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Partnering with Patients Howard 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  think tank and  catalyst for action  that  works across sectors and diseases  to improve the effectiveness and efficiency of the medical research system
Nonprofits as “De-Riskers” ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]
TRAIN Central Station www.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. ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]
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. Trial Activation Enrollment  361 247 74 120
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:  CoMentis Leading Anti-Amyloid Therapy
Biotechnology Founders Program:  CoMentis Leading Anti-Amyloid Therapy
Biotechnology Founders Program:  Allon Therapeutics Leading Anti-tangle Therapy
ADDF Biotechnology Founders Program:  Allon Therapeutics, Leading Anti-tangle Therapy  Listed on the Toronto Exchange (NPC.TSX) 2005, current market capitalization around $50M  Received return of note at time of IPO
[object Object],[object Object],[object Object],[object Object],ADDF Venture Philanthropy Fund Investments
ADDF Venture Philanthropy Fund Investments
 
Juvenile Diabetes Research Foundation ,[object Object],[object Object],[object Object]
Beta Cell Therapies  Immune  Therapies  JDRF’s Industry Partners DIABECELL® Encapsulated Cell Replacement Therapy
JDRF’s Industry Partners Glucose Control  Complications Therapies
JDRF’s Clinical Trial Awareness Activities: JDRF Type 1 Diabetes Clinical Trials Connection (www.trials.jdrf.org)
[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],JDRF’s BioBank: nPOD
JDRF’s FDA Activities ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]
JDRF’s Reimbursement Activities ,[object Object]
Why Does ALS TDI Exist? ,[object Object],[object Object],Drug Discovery Pre Clinical Development Phase 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
R&D Productivity Model Target ID Hit Lead  Optimization Preclinical Validation Phase I Phase II Phase III Submission Launch 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 ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],Preclinical Cost per NME Average:  $18.5M Capitalized:  $824M Cycle time:  5.5 years Clinical Cost per NME Average:  $245M Capitalized:  $954M Cycle time: 8 years P(TS) x WIP x V CT x C R&D Productivity =
ALS TDI Investment in ALS TDI 00846 Drug Discovery Pre Clinical Development Phase 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 Studies Efficacy Studies PD Studies 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 ,[object Object],[object Object]
ALS-TDI Facilitates Clinical Translation De-Risk the Opportunity & Broker the Deal Clinical Development Study Disease  Pre-Clinical Development   Build  Drug  Pre-Clinical Development Clinical Basic Research Drug Development Gap Find a Drug Target
[object Object],[object Object],[object Object],[object Object],Merit Cudkowicz, M.D. Mass General Hospital Harvard Medical School Jonathan Katz, M.D California Pacific  Medical Center (Forbes Norris) Stanley Appel, M.D. Methodist Neuro. Institute Cornell Medical School ALS TDI Patient Forum Preclinical Data Clinical Trials Experimental Therapies Patient Care Carey Goldberg Boston Globe  NY- Times,  LA- Times, AP ALS Forum Build and Empower a Community
Transforming Health through Genetics Promoting an Environment of Openness
Survey of Disease Advocacy Organizations ,[object Object],[object Object],[object Object],[object Object],[object Object]
DAO Demographics Characteristic N (%) Organization Age <5 years 2 (6) 5-10 years 13 (36) 11-20 years 15 (42) >20 years 6 (17) Paid Employees Yes 28 (76) Volunteer Network Yes 29 (81) Annual Budget <$100K 17 (49) $100K-$499K 11 (31) $500K-$999K 6 (17) $1M-$5M 1 (3) >$5M 1 (3)
DAO Involvement Types 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 Types Biorepository 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 Storage Characteristic 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 Organization Data and Sample Management Informed Consent Robust Clinical Information Sample Collection & Tracking Data and Sample Query Data Management Electronic & Paper Records Security & Access Control Patient Enrollment Disease  Sample and Clinical Banking Biological Samples Clinical Information Web based Boot camps Manuals Workbooks Wikis Webinars
Demonstrating Private Access’ Technology to Transform Patient Recruitment
Thank you ,[object Object],[object Object],[object Object]

