lecture_slides_ddc wse first lecture 2013

8/11/2019 Lecture_slides_DDC WSE First Lecture 2013

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


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Lecture Objectives

Course Introduction

Pharmaceutical and Biotechnology IndustryLandscape

Global Outlook: What Will the FuturePotentially Look Like?

Patient Centric Discovery


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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


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Pharmaceutical & Biotechnology

Industry Landscape


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Pharmaceutical Market 2011

955 billion US $

Source: IMS Health

NorthAmerica

36%

Europe

25%

Japan12%

China7%

Africa, Asia& Australia

13%

LatinAmerica

7%


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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 forrevenue growth over the next 12 months?

Source: PricewaterhouseCoopers LLP 15th Annual Global CEO Survey 2012www.pwc.com/gx/en/ceo-survey/.../15th-global-pwc-ceo-survey.pdf


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Fundamental Changes

Capacity to innovate is at the forefront of priorities for CEOs

Patent expirations and low R&D productivity are leaving manypharma with uncertain revenue streams

Major Changes to R&D: 1/3 of CEOs in pharmaceutical and lifesciences, 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 toovertake US levels before 2020



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Pressure for Process & Radical Innovation

Q: To what degree are you changing the emphasis of your companysoverall innovation portfolio in the following areas?



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New Molecular Entities Launched in

US 2002-2011

The Use of Medicines in the United States: Review of 2011Source: IMS Institute for Healthcare Informatics

34 New Molecular Entities launched in US, most in last 10 years


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FDA Approved

39*New Molecular Entities in 2012

Source: Bioassociate Consulting2012: The Winners and Losers of the Pharmaceutical Industry

FDA approved 39new drug in 2012

Almost twice the

number approvedin 2010

Highest numbersince mid 90s

Source: FDA


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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 Januskinase inhibitors, and the first new oral disease-modifying anti-rheumaticdrug for RA in more than 10 years

Lorcaserin

First weight loss prescription drug to reach market in 13 years, aninnovative molecule, Lorcaserin, a serotonin receptor agonist

Source: Bioassociate Consulting2012: The Winners and Losers of the Pharmaceutical Industry


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80% Prescriptions Dispensed as a

Generic in 2011 Generics dispensed 94% of

time where a generic form isavailable

Patent expiries thatoccurred in late 2011; notyet impacted utilization or

spending by end of year

Majority of increase drivenby expiries in 2010 andearly 2011, including

Lovenox, AriceptandEffexor XR.

The Use of Medicines in the United States: Review of 2011Report by the IMS Institute for Healthcare Informatics


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Spending in Leading Therapy Areas

The Use of Medicines in the United States: Review of 2011

Report by the IMS Institute for Healthcare Informatics

Absolute spendinggrowth gains highest forantipsychotics,antidiabetes, respiratoryagents, autoimmunediseases and lipidregulators

Many generics in

leading traditionalclasses including lipidregulators andantipsychotics

Specialty classspending up > 10% in

multiple sclerosis,autoimmune diseasesand HIV antivirals,

< 5% in oncology


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Top Medicines by Prescriptions

Leading active-ingredients oringredient fixed-combinations, includesboth branded &

generics

Includes all

prescriptionsdispensed through allretail pharmacies

90-day & 30-dayprescriptions both

counted as oneprescription

The Use of Medicines in the United States: Review of 2011Report by the IMS Institute for Healthcare Informatics


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Global Outlook

What Will the Future

Potentially Look Like?


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Opportunities & Threats

Opportunities

Strengthening scientific base

A lot more to learn about thehuman 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 LLChttp://www.pwc.com


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Challenges: Patent Expiration

Exposure & Impact - $127Bn

U.S.: $103Bn (44%) of 2011

Canada: 42% of spending

will be exposed

Other Developed markets23%

Patent expiration impact

- 13 of top 20 products

- 7 of the top 10 current

leading medicines such asLipitor, Plavix, AdvairDiskus, CrestorandNexium

Brand Spending Shift to Generics

Medicines Outlook Through 2016 IMS HealthSource: IMS Institute for Healthcare Informatics, May 2012


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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 brandgrowth

EU5: 19% to 13%

- Slower economic growth

- Aggressive cost containmentmeasures

Pharmerging markets: 14% to 30%

- Millions of people gain access tobasic medicines

Japan: 10% flat

- Biennial price cuts expected in2014 and 2016

Medicines Outlook Through 2016 IMS HealthSource: 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.


