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© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 11
Alicia D. Greenidge, Director General, IFPMAAlicia D. Greenidge, Director General, IFPMA
WHO Expert Working Group, Geneva, 12 Jan 2009WHO Expert Working Group, Geneva, 12 Jan 2009
IFPMA Presentation to WHO Expert Working Group on IFPMA Presentation to WHO Expert Working Group on Funding R&D for Diseases of the Developing World (DDW)Funding R&D for Diseases of the Developing World (DDW)
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 22
R&D IndustryR&D Industry
Generic IndustryGeneric Industry
ManufactureManufacture
R&D Industry
Product DevelopmentPartnerships (PDPs)
Research & Development
GovernmentsGovernments
FoundationsFoundations
AccessAccess(incl. Delivery)(incl. Delivery)
Tackling DDWs: a Combined Effort…Tackling DDWs: a Combined Effort…
AcademiaAcademia
Government AgenciesGovernment Agencies
R&D IndustryR&D Industry
Basic ResearchBasic Research
… … to benefit patients in developing countriesto benefit patients in developing countries
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 33
Industry is Committing Resources to DDW R&DIndustry is Committing Resources to DDW R&D
through multiple mechanisms…through multiple mechanisms…
Industry Dedicated DDW Industry Dedicated DDW R&D CentersR&D Centers
Independent EffortsIndependent Efforts PartnershipsPartnerships
(Current industry involvement & partnerships - illustrative only - not comprehensive)(Current industry involvement & partnerships - illustrative only - not comprehensive)
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 44
R&D for a New Medicine: 10+ years, $1 bn+R&D for a New Medicine: 10+ years, $1 bn+
Sources: Drug Discovery and Development: Understanding the R&D Process, www.innovation.org; CBO, Research and Development in the Pharmaceutical Industry, 2006(1) incl. cost of capital – from The price of innovation: new estimates of drug development costs, DiMasi et al, Tufts Center for the Study of Drug Development, 2002
IndefiniteIndefinite
DiscoveryDiscovery PreclinicalPreclinical Clinical TrialsClinical TrialsRegulatoryRegulatory
ReviewReviewScale-Up to Scale-Up to ManufactureManufacture
Post-MarketingPost-MarketingSurveillanceSurveillance
1Approved
NewMedicine
0.5 – 2 Years0.5 – 2 Years6 – 7 Years6 – 7 Years3 – 6 Years3 – 6 Years
Number Of Patients / Subjects
PhasePhaseII
PhasePhaseIIII
PhasePhaseIIIIII
55250250~ 5,000 – 10,000~ 5,000 – 10,000
CompoundsCompounds
Pre
-Dis
cove
ryP
re-D
isco
very
20 – 100 100 – 500 1,000 – 5,000
IND
Sub
mitt
ed
ND
A S
ubm
itted
… … a big challenge for addressing both developed and developing world diseasesa big challenge for addressing both developed and developing world diseases
$335 million $335 million (1)(1) $467 million $467 million (1)(1)
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 55
Industry R&D for DDW is GrowingIndustry R&D for DDW is Growing
32
43
6
50
8
58
9
0
10
20
30
40
50
60
2005 2006 2007 2008
32
43
6
50
8
58
9
0
10
20
30
40
50
60
2005 2006 2007 2008
Num
ber
of A
ctiv
e R
&D
Pro
ject
sN
umbe
r of
Act
ive
R&
D P
roje
cts
Sources: 2005 - Moran et Al “New Landscape…” 2006, 2007, 2008 – IFPMA Status ReportsSources: 2005 - Moran et Al “New Landscape…” 2006, 2007, 2008 – IFPMA Status Reports
# R&D Projects by IFPMA Companies, with Product Development Partnerships or alone# R&D Projects by IFPMA Companies, with Product Development Partnerships or alone
1 project = 1 compound in development 1 project = 1 compound in development OR 1 screening program for 1 diseaseOR 1 screening program for 1 disease
TDR 10 Priority DiseasesTDR 10 Priority Diseases
- Chagas- Chagas
- Dengue- Dengue
- Human African Trypanosomiasis- Human African Trypanosomiasis
- Leishmaniasis- Leishmaniasis
- Leprosy- Leprosy
- Lymphatic Filariasis- Lymphatic Filariasis
- Malaria- Malaria
- Onchocerciasis- Onchocerciasis
- Schistosomiasis- Schistosomiasis
- Tuberculosis- Tuberculosis
Medicine R&D projectsMedicine R&D projects
