Lecture - Slides - DDC WSE First Lecture 2013

Williams S. Ettouati, Pharm. D.
Director, Industrial Relations & Development

Health Sciences Associate Clinical Professor, N.S.
Lecture Objectives
Course Introduction
Pharmaceutical and Biotechnology Industry
Landscape
Global Outlook: What Will the Future
Potentially Look Like?
Patient Centric Discovery
DDC Course Objectives
Drug Discovery Process
From target to Investigational New Drug Application (IND)
Pharmaceutics & Life Cycle Strategic Plan
Clinical Development
- IND to New Drug Application (NDA) submission
- Clinical study design
Regulatory Requirements to File an IND and an NDA
Intellectual Property Strategy
Commercialization Strategies
Marketing strategy; Managed markets
Strategic partnership and business development
Work in Multifunctional Teams
Pharmaceutical & Biotechnology
Industry Landscape
Pharmaceutical Market 2011
955 billion US $
Source: IMS Health
North
America
36%
Europe
25%
Japan
12%
China
7%
Africa, Asia
& Australia
13%
Latin
America
7%
CEO Short-Term Confidence in the Economy
1,258 CEOs from 60 countries shared their view with PwC in 2012

Q: How confident are you about your companys prospects for
revenue growth over the next 12 months?
Source: PricewaterhouseCoopers LLP 15th Annual Global CEO Survey 2012
www.pwc.com/gx/en/ceo-survey/.../15th-global-pwc-ceo-survey.pdf
Fundamental Changes
Capacity to innovate is at the forefront of priorities for CEOs
Patent expirations and low R&D productivity are leaving many
pharma with uncertain revenue streams
Major Changes to R&D: 1/3 of CEOs in pharmaceutical and life
sciences, chemicals and technology industries
Pharma is shifting research resources to Asia
R&D spending in Asia has surpassed EU levels
A financial institution predicts R&D spending in Asia likely to
overtake US levels before 2020
Pressure for Process & Radical Innovation
Q: To what degree are you changing the emphasis of your companys
overall innovation portfolio in the following areas?
New Molecular Entities Launched in
US 2002-2011
The Use of Medicines in the United States: Review of 2011
Source: IMS Institute for Healthcare Informatics

34 New Molecular Entities launched in US, most in last 10 years
FDA Approved
39
*
New Molecular Entities in 2012
Source: Bioassociate Consulting
2012: The Winners and Losers of the Pharmaceutical Industry
FDA approved 39
new drug in 2012

Almost twice the
number approved
in 2010

Highest number
since mid 90s
Source: FDA
Few Breakthrough Drugs Available
For First Time in 2012
Ivacaftor
First treatment that targets underlying mechanism of Cystic Fibrosis
Tofacitinib
First Rheumatoid Arthritis (RA) treatment in the new class of Janus
kinase inhibitors, and the first new oral disease-modifying anti-rheumatic
drug for RA in more than 10 years
Lorcaserin
First weight loss prescription drug to reach market in 13 years, an
innovative molecule, Lorcaserin, a serotonin receptor agonist
Source: Bioassociate Consulting
2012: The Winners and Losers of the Pharmaceutical Industry
80% Prescriptions Dispensed as a
Generic in 2011
Generics dispensed 94% of
time where a generic form is
available
Patent expiries that
occurred in late 2011; not
yet impacted utilization or
spending by end of year
Majority of increase driven
by expiries in 2010 and
early 2011, including
Lovenox
, Aricept
and
Effexor XR
.
Report by the IMS Institute for Healthcare Informatics

Spending in Leading Therapy Areas

Absolute spending
growth gains highest for
antipsychotics,
antidiabetes, respiratory
agents, autoimmune
diseases and lipid
regulators
Many generics in
leading traditional
classes including lipid
regulators and
antipsychotics
Specialty class
spending up > 10% in
multiple sclerosis,
autoimmune diseases
and HIV antivirals,
< 5% in oncology
Top Medicines by Prescriptions
Leading active-
ingredients or
ingredient fixed-
combinations, includes
both branded &
generics
Includes all
prescriptions
dispensed through all
retail pharmacies
90-day & 30-day
prescriptions both
counted as one
prescription