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Spending Growth Driven By Innovation

Only anti-epileptics,contraceptives andantivirals (except HIVdrugs) grow fasterthan in the past 5

years

Specialty medicines

experience continuedgrowth in the mid-term, driven by:

Novel mechanisms

Improved efficacy

Relatively larger

patient populationsleading to increaseduptake


Compounded Annual Growth rate (CAGR): business and investing specific term for thesmoothed annualized gain of an investment over a given time period


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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

Source: PricewaterhouseCoopers LLChttp://www.pwc.com/gx/en/pharma-life-sciences/pharma2020/vision-to-decision.jhtml


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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 promotionresources

Healthcare payer, provider and patient, respectively decide on the new druginnovativeness:

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

T t t T f d b N d


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Treatments Transformed by New and

Existing Mechanisms

32-37 drugs expected to be

launched per year over the nextfive years

New mechanisms of action inAlzheimer's, autoimmune

diseases and various cancer,have the potential to transformdisease treatment

Not every therapy will becomeavailable or achieve its ultimate

clinical aims

Further developments in areas of

research where some therapiesalready exist like:

-

Hepatitis C- Multiple sclerosis

-

Prostate cancer

Have a potential to deliver better

efficacy, safety or convenientadministration



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Patient Centric Drug Discovery &

Development

Tomorrows challenge to develop new medicines that can

Prevent

orCure currently incurable diseases


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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 producttakes over 10 years

Inflexible:Drug development process is also very rigid and highly regulated byFDA, EMEA and others

Expensive:On average, companies spend well over US$1 billion to bring anapproved 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 theirintellectual property, companies fail to learn from experiences and the mistakesof others

Source: Beyond borders Global biotechnology Report 2012, Ernst & Young


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Source: PricewaterhouseCoopers LLCPharma 2020: Marketing the future: Which path will you take?


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Value Chains

Pharmaceutical and Biotech

Healthcare Provider

Healthcare Payer



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New Drug Development ParadigmInstead 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 andhealth care delivery are blurred; Share data and connect dots across the entire valuechain of companies

OPEN: openness members pool their strengths and assets. Involve sharing anyresulting output e.g., creating open standards, making insights available to allmembers and often to nonmembers as well

LEARNING: Learning rapidly, in real time, by connecting data from across theecosystem. 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 ofbig data requires the participation of diverse players from across the ecosystem


C f V l Ch i


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Convergence of Value Chain

Single Circular Value Chain: The Patient

Value Providers generatedepends on the revenuespayers raise and themedicines Pharma makes

Value Pharma generatesdepends on getting access tothe patients whom providers

serve and income from thepayers who fund thoseproviders

Relationship betweendifferent players is often quiteantagonistic and, while they

continue to clash, they arestruggling to retain theirrespective goals

Source: PricewaterhouseCoopers LLChttp://www.pwc.com/gx/en/pharma-life-sciences/pharma2020/vision-to-decision.jhtml


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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 pharmacoeconomicbenefits

Breast Cancer


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Breast CancerUnderstanding 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 otherpatient data streams, can coalesce into deep reservoirs of knowledge about entiredisease states and patient populations. an industry marketer

Source:PricewaterhouseCoopers LLC: From vision to decision, Pharma 2020www.pwc.com/pharma2020


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Which Stakeholders Decide if a New

Product is Truly Innovative?

The Payer


B ildi C ll ti I t t C N t k


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Building Collective Intent Care Networks

to Change Health Care Delivery


Create disease networks leveraging the creativemodels that many health care systems are nowpiloting

From accountable care organizations andpatient-centered medical homes in the US toprimary care trusts in the UK.

Focusing on outcomes, patient-centricapproaches and preventive care, such programsalready provide some of the key building blocksof a HOLNet approach

HOLNets could supplement such modelsby bringing a broader spectrum of constituents

from across the ecosystem

Potentially bring disease-specific focus and,create collective intent to cure or radicallyimprove outcomes within that disease


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FDA Approves Computer Chip for Humans

Implantable computer chip approved in 2004 for implantation in a patientsarm for medical purposes

Can speed vital information about patients medical history to doctors andhospitals

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, andrevealed, in a doctors office or hospital

With the code health providers can unlock that portion of a secure databasethat holds that persons medical information, including allergies and priortreatment

June 2007, the American Medical Association declared that "implantableradio frequency identification devices may help to identify patients

Source: FDA approved VeriChip for medical applications 2004

VeriChip


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Thank you

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

lecture_slides_ddc wse first lecture 2013

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