1200 100 pm Vice Chancellor for Science Policy and Strategy UCSF Director UCSF Precision Medicine Vice Dean for Research School of Medicine Professor Cellular amp Molecular ID: 734900
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Slide1
Medical Innovation 101
Tuesday, February 14, 2017
12:00- 1:00 pmSlide2
Vice
Chancellor for Science Policy and Strategy, UCSF; Director
, UCSF Precision Medicine;
Vice Dean for Research, School of Medicine; Professor, Cellular & Molecular Pharmacology, University of California San Francisco
Keith R. Yamamoto, Ph.D.Slide3
Role
of academia and basic research in the R&D continuum and precision
medicine
Keith
R.
Yamamoto
Vice Chancellor for Science Policy and Strategy
University of California, San Francisco
yamamoto@ucsf.edu
Medical Innovation 101
February 14,
2017Slide4
The result:
Biology is complicated. Therefore, so is disease.
Mutations in antennae or underlying interacting networks affect risk or course of disease.
Cellular “antennae” receive physiological and environmental
signals
that fine-tune
the
ways our genes work so each of us is unique
Biological process, disease process
(
e.g., increased cell division, blood glucose, etc.
)
And a corollary…
Biology is complicated, for a good reason
! Slide5
Fundamental biological processes are often solved in simple organisms
Simple organisms contain fewer antennae, simpler networks
Simple organisms allow simpler, more definitive experiments
It works. Look at the sources of big breakthroughs:
Breakthrough
Experimental organism
Gene regulation bacteria and their
viruses
Cell division baker’s yeast, clawed toad
Cancer genes chicken virus
Development fruit fly, sea urchin, fish
Aging and lifespan soil worm, pond scum
Learning and memory sea snail
Neuron-target connections chicken
Basic discoveries are gateways to human studies; critical elements of R&D continuum and precision medicineSlide6
A Google Maps-like knowledge network for precision medicine
Defines disease mechanisms
Drives drug development
Informs prevention and diagnosis-therapy decisions for individuals
Knowledge NetworkSlide7
Academic basic research, the R&D continuum
and Precision Medicine:
Impacts
Understand logic and mechanisms of biological processes
Knowledge
network builds iteratively, contributes continuously
Evidence-based prevention, diagnosis, therapy, cures
Prevent and control chronic, infectious, rare diseasesHealth and healthcare democratized, tailored to the individual
Smaller, faster, more successful clinical trialsFocused development/deployment of drugs and clinical testsReduce slope of healthcare cost curveSlide8
Vice President of Corporate Affairs, Celgene
Brian Gill Slide9
Our Mission and Vision
9
Celgene is building a preeminent global biopharmaceutical company focused
on the discovery, development and commercialization of
innovative therapies
for patients with cancer, immune-inflammatory, and other unmet medical needsSlide10
10
What it Takes to Make a MedicineSlide11
What it Takes to Make a MedicineSlide12
It takes $2.6 billion on average to develop a medicine
It takes an average of 12 years for an experimental drug to travel from the laboratory to your medicine cabinet Chances for success are less than 10 percent
Only 5 in 5,000 compounds that enter preclinical testing advance to human testing
Only 1 of those 5 compounds tested in people is approved by the FDAJust 2 in 10 FDA-approved medicines produce revenues that exceed average investment Developing Medicines is a Risky Proposition Slide13
Society Benefits from New Treatments and, Ultimately, Lower Costs
Developing a new medicine takes an average of 10-15 years; the Congressional Budget Office reports that “relatively few drugs survive the clinical trial process.” Innovative therapies have a limited time in their lifecycle to recapture investment and fund future innovation.
1
APPROXIMATELY
5,000
to
10,000COMPOUNDS
5COMPOUNDS
DISCOVERY AND
DEVELOPMENT
FDA
REVIEW
INNOVATOR
EXCLUSIVITY
GENERIC
$2.6 BILLION
3-6 YEARS
Preclinical
0.5-2 YEARS
FDA Review
FDA-Approved Therapy
FOREVER
6-7 YEARS
Clinical Trials
Source 10:
Pharmaceutical Research and Manufacturers of America. Drug Discovery and Development: Understanding the R&D Process. Available at
http://www.phrma.org/sites/default/files/pdf/rd_brochure_022307.pdf
. Accessed August, 2015. Congress of the United States Congressional Budget Office. Research and
Development in the Pharmaceutical Industry. October 2006. Available at
http://www.cbo.gov/sites/default/files/10‐02‐drugr‐d.pdf
. Accessed August, 2015. Tufts Center for the Study of Drug Development. Cost to Develop and Win Marketing Approval for a New Drug is $2.6 Billion. Available at
http://csdd.tufts.edu/news/complete_story/pr_tufts_csdd_2014_cost_study
. Accessed August, 2015.Slide14
Total personal healthcare spending
Source:
National Cancer Institute. Cancer Trends Progress Report – 2007 Update. Table L1: National Cancer Treatment Expenditures in Billions of Dollars (1963-2004). Available at
http://www.progressreport.cancer.gov/sites/default/files/archive/report2007.pdf
. Accessed August, 2015. Cutler, D. Are We Finally Winning the War on Cancer?
