The European MDS studies coordination office

Supporting Clinical Research, Education and Consulting in the field of MDS across Europe

EMSCO

Current MDS Clinical Trial Landscape in Europe

Multiple (69) MDS clinical trials are currently running across the EUOf 395 worldwide open trials with known status, 18 are in Germany, 26 in France, 19 are in Italy

The French Groupe Francophone des Myélodenysplasies (GFM) and the German MDS study group (GMDS-SG) sponsor 14 clinical trials in MDS

GMIHO (Dresden, Germany) is a specialist “Principal Investigator Network”, which pools scientific expertise to offer an efficient infrastructure for clinical trials. Sponsors 3 clinical trials in MDS

Concept for Cooperation

National Study Groups partner with the ELN-based EMSCO officeto carry out international MDS clinical trials

Groupe Francophone des Myélodenysplasies (GFM - France)

German MDS study groupGMDS-SG

Common MDS

Clinical Trials

Other

National MDS

Groups

The Industry has MDS candidate drugs in development in Phase II and will need to launch Phase III trials in the next 5 years.

*Market data from Global data 2012

1. International trials are required to enroll enough patients

2. EC statistics 2007 – 2009 showed a 10% drop in clinical trials in the EU and a 33% drop in the number of EU patients entering clinical trials

3. MDS classification has become more complex and changes in clinical trial structure must reflect that there are smaller subgroups which require more sites

Trends in MDS

The Industry has MDS candidate drugs in development in Phase II and will need to launch Phase III trials in the next 5 years.

*Market data from Global data 2012

Over the next 5-7 years new medications in MDS will come into clinical testing, international clinical trials will be necessary

International cooperation may be particularly important in the following cases:

- high risk MDS first line, to determine the best drugs to combine with HMA

- high risk second line, after HMA failure

- low risk non del 5q second line (after EPO failure)

- low risk del 5q second line (after LEN failure, or in case of TP 53 mutation)

Trends in MDS

Phase 2 (2013):

1. Sustainable and financially independent EMSCO –external funding

2. Website, logo 3. Common trials (GFM/GMDS-SG) are in planning

with the goal to identify hurdles and practical problems (e.g. submission to IRB and national authorities, insurance, central randomization, monitoring etc.)

www.emsco.eu

EMSCO: MDS Studies Coordination Office Phase 1:

Within the ELN, an Office has been set-up located in Dresden and administered by the GMIHO in Berlin

• European Platform for MDS trials

• Single-point of contact - efficient andcoordinated European trials in MDS

• Standardization of SOPs and treatmentparadigms – reduces regulatoryuncertainty regarding international trials

• Improvement in best practices andcomparability of trial results acrossnational borders

Consulting

Academic Clinical Research

Education

Phase 3 (2013/2014/2015)

Three Pillars of EMSCO

• Standardized Clinical Trial Documents - improving the quality andcomparability of clinical trials, eCRF

• SOPs for MDS trials

• Advantages: established by academia, research oriented, nooverhead costs, offers management services, impartial documentevaluation

ServicesSponsor FunctionSite recruiting / feasibilityEssential trial documents (protocol, regulatory submission)Medical WritingNative English proof-readingMDS specific eCFRs and SOPsMonitoring & BiostatisticsData ManagementCoordinator function for EU grants

Academic Clinical Research

• Conferences - organization of MDS specific conferences, symposium organization and strategy-development meetings

• Publications - coordination and conceptualization of common scientific, reviews, white papers and grants in the field of MDS

• Industry Career Development – Training sessions, video content

ServicesConference planningEducational seminars in MDSEducational symposia in MDSGCP training and refresher coursesWriting the Scientific Paper in EnglishTraining in MDS Diagnostics

Education

MDS Colloquium –

Berlin March 2014!

• Commentary on clinical trial design, research projects, grant programs, demographics and epidemiology

• Advice to the pharmaceutical industry and to regulatory agencies on MDS best practices

• Expert opinion on all MDS related questions

ServicesTreatment paradigms in MDSClinical Trial Design for MDSWhite Papers in MDSRegulatory Consultancy Feasibility StudiesGrant ReviewIntellectual property reviewAssessment of trial design for novel compounds

Consulting

1. Inter-european clinical trials in MDS (AVATAR, EUROPE, CMML DAC-HY)

2. European site mapping – key/prime sites for MDS clinical trials

3. Organization of Kick-off meeting – Paris Sept 18, 2013

4. Organization of MDS Colloquium Mar. 2014

5. Organization of 3rd French-German MDS meeting Berlin, Sep. 2014

6. Creation of „open source“ clinical trial documents (i.e. contract templates, eCRF)

7. Establish competency center for MDS

8. Invite sites and Investigators to participate in EMSCO and become listed on the website

Ongoing EMSCO Activities

www.emsco.eu

Websites: Educational material for MDS

Promotions

Newsletter: Reaching 2000

Hematologists in Germany

www.mdsdiagnosis.com

EMSCO - European MDS Studies Coordination Officepowered by GMIHO - Gesellschaft für Medizinische Innovation - Hämatologie und Onkologie mbH Alte Jakobstraße 77, 10179 BerlinTelefon: +49 351-259 33-283 | Telefax: +49 351-259 33-111

Thank you for your attention!

Mission Statement EMSCOThe European MDS studies coordination office

(EMSCO)- Facilitating academic clinical research, education and consulting in the field of MDS across

Europe

EMSCO builds bridges between academic and pharmaceutical clinical developers by:

• Facilitating research• Improving data quality• Accelerating translation and optimizing trial

recruitment in MDS to ultimately improve patient outcomes

The Group (Gesellschaft) for Medical Innovation in Hematology and

Oncology (GMIHO - Dresden, Germany)

Thank you for your attention!