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BIO 2010 Partnering with Patients

  • 1. Partnering with Patients Howard 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  
  • 2. nonprofit think tank and catalyst for action that works across sectors and diseases to improve the effectiveness and efficiency of the medical research system
  • 3.
  • 4. TRAIN Central Station www.fastercures.org/train
  • 5. 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.
  • 6.
  • 7. With over $140M raised to date, the MMRF serves as an end-to-end solution for drug development.
  • 8. Implementation of business solutions to trial barriers led to MMRC trials opening 35% faster and robust trial enrollment. Trial Activation Enrollment 361 247 74 120
  • 9. 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
  • 10. Accelerating Drug Discovery for Alzheimer’s Disease: The Alzheimer’s Drug Discovery Foundation
  • 11. Accelerating Drug Discovery for Alzheimer’s Disease: ADDF Venture Philanthropy
  • 12. Some Potential Advantages to Early Stage Biotechs of Venture Philanthropy Funding
  • 13. Biotechnology Founders Program: CoMentis Leading Anti-Amyloid Therapy
  • 14. Biotechnology Founders Program: CoMentis Leading Anti-Amyloid Therapy
  • 15. Biotechnology Founders Program: Allon Therapeutics Leading Anti-tangle Therapy
  • 16. ADDF Biotechnology Founders Program: Allon Therapeutics, Leading Anti-tangle Therapy Listed on the Toronto Exchange (NPC.TSX) 2005, current market capitalization around $50M Received return of note at time of IPO
  • 17.
  • 18. ADDF Venture Philanthropy Fund Investments
  • 19.  
  • 20.
  • 21. Beta Cell Therapies Immune Therapies JDRF’s Industry Partners DIABECELL® Encapsulated Cell Replacement Therapy
  • 22. JDRF’s Industry Partners Glucose Control Complications Therapies
  • 23. JDRF’s Clinical Trial Awareness Activities: JDRF Type 1 Diabetes Clinical Trials Connection (www.trials.jdrf.org)
  • 24.
  • 25.
  • 26.
  • 27.
  • 28.
  • 29.
  • 30. ALS-TDI Facilitates Clinical Translation De-Risk the Opportunity & Broker the Deal Clinical Development Study Disease  Pre-Clinical Development   Build Drug  Pre-Clinical Development Clinical Basic Research Drug Development Gap Find a Drug Target
  • 31.
  • 32. Transforming Health through Genetics Promoting an Environment of Openness
  • 33.
  • 34. DAO Demographics Characteristic N (%) Organization Age <5 years 2 (6) 5-10 years 13 (36) 11-20 years 15 (42) >20 years 6 (17) Paid Employees Yes 28 (76) Volunteer Network Yes 29 (81) Annual Budget <$100K 17 (49) $100K-$499K 11 (31) $500K-$999K 6 (17) $1M-$5M 1 (3) >$5M 1 (3)
  • 35. DAO Involvement Types 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)
  • 36. Sample Types Biorepository 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)
  • 37. Sample Use and Storage Characteristic 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)
  • 38. Disease Specific Organization Data and Sample Management Informed Consent Robust Clinical Information Sample Collection & Tracking Data and Sample Query Data Management Electronic & Paper Records Security & Access Control Patient Enrollment Disease Sample and Clinical Banking Biological Samples Clinical Information Web based Boot camps Manuals Workbooks Wikis Webinars
  • 39. Demonstrating Private Access’ Technology to Transform Patient Recruitment
  • 40.

Notas del editor

  1. For those that cited cost, all less than 500K budget, and approximately 75% that cited cost were below 100K.
  2. For the others - 2 is saliva for DNA; 1 is “not sure if we collect cell lines”
  3. Experiments (other) - 1 available to researchers, 1- biopsies used for studies, 1 diagnostics and research, 1 - just beginning to use in research; 1 all of the above and more Users (other) - 1 public health researchers, 1 researcher, 1 all data available online, 1 deidentified data, 1 all requests approved or declined Location (other) - 1 is GA BioBank; 1 is institutions outside of our control; 1 medical center; others really fit into the given categories.