Strategy, regulation and opportunity for pharma, biotech and investors in rare diseases - World Orphan Drug Congress USA

The global orphan drug industry marketplace

the leading international orphan drug industry gathering

Download the brochure

Join our weekly newsletter:
 
 

About the event

The 4th annual World Orphan Drug Congress USA is where industry, government and payers come to develop new strategies and partnerships to advance orphan drug development and patient access.

This is the largest, global event that stakeholders go to annually.

The event is dedicated to fostering partnerships and collaboration. It is about expediting orphan drug development and articulating its value, from discovery to patient access, so that manufacturers are guaranteed full and speedy reimbursement.

check out the World Orphan Drug Congress USA Heroes Wall  ›

Unconventional networking

We realize that you not only attend our conferences for their world-class content, but also to network and build relationships.

From the mobile event app, to 1-2-1 partnering, to Speed Networking, and our Networking Manager, we help you make lots of valuable new connections.

And don't miss the Neverland themed cocktail party and gala dinner at the end of day one! Embrace your inner child, and enjoy good food and entertainment, while networking with your peers in a relaxed and informal setting.

more info on the dinner & cocktail party  ›
book early and save $$$  ›

Who will attend

Confirmed participants include:

  • Emil Kakkis, CEO and President, Ultragenyx Pharmaceutical
  • Leonard Lance, Congressman, US House of Representatives
  • Pat Furlong, Founder and President, Parent Project Muscular Dystrophy
  • Anne Pariser, Associate Director for Rare Diseases, CDER, FDA
  • Pranesh Chakraborty, Director, Newborn Screening Ontario
  • Joanna Horobin, CMO, Verastem
  • Jimmy Lin, Founder and President , Rare Genomics Institute

view full list of speakers
more info on Patient Zone  ›

rare disease patient zone    Discover early stage orphan drugs in the pitch & partner session    Networking opportunities with orphan drug stakeholders

Interactive roundtables

Roundtable sessions are designed to stimulate debate and discussion between attendees. The roundtable leader presents a common industry challenge, and open debate ensues!

Exchange ideas with your peers, and crowd-source expertise around the industry's most pressing challenges, including:

  • How to price orphan drugs
  • How to optimize clinical studies
  • How to identify real drug targets
  • How to access rare disease patients
  • How to improve market access
  • How to build patient relationships

book now to attend
view full agenda  ›

Pitch & Partner

Discover promising early-stage orphan drugs and meet the scientists developing them in our pitch & partner sessions.

Pitching companies include:

  • Promethera Biosciences
  • Mobius Therapeutics
  • Lumos Therapeutics
  • National Institutes of Health
  • Jade Therapeutics
  • RXi Pharmaceuticals
  • ...and more!

Start-ups and investors will participate in one-to-one meetings after the round of pitches is complete.

register now  ›
more info on Pitch & Partner  ›

8 reasons to attend

  1. Hear how industry forerunners like Emil Kakkis envision the next 5 years
  2. Acquire a better understanding of how policies like the MODDERN Cures Act will impact new rare disease therapies
  3. Explore new approaches to sustain pricing and ensure reimbursement
  4. Access emerging markets such as MENA
  5. Leverage newborn screening and genetic sequencing to discover disease targets
  6. Learn best practice in low-scale manufacturing and analytics
  7. Streamline your development timeline by meeting patient group partners
  8. Engage with over 600 orphan drug stakeholders across 20+ countries

download brochure  ›
register now

 

 

sponsors
Platinum Sponsor
Alexion Pharmaceuticals at World Orphan Drug Congress USA
Platinum Sponsor
Genzyme Corp at World Orphan Drug Congress USA
Platinum Sponsor
ProMetic Life Sciences Inc. at World Orphan Drug Congress USA
Gold Sponsor
Clinigen Pharma Ltd at World Orphan Drug Congress USA
Gold Sponsor
Medunik Canada at World Orphan Drug Congress USA
Gold Sponsor
Pfizer at World Orphan Drug Congress USA
Gold Sponsor
PPD at World Orphan Drug Congress USA
Gold Sponsor
Premier Research at World Orphan Drug Congress USA
Gold Sponsor
Shire at World Orphan Drug Congress USA
Pitch & Partner Sponsor
Longitude Capital at World Orphan Drug Congress USA
Silver Sponsor
Clinart MENA at World Orphan Drug Congress USA
Silver Sponsor
Dohmen at World Orphan Drug Congress USA
Silver Sponsor
DRS Suporte Med at World Orphan Drug Congress USA
Silver Sponsor
Mapi Group at World Orphan Drug Congress USA
Silver Sponsor
Omnicare Inc at World Orphan Drug Congress USA
Silver Sponsor
Oncoprod at World Orphan Drug Congress USA
Silver Sponsor
Quintiles at World Orphan Drug Congress USA
Silver Sponsor
Sarepta Therapeutics Inc at World Orphan Drug Congress USA
Silver Sponsor
Siren Interactive at World Orphan Drug Congress USA
Silver Sponsor
Sonexus Health at World Orphan Drug Congress USA
Associate Sponsor
The Assistance Fund at World Orphan Drug Congress USA
Associate Sponsor
Catalent Pharma Solutions at World Orphan Drug Congress USA
Associate Sponsor
Genpharm Services at World Orphan Drug Congress USA
Associate Sponsor
LapidusData LLC at World Orphan Drug Congress USA