reg interstitial lung disease working group meeting
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Saturday May 16th, 16.30-18.00 (MST)Altitude Room; Crowne Plaza, Denver, Colorado
REG Interstitial Lung Disease Working Group Meeting
Working Group Members
Lead: Luca Richeldi
• Bruno Crestani• Martin Kolb• Toby Maher• Francesco Bonella• Fernando J Martinez• Ganesh Raghu• Ian Glaspole• Katerina M. Antoniou• Kevin Brown• Ulrich Costabel
• Moises Selman• Thomas Geiser• Nathan Steven• Mazzei Mariano• Vincent Cottin• Demosthenes Bouros• Mona Bafadhel• Carlo Vancheri
Blue indicates confirmed attendance at the time of slide preparation
REG Supporters in attendance
Confirmed• Daniel Mcbryan• Armin Furtwaengler• Lynn Hagger• Mark Milton-Edwards• Setareh Williams• Brooke Harrow• Gokul Gopalan• Glenn Crater• Keith Allan• Paul Michael Dorinsky
• Robert Fogel• Dorothy Keininger• David Evan• Karen Mezzi• Peter Schweikert• Guilherme Safioti
Tentative / Maybe• Mark Milton-Edwards• Carlo Vancheri
Agenda
Time Item Lead
16.30-16.35 Introductions All
16.35-16.40Overview of the Respiratory Effectiveness Group
Alison Chisholm
16.40-17.45
Research Ideas Luca Richeldi
• Characterizing healthcare resource utilisation and pathway in the period prior to an ILD (or IPF) diagnosis
• Electronic lung sounds – building an audio component into early ILD diagnosis
• Evaluation in the consistency of MDT diagnoses of ILD• Other ideas from the group...?
17.45-18.00 Other Opportunities for the Group Group Discussion
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
Effect‘efficacy’ Safety
High ‘internal’ validity feasible in clear-cut trial populations
APPROVAL
Real ‘external’ validity &generalisability by mirroring
real populations and healthcare practices
Medicines won’t work if peoplecan’t or don’t take them
Needs of Regulators
Effectiveness/Outcomes
Device‘to train, or not to train’?
Adherence
CAN IT WORK IN AN IDEAL POPULATION OPTIMALLY MANAGED?
DOES IT WORK IN REAL PATIENTS MANAGED IN ROUTINE CARE
SETTINGS?
Needs of Patients, Physicians, Payers
Efficacy vs Effectiveness
• Studies have shown that efficacy RCTs exclude about 95% of asthma and 90% of COPD routine care populations due to strict inclusion criteria.1
1. Herland K, et al. Respir Med 2005;99:11–19.
Limitations: RCTs inclusions/exclusions
COPD
Asthma
Patient RCT eligibility drop-off with sequential application of standard inclusion criteria
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
What sort of evidence do we have …?P
op
ula
tio
n
Broad
Narrow
Ecology of care FreeConstrained
Highly controlled Pragmatically controlled
Observational
Managed as...
Clinical diagnosis
Confirmed diagnosis
Registration RCTs
Long term phase III
Pragmatic RCTs
Observational studies
Roche N, Price D et. al 2013 Lancet Respir Med; 1(10):e29-30
Different research questions need different research approaches to answer them…
So…? Set up REG:Our wish: To raise the quality and profile of real-life (respiratory) research so that it can be used (appropriately) to inform guidelines and management of patients in routine care.How? Through:•International collaboration •Research•Standardizing methods •Setting quality standards
& Much more…
Prior REG / UK Department of Health Collaboration
• Jones RCM, et al on behalf of REG. Lancet Respiratory Medicine. 2014; 2:267-76
• Funding by the UK Department of Health and Research in Real Life Ltd.
REG Support & Research Funding
RESEARCH IDEA GENERATION
Working Groups Identify Research Priorities in their
respective fields of expertise
SECURING FUNDING
Nature of funding dictates future study course:• Single commercial funding source: an investigator
initiated study conducted external to REG• Non-product/brand specific grant(s): an REG
Collaboration carried by REG or in partnership with REG
REG Supporters
Non-supporter& wider
institutional grants
OR
REG develops idea in
collaboration with WG /
collaborators &
seek specific research grants
REG Grants awarded at FY end
REG Core Grants Awarded to Top Priority Ideas
(subject to available funding)
Mid October Core Grant submission
deadline
Ideas prioritised by REG Research Committee
If insufficient REG Funds, continue to look externally
Agenda
Time Item Lead
16.30-16.35 Introductions All
16.35-16.40Overview of the Respiratory Effectiveness Group
Alison Chisholm
16.40-17.45
Research Ideas Luca Richeldi
• Characterizing healthcare resource utilisation and pathway in the period prior to an ILD (or IPF) diagnosis
• Electronic lung sounds – building an audio component into early ILD diagnosis
• Evaluation in the consistency of MDT diagnoses of ILD• Other ideas from the group...?
17.45-18.00 Other Opportunities for the Group Group Discussion
Where does the ILD WG Fit…?• 2014: first two (first-in-class) treatments for IPF:
o Nintedanib & pirfenidoneo Slow disease progression; do not cure or reverto NEED FOR EARLIER DIAGNOSIS
• Many forms of ILD diseases, but small prevalence of eacho Need to aggregate data…
• All patients come through primary careo Time to start looking in primary care databases…
• Need to raise awareness of ILD among primary clinicians:o Put ILD on the diagnostic “agenda” o Develop diagnostic support tools
Idea I: Missed Diagnostic Opportunities
• There is a need to understand what happens to ILD patients before they receive their diagnosiso How and when do they engage with healthcareo What tests do they receive prior to their diagnosis?o What triggers the diagnosis?o Are there “red flags” that are being missed?
• Retrospective database study…?
Optimum Patient Care Research Database
Cohort Patients"Prevalence" within database population
Total Patient Population 2,414,621
Asthma Population 755,693 31.30%
COPD Population 134,281 5.56%
Pulmonary fibrosis (idiopathic)
2,955 0.12%
Interstitial lung disease 5,299 0.22%
UK primary care database available to REG:
Idea II: MDT diagnostic consistency
• IPF diagnostic pathway many steps, e.g. :o Local chest clinic i.e. secondary care diagnostic
work up o X-ray o HRCT scano Lung Volume and Airflow Studies o Simple Gas Exchange Studies o Simple Lung Function o Exercise Testing (e.g. six minute walk, shuttle walk)o Lung biopsyo MDT diagnosis
But what is the variation in the diagnosis of IPF across different MDTs at different centres / different countries?
Idea III: Prospective case finding
• IPF symptomso The classic sign of IPF is fine, dry, inspiratory crackles
(“Velcro crackles”) at both bases.o The remainder of the examination is normal until disease is
advanced, at which time signs of pulmonary hypertension and right ventricular systolic dysfunction may develop.
• Respiratory Assessment:o Listening to the chest is a standard component of a
respiratory assessment.o Where is the information documented…?o Discerning changes in chest “noises” requires a standard
approach to audio assessment & recording.o Listening is “cheap”, even with electronic tools.
Agenda
Time Item Lead
16.30-16.35 Introductions All
16.35-16.40Overview of the Respiratory Effectiveness Group
Alison Chisholm
16.40-17.45
Research Ideas Luca Richeldi
• Characterizing healthcare resource utilisation and pathway in the period prior to an ILD (or IPF) diagnosis
• Electronic lung sounds – building an audio component into early ILD diagnosis
• Evaluation in the consistency of MDT diagnoses of ILD• Other ideas from the group...?
17.45-18.00 Other Opportunities for the Group Group Discussion
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