ENTREPRENEURIAL INNOVATION BOOTCAMP IN

ORPHAN DISEASES

OCTOBER 23-25 2017 LEUVEN, BELGIUM

PROGRAM BOOK

MONDAY 23 OCTOBER 2017SESSION 1

Diane KLEINERMANSMinistry of Social Affairs and Health, Belgian Federal Government

POLICY ISSUES IN BELGIUM RELATED TO ORPHAN DISEASES

Agnès VEYRADIERAP-HP, Paris

DIAGNOSTICS IN TTP AND OTHER ORPHAN DISEASES - OVERVIEW AND UNMET NEEDS

KEY DRIVERS AND RESISTORS IN THE ORPHAN DISEASE DRUGS AND

DIAGNOSTICS LANDSCAPE

Carla HOLLAKAMC, Amsterdam

CHALLENGES LINKED TO DIAGNOSIS AND TREATMENT OF LYSOSOMAL STORAGE DISORDERS: ON THE INTERPLAY BETWEEN ACADEMICS, POLITICS, PATIENTS AND BIG PHARMA.

NETWORKING RECEPTION

Kristina LARSSONEuropean Medicines Agency

THE REGULATORY SYSTEM ON ORPHAN DISEASES WITHIN THE EU AND INTERACTIONS WITH EMA

Cees SMITPATIENT ORGANISATIONS: TENDENCIES TOWARDS TRUE PATIENT EMPOWERMENT

BUILDING A VALUE PROPOSITION AND SUSTAINABLE BUSINESS PLAN WITHIN

THE ORPHAN DISEASE ECOSYSTEM

TUESDAY 24 OCTOBER 2017SESSION 2

Walter VAN DYCKVlerick Business School Belgium

OPPORTUNITIES AND RISKS IN THE PROCESS OF CAPTURING VALUE FROM INNOVATION IN HEALTHCARE.

Ingrid MAESInovigate

OPTIMAL BUSINESS PLAN DEVELOPMENT WITH FOCUS ON SWOT ANALYSIS AND CRITICAL ASSUMPTION PLANNING.

SESSION 3

Frank VANDERDONCKAvivia

ORPHAN DRUG APPLICATIONS: A STRATEGIC OPTION FOR DRUG REPURPOSING

Hanspeter ROTTENSTEINERShire

ADVANCEMENT AND CHALLENGES IN GENE THERAPY FOR RARE DISEASES.

Luc KUPERSSanofi

OPPORTUNITIES AND PITFALLS IN THE MANUFACTURING OF BIOLOGICAL DRUGS FOR ORPHAN DISEASES

CHALLENGES IN DEVELOPING SOLUTIONS FOR ORPHAN DISEASES

WHEN MOVING OUTSIDE R&D

Hilde DE WINTER Ablynx

THE DEVELOPMENT OF CAPLACIZUMAB AS ORPHAN DRUG: COMPANY TESTIMONIAL AND KEY LEARNING EXAMPLE

University of MilanRANDOMIZED CLINICAL TRIALS IN RARE BLEEDING DISORDERS

Flora PEYVANDI

CLINICAL TRIAL DESIGN AND STRATEGIC PARTNERSHIPS ARE KEY TO ENSURE

SUCCESS

WEDNESDAY 25 OCTOBER 2017SESSION 4

Paul COPPOAP-HP, Paris

DRIVING OF CLINICAL TRIALS IN THROMBOTIC MICROANGIOPATHIES: BEHIND THE SCENES

Daan DIERICKX UZ Leuven

CHALLENGES IN CLINICAL TRIALS FOR ORPHAN HEMATOLOGICAL MALIGNANCIES

AmgenTBA

PfizerRARE DISEASES: UNIQUE CHALLENGES OR UNIQUE OPPORTUNITIES

FOR ORPHAN DRUG DEVELOPMENT?

Steven SIMOENS

Dirk VANDER MIJNSBRUGGE

KU LeuvenBENEFIT-RISK PREFERENCES FOR NEW MEDICINES IN

RARE DISEASE PATIENTS AND CAREGIVERS

ENSURING ACCESS AND AFFORDABILITY OF ORPHAN DISEASE DRUGS:

A CONTINUOUS EFFORT

SESSION 5

Huub SCHELLEKENSUniversiteit Utrecht

DISRUPTIVE TECHNOLOGIES FOR GENERATING AFFORDABLE BIOTHERAPEUTICS

Hans BÜLLERFair Medicine Initiative, Rotterdam

FAIR MEDICINES FOR ORPHAN DISEASES

Ana PALMA Sobi

THE IMPORTANCE OF FAST ACCESS TO ORPHAN DISEASE DRUGS

Kristof BONNARENSPharma.be

(SUPRA)NATIONAL INITIATIVES DRIVING RESEARCH AND DEVELOPMENT OF INNOVATIVE ORPHAN DISEASE THERAPEUTICS

Francis ARICKXVinciane KNAPPENBERG

RIZIVBeNeLuxA CONTRACTS AND MANAGED ENTRY AGREEMENTS

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