Global Outlook
What Will the Future
Potentially Look Like?
Opportunities & Threats
Opportunities
Strengthening scientific base
A lot more to learn about the
human body
Escalating demand for medicines
Trade liberalization
Wireless health

Threats
Poor scientific productivity
Tighter regulation
More difficult market conditions
Stronger price controls
Soaring healthcare costs
Patent cliff
Note: Figures for 2012 cover period up to 18 July 2012
Pharmas Financial Penalties in US
Source: PricewaterhouseCoopers LLC
http://www.pwc.com
Challenges: Patent Expiration
Exposure & Impact - $127Bn
U.S.: $103Bn (44%) of 2011

Canada: 42% of spending
will be exposed

Other Developed markets
23%

Patent expiration impact
- 13 of top 20 products
- 7 of the top 10 current
leading medicines such as
Lipitor
, Plavix
, Advair
Diskus
, Crestor
and
Nexium

Brand Spending Shift to Generics
Medicines Outlook Through 2016 IMS Health
Source: IMS Institute for Healthcare Informatics, May 2012
2016 Global Spending Reach $1.2 Trillion
From 2006 to 2016 drug spending

Developed markets: 73% to 57%
U.S.: 41% to 31%
- Patent expiries & slower brand
growth
EU5: 19% to 13%
- Slower economic growth
- Aggressive cost containment
measures

Pharmerging markets: 14% to 30%
- Millions of people gain access to
basic medicines
Japan: 10% flat
- Biennial price cuts expected in
2014 and 2016
Source: IMS Market Prognosis, May 2012
Spending by Geography
*Pharmerging markets include China, Brazil, India, Russia, Mexico, Turkey, Poland, Venezuela,
Argentina, Indonesia, South Africa, Thailand, Romania, Egypt, Ukraine, Pakistan and Vietnam.
Spending Growth Driven By Innovation
Only anti-epileptics,
contraceptives and
antivirals (except HIV
drugs) grow faster
than in the past 5
years
Specialty medicines
experience continued
growth in the mid-
term, driven by:
Novel mechanisms
Improved efficacy
Relatively larger
patient populations
leading to increased
uptake
Compounded Annual Growth rate (CAGR): business and investing specific term for the
smoothed annualized gain of an investment over a given time period
By 2020, Pharma Will be Making
Much More Diverse Range of Products
New product types
More complex manufacturing & distribution processes
Different supply chains for different product types
Shorter product lifecycles
http://www.pwc.com/gx/en/pharma-life-sciences/pharma2020/vision-to-decision.jhtml
What is Pharma & Biotech Innovation?
A new drug
Researcher identifies scientific potential of a particular target and molecule
Investor backs belief with $$$$$
Company commits resources development and production of treatment
Regulator approves label
New innovative drug reaches the market, company commits promotion
resources
Healthcare payer, provider and patient, respectively decide on the new drug
innovativeness:
Healthcare payer by paying a premium price for it
Provider by choosing it over other therapies
Patient by taking it as instructed or even pressing for a prescription
Treatments Transformed by New and
Existing Mechanisms
32-37 drugs expected to be
launched per year over the next
five years

New mechanisms of action in
Alzheimer's, autoimmune
diseases and various cancer,
have the potential to transform
disease treatment

Not every therapy will become
available or achieve its ultimate
clinical aims

Further developments in areas of
research where some therapies
already exist like:
- Hepatitis C
- Multiple sclerosis
- Prostate cancer
Have a potential to deliver better
efficacy, safety or convenient
administration
Patient Centric Drug Discovery &
Development

Tomorrows challenge to develop new medicines that can
Prevent
or
Cure currently incurable diseases
Challenges in Drug Discovery & Development
R&D productivity has not improved drug approvals have not increased significantly,
while development costs have escalated

Linear: Drug R&D is conducted in a stepwise manner
Slow: Taking a compound or molecule from early research to approved product
takes over 10 years
Inflexible: Drug development process is also very rigid and highly regulated by
FDA, EMEA and others
Expensive: On average, companies spend well over US$1 billion to bring an
approved drug to market (a number that includes the cost of products that fail
along the way)

