multi criteria value-maximization methods for the prioritization of r&d investments in global...
TRANSCRIPT
Multi-criteria value-maximization methods for the prioritization of R&D investments in global health product development
International Collaboration for Capitalizing on Life-Saving and Cost-Effective Commodities (i4C)
Limited resources invested in R&D for global health product development
• Neglected disease R&D funding 2007-2013 (G-FINDER 2014)
R&D investments key for sustaining product development pipelines
*BVGH (2007) Closing the Global Health Innovation Gap: A Role for the Biotechnology Industry in Drug Discovery for Neglected Diseases
A systematic prioritization of R&D investments is required
Which diseases?
• NTDs?
• The big three (HIV/AIDS, TB, malaria)?
• Emerging infectious diseases?
• Maternal and child health conditions?
• other?
What products?
• Vaccines?
• Drugs?
• Diagnostics?
• Vector control products?
• MCH essential commodities?
• Other medical devices?
What R&D areas?
• Basic research?
• Discovery?
• Preclinical?
• Clinical?
• Post-licensure?
Which actors?
• Industry?
• Academic and government research organizations?
• Product Development Partnerships?
Risk of misallocation of scarce resources in absence of systematic prioritization
© Can Stock Photo – csp23071257
Dearth of industry R&D investments due to time, risk, cost and lack of incentives
$236
$359
$391
$447$418
$401 $401
$0
$50
$100
$150
$200
$250
$300
$350
$400
$450
$500
FY 2007 FY 2008 FY 2009 FY 2010 FY 2011 FY 2012 FY 2013
Mili
on
s
Industry investments in neglected disease R&D (G-FINDER 2014)
Discovery & preclinical
Clinical I/II Clinical III Licensure
Basic research / translational researchBasic & translational
research
Average Time: 10 – 15 yearsAverage Risk: 95% chance of failure at discovery
Average Cost: $500mm - > $1.3bn
Variable concerns and motivations to R&D priority setting
x
CSR / reputational concerns?
Social returns?
Health returns?
Financialconstraints
R&D awareness
Technicalconstraints
Optimal R&D priority setting and investment decision making
Project outlineGoal- Assess whether a multi-criteria value-maximization tool can be applied for the prioritization of R&D investments in global health product development
Conceptual Framework
Global
Survey-based mapping of motivations, objectives and criteria for investing in
ND R&D
MCDA model development and testing for prioritizing global R&D
investments in NDs
ClusterMCDA model development and
testing for prioritizing funders’ R&D investments in PDPs
Organization-specific
MCDA model development and testing for prioritizing R&D
investments in a product developer’s R&D candidate portfolio
Methodological Framework
The partnership
Organization Name
Harvard Global Health Institute Suerie Moon
Harvard School of Public Health Joshua Salomon
Instituto de Evaluación Tecnológica en Salud Javier Guzman
K Marsh Consulting Ltd. Kevin Marsh
Technical University of Crete Constantin Zopounidis; Michael Doumpos
Universitair Medisch Centrum St Radboud Rob Baltussen
University of Bergen Ole Frithjof Norheim
University of Bergen Trygve Ottersen
University of California, San Francisco Dean Jamison
University of California, San Francisco Gavin Yamey
WHO Global Health R&D Observatory Riek Viergever
World Health Organization - PHI Zafar Mirza
World Health Organization - TDR Rob Terry
Stage 1: Towards a conceptual framework
Purposeconceptual framework
for systematic prioritization of R&D investments in global
health product development
Targeted review Nature of the prioritization
decision Type of options considered Who is making the choices What level of aggregation
choices are made at What concerns, objectives
and criteria influencing choices
What the model is for processing objectives and criteria
What additional processes are being deployed
Appraisal of pros / cons
identifying options identifying criteria valuing options
Issues emerging from priority setting literature in health research
Examples
• 3D CAM
• ENHR
• CHNRI
• COHRED
• WHO Ad Hoc Committee method
• Numerous informal methodologies
Emerging issues
• consensus building process around research issues requiring urgent attention
• making use of stakeholder judgments based on multiple values and objective criteria
• suitability of approaches varies according to
• level of application
• comprehensiveness of topics addressed
• mix of technical and interpretive techniques
• nature and degree of stakeholder involvement
Issues emerging from priority setting literature in health care more broadly
Examples
• PBMA
• health economic evaluation
• QALY league tables and BoDapproaches
• value of information approaches
• historical allocation processes
• partnership approaches
• clinical variations and payback approaches
