Public-Private Product Development Partnerships for Global Health R

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Public-Private Product Development Partnerships
for Global Health RDIssues Challenges

• Suerie Moon
• Giorgio Ruffolo Doctoral Fellow in Sustainability
  Science
• Doctoral Candidate in Public Policy
• Center for International Development, Harvard
  Kennedy School of Government
• suerie_moon_at_hksphd.harvard.edu
• Presentation to Designing Strategies for
  Neglected Disease Research
• Spring 2009
•  UC Berkeley Law 284.26/Public Policy 290, 190
• Professors Stephen Maurer Amy Kapczynski
• 10 March 2009

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Overview

• 1. Introduction History, Organizational Form,
  and Funding
• 2. Strengths and Weaknesses
• 3. Targeted Diseases  
• 4. Access Provisions Policies IP Management
• 5. PDPs in the broader RD Ecosystem
• 6. Governance Issues
• 7. Conclusions

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1.1 Introduction History of global health
product development

• 1900s-1950s National RD efforts led to
  trickle-down approach
• 1960s-80s International bifurcated
  public/private system
• Public e.g. UNICEF-UNDP-World Bank-WHO Special
  Programme for Research and Training in Tropical
  Diseases (TDR)
• Private e.g. Industry, globalizing intellectual
  property rights system
• 1990s Global health neglected diseases
• Linkages research, health, development
• Medicines as global public good access to AIDS
  treatment
• Global health needs 10/90 Gap and neglected
  diseases
• 2000s Global health for all?
• New system for Neglected Diseases (Type III)
• PDPs a fad and a sacred cow?
• Trust and networks built low-hanging fruit
  harvested
• Honeymoon period over?
• Diseases of global incidence? (Type I)

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1.2 Introduction History of PDPs
Source Ziemba, 2005.
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1.3 Introduction Definition Examples of
PDPsDefinition Public health driven
not-for-profit organisations that drive neglected
disease drug product development in conjunction
with industry groups (Moran et al., 2005)

• IDRI Infectious Disease Research Institute
• IOWH Institute for OneWorld Health
• IPM International Partnership for Microbicides
• LAPDAP LAPDAP Antimalarial Product Development
• MDP Microbicides Development Program
• MMV Medicines for Malaria Venture
• MVI Malaria Vaccine Initiative at PATH
• MVP Meningitis Vaccine Project at PATH (Program
  on Technologies for Health)
• PDVI Pediatric Dengue Vaccine Initiative
• PneumoADIP Pneumococcal Vaccines Accelerated
  Development and Introduction Plan
• RotaADIP Rotavirus Vaccine Program
• SAAVI South African AIDS Vaccine Initiative
• TB Alliance Global Alliance for Tuberculosis
  Drug Development

• Aeras Global Tuberculosis Vaccine Foundation
• BVGH BIO Ventures for Global Health
• CONRAD Contraceptive Research and Development
  Program
• CICCR Consortium for Industry Collaboration in
  Contraceptive Research
• DNDI Drugs for Neglected Diseases initiative
• EMVI European Malaria Vaccine Initiative
• FIND Foundation for Innovative New Diagnostics
• Gates/UNC Gates Foundation/University of North
  Carolina Partnership for the Development of New
  Drugs
• GMP Global Microbicide Project
• HHVI Human Hookworm Vaccine Initiative
• IAVI International AIDS Vaccine Initiative

Source Ziemba 2005
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1.4 Introduction Basic PDP model

• PDP is a non-profit entity that manages a
  globally-dispersed portfolio for a disease.
• Generalize with great caution
• PDPs vary on
• One disease or multiple
• Breadth or depth in portfolio management
• External or in-house research production
  capacity
• Relative emphasis on public vs private sector
• Level and diversity of funding sources
• Definition of core and secondary missions
• Approach to intellectual property management
• Governance structures and styles
• Scientific political challenges faced

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1.5 Introduction Basic PDP model