Siloed: R&D process is highly fragmented. Driven by the need to protect their
intellectual property, companies fail to learn from experiences and the mistakes
of others
Source: Beyond borders Global biotechnology Report 2012, Ernst & Young
Pharma 2020: Marketing the future: Which path will you take?
Value Chains
Pharmaceutical and Biotech
Healthcare Provider
Healthcare Payer
New Drug Development Paradigm
Instead of a linear, slow, inflexible, expensive and siloed drug development paradigm
Pharma need one that is iterative, fast, adaptive, cost efficient and open/networked
HOLNet approach represents a vastly different and inclusive approach to R&D

HOLISTIC: Boundaries between drug development, product commercialization and
health care delivery are blurred; Share data and connect dots across the entire value
chain of companies
OPEN: openness members pool their strengths and assets. Involve sharing any
resulting output e.g., creating open standards, making insights available to all
members and often to nonmembers as well

LEARNING: Learning rapidly, in real time, by connecting data from across the
ecosystem. Allows constituents to quickly adjust their approaches from clinical trials
to standards of care saving time and money and potentially increasing success rates

NETWORK: Radically reinventing R&D and unleashing the transformative potential of
big data requires the participation of diverse players from across the ecosystem
Convergence of Value Chain
Single Circular Value Chain: The Patient
Value Providers generate
depends on the revenues
payers raise and the
medicines Pharma makes
Value Pharma generates
depends on getting access to
the patients whom providers
serve and income from the
payers who fund those
providers
Relationship between
different players is often quite
antagonistic and, while they
continue to clash, they are
struggling to retain their
respective goals
http://www.pwc.com/gx/en/pharma-life-sciences/pharma2020/vision-to-decision.jhtml
Innovation!
Disruptive innovation
CURE a disease or PREVENT the disease and reduce mortality or morbidity

Incremental innovation
Reduce the cost of care
Improve the quality of life
Safer or easier to use
Improve patient compliance
Issue ME TOO drugs
Pharma race to develop new products, which all have the same mode of action
Maybe the third or fourth market entrant may be superior!
Payers are not going to pay a premium unless
Drug shows disruptive innovation
Demonstrate clear superiority in comparative trials with pharmacoeconomic
benefits
Breast Cancer
Understanding Being Transformed
Every patient experience now generates rivers of data which, if pooled intelligently,
can trace a detailed portrait of a patients health and, when aggregated with other
patient data streams, can coalesce into deep reservoirs of knowledge about entire
disease states and patient populations. an industry marketer
Source:PricewaterhouseCoopers LLC: From vision to decision, Pharma 2020
www.pwc.com/pharma2020
Which Stakeholders Decide if a New
Product is Truly Innovative?
The Payer
Building Collective Intent Care Networks
to Change Health Care Delivery
Create disease networks leveraging the creative
models that many health care systems are now
piloting

From accountable care organizations and
patient-centered medical homes in the US to
primary care trusts in the UK.

Focusing on outcomes, patient-centric
approaches and preventive care, such programs
already provide some of the key building blocks
of a HOLNet approach

HOLNets could supplement such models
by bringing a broader spectrum of constituents
from across the ecosystem

Potentially bring disease-specific focus and,
create collective intent to cure or radically
improve outcomes within that disease
Care Pathways
Healthcare providers in many parts of the world are developing
defined care pathways
Standardize treatment of patients with the same illnesses
Improve outcomes
Result in creation of defined healthcare packages for each disease
Pharma 2020: Supplying the future - Which path will you take?
FDA Approves Computer Chip for Humans
Implantable computer chip approved in 2004 for implantation in a patients
arm for medical purposes

Can speed vital information about patients medical history to doctors and
hospitals
Inserted under the skin
Silently and invisibly, dormant chip stores a code that releases patient-
specific information when a scanner passes over it
Chip contains no medical records, just codes that can be scanned, and
revealed, in a doctors office or hospital
With the code health providers can unlock that portion of a secure database
that holds that persons medical information, including allergies and prior
treatment
June 2007, the American Medical Association declared that "implantable
radio frequency identification devices may help to identify patients
Source: FDA approved VeriChip for medical applications 2004
VeriChip

Thank you

Williams S. Ettouati, Pharm. D.
Director, Industrial Relations & Development
Health Sciences Associate Clinical Professor, N.S.

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Williams S. Ettouati, Pharm. D.

Director, Industrial Relations & Development

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