Joseph The Innovation Quotient Prize

The Innovation Quotient (IQ) Prize:
A Milestone-Based Prize for Point-of-Care Diagnostics
Fondation Merieux Conference
September 14, 2011
Don Joseph, JD
Chief Operating Officer
Agenda
Introduction to BIO Ventures for Global Health
Need for Multiplex Diagnostics for Low-Resource Settings
Need for Biotech Engagement in Global Health R&D
A Solution: Milestone-Based Prize to Engage the Private Sector
Next Steps
2
BIO Venture for Global Health
To save lives by accelerating the
development of novel drugs, vaccines, and diagnostics
coming from the biotech industry that address the
unmet medical needs of the developing world
We have a unique perspective.
We look at health problems facing poor countries through a dual lens:
Expertise in Industry
Expertise in Global Health
BVGH and Global Health Incentives
BVGH works to create an enabling environment for the biopharmaceutical industry
to engage in global health R&D
Connecting innovators with critical information and resources for R&D
Assessing and developing R&D incentives
Assessing state-of-the-art science and technology in global health discovery,
research, & product development
4
There is an Urgent Need for New Diagnostics to
Differentiate Between Causes of Fever in Children
Pneumonia is the leading cause of mortality among children under five
Pneumonia kills more
than 2 million children
per year – almost all in
the developing world
Symptoms are often
misdiagnosed and
treated as malaria
A new diagnostic
could enable
appropriate use of
antibiotic and
antimalarial treatments
5
Source: UNICEF/WHO (2006) “Pneumonia: The forgotten killer of children”
There is an Urgent Need for New Diagnostics to
Differentiate Between Causes of Fever in Children
Patients present with symptoms, not diseases
Various illnesses share symptoms, making them hard to distinguish
o Diseases covered by IMCI and ICCM guidelines, such as malaria, pneumonia,
and diarrhea present with overlapping symptoms
o RDTs for malaria are increasingly available, however negative malaria tests do
not provide guidance for alternative diagnosis of the patient
Co-infection is common across febrile illnesses
o Malaria tests do not rule out co-infection with other infectious agents, such as
bacterial or viral pneumonia
o The rate of viral and bacterial co-infection in children with pneumonia is
estimated to be 45%1
Differential diagnosis of fever in children at the point-of-care is a
serious unmet need
1
Ruuskanen O, et al. (2011) The Lancet 377: 1264-75.
Recent Studies Highlight Limitations of Current
Malaria Rapid Diagnostic Tests
When a community believes that RDTs do not work, patients fail to adhere to
RDT test results, often demanding treatment regardless of a negative result.
Source:
Target Product Profile: POC Dx
Example: TPP for POC Fever Diagnostic
The TPP will:
Ensure the product is accurate,
inexpensive, portable, easy to use,
and durable
Focus industry effort toward a
product with developing-world utility
Spec
Goal of Test
Sensitivity
Be judged by an independent
panels of technical experts
Specificity
Note: Draft TPP is informed by over 100 leading
global health and industry stakeholder interviews,
and has been vetted by two global health NGOs
and an Industry Working Group
8
Specimen /
Sample
Optimal
Minimal
Differential diagnosis of the cause of fever for treatment, including in
children <5
P. falciparum – 90%
P. falciparum – 95%
P. vivax – 90%
P. vivax – 95%
Streptococcus pneumoniae
Streptococcus pneumoniae – 95%
– 90%
Staphylococcus aureus – 95%
Staphylococcus aureus –
Haemophilus influenzae B – 95%
90%
Supplemental pan-bacterial
Haemophilus influenzae B –
marker(s) that, in combination with
90%
pathogen-specific markers above,
Supplemental pan-bacterial
can identify all bacterial pneumonia
marker(s) that, in
with an overall sensitivity of 95%.
combination with pathogenSupplemental pan-viral marker(s)
specific markers above, can
– Best in class
identify all bacterial
Tuberculosis – Best in class
pneumonia with an overall
HIV – Best in class
sensitivity of 90%.