• Frequently-conducted functions include
• Finances research ( to and from private public
  sectors)
• Negotiates access to private sector compounds,
  experts, labs
• Reduces risk of projects
• Offers reputational benefits, CSR, employee
  morale
• Provides access to research with profitable
  spillovers (Amyris)
• Liaises with developing countries re clinical
  trials delivery
• Helps open up new emerging markets
• Focuses on adaptedness and affordability
• Advocates for more RD
• Sometimes
• Conducts in-house research (Aeras, IAVI)

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1.6 Introduction ND RD Funding to PDPs
Of 2.5 billion invested in ND RD in 2007, 23
(575 mln) routed through PDPs.
Source From Neglected Disease Research and
Development How Much Are We Really Spending?
Moran M, Guzman J, Ropars AL, McDonald A, Jameson
N, et al. PLoS Medicine Vol. 6, No. 2, e30
doi10.1371/journal.pmed.1000030
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1.7 Introduction PDP Funding 1994-2004
1994-2004
Source Ziemba, 2005.
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1.8 Introduction PDP Funding

• Neglected disease research 2.5 billion in 2007
• Overall global health research 125 billion/yr
• Largely financed by bilateral donors and
  foundations, Gates in particular
• Gates is majority funder of many PDPs (Aeras,
  FIND, IoWH)
• Funding short term uncertain
• Long-term strategic planning difficult
• Missed opportunities when rapid reaction needed
• Weakened negotiating leverage due to risk
• Dramatically increased future needs as products
  move through clinical trials
• Answers?
• Profit-making to underwrite research?
• Auctioning assets e.g. using PRV?
• UNITAID-type global tax?
• CGIAR-type donor support?

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Overview

• 1. Introduction History, Organizational Form,
  and Funding
• 2. Strengths and Weaknesses
• 3. Targeted Diseases  
• 4. Access Provisions Policies IP Management
• 5. PDPs in the broader RD Ecosystem
• 6. Governance Issues
• 7. Conclusions

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2.1 Strengths Weaknesses Challenges for
Evaluation

• Evaluating individual PDPs difficult because
• Varying disease profiles
• Scientific
• Economic
• Political
• Long lead times
• Complex expectations
• Varied approaches
• For a full discussion of the challenges of
  evaluating PDPs, see Toward a New Approach to
  Product Development Partnership Performance
  Measurement, June 2007, by FSG Social Impact
  Advisors.

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2.2 Strengths Weaknesses What does the
literature say?

• Strengths
• Renewed ND RD
• From 2000-2005 0 to 63 drug projects 8 or 9
  new drugs expected
• Affordability adaptedness as central criteria
  for new products
• Harnessing private sector capacity for public
  ends (CSR)
• Light networked structure
• Global coordination of scarce investments

• Weaknesses
• Untested institutional model will it work?
• Financial sustainability unclear
• Governance unclear (accountability,
  decision-making, transparency)
• Developing country participation is limited
• Risk that private interests undermine public
  goals
• Limited to ND (diseases without a market)

Sources Kettler Towse (2001), Ziemba (2005),
Moran et al. (2005), Widdus (various), Buse
(various), Turner Makgoba (2008), Moon
(forthcoming)
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2.3 Strengths Weaknesses Selected
Achievements

• New products
• 1.Paramomycin (V.leishmaniasis in India)
• 2.ASAQ (malaria)
• 3.ASMQ (malaria)
• 4.Pediatric AR-LU (Coartem for malaria)

• Phase III clinical trials/equiv
• Moxifloxacin (TB)
• PyronaridineAR (malaria)
• Dihydroartemisinin (malaria)
• Paramomycin (V.leishmaniasis in Africa)
• FastPlaque diagnostic (TB)
• LAM-based diagnostic (TB)
• Synthetic artemisinin (malaria)

Based on study of only 8 PDPs list is
non-exhaustive.
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2.4 Strengths Weaknesses Open Questions

• How many PDPs?
• One disease vs. Many?
• Compete vs Collaborate?
• Push vs Pull mechanisms?
• Access/affordability vs. Sustainability?
• Efficiency vs. Capacity building?
• Product development vs Delivery?

