Rare Diseases 

Learn about new market access, regulatory and innovative developments in the world of orphan drugs & rare diseases

RARE DISEASES, Wednesday 18 March 2020

Registration & refreshments

Morning Plenary

Wendy Jory
09:00

Global implications of pricing & access developments in the US

09:20

Rerserved for Covance

Bharat Tewarie
09:40

Unleash the Algorithms: Critical Success Factors to Reinvent Our Role in Delivering Patient Value

10:00

Reserved for Life Science Dynamics

10:20

Speed Networking

10:40

Morning refreshments

David Thompson
11:20

Virtual Research: What it Is & What it’s Doing in the Real-World Setting

Thomas Mueller
11:40

Orphan Drugs, Gene Therapies, Post-Marketing-Data: Latest developments in German legal framework for drug assessment and reimbursement

round tables
12:00

Roundtables

Roundtable 10: Accelerating access to treatments for rare diseases
Kate Adcock

Kate Adcock, Director of Research and Innovation, The Muscular Dystrophy U.K.

Roundtable 11: Does pricing innovative drugs fairly improve your bottomline?
Michael Schroter

Michael Schroter, Partner, Viopas Partners

Roundtable 12: Exploring reimbursement and healthcare systems across the Middle East
Rita Shorjian

Rita Shorjian, Market Access Head - Near East And North West Africa, AbbVie

Roundtable 14: Indication based pricing- where are we?
Nicki Catterick

Nicki Catterick, Senior Director, Global Market Access And Pricing, Merck Group

Roundtable 15: Access and HTA considerations for Turkey, Kazakhstan and Ukraine
Oresta Piniazhko

Oresta Piniazhko, Director, HTA Department, Ministry of Health of Ukraine

Roundtable 16: Exploring the value from repurposing drugs: Who Benefits?
Douglas Foerster

Douglas Foerster, Head Market Access Europe And Row, Santhera

Roundtable 17: Working together with NGOs, Advocates, Pharma & Government to improve outcomes for families?
Edmund Lim

Edmund Lim, Co-Founder, We CARE Journey

Roundtable 18: Exploring challenges and opportunities across Africa on achieving new affordable strategies for Universal Health Coverage
Sagie Pillay

Sagie Pillay, Chief Operating Officer, Wits Health Consortium

Roundtable 19: Exploring innovative reimbursement and payment models for orphan drugs
Sebastian Soluch

Sebastian Soluch, Head of Market Access and Pricing, Europe, Stemline Therapeutics Inc

Roundtable 1: Efficacy to Effectiveness - E2E – translating the promise of clinical trials to the real world
Chris Pashos

Chris Pashos, Vice President Of Global Evidence Strategy, AbbVie

Roundtable 20: Branded combo-pricing: Where to next?
Yves Samel

Yves Samel, Market Access Strategy Leader And Global Pricing Manager, Bayer

Roundtable 21: Exploring Patient Access in Belgium: challenges and opportunities
Katrien Van Geyt

Katrien Van Geyt, Head Of Market Access, Belgium And Luxembourg, Takeda

Roundtable 22: Achieving greater collaboration with HTAs: Insights from EUnetHTA & Industry
Marcus Gaurdian

Marcus Gaurdian, Chief Operating Officer, EUnetHTA

Roundtable 23: Exploring orphan drugs in emerging markets
Lisette Kaskens

Lisette Kaskens, Senior Global Market Access Manager, Advanced Accelerator Applications

Roundtable 24: Developments in pricing and patient access for speciality treatments
Keith White

Keith White, Vice President and Head, Global Market Access, Corbus Pharmaceuticals

Roundtable 25: Reserved for Syneos Health
Roundtable 26: Reserved for Life Science Dynamics
Roundtable 27: Exploring reimbursement models: Working with external stakeholders to improve outcomes
Gomathi Kaliappan

Gomathi Kaliappan, Global Market Access (Health Systems Strategy, Value Recognition), Roche

Roundtable 28: Oncology drug market: a high-growth, high-price therapeutic area
Elena Lungu

Elena Lungu, Manager, Policy Development, Government of Canada (Canada)

Roundtable 29: Game-changing policies for game-changing innovations: what regulatory changes to ensure the development, diffusion and sustainability of new technologies
Julien Patris

Julien Patris, Associate Director Of Market Access And Policy For Europe And Canada, Alnylam

Roundtable 2: How do you include the patient perspective in a outcomes-based contract?
Jolanda Koenders

Jolanda Koenders, Head of Market Access Netherlands, Takeda

Roundtable 30: Pricing Competition from Launch to Loss of Exclusivity
Adam Plich

Adam Plich, Managing Director, Plich Advisory

Roundtable 32: Exploring access and pricing challenges for low to middle income countries
Giovanny Leon

Giovanny Leon, Pricing And Market Access Director For Latin America And Canada, Novartis AG

Roundtable 33: Access and pricing considerations for CAR-T development
Nuno Prego Ramos

Nuno Prego Ramos, CEO, CellmAbs

Roundtable 34: Exploring Free of Charge supply model for an orphan drug at public healthcare coverage in Argentina
Marcelo Sal

