Chris MorrisSenior Research Fellow in Child Health

Meaningful health outcome measures

for children with neurodisability

Funding & disclaimer

Informing the NHS Outcomes Framework: what outcomes of NHS care should be measured for children with neurodisability?

NIHR Health Services and Delivery Research Programme 10/2002/16

This presentation describes independent research commissioned by the National Institute for Health Research (NIHR). The views and opinions expressed by the researchers, interviewees and other participants do not necessarily reflect those of the NHS, the NIHR, or Department of Health.

The full report is published in the NIHR Library.

Acknowledgements

Astrid Janssens Amanda Allard Valerie Shilling Bryony Beresford Jo Thompson-Coon Morwenna Rogers Andrew Fellows Crispin Jenkinson

Richard Tomlinson Jane Williams Stuart Logan Alan Tennant Karen Allen Colin Green PenCRU Family Faculty

What make health outcomes meaningful?

Valued by children/young people.

Valued by parent carers who seek children’s care.

Are realistic targets for health professionals.

Measurable using robust instruments.

NHS policy context (in 2010)

Sir Ian Kennedy:

lack of a “common

vision” between

families and health

professionals for

what services are

seeking to achieve

NHS policy context (in 2010)

Domain 2:

Health-related quality

of life for people with

long term conditions

Patient Reported Outcome Measures (PROMs)

Patient and/or carer

completed questionnaires.

No interpretation by

interviewer or clinician.

Produce quantitative score.

observational research health service performance indicators

clinical trial endpoints individual care

NHS policy context (in 2010)

RCPCH proposed…

there should be a

single PROM “for a

basket of conditions”

rather than aiming to

have one for every

diagnosis.

Research study aims

Seek a ‘shared vision’ regarding key health outcomes

for children and young people with neurodisability –

beyond mortality and morbidity.

Appraise the measurement properties of generic,

multidimensional PROMs to assess these outcomes.

PenCRU Family Faculty

Five parents involved, one was a co-applicant:

Co-investigator & parent advisory group meetings

Qualitative study: developed interview guide

Delphi Survey: drafting & reviewing Q&A

Interpretation of findings & recommendations

Dissemination – plain language summaries

Qualitative Study - participants

Council for Disabled Children networks, across England, socioeconomically diverse, various conditions represented.

54 children and young people: Focus groups: 50 (incl. Talking Mat group) Interviews: 4

53 parents: Focus groups: 47 Interviews: 6

Qualitative Study - methods

Topic guide

Explored ‘aspects of health’ improved if services were

‘ideally’ resourced and delivered.

Discussed commonly used PROM questionnaires:

KIDSCREEN, PedsQL, Health Utility Index, EQ-5D-Y

Analysis

Framework Approach, with ref to ICF and study aim.

Qualitative Study - findings

Aspect of health were viewed as

• Inter-related

and

• Hierarchical

Feedback on questionnaires…

Key outcomes - families

Communication Mobility Pain Self-care Temperament (including mood and anxiety) Relationships and interactions Community and social life Emotional wellbeing Gaining independence/future aspirations Sleep, behaviour & safety – parents mainly

Recruitment:

BACD child development team database

Then purposively through:

Societies and special interest groups

Participants registered online – contact email address.

Delphi Survey - professionals

Delphi Survey - professionals

Audiologist (1), C&A Psychiatrist (5), Child Development Worker

(1), Neurosurgeon (1), Nurse (9), Occupational Therapist (14),

Orthopaedic Surgeon (6), Orthotist (19), Paediatrician (76),

Paediatrician in Neurodisability (4), Paediatric Neurologist (5),

Paediatric Oncology (1), Paediatric Surgeon (2), Physiotherapist

(61), Prosthetist (2), Psychiatrist (1), Psychologist (8),

Rehabilitation Medic (1), Speech And Language Therapist (15),

Teaching Assistant (1).

Response:

R1: 233 / 284 (82%)

R2: 232 / 286 (81%)

R3: 227 / 285 (79%)

R4: 191 / 294 (67%)

Delphi Survey - professionals

Definition of neurodisability

A group of congenital or acquired long-term conditions that

are attributed to impairment of the brain and/or neuromuscular

system and create functional limitations. A specific diagnosis

may not be identified. Conditions may vary over time, occur

alone or in combination, and include a broad range of severity

and complexity. The impact may include difficulties with

movement, cognition, hearing and vision, communication,

emotion and behaviour.