Vaccine R&D projectsVaccine R&D projects
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 66
16 16
19 30
22 36
18 49
2005
2006
2007
2008
16 16
19 30
22 36
18 49
2005
2006
2007
2008
Industry DDW R&D is Increasingly CollaborativeIndustry DDW R&D is Increasingly Collaborative
Sources: 2005 - Moran et Al “New Landscape…” 2006, 2007, 2008 – IFPMA Status ReportsSources: 2005 - Moran et Al “New Landscape…” 2006, 2007, 2008 – IFPMA Status Reports
Companies working aloneCompanies working alone
Companies working with PDPsCompanies working with PDPs
# Medicine & Vaccine R&D Projects by IFPMA Companies, with PDPs or alone# Medicine & Vaccine R&D Projects by IFPMA Companies, with PDPs or alone
3232
4949
5858
6767
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 77
Current Industry DDW R&D is Mostly Early StageCurrent Industry DDW R&D is Mostly Early Stage
PreclinicalPreclinical 39 projects39 projects
ClinicalClinical 23 projects23 projects
RegistrationRegistration 5 projects5 projects
TOTAL (2008)TOTAL (2008) 67 projects67 projects
Non-industry R&D for DDW is also predominantly preclinical (or earlier) Non-industry R&D for DDW is also predominantly preclinical (or earlier)
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 88
Factors for Increased DDW R&DFactors for Increased DDW R&D
Large Scale Donor Funding for AccessLarge Scale Donor Funding for Access
Product Development PartnershipsProduct Development Partnerships
Increased Not-for-Profit R&D WorkIncreased Not-for-Profit R&D Work
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 99
Central Role of PDPs in DDW R&DCentral Role of PDPs in DDW R&D
Independent PDP-driven R&DIndependent PDP-driven R&D
Bringing partners together over the last decade…Bringing partners together over the last decade…
World Bank
Rockefeller
Bill & Melinda Gates
Wellcome Trust
USAID
DFID
PEPFAR
PMI
Grand Challenges
CIDA
Global Fund
GAVI
Doris Duke
Clinton Foundation
Carter Center
Basic Research OrganizationsBasic Research Organizations
TB medicines2000
TB treatments1998
Hookworm vaccines2000
Rotavirus vaccines2003
Pneumonia vaccines2003
Malaria, rotavirus, JE, pneumococcal & meningitis vaccines
1977
Malaria vaccines1999
Malaria medicines2000
Dengue vaccines2003
VL, malaria, diarrhoea& STH medicines
2000
HAT, VL, Chagas & malaria medicines
2003
TB vaccines2003
FocusFocusSinceSincePDPPDP
FundersFunders Product Development PartnershipsProduct Development Partnerships R&D ExecutionR&D Execution
(Funders, PDPs, Basic Research Organizations & Industry Players - illustrative only - not comprehensive)(Funders, PDPs, Basic Research Organizations & Industry Players - illustrative only - not comprehensive)
Industry playersIndustry players
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 1010
0
0
0
1
2
2
3
3
24
32
0 5 10 15 20 25 30 35
Schistosomiasis
Lymph Filariasis
Leprosy
Onchocerciasis
Chagas
Leishmaniasis
Dengue
HAT
Tuberculosis
Malaria
0
0
0
1
2
2
3
3
24
32
0 5 10 15 20 25 30 35
Schistosomiasis
Lymph Filariasis
Leprosy
Onchocerciasis
Chagas
Leishmaniasis
Dengue
HAT
Tuberculosis
Malaria
Some DDWs are More Neglected than OthersSome DDWs are More Neglected than Others
(HAT = Human African Trypanosomiasis or “Sleeping Sickness”)(HAT = Human African Trypanosomiasis or “Sleeping Sickness”)
Some “More Neglected Diseases” need R&D Some “More Neglected Diseases” need R&D more urgently than others and should be prioritized more urgently than others and should be prioritized
4 Approved Meds: ALL intravenous or intramuscular, 3 are v. old4 Approved Meds: ALL intravenous or intramuscular, 3 are v. old
0 Approved Meds0 Approved Meds
2 Approved Meds: 1) intravenous & side effects 2) v. expensive2 Approved Meds: 1) intravenous & side effects 2) v. expensive
2 Approved Meds: BOTH less effective against fatal late stage2 Approved Meds: BOTH less effective against fatal late stage
1 Approved Med: Effective, large donation program. Resistance?1 Approved Med: Effective, large donation program. Resistance?
