The role of feasibility and scoping studies in the development of the programme

grant: Does specialist teenage and young adult cancer care improve outcome?

Susie PearceHealth Services Researcher for Young People with

Cancer

Faith Gibson, Rachel Taylor, Lorna Fern, Catherine O’ Hara, Jeremy Whelan

Overview• Towards a programme of research:

• Background• Methodological underpinnings• Piloting and feasibility studies• The NIHR programme grant• Concluding thoughts

Background• Very little known about experiences of TYA with

cancer • 2000 new cases in the UK each year (13-24

year olds)• Cancer accounts for 11% of deaths in this age

group• Poorer outcomes compared to other age groups• Place of care, poor involvement in clinical trials,

delays in diagnosis, unfavourable tumour biology, all thought to contribute to these outcomes.

• Range of malignancies and treatment • Cancer superimposed the normal developmental

and psychosocial needs of TYA• Importance of disease and age appropriate

settings of care• Improving Outcomes Guidance (IOG) for

Children and Young people’s Cancer (NICE 2005)

• 13 Principal treatment centres in England.• However, as yet limited evidence for the

development of specialist services• National and international need for a substantial

body of work to identify the benefit

Aims• To develop a programme of research to

determine whether specialist care in England, for young people aged 13-24 years is associated with improved outcomes during and after treatment.

Challenges• How? What study design?

– Ideally randomised controlled trial, BUT• Services are already in place • Unethical to randomise to specialist care vs. not• Variation in services across country

• What sample?– Too much variability and small numbers to be

single centre– National cohort, BUT

• How do you identify ALL young people?• How do you recruit ALL young people?

• What should the outcomes be?• What exactly is TYA cancer care?

Methodological framework• Medical research council’s framework for

developing and evaluating complex interventions and theories of complex health care evaluation

• Specialist TYA cancer care - a complex health care intervention

• Evaluation requires a non standard, multifaceted approach

Complex intervention

• Several interacting components• Flexibility in the tailoring of delivery is

permitted• There are a number of outcomes• There are a number of groups and

organisational levels targeted by the complex intervention

MRC framework for evaluating complex interventions

Implementation Dissemination

Surveillance and monitoring Long term follow up

Development Identifying the evidence base Identifying/developing theory

Modelling processes and outcomes

Evaluation Assessing effectiveness

Understanding change processes Assessing cost effectiveness

Feasibility/ piloting: Testing procedures

Estimating recruitment/ retention

Determining sample size

Essence of Care: Phase IPhase 1

Commenced J une 2009

Understanding TYA cancer services

Methodological issues related to Phase 2

Review of longitudinal & panel surveys

in England

Management & Organisation of Phase 2

Interviews with

commissioners & documentary

analysis

Non-participant observation & interviews

with TYA & HP

Workshops with

YP & HP

Literature reviews of TYA cancer

services, outcomes & measures

Interviews with

young people

Evaluation of NHS datasets

& registries

Phase 1Commenced J une 2009

Understanding TYA cancer services

Methodological issues related to Phase 2

Review of longitudinal & panel surveys

in England

Management & Organisation of Phase 2

Interviews with

commissioners & documentary

analysis

Non-participant observation & interviews

with TYA & HP

Workshops with

YP & HP

Literature reviews of TYA cancer

services, outcomes & measures

Interviews with

young people

Evaluation of NHS datasets

& registries

Key Questions for this first phase of work

• What is the current service provision for TYA with cancer in England and what are the anticipated changes in the next 5 years?

• What is the nature of specialist cancer care?• What young people’s experiences of cancer care?• What are the key characteristics of a specialist unit?• What patient reported outcome measures are available and validated

for this age range?• How can we rapidly identify all new diagnoses of cancer in those aged

13-24?• Can clinical data be obtained accurately and timely from existing NHS

databases/ registries?• What are young people’s preferences for a longitudinal survey?• Is there a framework available to address the methodological

challenges of conducting a nationwide survey

Study 1: Current & future policy underpinning TYA cancer

• Semi-structured interviews with four commissioners of TYA cancer services

• Documentary analysis of implementation plans from all SHA’s– Progress reports to the National cancet Action

team from Sept ‘08 to Nov ‘09

What this informed…

• Aspects of study design:

– Multi-centre, national

– What TYA cancer care will look like in 5 years time.

The reality versus the policy.

– Emphasized importance of a work stream in main

programme of research to more fully explore the

varied contexts of TYA specialist care

Study 2: Nature of TYA cancer care

• Non-participant observation– Single principle treatment centre and shared

care units– Observation over 2 weeks of all key events– All areas providing TYA cancer care

• Semi-structured interviews– Three health care professionals– Three young people

What this informed…

• An in depth understanding of the complex nature of TYA cancer care

and processes of multidisciplinary working within one Principal

Treatment Centre

• The methodology, including the development of an observation

schedule for a multiple case study of half of the principal treatment

centres and networks of care in England.

