innovation in the biopharmaceutical pipeline-a multidimensional view
TRANSCRIPT
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“Innovation in the Biopharmaceutical
Pipeline: A Multidimensional View”
John Jeffrey JonesRegis University Class of 2016
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Topics• Continuing Need for New Medicines• R&D Pipeline
• Total Number of Medicines in Development, by Therapeutic Area
• Potential First-in-Class Medicines in Development• Orphan Diseases• Therapies Targeting Diseases with No Recently
Approved Therapies• Personalized Medicines in Development• Novel Scientific Strategies
• Conclusion
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Introductiono U.S. leads the world in development
of new medicines
o Improving the length and QOL and reduced disease burden for individuals and society
o Focus: NME Clinical testing stage in human volunteers
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Challenges of Drug Discovery
• Long/Complex Process – risk of failure at each step
• $1.2 billion cost to yield a single FDA-approved drug (includes cost of development failures)1
• 10-15 year span from R&D + FDA approval process time2
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Annual & Cumulative New Drug Approvals Since 20003
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Distribution of Products & Projects by Phase3
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Distribution of Products and Projects by Therapeutic
Area/Phase3
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Potential First-in-Class Medicines in Development, by Phase3
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Potential First-in-Class Medicines in Development, by
Therapeutic Area3
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Spotlight: DM II & Schizophrenia3
New Approach to Controlling Type II
Diabetes
Difficult-to-Treat Symptoms of
Schizophrenia
O Compound that controls blood sugar levels, or glycemia, independent of insulin pathways.
O Sodium glucose cotransporter-2 inhibitor
O Glycine reuptake inhibitor – could help normalize transmission of glutamate
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Orphan Disease Designation by the FDA Over Time3
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Spotlight: Idiopathic Pulmonary Fibrosis (IPF) 3
O Patients experience progressive difficulty breathing due to scarring of the lungs.
O There are currently no effective treatment options available, and the average patient with IPF dies within 3 years of diagnosis.
O A medicine in development targets connective tissue growth factor, which is elevated in the lungs of IPF patients.
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Projects for Selected Diseases/Conditions with No
Approvals in 10 Years3
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Spotlight on Therapies Targeting Diseases with No Recently Approved Therapies: Recent Progress
Against Lupus3
O Lupus: Complex, potentially fatal autoimmune disease that attacks healthy tissues from MULTIPLE organ systems
O At least 7 drugs in the last several years have suffered setbacks in clinical trials
O As of December 2011, there were 58 other projects in development to treat lupus or various aspects of lupus such as serious kidney complications.
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Personalized Medicine Trials by Condition & Therapeutic Area, 1993-
20083
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Distribution of Personalized Medicine Clinical Trials by
Therapeutic Area & Growth Over Time, 1993 - 20083
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Spotlight: Melanoma, Lung Cancer, & Cystic Fibrosis3
O Vemurafenib (Zelboraf®) – Melanoma
O Crizotinib (Xalkori®) - Lung cancer
O Ivacaftor (Kalydeco®) - Cystic fibrosis
O Each is indicated for patients expressing a specific genetic mutation where the drug is known to be effective.
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Examples of Selected Breakthrough Scientific Strategies3
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Spotlight: RNAi & Gene Therapy3
RNAi Targeted Therapy Approach for Duchenne
Muscular Dystrophy (DMD)
Gene Therapy as a Possible Approach for Parkinson’s Disease
O DMD: most severe genetic disorderO Affects 1 in 3,500
newborn boysO targeted
restoration of the function of dystrophin
O Use of adeno-associated virus (AAV) as a vector to deliver neurturin to restore cells; protection from further degeneration
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SummaryO Diverse clinical research programs
across many different therapeutic areas
O New treatments address both common conditions like cardiovascular disease and rare diseases like cystic fibrosis
O New hope for current and future patients
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ReferencesO 1. J.A. DiMasi and H.G. Grabowski. (2007) The
cost of biopharmaceutical R&D: is biotech different?. Managerial & Decision Economics. 28:469-479.
O 2. DiMasi, J.A., Hansen, R.W., and Grabowski, H.G. (2003) The price of innovation: new estimates of drug development costs. Journal of Health Economics. 22:151-185.
O 3. G. Long and J. Works. (2013) Innovation in the Biopharmaceutical Pipeline: A Multidimensional View. Analysis Group, Inc. 1-29.