Clinical, reimbursement & partnerships for the orphan drug industry

rare disease, orphan drug congress, clincial development, market access, partnershipsRare disease patient advocacy groups, payers, regulators and industry – Who will you make time to meet?

This year, the 3rd Annual World Orphan Drug Congress Europe has evolved to differentiate itself and provide the most comprehensive and focused event for the rare disease industry.

Wherever you are in your portfolio lifecycle and whatever your commercial objectives are for the coming year, there is a dedicated networking and learning track for you and your team.


Innovative trial design, faster clinical development, adaptive licensing, conditional approval for earlier regulatory success and clinical collaboration for all orphan drug developers & rare disease researchers

Meet patient advocacy groups, pharma, biotechs & regulatory officials

The Clinical Development and R&D track is focused to address the unique challenges of orphan drug clinical development faced by rare disease researchers striving to advance their orphan drug portfolio towards market authorisation and to the patients in need.

In this track you will:

· Learn how adaptive licensing can mean your orphan product gains approval at an earlier stage of development

· Learn how to integrate, evaluate and refine innovative approaches to your clinical trial design and generate powerful clinical data for a small patient population

· Hear how best to harness patient involvement during the clinical and regulatory procedure

· Uncover the reasons why some orphan drugs succeed and others do not during regulatory submission

· Discover how to transform a post marketing commitment constraint into an opportunity


Quick and early market access, harmonising Europe's fragmented reimbursement market, HTA for rare diseases, understanding the payer perspective and debating how to quantify the value of an orphan drug

Meet payers, reimbursement officials, HTA bodies, health economic and market access experts

The Market Access, Pricing & Reimbursement track is designed to constructively debate the growing reimbursement hurdle facing developers. Hear the priorities of payers, industry and patients alike when it comes to pricing an orphan drug, innovative strategies for gaining reimbursement and how to demonstrate the added value of your orphan drug in the context of new pricing models.

In this track you will:

· Gain crucial access to the reimbursement perspectives of France, Netherlands, Austria, Sweden, Italy, MEDEV and the European Commission

· Learn how HTA assesses relative value of orphan drugs and what are the current HTA requirements for different orphan drugs in different countries

· Hear how to engage and harness patient groups to drive reimbursement and market access

· Discover the up to date progress of the EU Transparency Directive in order to harmonise Europe's fragmented reimbursement landscape

· Learn from successful case studies where correct and compelling data was submitted to payers, demonstrating the value and creating a reimbursable treatment


Partnering strategies, sustainable orphan business models, innovative commercial strategies and access to funding for pharma & biotech business development executives, investors and patient advocacy groups

Meet potential industry partners, angel investors, VCs and patient advocacy groups

The Corporate Development & Partnerships track is dedicated to focus on new commercial partnerships, collaborative initiatives and portfolio expansion opportunities as a means to establish a leading and sustainable position within the orphan drug industry.

In this track you will:

· Understand when and why does a large company partner, how to get on the radar of a potential partner and what does a partnership look like at various stages of development

· Hear exclusively whether in hindsight, partnering with a large pharma has benefited orphan drug development

· Discover what the best strategic partnership looks like for you and exploit potential alternatives to drive forward the commercialisation of your orphan product

· Discover how patient groups, industry, academia and clinicians are collaborating to drive forward rare disease treatments

· Learn how some of the most innovative and high profile deals were achieved and how strategic alliances are formed based on your needs and their expertise

· Hear how VCs and Pharma Venture arms are viewing investments within the industry.

At this point, why not take a look at a some of last years presentations and discussions from David Meeker, CEO, Genzyme, Hans Schikan, CEO, Prosensa or Sharon Terry, CEO, Genetic Alliance. Registration is now open for the 2012 World Orphan Drug Congress and you can download the brochure here to view the speaker faculty and all three tracks in comprehensive detail.

Comments 1

Leave a Reply

Your email address will not be published. Required fields are marked *