October 18, 2016

Investment opportunitiesAn overview of the developing and emerging portfolios

Carmen Nitsche and John Wise

Partner logo if required

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Agenda

• Introducing new Portfolio Investment committee

• Developing portfolio– Unified chemistry data model– User experience/User centered design – Sequence analysis in the regulated domain

• Emerging portfolio– Wearables – Real-world data platform – IoT

• Other discussion topics– Identification of medicinal products (IDMP) – Non-Clinical Development Community of Interest – Blockchain

Standards• UDM• Wearables

Data sharing• RWD platforms• IoT• Non-clinical development

Best practices• UXUCD• Seq an. In reg dom • IDMP• Blockchain

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Introducing Portfolio Investment committee

Operations Board

Investment committee

Innovation committee

Advisory Panel

David Murray AZ

Angeli MöllerBayer

David NirschlBMS

Devendra DeshmukhMerck & Co

Simon ThornberGSK

Veit UlshoeferMerck Group

Mohit AgnihotriNovartis

Tom PiankoElsevier

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Developing PortfolioStandards• UDM

Data sharing

Best practices• UXUCD• Seq analysis (reg domain)

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Unified [chemistry] data model (UDM)

+Problem Statement: Collaboration and chemical information data exchange with partners and CROs is hampered when different ELNs are in use.• No common data model to describe chemical reactions • Thwarting the development/sharing of business rules for consistent

representation of reactions and IP capture

VARIATIONS STAGES+ +

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Standards•UDM

Data sharingBest practices

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Unified chemistry data model

• Status– Community of interest

established– Excellent example of

cross-community involvement

– Introductory materials posted on IP3

– Domain-aware, experienced PM hired

– Biz case underway• Jarek Tomczak

beginning interviews with stakeholders

• Next steps– Complete

biz case– Establish budget req.– Fundraise

• Estimate $150K required• Several pledges in

process• If this is of interest to

you:• Join the CoI• Contribute use-cases• Make a pledge!

Standards•UDM

Data sharingBest practices

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User experience/User centered designStandardsData sharing

Best practices•UXUCD

Problem Statement: “R&D in Pharma is quite hard enough without R&D IT getting in the way!”

(personal communication from a senior leader in R&D from a major biopharma company)

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User experience/User centered design

• Status– Active CoI– ~40 people/20 companies

at F-2-F joint EMBL-EBI workshop (Sep-16)

– 1st round fund-raising in process. $25K raised from:

• Bayer• GSK• Novartis• Roche• quattro research• (still in discussion with

others)

• Next steps– Contract a

domain-aware, experienced PM.

– Complete fundraising• Looking for $5K/co.

– Launch Phase 1• Build 3 high-priority

UX/UCD tools, • If this is of interest

to you:• Join project team• Make a pledge!

StandardsData sharing

Best practices•UXUCD

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Sequence analysis in regulated domain

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StandardsData sharing

Best practices• Seq analysis (reg domain)

Problem Statement: As sequence analysis becomes increasingly a part of clinical trials - so sequence analysis enters the realms of GCP, and must become compliant with all the concomitant regulations controlling the use of computer-based technologies, including 21 CFR Part 11 and Computer Systems Validation.

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Sequence analysis in regulated domain

• Status– Idea entered into

IP3– CoI emerging– Webinar on topic

presented in Sept 2016

– $20K seed money contributed by Roche to support concept development

• Next steps– Contract a

domain-aware, experienced PM.

– Identify needs and develop business case

• If this is of interest to you:– Join the CoI– Help refine/prioritize

the needs– Make a pledge!

StandardsData sharing

Best practices• Seq analysis (reg domain)

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Emerging PortfolioStandards• Wearables

Data sharing• RWD platforms• IoT

Best practices

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Wearables

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Problem Statement: There is an increasing use of wearables to collect phenotypic data in clinical trials. However, there are few standards to harmonise the data formats for transmission and storage of such data in clinical trials databases. This Community of Interest is working to understand how the Pistoia Alliance can identify best practices and “signpost” appropriate data and information standards.

