World Orphan Drug Congress USA 2022

July 11-13, 2022
Hynes Center, Boston, MA

The World’s Most Important Orphan Drug
and Rare Disease Event

Name: World Orphan Drug Congress USA 2022
Start: 2022-07-11T09:00-04:00
End: 2022-07-13T17:00-04:00
Location: Hynes Center

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Pre-Conference Day

Monday, July 11

1:00 - 4:00 pm

Day 1

Tuesday, July 12

8:30 am - 5:30 pm

Drinks Reception: 5:30 - 6:30 pm

Day 2

Wednesday, July 13

8:50 am - 4:30 pm

1,200+

Attendees

100+

Sponsors & Exhibitors

250+

Speakers

3

Days of Content

13

Themes of Content

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.

2022 Speakers

Peter Marks
Director, Center for Biologics Evaluation and Research (CBER)
FDA

Peter Marks speaking at World Orphan Drug Congress USA

Violeta Stoyanova-Beninska
Chair of Committee for Orphan Medicinal Products,
Medicines Evaluation Board
European Medicines Agency

Violeta Stoyanova-Beninska speaking at World Orphan Drug Congress USA

Yann Le Cam
Chief Executive Officer
Eurordis – Rare Diseases Europe

Yann Le Cam speaking at World Orphan Drug Congress USA

Rachel Yang
Secretary; Council Member,
RDI, Head of International Affairs
China Alliance For Rare Diseases (CHARD)

Rachel Yang speaking at World Orphan Drug Congress USA

Phillip Brooks
Deputy Director, Office of Rare Diseases Research
NCATS-NIH

Phillip Brooks speaking at World Orphan Drug Congress USA

Amy Nicole Nayar
Vice President of U.S. Patient Advocacy
& Government Affairs
Novartis

Amy Nicole Nayar speaking at World Orphan Drug Congress USA

Geoff MacKay
President & CEO,
AVROBIO

Geoff MacKay speaking at World Orphan Drug Congress USA

Christoph Glaetzer
Chief Global Value and Access Officer
Janssen Pharmaceutical Companies of Johnson & Johnson

Christoph Glaetzer speaking at World Orphan Drug Congress USA

Andrew Obenshain
Chief Executive Officer
bluebird bio

Andrew Obenshain speaking at World Orphan Drug Congress USA

Shannon Resetich
Global Franchise Head, Rare Diseases
Sanofi Genzyme

Shannon Resetich speaking at World Orphan Drug Congress USA

Emil Kakkis
Founder
President and Chief Executive Officer
Ultragenyx

Emil Kakkis speaking at World Orphan Drug Congress USA

Richa Poddar
Commercial Officer
Agios Pharmaceuticals

Richa Poddar speaking at World Orphan Drug Congress USA

Samarth Kulkarni
Chief Executive Officer
CRISPR Therapeutics

Samarth Kulkarni speaking at World Orphan Drug Congress USA

Amy Pott
Division Head of Gene Therapy
Commercial
Astellas Gene Therapies

Amy Pott speaking at World Orphan Drug Congress USA

Anne Kennedy
Chief of Policy and Advocacy
EveryLife Foundation for Rare Diseases

Anne Kennedy speaking at World Orphan Drug Congress USA

2022 Speakers

Peter Marks
Director, Center for Biologics Evaluation and Research (CBER)
FDA

Violeta Stoyanova-Beninska
Chair of Committee for Orphan Medicinal Products,
Medicines Evaluation Board
European Medicines Agency

Yann Le Cam
Chief Executive Officer
Eurordis – Rare Diseases Europe

Rachel Yang
Secretary; Council Member,
RDI, Head of International Affairs
China Alliance For Rare Diseases (CHARD)

Phillip Brooks
Deputy Director, Office of Rare Diseases Research
NCATS-NIH

Amy Nicole Nayar
Vice President of U.S. Patient Advocacy
& Government Affairs
Novartis

Geoff MacKay
President & CEO,
AVROBIO

Christoph Glaetzer
Chief Global Value and Access Officer
Janssen Pharmaceutical Companies of Johnson & Johnson

Andrew Obenshain
Chief Executive Officer
bluebird bio

Shannon Resetich
Global Franchise Head, Rare Diseases
Sanofi Genzyme

Emil Kakkis
Founder
President and Chief Executive Officer
Ultragenyx

Samarth Kulkarni
Chief Executive Officer
CRISPR Therapeutics

Richa Poddar
Chief Commercial Officer
Agios Pharmaceuticals

Amy Pott
Division Head of Gene Therapy
Commercial
Astellas Gene Therapies

Anne Kennedy
Chief of Policy and Advocacy
EveryLife Foundation for Rare Diseases

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?

BOOK YOUR PASS

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Companies that attend World Orphan Drug Congress USA

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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.

DOWNLOAD PROSPECTUS

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

The World’s Most Important Orphan Drug
and Rare Disease Event

TITLE SPONSOR

Pre-Conference Day

Monday, July 11

1:00 - 4:00 pm

Day 1

Tuesday, July 12

8:30 am - 5:30 pm

Drinks Reception: 5:30 - 6:30 pm

Day 2

Wednesday, July 13

8:50 am - 4:30 pm

1,200+

100+

250+

3

13

Breaking down silos and fostering collaboration to help rare disease patients

2022 Speakers

2022 Speakers

Our World-Class Conference Features The Following Themes

2022 Sponsors & Exhibitors

Companies that attend World Orphan Drug Congress USA

Sign Up for Event Updates

Sponsor the World Orphan Drug Congress USA

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

Claire Murphy
claire.murphy@terrapinn.com
t/ +1 646 619 1784

Marketing & Press Opportunities

Taylor Post
taylor.post@terrapinn.com
t/ +1 646 619 1785

Created by

The World’s Most Important Orphan Drugand Rare Disease Event

TITLE SPONSOR

Pre-Conference Day

Monday, July 11

1:00 - 4:00 pm

Day 1

Tuesday, July 12

8:30 am - 5:30 pm

Drinks Reception: 5:30 - 6:30 pm

Day 2

Wednesday, July 13

8:50 am - 4:30 pm

1,200+

100+

250+

3

13

Breaking down silos and fostering collaboration to help rare disease patients

2022 Speakers

2022 Speakers

Our World-Class Conference Features The Following Themes

2022 Sponsors & Exhibitors

Companies that attend World Orphan Drug Congress USA

Sign Up for Event Updates

Sponsor the World Orphan Drug Congress USA

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

Claire Murphy claire.murphy@terrapinn.com t/ +1 646 619 1784

Marketing & Press Opportunities

Taylor Post taylor.post@terrapinn.com t/ +1 646 619 1785

Created by

The World’s Most Important Orphan Drug
and Rare Disease Event

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

Claire Murphy
claire.murphy@terrapinn.com
t/ +1 646 619 1784

Taylor Post
taylor.post@terrapinn.com
t/ +1 646 619 1785