J Econ Persp.
2008:22(4);3-26. Mariotto AB, Yabroff KR, Shao Y, et al. Projections of the Cost of Cancer Care in the United States: 2010–2020.
J Natl Cancer Inst. 2011;103(2):117-128. Centers for Medicare and Medicaid Services. National Health Expenditures; Aggregate and Per Capita Amounts, Annual Percent Change and Percent Distribution: Selected Calendar Years 1960-2012. Available at http://www.cms.gov/Research-Statistics-Data-and-Systems/Statistics-Trends-and-Reports/NationalHealthExpendData/Downloads/tables.pdf. Accessed August, 2015. Bastian, A. The Rising Cost of Medical Care: Understanding the Problem and Exploring Solutions. GFK Market Access. Presented at American Society of Hematology Annual Meeting 2015.
1893
1955-66
1971
2015
1917
1988
1947
Cancer treatment spending
Billions
Bringing Medicine Costs into Context
Spending on cancer, relative to total healthcare spending, has remained constant
over the past half century
14Slide15
Executive Director,
Patient-Centered Outcomes Research Institute (PCORI)
Joe V. Selby, MD MPH Slide16
The Role of Comparative Effectiveness Research in Medical Innovation
Joe Selby, MD MPH
Executive Director, PCORI
February 14, 2017Slide17
Role of Comparative Effectiveness Research in Innovation
17
PCORI
OthersSlide18
CER Complements Precision Medicine Research
Approval
Targeted Medications;
Genetic Tests
Pre-Approval
Drug and Marker
DevelopmentIdentify potential Genetic Markers oftreatment Response, risk
Post-ApprovalCEREvaluate new tests, therapiesSlide19
CEO,
Avalon Health Economics LLC
John E. Schneider, Ph.D. Slide20
The Value of Biopharmaceutical Innovation
John E. Schneider, PhD
Avalon Health Economics | www.avalonecon.com
20Slide21
Pharmaceutical Innovation Requires Patience & Resources
2015 & 2016 Profile: Biopharmaceutical Research Industry
, PhRMA
Avalon Health Economics | www.avalonecon.com
21Slide22
Clinical & Economic Value
2015 Profile: Biopharmaceutical Research Industry
, PhRMA
Avalon Health Economics | www.avalonecon.com
22
There are many areas in which Rx innovations are good “value for money” because the utilization of a drug decreases costs elsewhere in the system:
Rx innovations in undertreated disease areas (HIV/AIDS, CF, MS, MM, etc.)Rx innovation in therapeutic areas where Rx substitutes for more expensive interventions, such as surgery or high volumes outpatient careRx Innovation in areas where there is poor adherence (e.g., a next-generation oral RA treatment replaces an injectable)Slide23
But there is also Direct Value to the U.S. Economy…
The Economic Impact of the U.S. Biopharmaceutical Industry
, PhRMA &
TEConomy
Partners, 2016
Avalon Health Economics | www.avalonecon.com
23At $57 billion, Rx R&D annual expenditures account for nearly 18% (!) of ALL domestic R&D expenditures (based on 2013 data)The U.S. pharma industry devotes nearly 11% of net sales revenue to R&D, which is more than 4-times the national industry averageNearly 20% of biopharma industry employment is devoted to R&D, again this is substantially above the US industry averageRx innovation remains a very human-capital intense industry– across major industry components (R&D, manufacturing, distribution, and administration), the US industry supports just under 1 million jobs (+ another 3 million indirectly)The net result is an economic impact of more than $1.2 trillion annuallySlide24
Senior Vice President of Strategic Initiatives,National Health Council
Eleanor
M. Perfetto, Ph.D., MS Slide25
Medical Innovation 101
Tuesday, February 14, 2017
12:00- 1:00 pm