27-28 May 2013, Munich, Germany

EuroBioForum 2013 2nd Annual Conference

Emmanuelle BenzimraGeneral Delegate

European PErsonalised MEDicine association EPEMED

Navigating the Challenges of Personalized Medicine Access in Europe

EuroBioforum - MUNICH27 May 2013

Emmanuelle BENZIMRA, General Delegateebenzimra@epemed.org

www.epemed.org

BiomarkersBiomarkers and PM are and PM are includedincluded in medical practices in medical practices sincesince the the 19th 19th centurycentury but but theirtheir developmentdevelopment has has acceleratedaccelerated in the last in the last

decadedecade

1960

Biomarkers of cancer : alpha-foetoprotein & CE

antigenCouple

Herceptin/Herceptest

(Roche, Genentech/Dako)

Amplichip Cyp450

(Roche/Affymetrix)

1848

Biomarker of myeloma: Ig chain (BJ)

in 75% cases

Ig : immunoglobulin

BJ : protein of Bence-Jones

PSA : prostate specific antigen

TPMT : thiopurine-méthyltransférase

1980

Biomarker of diabetes: glycemia

Biomarker of diabetes: Hb A1c

Couple Selzentry/Trofile

(Pfizer)

Biomarker of cancer:

Ovarian (CA 125)Prostatic (PSA)

1998

20102000 2005 2007

2006

Erbitux (Merck/Imclone) or Vectibix (Amgen) &

KRAS test

Dasatinib (BMS) & Philadelphia chromosome

« Omics » and start of the genome sequencing

1986

First use of Pharmacogenetic

definition

1959

Discovery of genetic polymorphism of TPMT

(Weinshilboum & Sladek)

1997

First use of Pharmacogenomic

definition

2011

Couple Zelboraf®/ Cobas® 4800

BRAFV600E mutation test (Roche/Daiichi)

2012

Couple Tysabri®/Stratify-JCV antibody Elisa

test (Biogen Idec/Quest)

Couple Xalkori®/Vysis® ALK

Break Apart test (Pfizer/Abbott)

DespiteDespite promisingpromising marketmarket forecastsforecasts and and currentcurrentdevelopmentdevelopment efforts, a efforts, a smallsmall numbernumber of of companioncompanion diagnostics have diagnostics have reachedreached the the marketmarket

< 3 to 5% of companionbiomarkers commercialized

30 to 50 % of Biomarkers

associated in development

•Utility, specificity and validation of biomarkers

•Some biomarkers are only developed for the drugs R&D

• Remaining hurdles associated to regulatory questions, market access (evaluation, coding, pricing and reimbursement ) of the RX-DX couple or of the CDx alone

• Hurdles associated to partnership model, commercialisation business model (alone/in association, distribution flows…)

Attrition rate of biomarkers within the pipeline of the pharma industry

Source: LEEM, Nature Reviews march 2009, « Biomarkers : The expanding global market », Yonker 2006

The The European Challenge: European Challenge: The The 27 Member States of the EU27 Member States of the EU

Personalised Medicine in EU: OverviewPersonalised Medicine in EU: Overview

Very few value-based reimbursement initiatives for innovative Dx

Many barriers and levels of complexity

Heterogeneous region in terms of regulation & reimbursement

Differences in centralised & de-centralised systems in the individual states

Differences in HTA (Health Technology Assessment) systems between and within

countries

The European ChallengeThe European Challenge

EPEMED White paper: Market access challenges in the EU for high medical value diagnostic testsIain Miller†1,2, Joanna Ashton-Chess1,3, Herman Spolders1,4, Vincent Fert1,5, Joseph Ferrara6, Werner Kroll1,7, Jon Askaa8, Patrick Larcier3, Patrick F Terry1,9, Anne Bruinvels10 & Alain Huriez1,3 Ref: Personalized Medicine (2011) 8(2), 137–148

European Opportunities for European Opportunities for PMPMQuality of the scientific research and medical practices

European Commission initiatives

Recast of IVD directive (input provided by EPEMED)

EMA initiatives (innovation task force, scientific advices for qualification of biomarkers, EMA "reflection paper on methodological issues associated with PGxbiomarkers)…(input provided by EPEMED)

High level quality Biobanks (IBBL, IMIDBB,…)

EC grants (FP7 health 2011), IMI programs

European associations (EuroBioforum, EPEMED, EDMA, EAPM, EBE ..)

Why EPEMED ?Why EPEMED ?

EPEMED is an independent, broad and inclusive not-for-profit organisation founded in 2009 and bringing together forces in personalised medicine in the EU.

A central point of communication for the different parties involved in progressing personalised medicine

Addressing issues in personalised medicine that confront the industry, regulators, payers & insurers as well as governments in Europe

Aiming to provide a platform for the harmonisation of personalised medicine development and implementation across Europe, focusing on the role of diagnostics, to make personalised medicine a reality

EPEMED’s Value PropositionEPEMED’s Value PropositionKey Value Points for

Members

• Forum to share best practices

• Publications, white papers, conferences, education & promotion on PersonalisedMedicine subjects

• Privileged access to European decision makers

• Input to policy makers on relevant legislation

Success Factors: EPEMED’s Success Factors: EPEMED’s propositions propositions

One of the major current issues related to the EU low market uptake of IVDPersonalized Medicine tests is the reimbursement process, which is complex,heavy, non-harmonised, and carried out by different commissions in everycountry, in most of cases handling also drugs dossiers.

There are not appropriate competences at payers level ensuring dossierssubmitted are understood and evaluated in a fair and knowledgeable manner.

Moreover, PM being one of the most striking examples of innovation, toolsshould be there to nourish research results and to help them turning intomarketable products.

Success Factors: EPEMED’s Success Factors: EPEMED’s propositions propositions

European wide coordination initiative is needed among health technology bodieswhich reimburse all diagnostics - including molecular diagnostics, companiondiagnostics, and personalised medicine products in order to ensure reimbursementallows innovation. Clarification and description of requirements to be met forreimbursement.

A more unified and coordinated accreditation procedures should be establishedfor European clinical testing laboratories, enabling to break the actual nationalprotectionism.

A more focused and coordinated effort should be established to make use ofpatient registries, patient biological samples, and patient outcome data in atargeted personalised medicine development program with results published in atimely manner.

EPEMED 2012EPEMED 2012--2013 European Market Access 2013 European Market Access StudyStudy

15 months collaboration: EPEMED & La Charité Institute for Social Medicine, Epidemiology, and Health Economics, Universitätsmedizin Berlin (Pr Stefan Willich & Dr Markus Buecheler)

Scope: Personalized Medicine in Europe - Navigating Market Access Challenges for Co-developed and further Combined Molecular Rx/Dx Technologies and Realizing the Potential

Aim: Devise recommendations how to shape a post-approval environment that facilitates broad and equitable access to CDx/Rx.

Approach: Description of the post-approval patient access pathways for CDx/Rx in 5 largestEU countries (Ge, UK, Fr, I, S)• Analyse best practices and barriers to patient access• Develop recommendations after interviews with KOLs Deliverables (starting Q4 2013): Study report, peer-reviewed paper, position paper, conferences.

EPEMED 2012EPEMED 2012--2013 European 2013 European Market Access StudyMarket Access Study

Preliminary findings will be presented on 28/29 October 2013 in Luxembourg (Health Economics Personalised Medicine Symposium) !

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Thank you for your attention !

For more information or to download recent study reports, white paper or webinars, please visit www.epemed.org

Contact: contact@epemed.org