3 Approved Meds: Effective, large donation program3 Approved Meds: Effective, large donation program
3 Approved Meds: Effective, large donation program3 Approved Meds: Effective, large donation program
1 Approved Med: Effective, large donation program1 Approved Med: Effective, large donation program
Source: 2008 IFPMA Status ReportSource: 2008 IFPMA Status Report
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 1111
Current DDW R&D Funding will be InsufficientCurrent DDW R&D Funding will be Insufficient as Pipelines Advance as Pipelines Advance
Dalberg Study for IFPMA (2007)Dalberg Study for IFPMA (2007) Total Funding for PDPs to 2007 = USD 0.5 billionTotal Funding for PDPs to 2007 = USD 0.5 billion
Est. funding to take current PDP pipelines to approval AND to augment Est. funding to take current PDP pipelines to approval AND to augment inadequate pipelines for Most Neglected Diseases = USD 8.3 billioninadequate pipelines for Most Neglected Diseases = USD 8.3 billion
Dalberg Study for IFPMA (2007)Dalberg Study for IFPMA (2007) Total Funding for PDPs to 2007 = USD 0.5 billionTotal Funding for PDPs to 2007 = USD 0.5 billion
Est. funding to take current PDP pipelines to approval AND to augment Est. funding to take current PDP pipelines to approval AND to augment inadequate pipelines for Most Neglected Diseases = USD 8.3 billioninadequate pipelines for Most Neglected Diseases = USD 8.3 billion
Estimated funding needs profile for Neglected diseases R&D drugs – 2008-2017USD B
Sources: Global plan to Stop TB 2006-2015; MMV financial plan 2008-2017; DNDi Business plan 2007-2014; Dalberg interviews and analysis.
0.4
2009
0.6
2010
0.9
2011
1.0
2012
1.1
2013
1.2
2014
1.1
2015
1.5
0.3
2008
1.0
2016
0.8
2017
Other diseases
HAT/ VL/ Chagas
Malaria
TB
0.0
1.0
0.5
Mid-range funding need for the next 10 years
USD 8.3 B
Estimated funding needs profile for Neglected diseases R&D drugs – 2008-2017USD B
Sources: Global plan to Stop TB 2006-2015; MMV financial plan 2008-2017; DNDi Business plan 2007-2014; Dalberg interviews and analysis.
0.4
2009
0.6
2010
0.9
2011
1.0
2012
1.1
2013
1.2
2014
1.1
2015
1.5
0.3
2008
1.0
2016
0.8
2017
Other diseases
HAT/ VL/ Chagas
Malaria
TB
0.0
1.0
0.5
Mid-range funding need for the next 10 years
USD 8.3 B
Source: Dalberg for IFPMASource: Dalberg for IFPMA
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 1212
Diseases TreatmentsStages of the R&D value chain
Portfolio management
Intermediaries & recipients
Malaria & TB
TDR list(1) - excluding those w/o PDPs
TDR list - excluding Malaria and TB
TDR list
TDR list + 7 diseases from NTD list(2)
Medicines
Medicines + Diagnostics
Medicines + diagnostics + vaccines
Clinical trials focus – Malaria and TB(3)
Discovery only – smaller diseases(3)
All stages of the value chain – VL, Chagas,
HAT(3)
Full Portfolio management with Fund
Partial Portfolio management with Fund
Pre-selection of intermediaries / recipients
All intermediaries - All recipients
Firm RecommendationFirm Recommendation
Initial recommendationInitial recommendation
OptionsOptions
Sustainable Funding
Push financing – spot commitments
Push financing – long term commitments
Push financing – IFF mechanism
Funding Prioritization
Spreading
Picking winners
Impact based sequence
Success based sequence
Notes: Notes: (1) TDR: WHO Special Programme for Research and Training in Tropical Diseases(1) TDR: WHO Special Programme for Research and Training in Tropical Diseases(2) NTD: WHO Control of Neglected Tropical Disease programme(2) NTD: WHO Control of Neglected Tropical Disease programme(3) Clinical trials only for Malaria and TB, discovery only for other diseases without existing PDPs, all stages of the value (3) Clinical trials only for Malaria and TB, discovery only for other diseases without existing PDPs, all stages of the value
chain for other diseases with existing PDPschain for other diseases with existing PDPs
Study Also Looked at Sustainable Funding StructureStudy Also Looked at Sustainable Funding Structure
Source: Dalberg for IFPMASource: Dalberg for IFPMA
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 1313
DDW R&D Trends that will Impact FinancingDDW R&D Trends that will Impact Financing
Maturing PipelineMaturing Pipeline
• More projects entering expensive later stages (clinical trials)More projects entering expensive later stages (clinical trials)
• Need to invest in trials infrastructure & regulatory frameworksNeed to invest in trials infrastructure & regulatory frameworks
− Regulatory delays can be significant (e.g. ~4 years in S. Africa)Regulatory delays can be significant (e.g. ~4 years in S. Africa)
Some Avenues of Research Prove FruitlessSome Avenues of Research Prove Fruitless
• From Development, return to Basic Research (e.g. Dengue)From Development, return to Basic Research (e.g. Dengue)
