© 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!