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Orphan Drugs and Rare Diseases

Orphan Drugs and Rare Diseases

SMi is proud to announce the return of their 5th annual Orphan Drugs event to London on the 19th -20th October 2016!

The global orphan drug market totaled nearly $123 billion in 2014 and will continue to grow to reach nearly $191 billion by 2019.* With such growth and innovation witnessed in this field now is the best time to discuss and strategize the best route to market.

In the past year (2015) alone, the Food and Drug Administration have approved 21 new entities. The orphan drug market has seen a dramatic increase in pharmacologic cures for rare diseases, such as Gaucher’s disease (Cerezyme/Imiglucerase), atypical Hemolytic Uremic Syndrome (Soliris/Eculizumab) and Non-Hodgkin’s Lymphoma (Rituxan/Rituximab). Such entities have revolutionized medicine and paved the way for other drug candidates to enter this space. *

SMi’s 5th annual Orphan Drugs event will bring together leading industry professionals to discuss the latest regulatory developments, explore how to reduce costs and learn from the latest innovations in the orphan drug landscape.


Day 1
8:30
Registration & Coffee
9:00
Chairman's Opening Remarks
Tim Miller, President & CEO, Abeona Therapeutics
9:10
Market access for orphan drugs
Sheela Upadhyaya, Associate Director - Highly Specialised Technologies , National Institute for Health and Care Excellence
9:40
Regulatory challenges for orphan products: Focus on emerging markets
Natalie Thomas, Consultant, Clinical Network Services (UK) Ltd
10:10
Morning Coffee
10:30
A new business model for developing rare diseases treatments
Tony Hall, Therapeutic Area Head, Orphan Diseases, Mereo BioPharma
11:00
Development of orphan drugs to prevent, diagnose and treat rare diseases
Tim Miller, President & CEO, Abeona Therapeutics
11:30
Challenges with paediatric orphan drug development
Cecile De Coster, Associate Director, Regulatory Affairs, Alexion Pharma International SÓrl
12:00
Networking Lunch
13:00
Rare disease clinical trials: Challenges & opportunities
Bindhya Cariappa, Chief Scientific Officer & Executive Vice President for Turkey. MEA & APAC., ClinTec International Ltd
13:40
Orphan drug industry and venture capital – key factors for a successful partnership
Robert Karl, Partner, RBV Capital
14:10
Global orphan drug regulatory strategy
Aaron Barzey, CEO, ADB Medical
14:40
An alternative development path for rare disease therapeutics
Chris Adams, CEO, Cydan
15:10
Afternoon Tea
15:30
A case study of patient foundations and industry working together
Michelle Berg, Vice President, Patient Advocacy, Abeona Therapeutics
16:00
A patient group’s perspective on patient recruitment and retention
Oliver Timmis, CEO, AKU Society
16:30
Developing a new model to fund drug repurposing for rare diseases.
Rick Thompson, Scientific Officer, Findacure
17:00
Chairman’s Closing Remarks and Close of Day One
Tim Miller, President & CEO, Abeona Therapeutics
Day 2
8:30
Registration & Coffee
9:00
Chairman's Opening Remarks
Tim Miller, President & CEO, Abeona Therapeutics
9:10
Building a patient centric model for rare disease drug discovery
Tim Guilliams, Founder & CEO, Healx
9:40
Rare disease clinical research: Collaboration the key to success
Judith Ng-Cashin, Chief Scientific Officer, INC Research
10:20
Morning Coffee
10:40
The challenges in defending orphan products pricing
Ad Rietveld, Founder & Director, RJW & partners Ltd
11:10
Setting reimbursement strategies - How to establish a foundation for reimbursement
Nigel Nicholls, Director and Country Manager UK/Ireland, BioMarin Europe Ltd
11:40
Advancing therapies for Duchenne Muscular Dystrophy (DMD)
Richard Pye, Senior Director, Corporate Affairs & Communications, Summit Therapeutics
12:10
Networking Lunch
13:10
The rise of one-off gene and cell therapy treatments for rare diseases: is the future commercially viable?
Diego Ardigo, ATMP Project and Clinical Program Leader, Chiesi Farmaceutici SpA
13:40
Why Turkey should feature early in launch plans for orphan drugs
Gulce Belgin, Director, Proceutica
14:10
Afternoon Tea
14:30
Panel Discussion:How can partnerships improve the orphan drug field?
Tim Miller, President & CEO, Abeona Therapeutics
14:30
Panel Discussion:How can partnerships improve the orphan drug field?
Nigel Nicholls, Director and Country Manager UK/Ireland, BioMarin Europe Ltd
14:30
Panel Discussion:How can partnerships improve the orphan drug field?
Robert Karl, Partner, RBV Capital
14:30
Panel Discussion:How can partnerships improve the orphan drug field?
Daniel O'Connor, Medical Assessor, MHRA
15:00
Chairman’s Closing Remarks and Close of Day Two
Tim Miller, President & CEO, Abeona Therapeutics

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