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World Orphan Drug Congress USA 2024 earlybird

 

Breaking down silos and fostering collaboration to help rare disease patients

The World Orphan Drug Congress brings together leading pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors and solution providers. The conference is a place to meet and brainstorm ways to advance orphan drug development and improve access to life-saving therapies.


Join us for three days in Boston for the most comprehensive program and inclusive gathering of rare disease stakeholders.

300+

Speakers

2,000+

Attendees

100+

Exhibitors

16

Streams of Content

2024 Speakers

 

 

Our World-Class Conference Features The Following Themes

patient data

PATIENT DATA

With so few patients, patient data/real-world-data (RWD/RWE) is the key element to advancing the rare disease space. What are best practices right now for data collection, ownership and sharing to benefit rare disease diagnosis, drug development and access.

next generation therapies

NEXT GENERATION THERAPIES

The rare disease space is a breeding ground for innovative approaches in drug development and platform technologies. What are the products and approaches that will be making headlines in 5-10 years from now?

payers

PAYERS

US-based insurers and single payer agencies around the world have a lot of impact of access and reimbursement for orphan products. Hear from top US and international payers on their strategies for orphan drugs.

clinical development & regulatory

CLINICAL DEVELOPMENT & REGULATORY

Understanding the regulatory and clinical landscape for rare disease drug development. How can companies, regulators and patient advocates work together to further patient voice, real world evidence, and decentralized clinical trials.

commercial

COMMERCIAL

What are the best commercial strategies for orphan products? How can companies bring products to new markets, build more efficient commercial operations, and develop effective launch strategies?

rare disease advocacy world

RARE DISEASE ADVOCACY WORLD

It all starts and ends with patients. So what are the challenges facing rare disease patients and patient organizations today? How can rare disease patient groups rethink funding strategies, partnerships with industry and advance policies around access?

global marketing & patient access

GLOBAL MARKET & PATIENT ACCESS

Access is often the crucial and challenges last step for rare disease patients and developers. How can all stakeholders work together to ensure rare disease patients have access to appropriate treatments globally.

pricing & reimbursement

PRICING & REIMBURSEMENT

Pricing orphan products, especially one-time treatments, is complex and a key element to ensuring timely access for rare disease patients. What are the current best practices for pricing orphan products, negotiating innovative contracts and working with payers around the world?

pitch & partner

PITCH & PARTNER

The orphan drug space is booming with innovation, M&A activity and partnerships. We play match maker for up and coming biotechs in the rare space and investors & pharma partners looking for their next blockbuster.

advanced therapies manufacturing

ADVANCED THERAPIES - MANUFACTURING

What are the current roadblocks in manufacturing cell and gene therapies and how can companies efficiently scale-up for commercial production?

advanced therapies clinical development

ADVANCED THERAPIES - CLINICAL DEVELOPMENT

The regulatory and clinical development landscape is ever evolving for cell and gene therapies. How are companies ensuring continued development of these potentially transformative products and what clinical hurdles remain?

rare oncology

RARE ONCOLOGY

As the rare cancer field evolves and diagnosis becomes more precise, how can all stakeholders work together to advance drug development for rare cancer patients?

advanced therapies commercialization

ADVANCED THERAPIES - COMMERCIALIZATION

Rare disease patients are uniquely positioned to benefit from the advancements of cell and gene therapies. What are the best strategies for commercializing these innovative products and how do health systems need to evolve to accommodate patient access and reimbursement?

digital health & artificial intelligence

DIGITAL HEALTH & ARTIFICIAL INTELLIGENCE

Approaches in AI and digital health span the full development life cycle of drug development and commercialization. What are the best approaches to implementing these technologies in patient finding, clinical development and commercialization for rare diseases?

diagonis

DIAGNOSIS

It takes an average of seven years for an accurate diagnosis. How can all stakeholders broaden access to Newborn Screening, Whole Genome Sequencing and Genetic testing to shorten the diagnostic odyssey for rare disease patients?

discovery

DISCOVERY

Technologies and processes are ever. How is the growing access to data, and use of AI and machine learning impacting drug discovery in the rare disease space?
 

 

Networking Is Our Sweet Spot

By App. By Facilitated Meetings. By RoundTables. By Hangin’ Out.


So you can: Exchange ideas, Build brand, Form friendships and partnerships, Grow your professional network, Explore the future, Uncover new opportunities.
 

