The James Lind Alliance and I

1
The James Lind Alliance - and I

• Lester Firkins
• Priority setting for Systematic Reviews
  Conference
• US Cochrane Center
• 11th July 2008

2
Introduction

• Who am I and how did I get here?
• What did the journey teach me?
• Is this the way research should be?
• There is another way
• The James Lind Alliance

3
Who is Lester Firkins?

• Middle aged, middle class, white, male
• 35 years in UK banking industry
• Conventional
• Content with rules and structure
• Performance and achievement driven
• Fairly well read - with a broad understanding of
  most things
• Medicine and research is looked after by others -
  and always in my best interest
• My money is well spent

4
My journey starts here

• Ellis Firkins
• First Class Honours graduate in Design Technology
• Teacher at major UK public school
• Died on 1 March 2001 from variant
  Creutzfeld-Jacob Disease (vCJD)

5
My journey

• Chair, Human BSE Foundation
• Co-chair, Prion-1 Clinical Trial, Medical
  Research Council (MRC)
• Co-chair, New Therapies Scrutiny Group
• Member, National Institute of Clinical Excellence
  (NICE)
• Chair, James Lind Alliance

6
Consumer workshop on CJD trials

• MRC and Department of Health (DH)
• To enable consumer understanding of clinical
  trial design and provide an opportunity for
  consumers and researchers to share ideas
• Strong support for trial design
• Strong support for collecting research
• Need for design to be flexible to accommodate
  emerging therapies
• Acknowledgement that it is good to be involved

7
MRC Prion1 Clinical Trial

• 2004 - A partially randomised patient preference
  trial to evaluate the activity and safety of
  quinacrine in human prion disease
• Trial Steering Committee
• Sir Iain Chalmers and Lester Firkins
• Why Quinacrine?
• Systematic review
• Daily Newspaper

8
New Therapies Scrutiny Group

• MRC Committee to look for emerging therapies
• Co- Chairs
• Sir Michael Rawlins and Lester Firkins
• Recommendations for future research funding and
  effort
• Theatre of conflict and dysfunction

9
The Missing Element

• Systematic Review
• ..of treated and untreated patients with human
  prion disease
• Published in Spring 2008
• Results too late to influence Prion-1 (now
  closed)
• Flupirtine may have been a wiser option than
  Quinacrine (but no media lobby)
• Clear evidence that publication had been hindered
  by vested interests

10

• A systematic review of therapeutic
  interventions in human prion disease

Project GroupLesley Stewart1, Larysa
Rydzewska1 Geraldine Keogh2, Richard
Knight3Advisory GroupIain Chalmers4, Lester
Firkins5, Sarah Walker1 1MRC Clinical Trials
Unit, 2National Prion Clinic, 3National CJD
Surveillance Unit, 4James Lind Library, 5MRC New
Therapies Scrutiny Group
11
The Unspeakable Element

• Minimal international collaboration
• Academic jealousies
• Directive from Chief Medical Officer on how UK
  institutions must work together
• Monthly meeting between Heads of Research Units
  to ensure that they work together

12
The Isolated Element

• Pentosan polysulphate
• Promoted by one man looking after his son
• 2003 - High Court agreement for (unapproved)
  therapy to be administered
• 3 patients still alive - longer than normal
• No coordinated monitoring whatsoever
• Mutual distrust leading to complete lack of
  learning and patient care

13
What have I seen?

• A hugely expensive trial can be set up with no
  formal understanding of what has been done in the
  past
• Media can influence decisions more than grounded
  knowledge
• Unless potential therapies find a friend their
  application can be frustrated
• Patient First promoted as an aspiration - but
  not if it conflicts with academic interests
• Dysfunction and jealousies betray the interests
  of patients and their families

14
What about a formal programme? One option

• James Lind Alliance

15
The James Lind Alliance

• Formed in 2004 by
• Royal Society of Medicine
• James Lind Library
• INVOLVE (formerly Consumers in NHS Research)
• Funded by DH and MRC till 2010, to see if it
  works
• Tackling treatment uncertainties - together
  (Patients and Clinicians)

16
What are our objectives

• To support Working Partnerships of patients
  (and their carers) and clinicians to prioritise
  Treatment Uncertainties into a list for
  research funders to address
• To support and raise the profile of proper
  involvement for all people with an interest in
  medical research
• To gain evidence on how best to prioritise with
  Patients and Clinicians collaborating

17
JLA - working partnerships

• Asthma
• JLA approached by British Thoracic Society and
  Asthma UK
• Worked together over many months.
• Harvesting Uncertainties
• Checking and verifying known unknowns
• Entering into a standard database DUETs
• Priority setting meetings - top ten list agreed
• What Next
• BMJ article in preparation
• Systematic review of inhaled and oral steroids
  commissioned
• HTA standing ready!

18
JLA - working partnerships

• Urinary Incontinence
• JLA approached by Incontact and University of
  Aberdeen (Cochrane)
• Approached 30 related organisations - of which 21
  have joined as formal partners
• Currently Harvesting Uncertainties - to conclude
  by 30th June
• Data cleaning and priority setting by December
  2008
• Schizophrenia
• Vitiligo
• Diabetes

19
NIHR HTA Annual Report 2007

• The HTA programme encourages patient involvement
  at every stage and it is continuing to take this
  forward and develop it
• . the HTA programme is working with the JLA to
  capture those priorities suitable for HTA
  research.

20
WIFM -Patients

• Real involvement in their problems
• Share of Voice
• Someone showing an interest
• Participation

21
WIFM Clinicians

• Opportunity to share their unique knowledge and
  perspective
• Getting their personal and professional concerns
  over Uncertainties acknowledged and addressed

22
WIFM Researchers

• Ideas that make a real difference
• Support in the ethics process
• Participation within the research itself
• Advocates and disseminators
• Seeing the difference in peoples eyes

23
Will the JLA work?

• Three years to find out!
• It needs to be seen as invaluable at every level
• It needs to find a permanent home
• It may stimulate others to do something better

24
Thank You

• Delighted and proud to be able to tell my story
• Will it make a difference?
• Could it work in America?
• No reason why it wouldnt be worth a try
• www.lindalliance.org