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Overview

• 1. Introduction History, Organizational Form,
  and Funding
• 2. Strengths and Weaknesses
• 3. Target Diseases  
• 4. Access Provisions Policies IP Management
• 5. PDPs in the broader RD Ecosystem
• 6. Governance Issues
• 7. Conclusions

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3.1 Target Diseases Overview

• Dengue PDVI
• Diarrheal Disease IOWH, Rotavirus RotaADIP
• Family Planning PATH, CONRAD, CICCR
• HIV IAVI, SAAVI
• Hookworm HHVI
• Malaria MMV, EMVI, MVI, IoWH, DNDi, LapDap, FIND
• Meningitis MVP
• Microbicides (HIV STIs) IPM, MDP, GMP
• Pneumonia PneumoADIP
• Tropical Neglected Diseases (Multiple) IOWH,
  DNDi, BVGH, FIND, Gates/UNC, IDRI
• TB Aeras, FIND, GATB

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3.2 Target DiseasesPriority-Setting

• Among diseases no norms for how to measure needs
  and translate into investments
• Within diseases PDP Scientific Advisory bodies

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Overview

• 1. Introduction History, Organizational Form,
  and Funding
• 2. Strengths and Weaknesses
• 3. Target Diseases  
• 4. Access Provisions
• 5. PDPs in the broader RD Ecosystem
• 6. Governance Issues
• 7. Conclusions

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4.1 Access provisions Affordability

• Affordability a central part of the mission
• But, most access provisions confidential (trust
  me approach) why?
• Fear of loss of competitive advantage among firms
• Loss of negotiating leverage for PDP
• Compromised patent application for firms
• Lack of detailed information on compound in early
  stages

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4.2 Access provisions Strategies

• Limited Experience
• Access strategies include
• Global market segmentation (tiered pricing)
• Country categories often undefined
• Challenge Middle income countries
• Public sector vs private markets in LMICs
• e.g. TB drugs in India
• Exclusive licensing
• Cost audits
• Target prices
• Open access (no patent or multiple licenses)
• DNDi

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2.3 Strengths Weaknesses Selected
Achievements

• New products
• 1.Paramomycin (V.leishmaniasis in India)
• 2.ASAQ (malaria)
• 3.ASMQ (malaria)
• 4.Pediatric AR-LU (Coartem for malaria)

• Phase III clinical trials/equiv
• Moxifloxacin (TB)
• PyronaridineAR (malaria)
• Dihydroartemisinin (malaria)
• Paramomycin (V.leishmaniasis in Africa)
• FastPlaque diagnostic (TB)
• LAM-based diagnostic (TB)
• Synthetic artemisinin (malaria)

Based on study of only 8 PDPs list is
non-exhaustive.
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4.3 Access provisionsIntellectual Property
Management

• Ideally PDPs get access to desired compounds
  technologies and maximize control over IP.
• Practically, will depend on negotiating leverage
  
• Size of firm,
• Stage of development,
• Disease area,
• PDP
• Who paid?
• How to strengthen leverage
• Priority review voucher?
• Mandatory donor access provisions?
• Reliable, plentiful funding?

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Source Giorgio Roscigno, Revolutionizing
disease control with innovation in new
diagnostics. June 2006. Available at
http//www.finddiagnostics.org/about/innov_new_dia
g_for_disease_control_june2006.pdf
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4.4 Access provisionsIP Management (contd)

• Generic Competition Affordability Factors to
  consider
• Non-patent barriers to entry eg technology
  transfer
• Economies of scale competitors cost audits
• License to multiple producers when significant
  benefits expected
• Follow-on innovation
• No norm (yet) of open access
• Information sharing across PDPs
• Giant sucking soundUnequal contributions by
  PDPs private actors?