93% agreement

Delphi Survey - questions

1. Which aspects of health do you try to influence and/or improve

when working with children and young people with

neurodisability?

2. (20 most frequently mentioned aspects of health presented)

Do you agree this list covers broadly the health outcomes that

the NHS should assess routinely for these children?

3. If you look at the list, would you agree that this list now

represents appropriate NHS health outcomes for children and

young people with neurodisability?

Key Targets – health professionals

Mental health

Confidence

Emotional stability

Anxiety/attention

Sleep

Pain

Toileting

Movement ability

Manual ability

Acquiring skills

Communication

Mobility

Self-care

Recreation & leisure

Excluding outcomes better assessed clinically, e.g. vision, gait, strength etc.

Priorities – health professionals

R4 - 23 discrete health domains from PROMs - to what

extent the NHS should be responsible for these outcomes?

(from ‘not at all’ to ‘completely’)

NHS ‘accountable’ for more medical outcomes rather

than social participation and well-being.

15 participants: young people, parents, professionals.

2 mixed groups and plenary discussions.

33 cards representing aspects of health.

Forced-choice task to prioritise outcomes.

Stakeholder prioritisation meeting

Q-sort task

LESS IMPORTANT - - - - - - - - - - - - - - - - - - - - - - - - - MORE IMPORTANT

(but none unimportant)

Stakeholder prioritisation - results

Stakeholder prioritisation - interpretation

Ranked most important, both groups:

Communication

Emotional wellbeing

Ranked highly, both groups:

Pain

Sleep

Moving about (mobility)

Independence

Worried (mental health)

Systematic review of PROMs

Identified 41 eligible multidimensional PROMs:

Catalogued characteristics

Mapped content to ICF-CY

Found 126 studies evaluating 25 PROMs

In general population or neurodisability samples

Rated methodological quality (COSMIN)

Rated measurement properties (standard criteria)

Measurement properties

Systematic review - findings

Broad & inconsistent coverage of ICF-CY domains.

Evidence lacking in neurodisability conditions.

Poor evidence of parent-proxy reliability.

No evidence of responsiveness to change.

Stronger evidence: KIDSCREEN, DISABKIDS,

HealthyPathways, CHU-9D.

Proposed core suite of outcomes

Communication

Emotional wellbeing

Pain

Sleep

Mobility

Self-care /

independence

Social activities

Mental health

Behaviour

Toileting

Safety

Unfortunately…

No single PROM assesses the key outcome domains

for children & young people with neurodisability.

Communication not assessed in any PROM

Problems with appropriateness and acceptability of

content and style to families.

Limitations

Not complete agreement on key outcomes.

Potential sample biases?

We did not review ‘domain-specific’ PROMs.

Further policy developments…

Further policy developments…

Recommendations

Build consensus between families and professionals

(and between professionals) on shared goals.

Separate PROMs for children/young people and parents,

capturing different priorities.

Formats for administering PROMs should be inclusive,

attractive and expedient – computer-based.

Evidence of measurement properties so we can interpret

meaningful change (versus measurement error).

Read all about it

Read all about it

Janssens, A., Williams, J., Tomlinson, R., Logan, S., Morris, C. (2014). Health outcomes for children with neurodisability: what do professionals regard as primary targets? Archives of Diseases in Childhood 99:927-932.

Allard, A., Fellowes, A., Shilling, V., Janssens, A., Beresford, B., Morris, C. (2014). Key health outcomes for children and young people with neurodisability: qualitative research with young people and parents. BMJ Open (4:e004611).

Morris, C., Janssens, A., Tomlinson, R., Williams, J., Logan, S. (2013). Towards a definition of neurodisability: a Delphi survey. Developmental Medicine and Child Neurology 55(12),1103-1108.

Plain language summaries www.pencru.org/projectsmeetings/plain_language_summaries/

More papers in preparation…

www.pencru.org

christopher.morris@exeter.ac.uk

Questions & discussion