Standards•Wearables

Data sharingBest practices

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Wearables

• Status– Wearables are a hot

topic our members are highlighting

– Conference session at PA 2016 conference London

– 2 webinar hosted in 2016

• Smart glasses• Clinical trials&

wearables– Initial idea entered

into IP3

• Next steps– Develop the

CoI– Submit idea to

investment committee

• Seed $ to hire PM• If this is of interest

to you:– Join the CoI– Make a pledge!

Standards•Wearables

Data sharingBest practices

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Real-world data

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Problem Statement: The need for real world data (RWD) means a need for rapid integration of structured, semi-structured and unstructured data. Individual approaches are suboptimal. This community of interest will explore what is needed to create a more shared RWD environment, to improve access and mining of such data.

Standards

Data sharing• RWD platforms

Best practices

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Real-world data

• Status– Idea submitted to

IP3– Preliminary polling

of PA members suggests broad interest in topic

– New position paper submitted to PA comms company

• Next steps– Organize a PA

debates webinar – Establish a CoI

• Share case studies• Discuss requirements• Identify best practices and • opportunities for

precompetitive collaboration– Submit idea to investment

committee• If this is of interest to you:

– Join the CoI– Comment on IP3 to help

focus the discussion– Make a pledge!

Standards• Wearables

Data sharing• RWD platforms• IoT

Best practices

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Internet of Things (IoT)

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Problem Statement: Internet of Things is still an immature field within Pharma. This community of interest will share use cases and identify tangible, common opportunities, including those in the laboratory, that could be addressed with a pre-competitive model.

Standards

Data sharing• IoT

Best practices

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Internet of Things (IoT)

• Status– Preliminary idea

entered into IP3– Session at 2016 PA

conference in Boston

• Next steps– Establish CoI

• Share case studies• Identify best practices &• opportunities for

precompetitive collaboration

– Submit idea to investment committee

• If this is of interest to you:– Join the CoI– Share case studies– Make a pledge!

Standards

Data sharing• IoT

Best practices

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Other discussion topics

Standards

Data sharing• Non-clinical development

Best practices• IDMP• Blockchain

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IDMP

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Problem Statement: Mandatory implementation of IDMP (identification of medicinal products - the ISO standard) is upon us. The Pistoia Alliance has the capability to identify pre-competitively and define best practices to implement this standard across the R&D pipeline through establishment of a Community of Interest.

StandardsData sharing

Best practices• IDMP

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IDMP

• Status– Personal

communication with a regulatory agency official... “What is the Pistoia Alliance doing about IDMP?”

– Write-up being prepared

• Next steps– Establish CoI– Submit idea to

investment committee• If this is of interest to

you:– Consider championing

the idea!– Submit comments to

IP3– Join the CoI– Make a pledge!

StandardsData sharing

Best practices• IDMP

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Non-clinical development CoI (NCD CoI)

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Problem Statement: Currently, there is no cross-industry community of interest focusing on optimizing non-clinical development processes. For example, Much time and money is expended by NCD functions replicating protocol creation and validation. Collaborating in this area could save time, decrease costs and increase quality.

Standards

Data sharing• Non-clinical development

Best practices

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Non-clinical development CoI

• Status– Initial IP3 idea

submitted– F2F discussion

planning underway

• Next steps– Hold first CoI

meeting in November

– Submit idea to investment committee

• If this is of interest to you:– Join the CoI– Submit discussion

topics on IP3– Make a pledge!

Standards

Data sharing• Non-clinical development

Best practices

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Blockchain

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Problem Statement: Blockchain has come on our radar recently, but we don’t know enough about it yet. For example can it enhance compliance in clinical trials and the clinical trials supply chain? If this topic is of interest to you, please let us know and consider entering your thoughts into IP3.

StandardsData sharing

Best practices• Blockchain

info@pistoiaalliance.org @pistoiaalliance www.pistoiaalliance.org