• These case studies will use non-participant observation and semi

structured interviews and document analysis

• Guided the sampling strategy for a multiple case study

Studies 3 - 5

• Study 3: Towards a definition of TYA cancer care unit

• Study 4: Identifying the professional competencies for TYA cancer care

• Study 5: Young people’s experiences of cancer care

Workshops with young people ( n=11) and health professionals ( n=22)

What studies 3-5 informed…• Importance of young people as co-researchers

– Involve young people in study design

– Involve young people in study conduct and analysis

• Highlighted the pattern of communication in TYA

healthcare

• Initial survey for an international Delphi survey in

professional competency in TYA cancer care

• Quality of life as important as survival– Implication for primary outcome

Study 6: Self-reported outcome after treatment for cancer

• Literature review– Search terms related to outcome, i.e., quality

of life– Cochrane + 4 databases– Young people aged 13 – 45 years with cancer

(US definition of ‘young adult’)– Any study design– Study quality assessed with

Cesario/CONSORT/STROBE guidelines

What this informed…

• Identify significant non-medical outcomes• Understand young people’s experiences of

cancer [care]– Underpin content of the Essence of Care,

‘Brightlight’ Survey

Study 7: Outcome measures validated for young people aged 13

– 24 years• Literature review

– Search terms related to outcome, i.e., quality of life

– Cochrane + 4 databases– Participants <16 years and ≥19 years– Only quantitative designs– Instrument quality assessed with McMaster

Outcome Measure Rating Form

What this informed…

• Identify outcome measures that can be used in the cohort study.

• Develop a toolkit of outcome measures used in TYA, with a validity & reliability profile, for use in future research

Study 8: Young people’s preference for participation in a longitudinal

study• Recruitment in two principal treatment

centres• Six young people aged 17 – 21 years• Complete outcome measures

– PedsQL, EORTC, INPATSAT, HUI2, EQ5D– Feasibility, acceptability

• Semi-structured interviews

What this informed…

• Primary and secondary outcome measures

• Study design issues particularly around acceptable methods of data collection in a cohort study

• Importance of involving young people in study design

Study 8: Evaluation of NHS datasets

• Aims– Identify all young people within 90 days of

diagnosis– To provide clinical data

• Review national datasets• Criteria:

– Minimum data: NHS number, name, gender, age, address, diagnosis & place of care

Feasibility study of processes

Results: identification of a dataset

What this informed…•Cancer Waiting Time dataset had overall completeness of 99%

–Young people can be identified within 90 days•Only suspected cancer therefore needs confirmation

•Additional data in other datasets–Clinical data available without relying on medical notes

•Quality of data is not confirmed•Missing data•Lack of reliable death data

•Further study in programme grant to validate the national data sets through the cohort study

Study 10: Framework for the Essence of Care study

• Challenges for Phase II– Can we identify all newly diagnosed young

people in England in 2012? – How can they be recruited??????– How can data be collected??????

Yes

Solutions

• NIHR funded project = access to NCRN nurses– Will they recruit young people?

• Search existing national cohort studies (e.g. Millennium Cohort Study)

• How do they data collect?– Commercial research companies– Is this economically feasible?

Yes

Yes

Developing the programme grant• Identification of co-applicant team• Feeding pilot studies into programme grant

development• Regular co applicant team meetings through the

feasibility phase• Ongoing refinement and stages of submission • Successful NIHR Applied health programme

grant £1999,957. ‘Do Specialist Cancer Services for TYA add value’ Cohort to begin recruitment 1/7/12.

The 2012 TYA Cancer Cohort Study: developing team working

UCLH:• Dr Jeremy Whelan (CI)• Susie Pearce• Martin LernerUCL:• Dr Julie Barber • Professor Steve Morris• Professor Rosalind Raine• Dr Rachel TaylorUniversity of Leeds:• Dr Richard FeltbowerSt James’ University Hospital• Dr Dan Stark

Cancer Research UK• Dr Lorna Fern GOSH/LSBU• Professor Faith Gibson NCAT• Louise HookerNWCIS• Dr Tony Moran• Dr Catherine O’HaraNCRI CSG TYA CCG:• Hannah Millington

Workstream 1: the description of specialist TYA cancer care

• Explore the culture of care through non-participant observation, interviews and documents analysis

• Identify the specialist competencies and added value of specialist health professionals through a Delphi survey

• Validate a bespoke scale to categorise 3 levels of TYA care (TYA Cancer Specialism Scale)

Workstream 2: cohort study of all TYA diagnosed during an 18 month period

• Accrual within 3 months of diagnosis and follow-up will continue for 3 years

• Relate the level of specialist care received to: – Patient reported outcome. QL, satisfaction with care.

– Clinical processes and outcomes

– Experience of cancer care.

– Achieving social and educational milestones

– Geographic and socio-demographic inequalities

Workstream 3: health economics

• Compare costs to the NHS and personal social services between TYA receiving different levels of specialist care

• Estimate the cost to TYA and families of specialist care

• Calculate the cost effectiveness of different levels of TYA care

Workstream 4: intervention development & service evaluation

• Workshops with clinicians and policy makers

• Aiming to throughout the programme of research to feed findings into practice, service development, policy making and the development of new areas of research

Summary

• Complex evaluation of health care need complex study designs

• MRC framework for evaluating complex interventions– Importance of feasibility & pilot studies– Importance of understanding processes and

context• Importance of these stages for successful

programme development grant

Conclusions• Challenge of the development of feasibility studies

in synchrony with the development of the programme submission

• Feasibility and development work ongoing• Importance of the development of team working

(and the most appropriate team) through this process.

• Role of nurses in understanding the complexities of health care; leading practice and service delivery.

• Nurses skills as researchers are thus crucial to the facilitation, development, management and leadership in programmes of research evaluating service delivery and patient experience and outcome.