respiratory effectiveness group inaugural collaborators’ meeting
DESCRIPTION
Respiratory Effectiveness Group Inaugural Collaborators’ Meeting. Venue: Franklin Hall 3; Marriott Downtown, Philadelphia Date: Sunday 19 May, 2013 Time: 7.30–10.00am Contact: Alison Chisholm, REG Director: alison @ effectivenessevaluation.org. - PowerPoint PPT PresentationTRANSCRIPT
Respiratory Effectiveness Group Inaugural Collaborators’ Meeting
Venue: Franklin Hall 3; Marriott Downtown, PhiladelphiaDate: Sunday 19 May, 2013Time: 7.30–10.00am
Contact: Alison Chisholm, REG Director: [email protected]
Part 1
Respiratory Effectiveness ½ Year in Review OPEN SESSION FOR REG COLLABORATORS; CURRENT AND POTENTIAL SPONSORS WELCOMED
7.30 am – 8:15 am
Meeting Objectives• Meet each other
• Reviewo The motivation for the groupo What’s been achieved so faro What REG is funding in its first year
• Share experienceo What other real-life work REG collaborators are
involve in?
• Definition and Standards o Discuss and agree the framework for a standards
document for all types of real-life research
• Guidelines Groupo Discuss how REG can work with guideline groups to
see real-life data better integrated within the guidelines
Agenda: Session 1 (open session)
Agenda: Session 2 (closed session)
The Origins of the Respiratory Effectiveness Group (REG)David Price, University of Aberdeen and REG founder
7.30 am – 7:40 am
Evolving landscape: timeline
• Brussels Declaration on Asthma: stated a need to include evidence from real world studies in treatment guidelines
• Michael Rawlins (NICE Chairman): RCTs should be complemented by a diversity of approaches that involve analysing the totality of the evidence base
2008
ATS/ERS
Large, prospective studies in ʻreal-worldʼ settings (e.g., trials designed pragmatically to reflect everyday clinical practice) to ensure they provide content validity as well as reflect clinically meaningful outcomes
2009
ARIA / GA2LEN
Proposed the use of composite measures when evaluating asthma control and called for the measurement properties to be validated in clinical trials
2010
NHLBI expert workshopHighlighted areas that need strengthening in order to optimize the potential of real-life/comparative effectiveness (CER) research in pulmonary diseases, sleep, and critical care.
2011
REG was founded!
2012
Drivers for change: EU perspective• Clinical drivers:
o On-going need to improve patient outcomeso Evidence-based decisions require representative data
• Budget pressures increasing the need to demostrate:o Affordability of therapieso Value of therapies
• Pharmao Bringing products to market is only the first step
– Licensing ≠ approved– Licensing ≠ reimbursement– Licensing ≠ usage
o Increasing need to invest in demosntrating the value proposition for new and existing products
Population• Reversibility • Severity• Age / sex mix• Attitude to disease
• Smoke exposure• Concomitant
disease• Individual variation
in response
“real-life” population of patients
Efficacy x
Effectiveness
Clinical drivers: representative data
Clinical drivers: representative dataCriteria for selecting asthma patients to a clinical trial:o Lung function 50–80% predictedo Bronchodilator reversibilityo No co-morbiditieso Non smokers or ex-smokers <10 pack yearso Good treatment complianceo Symptomatic and regular relief medication useo Good inhaler technique
Travers et al. Thorax 2007
Norwegian study of asthma patients to identify who would be eligible for standard clinical trials
1.2%
Herland K, et al. Respir Med. 2005;99:11-9
Patient population
Does it matter if we exclude patients with:o Lesser reversibility than 20%o Active rhinitiso Smokerso Lower adherence o Poor inhaler technique
ANDo Design the study not like real-life?o Study only lasts 3 months?
Clinical drivers: representative data
Yes!
Real-life research: Is it new?
• Existing fields and terminology linked to real-life research:o Comparative effectiveness research (CER)o Epidemiology (pharmacoepidemiology)o Health economics (pharmacoeconomics)o Health policy (drug policy)o Pragmatic randomized controlled trialso Personalized medicineo Outcomes research (patient-centered
outcomes research)o Treatment “value proposition”
Evidence
Theoretical
Theoretical model provide
rationale
Classical double-blind
double-dummy RCTs
Gold standard, large range of
outcomes. But not “real-life” patients, compliance
and represent <10% of patients
Pragmatic trials
More real-life Broader
inclusion criteria Allow normal
factors to occur usually
randomised. Simple
outcomes, but still consent &
rigorous
Observational Data
Real-life patients Not randomised
Routine data Normal decisions
Difficult to ensure group comparability
Matching of case controls,
adjustment
Real-life studies
Clinical drivers: representative data
RCTs Pragmatic Observational
trials studies
Experiment, observation, mathematics, individually and collectively, have a crucial role in providing the evidential basis for modern therapeutics. Arguments about the importance of each are an unnecessary distraction. Hierarchies of evidence should be replaced by accepting – indeed embracing – a diversity of approaches.
Sir Michael Rawlins, Head of NICE, Lancet 2008
Contemporary View
Evidence continuum – complementary study designs
Evidence base: hierarchy—continuum
Traditional View
RCTs
Pragmatic studies
Observational studies
Commercial drivers…
• Testing What We Think We Know. New York Times - August 19, 2012
“The truth is that for a large part of medical practice, we don’t know what works. But we pay for it anyway.”
H. Gilbert Welch, MD,Geisel School of Medicine at Dartmouth
Development of guidelinesInterventionalstudies(RCT):
Efficacy
Safety
Effectiveness
Observationalstudies(pharmaco-epidemiology):
Safety
Cost-effectiveness
Effectiveness