John Wise, Business Development, Pistoia Alliance

Collaborate 2016: PerkinElmer User Conference

Basel 4th November 2016

Current Trends in Pre-Competitive Collaborations

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1. Introduction – the challenge for, and the status of, the

biopharmaceutical industry

2. R&D innovation and efficiency and pre-competitive

collaborations.

3. Collaborations necessitate information exchange

standards.

4. Understanding alliances; their roles, their overlaps and

the gaps.

5. In which alliances should one invest?

6. The Pistoia Alliance

Presentation Outline

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• [Pharma] will have to learn much more about how the human body functions at the molecular level and the pathophysiological changes disease causes.

• Only then will it be able to develop a better understanding of how to modify or reverse these changes.

• This is a huge undertaking – and one that Pharma cannot complete alone.

• It will require the support of academia, governments, technology vendors, healthcare providers and the regulators.

• Patients must play their part, too; without access to medical data and volunteers for clinical studies, the industry will be unable either to make theoretical advances or to translate those advances into practice.

The Challenge for Pharma

PwC’s Pharma 2020: Virtual R&D Which path will you take?

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The terms ―efficacy‖ and ―effectiveness‖ have very different

meanings.

Efficacy refers to the extent to which a drug does more good than

harm in clinical trials where patients are carefully selected and

monitored

Effectiveness refers to the extent to which a drug does more good

than harm in real life where patients are not so narrowly selected and

often not closely monitored.

Hans-Georg Eichler, M.D., M.Sc.

Senior Medical Officer at the European Medicines Agency in London, United Kingdom

The Tale of Health Care Reform - DIA Global Forum December 2010 p20

Drug Efficacy is not the same as Drug Effectiveness

Challenges in a Changing Landscape

[Today] ―Pharma is developing drugs that bring incremental benefits, but at a

premium price. This has given rise to the debate between the providers and

payers—what is the value of the extra benefit?‖

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The Challenge for Pharma

The context in which pharma operates has

changed dramatically

From vision to decision Pharma 2020

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McKinsey: Evolution or revolution? http://www.mckinsey.com/~/media/mckinsey/dotcom/client_service/Pharma%20and%20Medical%20Products/PMP%20NEW/PDFs/Ev

olution_or_revolution_Compendium_2012.ashx

(EHI) - Electronic health information / (HTA) – Health Technology Assessment

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http://nuffieldbioethics.org/wp-content/uploads/Biological_and_health_data_web.pdf

Isambard Kingdom Brunel, (1805-1859)

The 'Great Eastern', or 'Leviathan', launched 1858.

Length 692 feet. Displacement 32,000 tons.

Not equalled in size for another 50 years.

Paddle and screw propulsion & designed to carry 4,000 passengers.

Made only nine Atlantic crossings before her conversion to a cable-laying ship.

The Great Eastern later went on to lay the first transatlantic cable in 1866

http://www.brunel.ac.uk/about/history/ikb/greateastern

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And the status of…

• U.S. healthcare expenditures amounted to 17.5% of GDP in 2014 • Stronger economy and the newly insured in USA mean increased spending

growth for healthcare.

• However, health spending continues to outpace GDP mandating sharp focus on productivity, efficiency, and better value for purchasers.

• The Western European average, 10.5% of GDP, is unsustainable. (UK ~ 9.1%)

• Specialty drugs spending in the US projected at an annual growth > 20%

• The average top-line growth of the top 10 pharmas decreased from ~5.0% in 2008 to –0.8% in 2013.

• The emergence of health technology assessment (HTA) practices in many healthcare systems makes it harder to achieve an attractive reimbursement value for newly launched products.

Year Spending $ Billions on Speciality Drugs

PwC’s report: “Medical Cost Trend: Behind the Numbers 2015”

2012 87.1

2016 192.2

2020 401.7

• Between March 2000 and April 2014 in the UK, the National Institute for Health and Care Excellence (NICE) gave:

• 36 negative recommendations out of 141 single technology appraisals - a rejection rate of 26%.

