Multi criteria value-maximization methods for the prioritization of r&d investments in global health product development nrc

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)

Women and children particularly at risk

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

A Complex funding flow structure

Funders

Fund Managers

Product Developers

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