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Conference on Rare Disease and Orphan Drugs

1st and 2nd October
10+ Speakers
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Event   Details

The orphan drug market is continuously growing over the years now at twice the growth rate of overall prescription drug market due to the arising interest from pharmaceutical companies for underlying benefits and involvement of many smaller biotechnology start-up companies who are primarily focused on rare diseases. However still there are lot of attention that needs to be given since the ratio of one having rare disease is 1 in 2000 leading to the lack of knowledge about disease mechanisms, along with FDA/Marketing approvals which take years.

The goal of this Conference is uniting all well-known clinical researchers, biotech professionals, Medical Advisories, Academicians and decision makers to discuss possible ways to accelerate orphan drug development and access to rare disease patients, including the introduction of recent technologies and products to help aid the access of orphan drugs.

In the pioneer edition of DBMR Conference on Rare Disease and Orphan Drugs come join us in this enriching knowledge fest where experts will speak on new technologies and market dynamics perceptions. Together let’s move ahead in the future of medical science and technology.

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Key Highlights

Key Highlights

Who Should Attend?

Who Should Attend?

Why Attend?

Why Attend?

Our Speakers

Conference Program

8:00 AM EST

Registration (Telephonic And Virtual)

8:30 AM EST

Introduction To DBMR

9:00 AM EST

the Rare Disease Community for the Long Haul

Dan Donovan

Founder & CEO - rareLife solutions

10:00 AM EST

Rethinking our approach to rare disease communications during COVID-19 and beyond

Jonathan Morton

Rare Disease Consultant - AMICULUM
Agency Lead - Comradis Limited

11:15 AM EST

Every BODY is unique: A patient perspective into precision medicine

Christine Von Raesfeld

Founder and CEO - People with Empathy
Technical Expert Panelist - Centers for Medicare & Medicaid Services

12:30 PM EST

Advocacy and the need to hear the rare disease patients voice within strategic efforts

Lydia L. Seiders

Patient Education Council Member Aplastic Anemia and MDS International Foundation
Advisor - Liv4TheCure
Advisor - Rare Disease Ghana Initiative

1:45 PM EST

A Case Study (Epidiolex) Demonstrating How to Use Special FDA Designations to Efficiently Reach the US Market

Kelly Austin

Founder, Regulatory Specialist (ODD, Fast Track, Breakthrough, RMAT)
KLA Breakthrough Consulting

3:00 PM EST

The Future of Rare Diseases: Developing and Accessing the Next Generation of Therapies

Rich Horgan

Founder and President - Cure Rare Disease

4:15 PM EST

Federated data solutions for Accessing rare genomes across borders

Alice Rathjen

Co-Founder and CEO - DNA Compass

5:15 PM EST

How to Commercialize an Orphan Drug

6:15 PM EST

Rare vs. Common Diseases

7:00 PM EST

DBMR Quantum Leap Awards

8:30 AM EST


9:30 AM EST

Challenges being faced by Orphan drug development Companies

10:00 AM EST

Succeeding with Star Wars Medicine - Translating the science and value of Gene Therapies

Gavin Jones

Director of Rare Disease - OPEN Health

11:15 AM EST

Gene Therapy, a New Paradigm in Ocular Rare Diseases

Hector De la Riva

Commercial Lead Ocular Rare Disease – Gene Therapy

12:30 PM EST

Treatment and Advanced Therapies for Rare Diseases

Shahar Keinan

CEO and Co-Founder - POLARISqb

1:45 PM EST

Social Entrepreneurship in Rare Diseases: It takes concerted efforts between technology and advocacy to accelerate therapies for rare diseases

Harsha K Rajasimha

Founder and CEO - Jeeva Informatics Solutions
Founder and Chairman IndoUSrare
Member of the Board of Advisors - Synergy

3:00 PM EST

Patient involvement in drug development for rare diseases

Diana Stefani-Hunyady

Head of Global Medical Affairs - Viela Bio
Co-Founder - Life Key Sciences LLC

4:15 PM EST

Examining industry strategies for leveraging patient data and AI to advance rare disease patient identification, R&D, and commercialization

5:15 PM EST

Regulatory Framework for Drugs for Rare Diseases

6:15 PM EST

Rare Mental and Behavioural Disorders

7:00 PM EST

DBMR Closing Remarks

Gold Sponsor

rareLife solutions

Media Partner

Healthcare Sales and Marketing



Speaking Partner

DNA Compass

KLA Breakthrough Consulting


Cure Rare Disease

Jeeva Informatics Solutions


People with empathy

Association Member