• 24 negative recommendations out of 57 for new oncology products - a rejection rate of 42%

• 2011 - Germany’s Act on the Reform of the Market for Medicinal Products (Arzneimittelmarktneuordnungsgesetz, or AMNOG), came into effect.

• Between January 2011 and December 2013, 51 out of 62 assessments found less than a significant incremental benefit - a rejection rate of 82%.

• Pharma needs to move to an integrated evidence development model that brings together randomized controlled trials (RCTs) and real world evidence (RWE) to enable an adaptive licensing approach along the full life cycle of each drug.

• The burden is now on pharma R&D organizations and their corporate leadership fundamentally to overhaul the evidence development model.

And the status of…

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Escaping the sword of Damocles:

Toward a new future for pharmaceutical R&D (McKinsey)

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Blue Line – The number of NMEs / BLAs per company in 10 years

How Much Does Pharmaceutical Innovation Cost?

A Look At 100 Companies

http://www.forbes.com/sites/matthewherper/2013/08/11/the-cost-of-inventing-a-new-drug-98-companies-ranked/

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4,000

6,000

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Number of NMEs and the average R&D costs / NME

Blue Arrow – Member of the Pistoia Alliance

Brown Line – Average cost to develop each NME / BLA over 10 years

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Figure 3 | R&D productivity model: parametric sensitivity analysis. This parametric sensitivity analysis is created from an R&D model that calculates the capitalized cost per

launch based on assumptions for the model’s parameters (the probability of technical success (p(Ts)), cost and cycle time, all by phase). When baseline values for each of the

parameters are applied, the model calculates a capitalized cost per launch of Us$1,778 million. This forms the spine of the sensitivity analysis (tornado diagram). At the top of the

graph are the parameters that have the greatest effect on the cost per launch, with positive effect in blue (for example, reducing cost) and negative effect in red. Parameters

shown lower on the graph have a smaller effect on cost per launch.

How to improve R&D productivity: the pharmaceutical industry’s grand challenge Steven M. Paul et al.

MARCH 2010 | VOLUME 9 http://www.nature.com/nrd/journal/v9/n3/full/nrd3078.html 16

No employer today is independent of those about

him. He cannot succeed alone, no matter how

great his ability or capital. Business today is more

than ever a question of cooperation.

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Orison Swett Marden (1850–1924)

writer, physician, and hotelier

R&D innovation and efficiency and

pre-competitive collaborations.

A rapidly evolving ecosystem

Big Life

Science

Company

Yesterday Today Tomorrow

Yesterday Today Tomorrow

Innovation

Model Innovation inside Searching for Innovation

Heterogeneity of collaborations. Part

of the wider ecosystem

IT Internal apps & data Struggling with change

Security and Trust Cloud/Services

Data Mostly inside Inside Outside Distributed

Portfolio Internally driven and owned Partially shared Shared portfolio

Borrowed from Nick Lynch, Pistoia Alliance and Curlew Consulting

Strategic Alliance in Life Sciences

“Currently there is a lack of adequate alliance setup and first-hand experience of alliance management which will most likely only serve to inhibit the industry’s ability to capture the full value of strategic alliances.

To realise the true potential of strategic alliances an increase in the number of alliance engagements as well as a high commitment – be it financial or not – is required.”

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Deloitte: Executing an open innovation model

Cooperation is key to competition for biopharmaceutical companies http://www2.deloitte.com/content/dam/Deloitte/us/Documents/life-sciences-health-care/us-lshc-open-innovation.pdf

Benefits of open innovation A 3-fold increase in probability of success when drugs are sourced via OI

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New Molecular Entities World Sales $Bn R&D Spend $Bn

EvaluatePharma® World Preview 2015 http://info.evaluategroup.com/rs/607-YGS-364/images/wp15.pdf

Summary of NDA Approvals & Receipts, 1938 to the present http://www.fda.gov/aboutfda/whatwedo/history/productregulation/summaryofndaapprovalsreceipts1938tothepresent/default.htm#Notes

~33% of 2014 launches were for rare indications, mainly

within Anti-cancer.

~65 % were specialty drugs for the treatment of Anti-cancer,

Hepatitis C Virus and HIV.