2024 Sponsors & Exhibitors

 

  • Title Sponsor

    Sanofi at World Orphan Drug Congress USA 2024

    Diamond Sponsor

    Parexel at World Orphan Drug Congress USA 2024
  • Diamond Sponsor

    Eversana at World Orphan Drug Congress USA 2024

    Diamond Sponsor

    Sciensus at World Orphan Drug Congress USA 2024
  • Platinum Sponsor

    Pfizer at World Orphan Drug Congress USA 2024

    Platinum Sponsor

    Premier Research at World Orphan Drug Congress USA 2024
  • Platinum Sponsor

    Red Nucleus at World Orphan Drug Congress USA 2024

    Gold Sponsor

    Allucent at World Orphan Drug Congress USA 2024
  • Gold Sponsor

    ICON PLC at World Orphan Drug Congress USA 2024

    Gold Sponsor

    Halloran Consulting Group at World Orphan Drug Congress USA 2024
  • Silver Sponsor

    Alethium Health at World Orphan Drug Congress USA 2024

    Silver Sponsor

    BlueGene Search at World Orphan Drug Congress USA 2024
  • Silver Sponsor

    HEOR Strategies at World Orphan Drug Congress USA 2024

    Gold Sponsor

    Lannett CDMO at World Orphan Drug Congress USA 2024
  • Gold Sponsor

    P2P Syncro at World Orphan Drug Congress USA 2024

    Gold Sponsor

    PCM Trials at World Orphan Drug Congress USA 2024
  • Gold Sponsor

    Splash Clinical at World Orphan Drug Congress USA 2024

    Gold Sponsor

    TREND Community at World Orphan Drug Congress USA 2024
  • Gold Sponsor

    Worldwide Clinical Trials at World Orphan Drug Congress USA 2024

    Gold Sponsor

    York Health Economics Consortium at World Orphan Drug Congress USA 2024
  • Silver Sponsor

    Anovo at World Orphan Drug Congress USA 2024

    Silver Sponsor

    Costello Medical at World Orphan Drug Congress USA 2024
  • Silver Sponsor

    Orphan Drug Consulting at World Orphan Drug Congress USA 2024

    Silver Sponsor

    PharmaBlue at World Orphan Drug Congress USA 2024
  • Silver Sponsor

    Scout Clinical at World Orphan Drug Congress USA 2024

    Silver Sponsor

    Veristat at World Orphan Drug Congress USA 2024
  • Exhibitor

    Caidya at World Orphan Drug Congress USA 2024

    Exhibitor

    CENTOGENE at World Orphan Drug Congress USA 2024
  • Exhibitor

    ExperTrials at World Orphan Drug Congress USA 2024

    Exhibitor

    Genome Medical at World Orphan Drug Congress USA 2024
  • Exhibitor

    PDC CRO at World Orphan Drug Congress USA 2024

    Exhibitor

    Rare Disease Research, LLC at World Orphan Drug Congress USA 2024
  • Exhibitor

    Rare Patient Voice, LLC at World Orphan Drug Congress USA 2024

    Exhibitor

    TMC Pharma at World Orphan Drug Congress USA 2024
  • Start-Up

    HealthLumen at World Orphan Drug Congress USA 2024

    Start-Up

    jCyte Inc at World Orphan Drug Congress USA 2024
  • Start-Up

    Varient at World Orphan Drug Congress USA 2024

    Poster

    Adcum - association of urea and metabolic cycle disorders Peru at World Orphan Drug Congress USA 2024
  • Poster

    Catailyst Inc at World Orphan Drug Congress USA 2024

    Poster

    Fibrous Dysplasia Foundation at World Orphan Drug Congress USA 2024
  • Poster

    JEEVA INFORMATICS SOLUTIONS INC at World Orphan Drug Congress USA 2024

 

Sponsor the World Orphan Drug Congress USA

 

 

Get your product or solution in front of the leaders in the rare disease industry — from pharma, biotechs, governments, payers, investors and patient/patient advocates.


The World Orphan Drug Congress USA is a place where innovation and expertise is showcased, solutions are found and learning is done.

 

pharma, biotechs, governments, payers, investors and patient patient advocates.

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Companies that attend World Orphan Drug Congress USA

 

 
World Orphan Drug Congress USA
"If there is one event you want to go to as a stakeholder in rare diseases, it is the World Orphan Drug Congress USA. This conference has helped to define the world of rare diseases, it’s not just a leading event, it has actually helped create the future for rare diseases"

President And Chief Executive Officer, Canadian Organization For Rare Disorders
World Orphan Drug Congress USA
"The support, kindness, courtesy and interest shown by WODC Team in our company’s drug development program for orphan disease glioblastoma, to aid us to secure funding to run registration trials"

Chief Executive Officer, Tactical Therapeutics Inc

New Location:
Boston Convention & Exhibition Center
Boston, MA

April 23-25, 2024

 

Get Involved At World Orphan Drug Congress USA

 

To Sponsor Or Exhibit

 
 

Justin Franks
justin.franks@terrapinn.com
t/ +1 914 819 3506

 

 

 

To Speak

 
 

Gina McHugh
gina.mchugh@terrapinn.com
t/ +1 646 619 1775

 

 

 

Marketing & Press Opportunities

 
 

Kaylie Brogan
kaylie.brogan@terrapinn.com