Partnering with Patients to Increase Engagement and Improve Outcomes
BIO 2010 Partnering with Patients
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Transcript of BIO 2010 Partnering with Patients
Partnering with PatientsHoward Fillit, MD Executive Director , Alzheimer’s Drug Discovery Foundation Richard Insel, MD Executive Vice President, Research , Juvenile Diabetes Research Foundation
James C. O’Leary Chief Innovation Officer , Genetic Alliance Louise Perkins, PhD Chief Scientific Officer, Multiple Myeloma Research Foundation Steven Perrin, PhD Chief Executive Officer and Chief Scientific Officer , ALS Therapy Development Institute Kristin Schneeman - Chair Program Director, FasterCures
nonprofit nonprofit think tank and think tank and catalyst for action catalyst for action that that
works across sectors and diseases works across sectors and diseases to improve the effectiveness and efficiency of to improve the effectiveness and efficiency of
the medical research systemthe medical research system
Nonprofits as “De-Riskers”• Developing pre-clinical tools• Targeting research to support translation• Funding mechanisms that bring in industry• Managing academic science• Access to patient community• New indications for existing drugs• High-throughput screening of promising
compounds• Access to scientific expertise• Advocating with FDA
TRAIN Central Stationwww.fastercures.org/train
An effort designed to facilitate cross-sector collaborations needed to turn a scientific discovery into an accessible therapy.
Be a part of an effort that brings together people with the expertise, experience, and creativity needed to transform the medical research system.
The MMRF and MMRC are sister organizations contributing to all phases of drug development.
• Funds worldwide research • Builds myeloma community• Partners with MMRC
• Supports 13 institutions• Conducts tissue banking• Facilitates Phase I/II trials
With over $140M raised to date, the MMRF serves as an end-to-end solution for drug development.
Implementation of business solutions to trial barriers led to MMRC trials opening 35% faster and robust trial enrollment.
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Jan Feb Mar April May June July Aug Sep Oct Nov Dec
2007 2008 2009 2010
Trial Activation Enrollment
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The MMRF Patient Navigator program aims to drive clinical trial accrual with specific benefits for late-stage trials.
Program Highlights
Up-to-date searchable database
Trained clinical specialists
Late-stage trials prioritized
Site support
Marketing
Targeted communications to stage of disease and geography
MMRF Newsletter articles, ads
Ads in MMRC, cancer publications
Social networking/communities
Accelerating Drug Discovery for Alzheimer’s Disease: The Alzheimer’s Drug Discovery Foundation
Accelerating Drug Discovery for Alzheimer’s Disease: ADDF Venture Philanthropy
Some Potential Advantages to Early Stage Biotechs of Venture Philanthropy Funding
Biotechnology Founders Program: CoMentisLeading Anti-Amyloid Therapy
Biotechnology Founders Program: CoMentisLeading Anti-Amyloid Therapy
Biotechnology Founders Program: Allon TherapeuticsLeading Anti-tangle Therapy
Listed on the Toronto Exchange (NPC.TSX) 2005, current market capitalization around $50M Listed on the Toronto Exchange (NPC.TSX) 2005, current market capitalization around $50M
Received return of note at time of IPOReceived return of note at time of IPO
ADDF Biotechnology Founders Program: Allon Therapeutics, Leading Anti-tangle Therapy
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– Start-up biotech company focused on development of small molecule mimetics of neurotrophins (NGF, BDNF) to treat AD, PD, and other neurodegenerative diseases
