Personalized Medicine – once seen as a threat to the Pharma Industry – is today viewed as a growth opportunity. In the future, the sale of pharmaceuticals must be redefined away from a bulk business as patients and treatments become individual and unique. Consequently, as the industry learns to deal with small populations, the development of orphan drugs becomes increasingly interesting.

In Europe, between 5,000 and 8,000 people suffer from a rare disease, most of them life-threatening or chronically debilitating. Orphan indications affect between 6% and 8% of the population in total. This is between 27 and 36 million people in the European Union who represent an interesting target group for the industry. However, the research, development, and marketing of orphan drugs must keep to strict guidelines.

This BIOCOM Event offered a unique opportunity to gain insight into the strategies of pharma and biotech companies. Leading experts in the field will shared their knowledge on how to successfully bring orphan drugs to the market. Representatives from biotech and pharmaceutical companies, researchers, business developers, clinical trial- and marketing experts, as well as investors, advisers and patent lawyers attended this high-level event in the standout location of the Spanish Embassy in Berlin.

Watch the conference video on Youtube.

Programme

9:00

Welcome Address

Rafael Dezcallar y Mazarredo, Spanish Ambassador
Dr. Hans-Rainer Jaenichen, Partner, Vossius & Partner



 

Keynote

Orphan Drugs to Combat the Patent Cliff

Maggie Shafmaster, SVP, Chief Patent Counsel, Genzyme Corp. (Boston)



 

NGOs

Programmes and organisations promoting R&D in Orphan Drugs

Prof. Dr. Manfred Stuhrmann-Spangenberg, Hannover Medical School, German Coordinator Orpha.net



 

Drug Development

Prosensa and the GSK Experience

Luc Dochez, CBO, Prosensa Therapeutics BV



10:15

Coffee Break

10:45

Research

Using the Experience of Online Expert Communities

Fabio Cavalli, CEO, Mondobiotech AG

 

 

Clinical Trials

Pharmacogenetics-based Patient Stratification

Prof. Ivar Roots, Charité - Universitätsmedizin Berlin



 

Development

Orphan Indications – an Entrance for Biotechs

Sascha Tillmanns, Medical Director, SuppreMol GmbH



 

Patents

Patenting in Theranostics - Drug Diagnostic Combinations"

Dr. Christian Kilger, Patent Attorney, Vossius & Partner



12:00

Lunch

13:00

Focus on Spain

Research on Rare Diseases

Francesc Palau, Director, CIBERER



 

Focus on Spain

Nanomedical Approach for Orphan Therapies

Dr. Simo Schwartz, Coordinator of CIBBIM-Nanomedicin of Valle de Hebron Hospital



 

Focus on Spain

Genetically Corrected Blood Cells for Fanconi Anemia

Juan Antonio Bueren, Group Leader, CIEMAT



 

Focus on Spain

Orphan Therapies in Oncology – The Aplidin Example

Ana Irigaray, Regulatory Affairs Group Head PharmaMar



 

Regulatory

A Special Case: Getting Orphan Drugs Approved

Dr. Jürgen Regenold, CEO, Regulanet



14:30

Coffee Break

15:00

Marketing

Early Access to Orphan Drugs – the rationale, challenges and options available

Mark Corbett, Global Strategic Planning Director, Clinigen Group



 

Marketing

Charity Organisations as a Partner

Jasmina Begovic, Senior Specialist Market Access & Charitable programs,
Genzyme Corp.



 

Marketing

Physicians Academy – Educating the Market

Samantha Parker, Manager Orphan Academy and External Affairs,
Orphan-Europe



16:00

Discussion, Guided Embassy Tour, Cocktail Reception

18:00

End

When & where

CONTACT & INFORMATION

 

 

Uta Holmer

Dipl.-Volkswirtin

Veranstaltungsmanagement

 

 

Telefon: +49-30-264921-53

events[at]biocom.de