Each individual pathogen above,
Each individual pathogen
85%
above, 80%
One of the following sample types:
blood, saliva, sputum, mouth swab,
or urine
One or more of the following
sample types collected in a
single patient visit: blood,
saliva, sputum, mouth swab,
urine
Time to result
<10 minutes
<30 minutes
Test/Platform
size
Handheld device;
<5 lbs / 100 tests
Portable device;
<10 lbs / 100 tests
$2-5
$2-5
Target ExWorks Price
BVGH Conducted Over 100 Interviews to Inform
Our Work on a the IQ Prize
Academics and
Clinicians
Brian Wright, UC Berkley
Dave Brown, Consultant
Doreen Ramogola-Masire, UPenn/Bostwana
Elizabeth Molyneux, World Child Cancer
George Rutherford, UCSF
Gerald Kost, UC Davis
Groesbeck Parham, Clinician in Zambia
Kara Palamountain, Northwestern
Julia Gage, National Cancer Institute
Lynette Denny, University of Capetown
Madhukar Pai, McGill University
Malcolm Molyneux, LSTMH
Mark Schiffman, National Cancer Institute
Paul Yager, PhD, University of Washington
Paul Wilson, Columbia University
Philip Castle, National Cancer Institute
Rebecca Richards-Kortum, Rice University
Rosanna Peeling, LSHTM
Serigne Diene, FANTA , AED Center for
Nutrition
Talha Syed, UC Berkley
Temina Madon, UC Berkley
*Names in bold participated in
the Industry Working Group
meeting
9
Industry Executives
and VCs*
Alex Rubido, Consultant
Anthony Lakavage, Becton Dickinson
Bala Manian, ReaMetrix
Benjamin Whitesides, Diagnostics for All
Blessed Okole, TIA, South Africa
Candice Pillay, TIA, South Africa
Chris Collwell, Becton Dickinson
Daryl Pritchard, BIO
David Mack, Alta Partners
David Friedman, Ativa Medical
David Steinmiller, Claros Diagnostics
Doug Dolginow, Ignite Institute
Elizabeth Bailey, Commons Capital
Geoff McKinley, Consultant
Jack Wilkens, GeneEx
James Geraghty, Genzyme
Jason Kim, Quidel
Jean-Francois de Lavison, Ahimsa
John Clarkson, Atlas Genetics
John Hurvitz, Covington & Burling
John McDonough, T2 Biosystems
Karen Hedine, Micronics
Knut Seifert, Roche (South Africa)
Krista Thompson, Becton Dickinson
Leighton Read, Alloy Ventures
Nair Chandrasekhar, BigTec Labs, India
Natarajan Sriram, Tulip Group
Neil Butler, formerly Vivacta
Patrick Beattie, Diagnostics for All
Peter Chun, EASE-Medtrend
Peter Daily, Cepheid
Robert Schueren, Agilent Technologies
Robert Wallis, Pfizer
Sarah Smiley, Advamed
Sharma Bhat, Bhat Biotech, India
Susan Bromley, Novartis
Teva Rothwell, Consultant
Thomas Lowry, T2 Biosystems
Una Ryan, Diagnostics for All
Wendy Woods, BCG
William Rodriguez, Daktari
Non- Profit and
Government Reps
Alan Magill, Walter Reed
Bernhard Weigl, PATH
David Wholly, Foundation for NIH
Hannah Kettler, Gates Foundation
Hellen Gellband, RFF/GARP
Jane Rowley
Kevin Kain, MRC Global
Mark Perkins, FIND
Mary Moran, Policy Cures
Maurine Murtagh, Murtaugh Group
Patrick Lammie, CDC
Peter Singer, MRC Global
Rachel Nugent, CGD
Rich Thayer, Catalysis Foundation
Robert Hecht, R4D
Ruth Levine, USAID
Tala de los Santos, PATH
Ted Roumel, formerly NIH/Phrma
Tido von Schoen-Angerer, MSF
Travis Carley, CCS
Trevor Peter, Clinton Foundation
Francis Moussy, WHO/TDR
Francis Collins, NIH
Anthony Fauci, NIAID
Hugh Auchincloss, NIAID
Carole Hudgins, NIAID
Carole Heilman, NIH
John McGowan, NIAID
Lee Hall, NIAID
Gray Handley, NIAID
Maria Giovanni, NIH
Daniel Singer, NICHD
Tonse Raju, NICHD
Sybil Philip, NICHD
James Hanson, NICHD
Rajiv Shah, USAID
Amie Batson, USAID
Wendy Taylor, USAID
Neal Brandes, USAID
Patricia Stephenson, USAID
Elizabeth Higgs, GHI
Kamiar Khajavi, GHI
Timothy Ziemer, PMI
Larry Barat, PMI
Alan Rudolf, DTRA
Jason Paragas, DTRA
Daniel Wattendorf, DARPA
Nils Daulaire, OGHA
Jonathan Seals, BARDA
Richard Hatchett, BARDA
Impact of a Point-of-Care Fever Diagnostic
Point-of-Care
Fever
Diagnostic
35M fewer
inappropriate
prescriptions per year
in India alone
Up to 20% reduction in
pneumonia deaths of
children under 5
350,000 children under 5
saved annually; costeffective per World Bank
benchmark
10-20% reduction in
inappropriate antibiotic
prescriptions in the
developing world
Biotech Companies are a Major Source of Innovation
Source of New Drugs Approved by US
FDA, 1998-2007
Over a recent 10 year period,
biotechs have been involved in
the development of:
134
9%
11%
54
100%
23%
36%
* 9%
o ~60% of all orphan drugs
approved by the US FDA
o ~50% of all novel drugs
approved by the US FDA
118
25%
22%
* 8%
* 71%
*12%
*44%
*29%
Followon Drugs
Novel
Drugs
Orphan
Drugs
University (tech transfer to biotech)
Biotech
University (tech transfer to pharma)
11
Pharma
Source: Kneller, Robert, “The importance of new companies for drug discovery: origins of a decade of new drugs.” Nature Review. Volume 9. November 2010.