• conjoint analysis and DCE methods
• MCDA methods
• ethical frameworks
Emerging issues
• a process of rationing limited resources to meet target group needs
• stakeholder preferences matter and how target groups are approached is critical, including ethical considerations
• central are also issues of:
• what evidence is provided to inform the prioritization process
• what analytic methods are applied to assess performance of options against a range of values and quantitative criteria
Issues emerging from priority setting literature in public policy (linked to health)
Examples
•category-based (cost-benefit) models
•user-based models
• institutional models
•political models
Emerging issues
• systemic process of benefit-cost trade-offs between preferred areas of science towards certain national goals,
• with interplays occur between user-based, institutional, and political forces
• dynamic process whereby priorities change with goals
• recognition of complex political interactions involving multiple stakeholders
• who makes the choices, at what level of decision making and in what relation to national goals and needs also central
• consideration of funding outcomes transform priorities from ways of stressing issues for attention to methods for managing resource allocation
Issues emerging from priority setting literature in R&D management
Examples
• capital budgeting and pharmacoeconomicmodels
• efficiency and productivity analysis models
• optimization and simulation models
• MCDA models
Emerging issues
• organization-specific decision problem of R&D project selection or portfolio management
• under specified constraints and criteria
• accompanied by good managerial judgment
• earlier models of limited practical application due to over-emphasizing mathematical sophistication while being contextually naïve
• recent models recognize importance of stakeholder interactions in resource allocation decisions
• with managerial perception and satisfaction acquiring a central role
Common issues emerging across disciplines
Priority setting can be seen as a resource allocation problem and as an investment decision aiding process when funding outcomes are considered
Priority setting dependent on data for observing reality, and on preference relations for interpreting data in a meaningful way
Valuation of options conducted against pre-defined or interactively constructed metrics to which a specific type of trade-off is always associated
Suitability of empirical methods varies, depending on context of application & balance between methodological sophistication and intuitive judgment of stakeholders involved
Conceptual Framework: Next steps
x 5,492 records identified
391 duplicates removed
5,101 records screened by title/abstract
5,010 records excluded
91 records pre-selected for full
text review
Google scholar
views
1,000 records viewed13 additional records selected
for full text review
Excluded if:
Not a method for pharma R&D priority setting;
Theoretical approach without application on pharma R&D priority setting;
Method from chemical or biology perspective without focus on strategy or resource allocation problems
Database searches
Stage 1: Towards a Methodological Framework
• Research already conducted
– Viergever R, Gouglas D (Draft). The role of multi-criterion decision analysis (MCDA) in health research priority setting. (Eds) Rob Baltussen, Kevin Marsh ‘Multi-criteria Decision Analysis for Health Interventions’ Springer Editions. 2014.
• Relevant findings:
– many health research priority setting approaches apply MCDA elements
– better use of well-established MCDA approaches can increase transparency, explicitness, and analytic robustness of methods deployed
• Next step: Determine how to apply MCDA in R&D priority setting and investment decision making for global health product development
Method Papers (No)
MCDA / Value measurement methods 46
Scoring / weighted sum model (CHNRI methodology) 24
Scoring model (not weighted) 14
Scoring / weighted sum model 7
Nominal Group Technique (not weighted) 1
Qualitative 31
Consultative group process 14
Consensus-based 9
Nominal Group Technique (consensus-based) 4
stepwise approach (literature review, key informant interviews, consultative group process) 2
Survey 2
Mixed methods 21
Listing / Sorting model 11
ENHR 7
stepwise approach (literature review, key informant interviews, consultative group process) 2
Concept mapping 1
MCDA / Qualitative 20
Listing / Sorting model 14
CAM 3
Consensus-based 3
39%
26%
18%
17%MCDA /Qualitative
MCDA / Valuemeasurementmethods
Mixedmethods
Thank you
Dimitrios GouglasAdviser / StipendiatNorwegian Institute of Public HealthPostbox 4404 Nydalen, 0403 OsloNorwayEmail: [email protected]: +47 2107 8013www.fhi.no