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Respiratory Effectiveness Group: Steering Committee MeetingThe Arch Hotel, London15 February, 2013 (8.30–16.00, The Martini Library)
Meeting Objective
• Devise an action plan!
o Ideas: What are we going to do?
o Details: How are we going to do it?
o Priorities: When are we going to do it?
o Collaborations: Who’s going to do it?
o Funding: How can we make sure we can
afford it?
Morning Agenda: 8.30–11:40• 8:30–9:00: Introductions & Updates (David /
Alison)
• 9:00–10:20: Sharing Research interests o Jon: 9:00–9:20 Comparative effectiveness Research: an
overview from the USo Jerry: 9:20–9:40 Analytic hierarchy to define priorities for
effectiveness/implementation researcho Richard: 9:40–10:00 The RO1 NIH submission / COPD gene group –
potential collaborationso Leif: 10:00–10:20: Real-life effectiveness of different treatment
modalities of asthma or COPD in patients with significant co-morbidities
• 10:20–10:40 BREAKo Andrew: 10:40–11:00 e-Health in COPD and asthma:
trackingexacerbations, guideline dissemination and physician education
o Gary: 11:00–11:20 The fallacy of asthma guidelines: Discrepancy between science and practice
o Marc: 11:20–11:40 The Respiratory Effectiveness Group in COPD
• 11:40–12:30 LUNCH
Afternoon Agenda: 12.30–16.00
Introductions• Steering Committee
o David Priceo Jon Campbello Richard Martino Jerry Krishnano Andrew McIvor o Leif Bjermero Marc Miravitlleso Gary Wong
• Implementation Manager: Alison Chisholm
• Absent Committee Members:
Christian Virchow; Guy Brusselle; Nicolas Roche; Alberto Papi;
Nikos Papadopoulos; Stephen Holgate, Elliot Israel
Welcome and updateEvolving landscape: David Price What have we achieved so far: Alison ChisholmStudies underway already: David Price
8:30–9.00
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 demonstrate: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 demonstrating the value proposition for new and existing products
“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
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
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
Commercial drivers…
• Drugs must be shown to:o Be safeo Be efficaciouso Address an unmet
clinical need
But also to be:o Cost-effective o Affordableo …“value propositions”
Development of guidelinesInterventionalstudies(RCT):
Efficacy
Safety
Effectiveness
Observationalstudies(pharmaco-epidemiology):
Safety
Cost-effectiveness
Effectiveness
The future of drug research
• Multiple rich heterogeneous and intricately constructed ‘real world’ data sets of Electronic Medical Records and Transactional Claims databases o Surveillance approaches are now being innovatively
applied to such data o Several international initiatives and partnerships
doing essential foundational work in the field o Challenging how to determine how to best utilise this
wealth of data, and how to best incorporate such analyses into overall safety strategies
• Analysis of real world data is only one potential component of an overall continual assessment of risk benefit
However… there are challenges
Obstacle Solution?
No standard methods or endpoints
Carry out validation studiesSet standardsEnforce minimum planning by offering study registration
Concerns around internal validity and confounding of results
Match patientsExplore different matching methods; set gold standard
Cynicism around retrospective data mining
Promote and facilitate a priori study registration
Limited understanding of how to interpret the data
Educate and raise awareness
Limited penetration of the data:– to high impact journals– clinical guidelines
Address the issues above, and the data will start to talk for itself…?
Set up REG an international, collaborative approach
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