Opt-in, Opt-out,

& Patient Led DatabasesAccelerating Treatment

Lowering Costs

Our Partners, Thank You!

Questions? Tweet us!

#EHFG2015

MAPPs

Medicines Adaptive Pathways to Patients seek to

foster access to beneficial treatments for the right

patient groups at the earliest appropriate time in

the product life-span in a sustainable fashion

www.adaptsmart.eu

• Demonstrated potential benefit

• Continuous monitoring of a new therapy

• Measurement and adjustment over the entire

lifecycle

• Objective measures, including real world evidence

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Breakthrough Designation

Demonstrate[s] substantial improvement over

existing therapies on one or more clinically

significant endpoints, such as substantial treatment

effects observed early in clinical development.

FDA will expedite the development and review of

such drug. All requests for breakthrough therapy

designation will be reviewed within 60 days of

receipt, and FDA will either grant or deny the

request.

http://www.fda.gov/RegulatoryInformation/Legislation/SignificantAmend

mentstotheFDCAct/FDASIA/ucm329491.htm

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Cancer Survival Rates in Europe:

Survival of Cancer Patients in Europe, 1999 - 2007: The EUROCARE-5 Study. Eds: P. Minicozzi, R.

Otter, M. Primic-Žakelj, S. Francisci, European Journal of Cancer, vol.51, issue 15, October 2015, pp

2099-2266.

#EHFG2015

Cancer Survival Rates in Europe:

Survival of Cancer Patients in Europe, 1999 - 2007: The EUROCARE-5 Study. Eds: P.

Minicozzi, R. Otter, M. Primic-Žakelj, S. Francisci, European Journal of Cancer, vol.51,

issue 15, October 2015, pp 2099-2266.

#EHFG2015

Gordon McVie,

Senior Consultant,

European Institute of Oncology,

Secretary European Alliance for

Personalised Medicine (EAPM)

Stanimir Hasurdjiev

General Secretary of

the Patient Access

Partnership

Carole M Longson,

Director Centre for

Health Technology

Evaluation,

NICE

Amy M Miller,

Executive VP,

Personalized Medicine

Coalition

Chris Hoyle,

Director, Health

Economics & Payer

Analytics (Oncology),

AstraZeneca

Duane Schulthess,

Managing Director,

Vital Transformation

(moderator)

#EHFG2015

Personalised Medicine- Patient Empowerment(The Patient Centred

Approach)

Professor Gordon McVieeditor, ecancer.org

“Who is” the patient?

Precision Medicine – Aggregation andMapsIntegration of Health Data > Google

of Health

Ref: Toward Precision Medicine: Building a Knowledge Network for Biomedical Research and a NewTaxonomy of Disease National Research Council of the National Academies of Sciences, USA 2012

Each patient can exercise his right to choose his / her treatment if and only if:

He/she is able to understandthe available choice options

He/she is capable of expressing his opinion

The right to CHOOSE

Pathways for Personalised

Medicine – Facilitating Innovation through regulatory change

Gordon McVie, EAPM Secretary,

King's College London,

Key facts about EAPM

• The European Alliance for Personalised Medicine (EAPM) brings together European healthcare experts and patient advocates involved with major chronic diseases

• EAPM aims to improve patient care by accelerating development, delivery and uptake of personalised medicine and diagnostics. There are over 40 members in the Alliance.

• The EAPM is chaired by Helmut Brand, President of EHFG and David Byrne, Former EU Commissioner for Health. It was launched in March 2012.

Political/

Insititutional

EAPM

Membership

Pillars

ACADEMIA

Political Arena

Healthcare Plannes

PATIENTS’ORGANISATIONS

Industry

REGULATORS

Commission

EMA

Parliament

National Stakeholders

Stakeholder Engagement- Communication

EAPM Stakeholders and Engagement

Concerted action is required across three foundational sets of policy actions

Technology generates new data andsilos

• Genome sequencing– Costs in 2-3 years below

CHF 500.00

– Data contain important

personal information

• Effectiveness of drugs

• Health risks

• Mobile Health–

–

> 40’000 health applications

Longitudinal health

monitoring without a visit to

the doctor ’s office

New data silos, no data

integration

–

20“The Anatomy Lecture of Dr. Nicolaes Tulp” – Rembrandt, 1632

21

Molecular portrait of cancer

Survival analysis was done on the 51 doxorubicin treated patients only

Breast Phenotype and Outcome

Sorlie, PNAS

Eligibility for Trastuzumab Therapy

HER2 status

IHC

FISH/CISH+

FISH/CISH–

Eligible for Trastuzumab

Joint Analysis: Disease-Free

Survival: Addition of “H”

87%

67%

75%

N Events

ACT 1679 261

ACTH 1672 134

%

HR=0.48,

2P=3x10-12

ACTH H

ACT

Years From Randomization B31/N9831

Poster Children for the PM Generation

Herceptin in Breast Cancer Glivec in Chronic Myeloid Leukemia

And its not just in cancer!

