed at the end of Round 2. This will be measured by comparing the percentage of participants in each group who rate individual metrics as extremely valuable (scores 7, 8, or 9 on a 1–9 Likert scale) in the questionnaires. 2. The extent of variation in consensus on candidate metrics based on the demographic characteristics of Delphi panel members, assessed at the end of Round 2. This will be measured by analysing the percentage of participants in different demographic subgroups who rate individual metrics as extremely valuable (scores 7, 8, or 9 on a 1–9 Likert scale) in the questionnaires.	—

Measure

Time frame

1. The extent to which consensus levels differ between people living with rare diseases (or their carers) and professional stakeholders, assessed at the end of Round 2. This will be measured by comparing the percentage of participants in each group who rate individual metrics as extremely valuable (scores 7, 8, or 9 on a 1–9 Likert scale) in the questionnaires. 2. The extent of variation in consensus on candidate metrics based on the demographic characteristics of Delphi panel members, assessed at the end of Round 2. This will be measured by analysing the percentage of participants in different demographic subgroups who rate individual metrics as extremely valuable (scores 7, 8, or 9 on a 1–9 Likert scale) in the questionnaires.

—

Countries

England, United Kingdom

Contacts

Public ContactAimee Walker-Clarke

aimee.walker-clarke@coventry.ac.uk-

Outcome results

None listed

Source: ISRCTN (via WHO ICTRP) · Data processed: Feb 4, 2026