Marcelo Sal, Head of Market Access and Pricing, Argentina, PTC Therapeutics

Roundtable 6: A tale of two diseases: how orphan drugs can navigate NICE
Lindsay Weaver

Lindsay Weaver, Chief Executive Officer, Metabolic Support UK

Roundtable 7: Achieving positive reimbursement negotiations
Hassan Bruneo

Hassan Bruneo, Associate Director, Alnylam Switzerland Gmbh

Roundtable 8: The development of a patient advocate – How to find experienced and qualified patient advocates- The Continual Professional Development approach
Nadia Bodkin

Nadia Bodkin, Rare Disease Advocacy Professional, Rare Advocacy Movement

Roundtable 9: Using Health Economic Studies to Support Access and Value
Fabrizio Gianfrate

Fabrizio Gianfrate, Professor Of Health Economics, Market Access Advisor, Universities of Rome and Ferrara

12:50

Networking lunch

RARE DISEASES

Reimbursement & Value

Friedhelm Leverkus
RARE DISEASES
14:15

Registries and reimbursement for orphan drugs in Germany

RARE DISEASES
14:35

Slot reserved for DRG Group

Diann Potestio
RARE DISEASES
14:55

The continuing changing landscape in creating value beyond price for rare disease products in the US

RARE DISEASES

Sustainability of Orphan Drugs

Diego Ardigo
RARE DISEASES
15:20

Developing advanced therapies for rare diseases: is this sustainable?

RARE DISEASES
15:40

Sponsor presentation

Saira Jan
RARE DISEASES
16:00

Management and sustainability of Orphan Drugs in the US

16:20

Afternoon Refreshment Break

RARE DISEASES

Patient Engagement

RARE DISEASES
16:50

Chair’s remarks

Lara Pippo
RARE DISEASES
16:55

The patient’s journey for neurological diseases

Stefan Holmstrom
RARE DISEASES
17:15

Patient engagement within the HTA process for orphan drugs

Josie Godfrey
RARE DISEASES
17:35

The Hercules Project: Patient-led Access in Rare Diseases

17:55

Close of conference and networking drinks reception

last published: 11/Nov/19 14:55

RARE DISEASES, Thursday 19 March 2020

Morning Plenary

09:20

Sponsor presentation

Panel discussion
10:20

Morning refreshments

11:00

Roundtable 10: How to establish a market access department from scratch?

11:00

Roundtable 12: Exploring the international reference pricing landscape

11:00

Roundtable 13: Patient Assistance Programs and Access Structure in the US

11:00

Roundtable 14: Achieving better alignment amongst stakeholders regarding definitions and concepts of value in diagnostics

11:00

Roundtable 5: Exploring the evolution of the value framework to assess drugs in Italy

11:00

Roundtable 7: Exploring Biosimilars in the USA and latest developments

11:00

Roundtable 8: Accelerating Access to treatments for Rare Diseases

Rute Fernandes, Head Of Rare Diseases Of Europe And Canada, Takeda
11:00

Roundtable 9: How to involve patients in drug research and market access?

11:00

Roundtables

11:00

Roundtables 1: Innovation in Contracting

11:00

Roundtables 2: Achieving greater collaboration amongst payers, HTAs, pharma and patients to improve outcomes

11:00

Roundtables 3: Exploring innovative payment and evidence development models for rare diseases

11:00

Roundtables 4: How to improve the environment for value-added medicines in Europe

RARE DISEASES

Innovation in Rare Diseases

Ines Alves
RARE DISEASES
12:00

Chair’s remarks

Mark Macgregor
RARE DISEASES
12:05

Innovation in treating rare diseases: HTA approach in Scotland

Rute Fernandes
RARE DISEASES
12:25

Immediate Access to treatment for Rare Disease patients

Rute Fernandes, Head Of Rare Diseases Of Europe And Canada, Takeda
Simon Shohet
RARE DISEASES
12:45

Innovative payment and evidence development models for rare diseases

13:05

Networking Lunch

RARE DISEASES

Regulatory Landscape of Orphan Drugs

Ana Palma
RARE DISEASES
14:10

The incentives’ review by the EU Commission and its implication on OMPs

Durhane Wong-Rieger
RARE DISEASES
14:30

Developing a modern regulatory framework for orphan drugs

Durhane Wong-Rieger, President And Chief Executive Officer, Canadian Organization For Rare Disorders
Anna Bucsics
RARE DISEASES
14:50

Where do we go from here - bridging the gap from regulatory approval to market access using MoCA

15:10

Afternoon Refreshments

RARE DISEASES

Collaborating with Payers & Patients to improve Access

Natasa Zibelnik
RARE DISEASES
15:45

Working with stakeholders to improve access to rare disease treatments

RARE DISEASES
16:05

Bridging the Gap between Payers, Patients & the Advocates Navigating Access to Care

Peter Rutherford
RARE DISEASES
16:25

Using RWE and patient advocacy to ensure patient access in rare disease drug launch

RARE DISEASES
16:45

Chair’s Closing Remarks

last published: 11/Nov/19 14:55

Contact us

To sponsor or exhibit:
Edmond Rama
+44 (0)207 092 1052

edmond.rama@terrapinn.com

To speak:
Chris Shanks
+44 (0)207 092 1151

chris.shanks@terrapinn.com