Ivacaftor in Cystic Fibrosis NT-proBNP in Diabetes

Genes unsupervised analysis: clusteringOne drug for the whole

An

gio

gen

esis

inh

ibit

ors

Bip

ho

sph

on

ates

Stro

ma-

targ

etin

g d

rugs

New

ch

emo

ther

apie

sM

od

ula

tio

n o

f d

rug

sen

sitv

ity

(in

cl IG

F1r

Iinh

)C

ance

r va

ccin

es

TrastuzumabT-DM1

PARP inh ?Cisplatin?

Subtype-specificFirst-in class

AI +everolimus? TKI ?

or new subdivision

according to molecular events

TAM

Small populationHigh sensitivity

Second-in classIn a specific subtype:

To do betterTo reverse resistance

Molecular NicheGlobal trials

mTOR inhibitors small TKIPertuzumab/trast

CHK1 inh ?

Patient with bone marrow mets from a pancreatic G2 NET treated with PRRT with 177Lu-DOTATATE (25 GBq)

Basal 68Ga-DOTATOC PET/CT Final 68Ga-DOTATOC PET/CT

We Are Getting Faster!!!

KRAS

EGFR

EML4-ALK

Slebos 1990

Kwak 2010

Many 1990s

ROS-1

Bergathon 2012

BMJ 2003;327:1459

Four Pillars to EAPM Activities

EAPM WG on Education of HCP

EAPM WG on Big Data

EAPM WG on Access & Value in

Innovation

TF on Regulatory Affairs which

includes – Clinical Trials, Data

Protection, MAPPs & In-Vitro

Diagnostics

Work over the last has culminated

in having Council Conclusions for

Personalised Medicine to be

published – December 9th, 2015

If we take one activity – Research &

Clinical Trials and the recent Cancer

Congress in Vienna a few days ago

Charting a Way Forward for

Personalised Medicine (PM)

EAPM Research Policy Roadmap

Launched at EAPM Italian EU

Presidency Event (Brussels, September

2014)

An empowerment tool to help drive PM

integration into health systems in

Europe

EAPM Action on Clinical Trials and

Research

Assembled a multi-stakeholder panel

Address how Personalised Medicine

enabled models of clinical

research/clinical trials can underpin

better outcomes for (European) Cancer

Patients

Outputs to be published in Special

Issue of Public Health Genomics

Special Issue Topic Areas I

Editorial Horgan, D., Brand A.

Personalised and Precision

Medicine in Cancer Clinical

Trials : Panacea for Progress or

Pandora’s Box

The challenges of Precision

Oncology Drug Development and

Implementation

Establishing the evidence bar for

biomarker integration into

personalised cancer care

Cancer clinical trials in the era

of the new EU regulations

Personalised and Precision

Medicine in Cancer : Panacea for

Progress or Pandora’s Box

Authors: Lawler M, Sullivan R

PPM: Opportunities and Challenges

Cancer Clinical Trials: The Wheel is Broken –

so lets fix it!

Clinical Gain needs to be transformative, not

incremental

Evidence based interpretation of therapeutic

“value”

Converting cost to value: Charting a course to

Affordable Cancer Care

The challenges of Precision Oncology

Drug Development and Implementation

Authors: Hollingsworth SJ, Biankin AV

PM needs to be effective and sustainable

Innovative trial design: Right patient –

Right Trial

New Partnership models– leveraging

international collaboration and multi-

Pharma collaborative networks

Operational integration

Multi-target biomarker screening

Need for integrated molecular pathology

“Fail early”, “Fail Cheaply”

Innovative Flexible Adjustable Algorithms

Establishing the evidence bar for

biomarker integration into

personalised cancer care

Authors: Schneider D, Bianchini G, Horgan D,

Michiels, S, Witjes W, Plun-Favreau J, Lawler M

Paucity of validated biomarkers

Need to define evidence requirements for clinical

adoption/reimbursement

The ideal biomarker?

Actionable mutations and clinical utility

Valuing both Clinical Trial and Cohort samples

Economic validation

Companion Diagnostics regulatory framework

Reimbursement and value based pricing

For more information about

EAPM, please contact:

Denis Horgan, EAPM

Executive Director,

Email: denishorgan@euapm.eu

Ph: 0032 47 25 35 104

Leading cancer

communication in Europe

Information resource for professionals worldwide

Peer to peer education and information

Latest thinking delivered in an engaging format

2. Oktober 2015

Gordon McVie,

Senior Consultant,

European Institute of Oncology,

Secretary European Alliance for

Personalised Medicine (EAPM)

Stanimir Hasurdjiev

General Secretary of

the Patient Access

Partnership

Carole M Longson,

Director Centre for

Health Technology

Evaluation,

NICE

Amy M Miller,

Executive VP,

Personalized Medicine

Coalition

Chris Hoyle,

Director, Health

Economics & Payer

Analytics (Oncology),

AstraZeneca

Duane Schulthess,

Managing Director,

Vital Transformation

(moderator)

#EHFG2015