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Overview

• 1. Introduction History, Organizational Form,
  and Funding
• 2. Strengths and Weaknesses
• 3. Targeted Diseases  
• 4. Access Provisions Policies IP Management
• 5. PDPs in the broader RD Ecosystem
• 6. Governance Issues
• 7. Conclusions

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5.1 PDPs in the RD Ecosystem

• Delicate balance charitable vs profitable
  enterprise
• Effects of new market-based incentives? (AMC,
  PRV)
• Firms may seek market-value remuneration if
  available
• Small firms may enter disease areas and reduce
  PDPs ability to build portfolios and synergies
  across firms/research groups
• Priorities of PDPs may shift toward products that
  can generate revenue to keep an organization
  running
• PDPs may have stronger leverage with PRV
• PDPs may be reluctant to share information with
  each other if competing for the same valuable
  reward (e.g. PRV).

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5.2 RD Ecosystem Unintended Consequences of
Market-based Incentives

• We note that commercialising low-value neglected
  disease markets, for example, through the use of
  advance purchase commitments or roaming patent
  extensions, is likely to increase industry
  activity (particularly by small companies), but
  at the cost of curtailing these positive
  behaviours and returning RD to the more
  secretive and non-collaborative approaches that
  are characteristic of commercial RD. (Moran et
  al., 2005)
• Prizes, AMC, PRV could make a big difference,
  but
• Careful attention to system-wide effects of new
  incentives

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Overview

• 1. Introduction History, Organizational Form,
  and Funding
• 2. Strengths and Weaknesses
• 3. Targeted Diseases  
• 4. Access Provisions Policies IP Management
• 5. PDPs in the broader RD Ecosystem
• 6. Governance Issues
• 7. Conclusions

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6.1 Governance Transparency

• Transparent re
• Composition of Board, Scientific Advisory,
  Stakeholders, Staff,
• Limited transparency re
• Funding sources
• Budgets Spending
• Very little transparency re
• Decision-making procedures
• Agreements between PDPs and industry
• Cost of RD
• How much transparency should we expect or demand
  from a hybrid public-private entity? From a
  non-profit organization?

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6.2 GovernanceEndemic country involvement

• Clinical trials
• Research
• Production
• Governance
• BRICS vs LDCs?

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Source Ziemba, 2005.
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6.3 Governance Sustainability

• Scientific need to invest in capacity of more
  countries to contribute
• Financial need innovative financing model
• Political need broader base of support
• How do PDPs fit into a rapidly changing
  institutional landscape?

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6.4 Governance Accountability

• Accountable to mechanism
• Donors future funding
• Partner firms future partners
• Patients reputation
• Public (as taxpayers and potential
  beneficiaries) reputation
• Hybrid structure unclear expectations of
  accountability
• Example FDA Priority Review Vouchers
• New neglected disease product gets tradable
  voucher for accelerated FDA review
• Worth up to 300 million USD
• Who decides how to spend, how to invest, how to
  ensure public interest?

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6.5 GovernancePublic goals and private partners

• Tension between public private objectives
• Impacts governance (secrecy)
• Impacts innovation (secrecy, patent applications,
  competitiveness, delays moxifloxacin)
• Impacts affordability (price discrimination)
• Impacts financial position direct tradeoff
  between control over end-user access and PDP
  investment
• Impacts public trust public money to private
  sector
• Private free-riding on PDP knowledge-generation
• Unintended consequences of private incentives on
  public-private cooperation?

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7. Conclusions

• Advances of PDPs over old pure public or private
  RD
• Increased resources for RD dedicated to diseases
  affecting worlds poorest
• Improved emphasis on access adaptedness
• Increased transparency (compared to private)
• Key questions re PDP model
• Financing how to sustain?
• Governance Who sets the agenda? How is IP
  managed? Transparency? Public accountability?
• Sustainable how to achieve scientific,
  financial, political sustainability of global
  public goods provision?
• How do we manage the tensions inherent in
  harnessing private actors for public ends? What
  expectations should we have?

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Thank you

• Questions to suerie_moon_at_hksphd.harvard.edu