• Implications for total funding and for overall timeframeImplications for total funding and for overall timeframe
New Funding Sources / IncentivesNew Funding Sources / Incentives
• Advanced Market Commitment (AMC)Advanced Market Commitment (AMC)
• Priority Review Voucher (PRV)Priority Review Voucher (PRV)
− Recognition of Importance of DDW R&DRecognition of Importance of DDW R&D
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 1414
Enhanced Industry Contribution: Potential Next Steps Enhanced Industry Contribution: Potential Next Steps
Consult with WHO & other partners to prioritize DDW R&D needsConsult with WHO & other partners to prioritize DDW R&D needs
Explore working with African Network for Drugs Discovery & Explore working with African Network for Drugs Discovery &
Innovation (ANDI)Innovation (ANDI)
IFPMA working on a Developing Country Young Researcher AwardIFPMA working on a Developing Country Young Researcher Award
IFPMA exploring ways to facilitate IFPMA exploring ways to facilitate increased availability of industry increased availability of industry
resourcesresources
IFPMA Members looking at additional activities in partnership with IFPMA Members looking at additional activities in partnership with
DNDi DNDi et alet al
Consulting on other possible collaborationsConsulting on other possible collaborations
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 1515
ConclusionsConclusions
DDW R&D has increased substantiallyDDW R&D has increased substantially
• Increased activity by Product Development PartnershipsIncreased activity by Product Development Partnerships
• Increased in-kind & not-for-profit contribution by industryIncreased in-kind & not-for-profit contribution by industry
Current DDW R&D is mainly in less expensive earlier stagesCurrent DDW R&D is mainly in less expensive earlier stages
• Funding requirements will increase as more projects move into clinical trialsFunding requirements will increase as more projects move into clinical trials
Industry is working to help expand and improve current DDW effortIndustry is working to help expand and improve current DDW effort
Current volume of DDW R&D inadequate to deliver enough new Current volume of DDW R&D inadequate to deliver enough new medicines for all the most needy diseasesmedicines for all the most needy diseases
• Shortfall is significant – funding to date perhaps 10 % of what is neededShortfall is significant – funding to date perhaps 10 % of what is needed
Additional funding critical to achieve adequate range of new Additional funding critical to achieve adequate range of new medicines & vaccines for Most Neglected Diseasesmedicines & vaccines for Most Neglected Diseases
DDW R&D has increased substantiallyDDW R&D has increased substantially
• Increased activity by Product Development PartnershipsIncreased activity by Product Development Partnerships
• Increased in-kind & not-for-profit contribution by industryIncreased in-kind & not-for-profit contribution by industry
Current DDW R&D is mainly in less expensive earlier stagesCurrent DDW R&D is mainly in less expensive earlier stages
• Funding requirements will increase as more projects move into clinical trialsFunding requirements will increase as more projects move into clinical trials
Industry is working to help expand and improve current DDW effortIndustry is working to help expand and improve current DDW effort
Current volume of DDW R&D inadequate to deliver enough new Current volume of DDW R&D inadequate to deliver enough new medicines for all the most needy diseasesmedicines for all the most needy diseases
• Shortfall is significant – funding to date perhaps 10 % of what is neededShortfall is significant – funding to date perhaps 10 % of what is needed
Additional funding critical to achieve adequate range of new Additional funding critical to achieve adequate range of new medicines & vaccines for Most Neglected Diseasesmedicines & vaccines for Most Neglected Diseases
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 1616
R&D IndustryR&D Industry
Generic IndustryGeneric Industry
ManufactureManufacture
R&D Industry
Product DevelopmentPartnerships (PDPs)
Research & Development
GovernmentsGovernments
FoundationsFoundations
AccessAccess(incl. Delivery)(incl. Delivery)
Combined Effort Needed to AdvanceCombined Effort Needed to Advance Existing & Future Pipeline for DDWs Existing & Future Pipeline for DDWs
AcademiaAcademia
Government AgenciesGovernment Agencies
R&D IndustryR&D Industry
Basic ResearchBasic Research
… … to benefit patients in developing countriesto benefit patients in developing countries
© IFPMA 2009© IFPMA 2009IFPMA to WHO EWG 12 Jan 09IFPMA to WHO EWG 12 Jan 09 1717
Thank You!Thank You!