CMR INTERNATIONAL 2015 PHARMACEUTICAL R&D FACTBOOK

Benefits of open innovation – some green shoots perhaps?

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Mid-Sized Pharmas externalise less of their R&D than Big

Pharmas

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R&

D F

TE

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er

€1M

Spend

http://www.novasecta.com/insights/white-papers/the-novasecta-european-midpharma-report-2016

2.4

3.9 3.8 3.2

Racing to define pharmaceutical R&D external innovation models Liangsu Wang, Andrew Plump, Michael Ringel

Drug Discovery Today, March 2015

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Racing to define pharmaceutical R&D external innovation models Liangsu Wang, Andrew Plump, Michael Ringel

Drug Discovery Today, March 2015

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An increasingly crowded

Information Ecosystem Alliance Space!

Product X-licensing

Co-Development

Partners

Regulators

Public / Private

Partnerships

in silico

in vitro in vivo in patient

Ecosystem Semantic Interface

Web Interface

Standards-based, Scalable, Analytical Processing, Workflow and Knowledge Management

Scientific Strategy

R&D Decision

Support

Project managers

Resource, process

management

Research Teams Regulatory Strategy

Risk Management /

Signal Detection Scientific Analytics

Virtual

Pharma

On the way to the bio-pharma Information Ecosystem

The Pistoia Alliance

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Pistoia Alliance

Lowering the barriers to innovation in life

science R&D

by improving inter-operability of business

processes

through pre-competitive collaboration

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Pistoia Alliance Value Engine

Retaining existing

members and attracting new

members

Running exciting, high profile Innovation

Challenges and Competitions

Establishing Interesting &

Innovative Projects & Activities

Conversations

Conferences

Webinars

Communities of Interest

Life Sciences companies

Technology community

Academic community

Startup Challenges

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Pistoia Alliance paying members (July 2016)

• Accenture

• Agrimetrics

• Amgen

• AstraZeneca

• Bayer

• BioReference

• Biovariance

• Boehringer-Ingelheim

• Bristol-Meyers Squibb

• ChemAxon

• Dassault (BIOVIA)

• DataBiology

• DNAnexus

• Dotmatics

• Elsevier

• GSK

• Hewlett Packard

• Instem

• Ionis

• Johnson & Johnson

• KNIME

• KWS

• Merck Inc.

• Merck KGaA

• Novartis

• Novaseek

• Osthus

• Perkin Elmer

• Pfizer

• quattro research

• Roche

• Scitegrity

• Takeda

• Tessella

• The Hive

• Thomson Reuters

• UCB

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Overall ROI for Pistoia Alliance Members

Inform

• Attend member-only meetings, receptions and networking events

• Learn of emerging, disruptive, technology trends

• Get broader perspectives from technologists in different industry verticals

Innovate

• Amplify your R&D innovation spend

• Solve common challenges by collaborating across Life Sciences R&D

• Support start-up challenges through judging and mentorship

Influence

• Initiate cross-company work streams

• Contribute to consensus-based, cross-company requirements

• Demonstrate industry leadership

• Stand for election to the Innovation Committee

Implement

• Access to the unique, “on-demand” Pistoia Alliance innovation platform

• Participate in project specification workgroups & active project teams

• Help drive direction & portfolio by standing for election to the Board or Portfolio Investment Committee

The Pistoia Alliance membership fee is just a small part of the overall member commitment. For

maximum return on investment, members should invest time money and passion into strategic

and impactful projects that leverage this unique, inclusive, precompetitive collaboration model.

