Adam Heathfield, PhD Adam Heathfield, PhD Senior Director, Worldwide Policy, Pfizer Inc.Senior Director, Worldwide Policy, Pfizer Inc.

September 25, 2013September 25, 2013

Personalised Medicine – an industry Personalised Medicine – an industry perspectiveperspective

The case for Personalised Medicine has been made

…..and clearer safety signals too

Multiple domains and collaborations

Data handlingComprehensive, accessible and interoperable datasets must be generated to support the development of a new disease taxomony and allow for its ongoing refinement and application.

Interdisciplinarity, participation and translational researchEmphasis must be placed on stakeholder participation, interdisciplinary interaction, public-private and pre-competitive partnerships and translational research in order to develop the frameworks that support the vision of personalised medicine and healthcare.

Policy work is essential

UK has good collaborative environment for organising tests and sharing research data, but other countries have higher rates of testing

Emerging findings in the Cancer Research UK Stratified Medicines Programme Shaw et al (ASCO poster)

Diagnostics a key component

Unlocking the value of personalised healthcare in Europe—breast cancers tratification Walter Van Dyck at al Health Policy and Technology (2012) 1, 63–68

PM impacts across care pathways

Clinical Trial: PROFILE 1005 (n = 261 mature patients)References: 1. Kim et al. Poster at ASCO, 2012 (Abstract 7533). 2. XALKORI® Summary of Product Characteristics. 7

Results from an open-label, single-arm, non-comparative Phase II study investigating the safety, tolerability, pharmacokinetics and anti-tumor activity of crizotinib in 261 patients with advanced ALK-positive NSCLC progressed on standard therapy. Patients received oral crizotinib 250mg twice a day in 21-day cycles. Efficacy endpoints included: objective response rate, disease control rate, duration of response, time to response, overall survival and progression-free survival.

• Crizotinib demonstrated marked clinical activity in advanced ALK+ NSCLC (ORR ~60%, median PFS 8.1 months)1,2

• More than 90% of crizotinib patients achieved tumor shrinkage1

Early promise and rapid uptake – or not?

Lots of opportunities for collaboration:• Basic research• Translational medicine• Access and quality of real world data• Diagnostic infrastructure• Early access for patients

Personalised Medicine has moved on from an R&D topic

Integrated approach needed for optimal care and future investment

Summary

Back Up

Evolving Personalized Paradigm

Metastatic disease (stage IIIB/ IV)

EGFR K-RAS ERCC1 ALK TS B-RAFHER-2

Traditional Paradigm

Non-squamous cell carcinoma

Metastatic disease (stage IIIB/ IV)

Squamous cell carcinoma

Diagnostic challenges

Oncologist sole treatment decision maker

Treatment decisions depend on histology

More complex decisions involving more stakeholders beyond oncologist (surgeon, pathologist)

Education required to integrate molecular diagnostics into treatment decisions

Need for multiple molecular Dx creates competition for available tissue, budget, manpower

Not a “simple” issue of a single drug-diagnostic combination

Multiple test options