• “holy grail” of neuroprotection
– Founder is Professor Frank Longo, Chairman, Neurology and Neurosciences at Stanford
– PTX chose the ADDF Venture Philanthropy Fund as preferred founding investor
ADDF Venture Philanthropy Fund Investments
ADDF Venture Philanthropy Fund Investments
Juvenile Diabetes Research Foundation• Mission: To find a cure for type 1 diabetes and
its complications• Strategy: Proactively accelerate the discovery,
development, and delivery of drugs and devices to cure, better treat, or prevent type 1 diabetes and its complications
• Core Principles: People with T1DM, Pipeline, Priorities, Partners
Beta
Cel
lTh
erap
ies
Imm
une
Ther
apie
s
DIABECELL® Encapsulated Cell Replacement Therapy
JDRF’s Industry Partners
Glu
cose
Con
trol
Co
mpl
icati
ons
Ther
apie
s
JDRF’s Industry Partners
JDRF’s Clinical Trial Awareness Activities:JDRF Type 1 Diabetes Clinical Trials Connection (www.trials.jdrf.org)
Biobank of pancreata and related tissues from cadaveric organ donors with:
• Recent onset type 1 diabetes• Established diabetes including long-standing
(>50yrs)• At-risk, islet autoantibody positive
Tissues being used by > 50 groups to address key immunological, histological, viral, and metabolic questions to better understand type 1 diabetes
www.jdrfnpod.org
JDRF’s BioBank: nPOD
JDRF’s FDA Activities• FDA-NIH-JDRF Workshops:
– How Good Does a Beta Cell Have to Be: November 2009– Towards An Artificial Pancreas: July 2008– Obstacles and Opportunities on the Road to Artificial Pancreas:
Closing the Loop: December 2005• Artificial Pancreas placed on Critical Path list in 2006;
Artificial Pancreas Review team received FDA “Leveraging/Collaboration Award” in 2007
• FDA acceptance of in silico model to test algorithms – Algorithms can be extensively tested on in silico ‘patients’ prior
to clinical study and allows elimination of animal testing
JDRF’s Reimbursement Activities JDRF funded a multicenter phase IV clinical trial of
effectiveness of Continuous Glucose Monitors in Type 1 Diabetes → Change in reimbursement policy (United Healthcare, Aetna, Cigna, Wellpoint, Kaiser Permanente) and clinician and patient adoption
Why Does ALS TDI Exist?
• Nature Drug Discovery, February 2010• Nature Drug Discovery, March 2010
Drug DiscoveryPre Clinical
DevelopmentPhase I Phase II Phase III
3.1 yrs 2.5 yrs 1.5 yrs 2.5yrs 3.0 yrs
Prototypical Pharmaceutical Timelines
* Average cost to commercialization: $1.8B
TargetID Hit Lead
OptimizationPreclinicalValidation
Phase I Phase IIPhase III
SubmissionLaunch
80%
24.3
$1
1.0
3%
$94
75%
19.4
$2.5
1.5
6%
$166
85%
14.6
$10
2.0
17%
$414
69%
12.4
$5
1.0
7%
$150
54%
8.6
$15
1.5
15%
$273
34%
4.6
$40
2.5
21%
$319
70%
1.6
$150
2.5
27%
$314
91%
1.1
$40
1.5
5%
$48
P(TS)
WIP per Launch
Cost per WIP per Phase
Cycle Time (Years)
Cost per Launch
% Total Cost per NME
• P(TS) : probability of technical success• WIP : work in progress• V : value• CT : cycle time• C : cost
R&D Productivity Model
• Nature Drug Discovery March 2010
Preclinical Cost per NMEAverage: $18.5MCapitalized: $824MCycle time: 5.5 years
Clinical Cost per NMEAverage: $245MCapitalized: $954MCycle time: 8 years
P(TS) x WIP x VCT x C
R&D Productivity =
ALS TDI Investment in ALS TDI 00846
Drug DiscoveryPre Clinical
DevelopmentPhase I Phase II Phase III
3.1 yrs($10M)
1.4 yrs($20M)
1.7 yrs($10M)
2.7 yrs($30M)
1.9 yrs($100M)
Discovery Tools
Pk StudiesEfficacy Studies
PDStudies
Tolerability Studies
Disease Models
Clinical Samples
Therapeutic Development
Prototypical Pharmaceutical Cost & Timelines
0.75 yrs($0.6M)
1 yr($0.8M)
1.5 yrs($1.2M)
ALS TDI Cost & Timelines
