Setting a research agenda with the JLA Priority Setting ... - Findacure

Setting a research agenda with the JLA Priority Setting Partnership David White Chair, Trustees of Cavernoma Alliance UK

www.cavernoma.org.uk/psp/

My brief from Flóra was to present “the JLA process and how this facilitated our partnership with researchers”. 1. The JLA Process = Priority Setting Partnerships (PSPs) 2. Our partnership with researchers

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Findacure 24 June 2016

A Priority Setting Partnership is A partnership of clinicians, patients and carers to identify a prioritised list of uncertainties for a given condition (An uncertainty is a question to which it is known that the answer is unknown) I’ll use the Cavernoma PSP for examples and start with a brief introduction to cavernoma 3


Cavernoma

• Bloody caverns in the brain / spinal cord • Impact on nerve tissue causing neurological problems • Symptoms depend on location Epilepsy

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Cavernoma

• Bloody caverns in the brain / spinal cord • Impact on nerve tissue causing neurological problems • Symptoms depend on location • • • • 5

slurred speech difficulty in walking, tremor on his right side double vision Findacure 24 June 2016

Cavernoma • 1/600 have cavernoma 100,000 • 1/12,500 have symptomatic cavernoma 5,000 • Symptomatic cavernoma: a rare condition • About 25% genetic

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Cavernoma

• Treatment options? • Treat symptoms (if possible) • Remove surgically (if safe) • Gamma knife ‘surgery’ • Very poor evidence base for the best treatment

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SO How can we find out what clinicians and patients/carers identify as the important research questions?

ONE ANSWER The James Lind Alliance was formed to provide a framework to do this via a Priority Setting Partnership (PSP)

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JLA • Organisation set up in 2004, now hosted by the National Institute for Health Research (NIHR) • Sets procedures = quality

David White, Amy Street, Katherine Cowan, Caroline Whiting, Beccy Maeso, Ian Stuart

• Originally a PSP just considered clinical care and treatment, but topics can be broader now • Outputs – prioritised list of 10 uncertainties • Published and promoted to researchers and research funders 14


Interested parties • Clinicians • Patients (and their carers/relatives) • Researchers • Applied: Clinical issues • Blue-sky

• Commercial organisations • Drug Companies, medical aids … …

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JLA PSP works with • Clinicians • Patients (and their carers/relatives)

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but not with: • Clinicians • Patients (and their carers/relatives) • Researchers • Applied: Clinical issues • Blue-sky

• Commercial organisations • Drug Companies, medical aids … …

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To get started

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Costs

Data is for Cavernoma PSP

• Project Manager (appointed by JLA)

£5,000

Low

• Information specialist (CRITICAL)

£4,500

VERY low

• Administrator

£2,000

Lowish

• Administration

£500

Low

• Steering Group Meetings Travel Subsistence

£2,000

Depends

• Final Workshop Travel Subsistence Lunch etc

£2,500

Normal

• Final Workshop Facilitators

£2,300

Normal

• Room hire

£0

Depends

• Web site

£0

Depends

• Publication TOTAL

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£2,000 Findacure 24 June 2016

£20,800

Not normal

Steering Group

• Relevant Clinicians

• Patients • Carers • Relevant Charities • NOT Researchers • NOT Industry

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Protocol for PSP

• What you are going to do • How you are going to do it • Who’s going to do it • What are you going to do with it when you’ve go it • What are you going to do later

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The JLA Process

CONSULTATION

Gathering Questions

COLLATION

Sifting / formatting

Long List

PRIORITISATION

Short List Output : Top 10

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OUTPUT

DUETS


Consultation

Gathering Questions

Collect ideas from all individuals with an interest e.g. • • • • •

Clinicians (e.g. via professional organisations) Patients (from support charities, via clinicians. …) Carers (from support charities, via clinicians. …) Support organisations The research literature

Collect

• Via survey(s) • Specialist to read the literature 24


Research Categories

• Aetiology (cause)

• Clinical Issues: • Management and Treatment • Diagnosis • Prognosis

• Therapeutics

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The JLA Process

CONSULTATION

Cavernoma PSP Survey

Literature

2268

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Gathering Questions

2302

COLLATION

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COLLATION


The Questions were not all unique • Many asked the same question

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For example  “Are there any activities to be avoided?”  “Am I able to lead a normal life?”  “What activities should my daughter avoid at school if she has a cavernoma?” These, and 139 other original questions became grouped under:

 “Is there any evidence that specific physical activities can trigger cavernoma symptoms?”

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COLLATION


The Questions were not all unique • Many asked the same question The Questions were not all “unkowns” • Patients especially often asked questions whose answers were known, but not by them! • The Information specialist dealt with this

Some ‘Questions’ were not questions

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The JLA Process

CONSULTATION


Literature

2268

34

Gathering Questions 2302

COLLATION

Sifting / formatting 79

Long List

PRIORITISATION

Short List Output : Top 10

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Prioritisation

Long List

Generated by the Steering Group Generated from a survey

Short List

Output : Top 10 31

• All those who submitted questions were asked to give each Long-List question a score on a 15 scale • The mean score was determined for each question for each of Clinician, Patients , Carers/others • The rank orders combined to produce a Short List

Generated at a full-day workshop with JLA facilitators Findacure 24 June 2016

The JLA Process

CONSULTATION


Literature

2268

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Gathering Questions 2302

COLLATION

Sifting / formatting 79

Long List 54

PRIORITISATION

Short List 29 Output : Top 10 10 / 27

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OUTPUT

DUETS


Submission to UK DUETS The main steps involved for adding the uncertainties to the JLA website and giving more detail to researchers and research funders were: • Preparation of the questions in PICO (Patients, Intervention, Comparison, Outcome) format

• Identifying existing systematic reviews or guidelines relevant to each question • Identifying the source of each question (patients, carers, clinicians, published source) 33


The Benefits to us

• Identified the priorities • Gave us fantastic new contacts with clinicians

• Gave an impetus to answering the research / uncertainties • FAQs for patient/carer community • New opportunities open

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CAUK’s Research Needs The Research Questions identified from the PSP require research at different levels: Category Treatments Aetiology / causes Diagnosis Self-management Care & support Prognosis Genetics General questions

Top10 2 2

RCT: Randomised clinical trials 35

1 4 1

Top27 8 4 2 1 2 5 2 3

Type of Research RCT / Clinical DB / Drugs Basic Research Clinical DB + MRI scans Clinical DB Clinical DB Clinical DB Applied Research Basic / Clinical DB

Clinical DB: Clinical Database


Potential Researchers Category

Type of research

‘Academic’ Researchers

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Blue sky

Inquisitiveness with potential development

Applied

En route to commercial

Clinicians

Randomised Clinical Trials Observational Studies

Commercial organisations

Drug development


Types of Research Randomised Clinical Trials (RCT) • Numbers may make this impossible, or not stand-alone, but under investigation with survey of CAUK members Controlled Clinical observation onto Cavernoma Database • Urgent to get started. Should be developed with wide range of users/uses in mind: clinical, natural and social science (e.g. health economics) Cell biology and genetics of development, growth and genetics of cavernoma • Happening in USA, but not (as far as I know) in UK Drug development: Basic  Applied  Commercial  RCT • Happening in USA, but not (as far as I know) in UK 37


www.cavernoma.org.uk/psp/ includes access to supporting files

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