avoiding waste in research: the role of public involvement iain chalmers coordinator james lind...
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Avoiding waste in research: the role of public involvement
Iain ChalmersCoordinator
James Lind Initiative
NCRN Consumer Liaison Group MeetingLeeds, 25 June 2013
better, more equal
‘The Liberati Manifesto’, 2011
Reasons for wanting to participate in clinical trials
Self-interest, albeit with some altruism thrown in
(2012)
7
2001-2005
1977-1985
Patients
1 Demand clinical trials addressing unanswered questions important to you
2 Demand and help to reduce waste in research
Patients
1 Demand clinical trials addressing unanswered questions that are important
2 Demand reduced wastein research
1 Demand clinical trials addressing unanswered questions that are important
2 Demand reduced wastein research
To promote Priority Setting Partnerships involving patients and clinicians to identify and promote their shared priorities for therapeutic
research
The UK Database of Uncertainties about the Effects of Treatments
To publish uncertainties about the effects of treatments which cannot currently be answered by referring to relevant and reliable, up-to-date
systematic reviews of existing research evidence
JLA Priority Setting Partnerships
Completed Current •Asthma•Urinary incontinence•Vitiligo•Prostate cancer•Schizophrenia•Type 1 diabetes•ENT aspects of balance•Life after stroke•Eczema•Tinnitus•Cleft lip and palate•Lyme disease
•Acne•Childhood disability •Dementia•Dialysis•Head and neck cancer•Inflammatory bowel disease•Multiple sclerosis •Pressure ulcers•Pre-term birth•Sight loss and vision •Skin - Hidradenitis Suppurative•Hips and Knees - surgical interventions•Spinal Cord Injury•Palliative Care
Involving patients, carers and clinicians in research priority setting
The JLA’s principles•Inclusive
•Balance of perspectives•Accessible to all
•Supportive•Recognising a range of capacities and skills
•Transparent and democratic•Data sharing•Agreed protocol•Declaration of interests•Neutral facilitation •Communication and feedback
Research priority themes [across asthma,
incontinence, vitiligo, eczema, stroke, prostate cancer, schizophrenia, aspects of balance, and type 1 diabetes]
• Assessment of long-term effects (wanted and unwanted) of treatments
• Assessment of safety and adverse effects of treatments
• Assessment of complementary and non-prescribed treatments
• Assessment of strategies to improve early diagnosis and treatments, and harmonisation of practice
• Assessment of the effectiveness and safety of self-care
23
397
689
29
332
89
69
307
20
0%
10%
20%
30%
40%
50%
60%
70%
80%
90%
100%
JLA patient-clinicianPriority Setting
Partnerships
Registered non-commercial trials
Registeredcommercial trials
Education and training, servicedelivery, psychological, physical,exercise, complementary, diet,other
Radiotherapy, surgery andperioperative, devices, anddiagnostic
Drugs, vaccines and biologicals
Interventions mentioned in research priorities identified byJames Lind Alliance patient-clinician Priority Setting Partnerships,and among registered trials, 2003-2012. (Crowe et al. forthcoming)
www.jla.nihr.ac.uk
Measuring outcomes that matter to patients
Which outcomes of the effects of treatments do cancer patients rate as most important to them?
• Cure• Life expectancy• Freedom from symptoms• Quality-Adjusted Life Years (QALYs)• Tumour size• X-rays/MRI images• Blood/urine test results
1 Demand clinical trials addressing unanswered questions that are important
2 Demand reduced wastein research
Low priority questions addressed
Important outcomes not assessed
Clinicians and patients not involved in setting research agendas
Questions relevantto clinicians &
patients?
Over 50% studies designed without reference to systematic reviews of existing evidence
Over 50% of studies fail to take adequate steps to reduce biases, e.g. unconcealed treatment allocation
Appropriate design and methods?
Over 50% of studies never published in full
Biased under-reporting of studies with disappointing results
Accessible full publication?
Over 30% of trial interventions not sufficiently described
Over 50% of planned study outcomes not reported
Most new research not interpreted in the context of systematic assessment of other relevant evidence
Unbiased and usable report?
50%
85% Research waste = over $85 Billion / year
50%
50%
Overall conclusion:
Patients are suffering and dying unnecessarily because of avoidable
waste in research.
Low priority questions addressed
Important outcomes not assessed
Clinicians and patients not involved in setting research agendas
Questions relevantto clinicians &
patients?
Over 50% studies designed without reference to systematic reviews of existing evidence
Over 50% of studies fail to take adequate steps to reduce biases, e.g. unconcealed treatment allocation
Appropriate design and methods?
Over 50% of studies never published in full
Biased under-reporting of studies with disappointing results
Accessible full publication?
Over 30% of trial interventions not sufficiently described
Over 50% of planned study outcomes not reported
Most new research not interpreted in the context of systematic assessment of other relevant evidence
Unbiased and usable report?
50%
85% Research waste = over $85 Billion / year
50%
50%
Sources of waste in deciding what research to do
Studies designed without reference to systematic
reviews of existing evidence, published and unpublished
Analysis of Introduction sections of all reports of controlled trials in May 2009 and May 2012 in Lancet, New Eng J Med, BMJ, JAMA, & Ann Int Med (Clarke et al. forthcoming)
2009
(n=29)2012
(n=35)First trial addressing the question 5 5
Contains an updated systematic review, which was used to inform the design of a new trial
1 1
Discusses a relevant systematic review, which was not used to inform the design of a new trial
10 13
Refers to other randomized trials 4 10
Although not first trial, does not refer to other randomized trials
9 6
"It is essential that existing sources of evidence, especially systematic reviews, are considered carefully prior to undertaking research…
Research which duplicates other work unnecessarily, or which is not of sufficient quality to contribute something useful to existing knowledge, is in itself unethical."
(Department of Health 2001)
Sometimes the most important advances in knowledge come from analyses of existing evidence
Low priority questions addressed
Important outcomes not assessed
Clinicians and patients not involved in setting research agendas
Questions relevantto clinicians &
patients?
Over 50% studies designed without reference to systematic reviews of existing evidence
Over 50% of studies fail to take adequate steps to reduce biases, e.g. unconcealed treatment allocation
Appropriate design and methods?
Over 50% of studies never published in full
Biased under-reporting of studies with disappointing results
Accessible full publication?
Over 30% of trial interventions not sufficiently described
Over 50% of planned study outcomes not reported
Most new research not interpreted in the context of systematic assessment of other relevant evidence
Unbiased and usable report?
50%
85% Research waste = over $85 Billion / year
50%
50%
Alessandro Liberati
Proportion (%) of clinical trials registered by 1999 and published by 2007
(from Ross et al. PLoS Med 2009;6(9): e1000144).
Country
Size
Phase
Funder
UK HTA program
www.alltrials.net
“(It) is my belief that by taking part in research, I will help other patients in future. For that to happen, the results of the research must be made available. Even if the research isn't finished or the results aren't as expected, the data and information are still of value and should be made available…. Patients become participants to add to knowledge and to eliminate uncertainties. Hiding results, no matter what the reason, isn't in that spirit at all.”
Richard Stephens, 12 June 2013
In summary, patients should require the research community:
• to seek help from patients in selecting questions to address in research
• to review existing evidence systematically before planning new research
• to publish the results of research, whatever their direction or strength
What are patient groups doing to address these problems?
Be more discriminating about what research and
which clinical trials to support
BMJ 2010;340:c725
Chalmers I. Lancet 2000;356:774
www.testingtreatments.org
www.testingtreatments.org
Promote research on the effects of treatments…
…but only if it meets scientific and ethical principles
Promote research on the effects of treatments…
better, more equal