• ALS TDI 00846 preclinical development: $2.5M• 40% of investment is in therapeutic development, pk, and tolerability
ALS-TDI Facilitates Clinical TranslationDe-Risk the Opportunity & Broker the Deal
Clinical Developme
nt
Study Diseas
e
Pre-Clinical Developme
nt
Build Drug
Pre-Clinical Development
ClinicalBasic Research
Drug Development Gap
Find a Drug Target
• Largest Online Discussion of ALS Therapeutics• 3,000+ Members, 40,000 posts•Topics include Therapeutics & Clinical Trials to Living With ALS and Advocacy• Free, anonymous and open to the public
• Largest Online Discussion of ALS Therapeutics• 3,000+ Members, 40,000 posts•Topics include Therapeutics & Clinical Trials to Living With ALS and Advocacy• Free, anonymous and open to the public
Merit Cudkowicz, M.D.Mass General HospitalHarvard Medical School
Jonathan Katz, M.DCalifornia Pacific Medical Center(Forbes Norris)
Stanley Appel, M.D.Methodist Neuro. InstituteCornell Medical School
ALS TDIPatient Forum
PreclinicalData
ClinicalTrials
ExperimentalTherapies
PatientCare
Carey Goldberg
Boston Globe NY- Times, LA- Times, AP
ALS ForumBuild and Empower a Community
Transforming Health through Genetics
Promoting an Environment of Openness
Survey of Disease Advocacy Organizations• 37 organizations responded to the survey• 13 organizations (35%) were involved with a
biorepository, and 15 organizations (41%) were involved with a registry
• Half of biorepositories and 73% of registries have been in operation for more than 5 years
• Of those organizations without biorepositories or registries, 65% were interested in establishing a biorepository, and 86% were interested in establishing a registry
• Cost was the major reason for not establishing a biorepository or registry
DAO DemographicsCharacteristic N (%)Organization Age
<5 years 2 (6)5-10 years 13 (36)11-20 years 15 (42)>20 years 6 (17)
Paid EmployeesYes 28 (76)
Volunteer NetworkYes 29 (81)
Annual Budget<$100K 17 (49)$100K-$499K 11 (31)$500K-$999K 6 (17)$1M-$5M 1 (3)>$5M 1 (3)
DAO InvolvementTypes of Engagement N (%)
Established Biorepository/registry 12 (67)
Own Biorepository/registry 9 (53)
Provide Financial Support 10 (56)
Help Obtain Financial Support 10 (56)
Recruit Participants 17 (94)
Obtain Informed Consent 11 (61)
Sample TypesBiorepository Collects N (%)
Blood 13 (72)
Tissue (Frozen) 10 (56)
Tissue (Formalin) 9 (50)
Cell Blocks 5 (28)
Cell Lines 7 (39)
Other Biological Material 3 (17)
Desire to Collect other Types 13 (81)
Sample Use and StorageCharacteristic N (%)Experiments DNA/genomics Analysis 9 (64) RNA Expression 1 (7) Protein/immunohistochemistry 2 (14) Other 5 (36)Users Research Consortium 5 (31) Any Qualified Researcher 12 (75) Other 5 (31) Costs Passed on to Researcher 9 (50)Location Nonprofit Organization 3 (21) Commercial Lab 3 (21) University/research Lab 8 (57) Government Lab 1 (7) Other 3 (21)
Disease Specific OrganizationData and Sample Management
Informed ConsentRobust Clinical Information
Sample Collection & TrackingData and Sample Query
Data ManagementElectronic & Paper Records
Security & Access Control
Informed ConsentRobust Clinical Information
Sample Collection & TrackingData and Sample Query
Data ManagementElectronic & Paper Records
Security & Access Control
Patient Enrollment
Disease Sample and Clinical
Banking
Disease Sample and Clinical
Banking
Biological SamplesBiological Samples
Clinical InformationClinical Information
Web basedBoot camps
ManualsWorkbooks
WikisWebinars
Demonstrating Private Access’ Technology to Transform Patient Recruitment
Thank you• James C. O'Leary
Chief Innovation Officer
• Genetic Alliance4301 Connecticut Ave. NWSuite 404Washington, DC 20008-2369
• (202) 966-5557 [email protected]