Many Dx Companies Have Detection Platforms That
Could Apply to the Developing World
Adaptations of Existing
Detection Platforms
New Detection Technologies
GeneXpert
Platform:
RT-PCR (Real time PCR, not
limited to Reverse Transcriptase
PCR)
Differentiating Features:
Ease of use in a fully-integrated
cartridge; one cartridge can be used
across a broad range of platforms
Scalable, modular instrument
design
Innovation:
Fully automated and integrated
nucleic acid extraction,
amplification, and detection
Developing World Application:
Labs
Microscopy centers
NA
Small Sample
Volume
No or Auto Sample
Preparation
*
Non-Optical
Detection
High Sensitivity
?
Rapid Result
Simple to Use
Low Cost
?
?
?
?
?
?
?
?
?
?
?
?
?
?
?
Minimal Power
Required
No Water Required
Reagents Stable at
Room Temp
Note: Ionian Technologies device does not require sample preparation for most sample types, with the exception of whole blood, for which preparation is required
?
How the Prize Works: POC Dx
13
Our Milestone-Based Prize Incorporates These Benefits
Pay-for-success: Fixed number of awards available at each milestone following
successful developments
Milestone-based: Timely return on investment for developers at key inflection
points in the development process
Competitive: Open to multiple diagnostic firms who compete in a "race to the
finish" at each milestone
Objective: A panel of independent experts will determine whether TPP criteria
have been met at each phase
Demand-driven: Innovative design informed by over 100 industry experts and
global health professionals
14
Other Aspects
Proposed structure also addresses:
•Intellectual property
•Supply and manufacturing
•Hosting/administration
•Possibility of purchase contract(s) with
relevant purchaser(s)
•And other features of the Prize program
More information can be found at:
http://www.bvgh.org/What-We-Do/Incentives/IQ-Prize.aspx
15
Next Steps
We are engaged in discussions toward potential funding of the POC
Dx program, with positive reactions
WHO’s Consultative Expert Working Group (CEWG) has identified
the IQ Prize as meeting most of its criteria for innovative financing
We are advancing another application of the IQ Prize, in the area of
Chagas disease therapeutics
We look forward to your comments and feedback--thank you for your
time and attention!
16
Back up
17
There is an Urgent Need for New Diagnostics to
Differentiate Between Causes of Fever in Children
Children under 5 account for 85% of malaria deaths
Malaria affects 250
million people per year
and results in more
than 800,000 deaths
Approximately 89% of
all malaria deaths
occur in Africa,
primarily in children
under five years old
A new diagnostic
could enable
appropriate use of
antibiotic and
antimalarial treatments
Source: WHO (2009) World Malaria Report 2009
18 UNICEF/WHO
(2006) “Pneumonia: The forgotten killer of children”
Recent Studies Highlight Limitations of Current
Malaria Rapid Diagnostic Tests
Country
Zambia
Tanzania
Tanzania
Kenya
RDT Study Result
58.4% of patients with a negative microscopy
result and 35.5% of patients with a negative
RDT result received an antimalarial medicine
51% of patients with a negative microscopy
result and 54% with a negative RDT result
received an antimalarial medicine
7% of patients with a negative RDT result
received an antimalarial, however 78% of
patients with a negative RDT received an
antibiotic
60% of RDT negative patients purchased an
ACT
Hamer DH et al. (2007) JAMA 297: 2227–2231.
Reyburn H et al. (2007) British Medical Journal 334: 403.
D’Acremont V et al. (2011) Malaria Journal 10: 107.
Cohen J, unpublished data presented in: WHO (2010) Global Plan for Artemisinin Resistance Containment
IQ Prize Advantages
Donors
•Pay for success
•Novelty
•USG: Responsive to
Congress and
Administration
•Companies have “skin in
the game” – Shared risk
•Don’t have to choose
winning technologies in
advance
Companies
•Operates more like
commercial models
•Open competition
•Clear rules
•No moving target on
target product profile
•Allows financial
calculation for BOD
•Provides for risk sharing
Funding Requirements Over Time
17.5
Milestone 1: $58.5M
Milestone 2: $23.4M
16.9
Milestone 3: $65.7M
Milestone 4: $0.6M
16.9
Administration: $4.5M
37.6
16.9
8.3
7.8
8.3
7.8
16.4
152.7
16.4
0.5
58.5
16.4
16.4
23.4
29.3
29.8
65.7
7.8
29.3
4.5
Year 1
Year 2
Year 3
Year 4
Year 5
Year 6
Year 7
Year 8
Year 9
Note: funding requirements over time drawn from output summary in the financial model
Total