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Interactive Project Portfolio Platform (IP3)

• Collection of all our

project ideas

• Highlights our active

and funding portfolios

• Supports discussion

around new ideas

• Provides updates and

status reports

• Fully open to everyone

• Please contribute your

ideas and comments

IP3 enables open innovation and collaboration

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Pistoia Alliance Debate Webinar Program

• Debate webinar series presents ―hot topics‖ relevant to

Life Sciences R&D community

• 2016 programs so far have attracted >1,000 global

registered attendees from over 12 countries with appx

50% attendance

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Date Topic Registered Attended

Nov-16 CRISPR: what it is, and why it is having a profound impact on human health 138

Sep-16 Sequence Analysis in the Regulated Domain of the Biopharmaceutical Industry 72 36

Jul-16The PhUSE Framework for the Adoption of Cloud Technology in the Regulated Life

Sciences Industry159 80

May-16 Moving Research Informatics to the Cloud 324 150

Mar-16 SEND, the CDISC Standard for Exchange of Nonclinical Data 149 93

Feb-16 Smart Glasses, Smart Scientists 85 42

Jan-16 Analytics for Clinical trials and wearables 226 104

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Webinar Outreach

Pistoia

Alliance

Membership

Introduction

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Pistoia Alliance Project Overview

Active Projects

1. HELM (Hierarchical Editing Language for Macromolecules)

2. Controlled Substance Compliance Expert Community

3. Chemical Safety Library

4. Ontology Mapping

Emerging Projects

1. Expanding Antibody 3D Structures in PDB

2. Sequence Analysis in the Regulated Domain

3. User Experience / User-Centred Design

4. Unified Chemistry Data Model

Other project ideas

1. Wearables & Internet of Things

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30 second Introduction

N

NH

O

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N

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Small

Molecules

Sequences Biomolecules

Small Molecule Tools Sequence-Based Tools

The representation language and

toolkit that remediate this gap :

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Chemical Safety Library (CSL)

Project Rationale

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Chemical Safety Library

Project Team

1. Amgen 2. AZ 3. Bayer 4. BMS 5. Chemaxon 6. Cornell University 7. Dotmatics 8. Elsevier 9. GSK 10. Merck 11. Merck Group 12. PerkinElmer 13. Pfizer 14. SiAl

Steering Committee

1. Bayer 2. BMS 3. GSK 4. Merck Group 5. Merck INC 6. Pfizer

The Chemistry Safety Library

project will capture and share

previously inaccessible cross-

industry hazardous reaction

incident information.

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Steering Committee

1. Amgen 2. AstraZeneca 3. Bayer 4. GSK 5. Merck 6. Novartis 7. quattro research 8. Roche

User Experience / User-Centred Design

F-2-F Workshop – Sep 2016

~ 40 delegates

~ 20 companies

Recruiting an experience,

domain-aware, project manager

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Selected Bibliography

1. Robotics I, Robot – I, The Future Richard Holland, DDW Volume 17 Issue 4 - Fall 2016

2. Smarter ELNs and Smarter Labs Make for Smarter Science Joe Stanganelli – 31st October 2016 - BioIT World

http://www.bio-itworld.com/2016/10/31/smarter-elns-and-smarter-labs-make-for-smarter-science.aspx

3. Pre-competitive Collaboration in Pharma- An Overview Study Eric Gastfriend and Bryan Lee - 24th February 2015

https://www.google.co.uk/url?sa=t&source=web&cd=2&ved=0ahUKEwjG442T24DQAhVpDMAKHZW0Ad8QFggXMAE&u

rl=http%3A%2F%2Ffutureoflife.org%2Fdata%2Fdocuments%2FPreCompetitiveCollaborationInPharmaIndustry.pdf&usg=

AFQjCNGEFaIt_eXo2ZvJDMuNwolwEd5g1Q

4. Precompetitive Collaboration: Emerging Realism in the Pharma World Sandeep Pingle - 6th November 2014

http://sciberomics.com/precompetitive-collaboration-pharma/

5. Why do Pistoia Alliance members contribute to it voluntary projects? (LOUGHBOROUGH MBA PROGRAMME)

Claire Bellamy – 24th September 2014

http://www.pistoiaalliance.org/wp-content/uploads/2015/10/Claire-Bellamy-Pistoia-Alliance-MBA-Dissertation.pdf

6. Collaboration for innovation is the new mantra for the pharmaceutical

industry. Professor Jackie Hunter - Spring 2014 Drug Discovery World

http://www.ddw-online.com/business/p217613-collaboration-for-innovation-is-the-new-mantra-for-the-pharmaceutical-

industry-spring-14.html