2nd Edition MarketsandMarkets

Digital Event on Orphan Drugs and Rare Diseases (Time zone- Greenwich Mean Time (GMT)

3rd-4th December 2020

New medicine for orphan and Rare diseases has the capability to undoubtedly impact the lives of patients, where historically there were few or no treatments have been provided to control the sickness. The Orphan Drug Act (ODA) established in 1983 compelled producers to expand scale to treat uncommon/orphan diseases, and the orphan drug panorama has advanced drastically. Approvals for orphan drugs has doubled in the past decade, and there are over 500 applicants in the pipeline for orphan diseases.

MarketsandMarkets Digital Event on Orphan Drugs and Rare Diseases scheduled to be held on 3rd -4th December 2020 will provide valuable and necessary platform for uplifting knowledge at the forefront of drug analysis. Over the course of the conference, internationally renowned speakers can describe how their analysis journeys have developed the response towards up to date challenges. The attending specialists and trade partners will offer an incredible networking expertise.
 

This conference will gather scientists, researchers, entrepreneurs, academicians, medical officers, CEO’s, CSO’s and technologists from everywhere around the globe to discuss on the newest scientific advances within the field of rare diseases & orphan drugs that facilitate to form current and future challenges in drug analysis.

WHAT TO EXPECT

  • The growth of orphan drugs for rare diseases
  • Challenges and opportunities in treatment
  • Improving clinical trials for rare diseases
  • Classification by types of rare diseases and therapy options
  • Commercialization and pricing strategies for orphan drugs
  • Discussion on current regulatory measures for manufacturing orphan drugs

  • 100+ experts attending
  • Participate in constructive discussion with leading industry experts and decision-makers from pharmaceutical manufacturers who will tackle the most important issues in Rare disease diagnosis.
  • Best opportunity to showcase your work and research in front of industry leaders.
  • Develop your skills by learning new competencies and acquire knowledge from pioneers all around the world.

CONFERENCE AGENDA

Online Registration

13:30 - 13:50

Welcome note from MarketsandMarkets

13:50 - 13:55

Opening remarks from the Chairman

13:55 - 14:00

THE GROWTH OF ORPHAN DRUGS FOR RARE DISEASES

Keynote Presentation - Rare Disease Research: The Changing Landscape

Stephen Groft

Stephen Groft , Senior Advisor to the Director NCATS, , NIH

14:00 - 14:30

Keynote: Orphan Drug Designation and Market Exclusivity

 Dairine Dempsey

Dairine Dempsey, Non-Executive Director, Omnispirant Therapeutics

14:30 - 15:00

Why can’t anyone find the undiagnosed 50% of patients? AI-based identification to drive rare disease diagnosis

Christopher Rudolf

Christopher Rudolf, Founder & CEO, Volv Global

15:00 - 15:30

Realizing the promise of Gene Therapy

Sebastian Waldmeier

Sebastian Waldmeier, Brand Director Gene Therapy/Luxturna, Region Europe, Novartis

15:30 - 16:00

Networking Break | One-on-One Meetings

16:00 - 16:20

Making the Connection - in Rare Diseases

Alex Monaghan

Alex Monaghan, Senior Director, Business Partnerships , Within3

16:20 - 16:50

CLASSIFICATION BY TYPES OF RARE DISEASES

Lesson learned during start-up of clinical study during Covid-19

Mariusz Olejniczak

Mariusz Olejniczak, Chief Executive Officer, WPD Pharmaceuticals Inc

16:50 - 17:20

Closing remarks from Chairman- End of Day 1

17:20 - 17:20

Online Registration

13:30 - 13:50

Welcome note from MarketsandMarkets

13:50 - 13:55

Opening remarks from the Chairman

13:55 - 14:00

CHALLENGES AND OPPORTUNITIES IN TREATMENT OF RARE DISEASES

Keynote Presentation: Improving clinical trials for rare diseases

Monica Vinhas de Souza, MD, PhD

Monica Vinhas de Souza, MD, PhD , Executive Member of the research and ethics committee, Hospital de Clínicas de Porto Alegre (HCPA)

14:00 - 14:30

Launching an Orphan Drug: Do’s And Don’ts

 David Garzon Lafuente

David Garzon Lafuente, Head Iberia Rare Diseases, Chiesi

14:30 - 15:00

Accelerating Diagnosis Implementing Patient Finding at Scale

Hadley Mahon

Hadley Mahon , Head of Commercial & Partnerships , Mendelian

15:00 - 15:20

Changing market access challenges for Orphan Drugs in Europe

Rudiger Schulze

Rudiger Schulze, Vice President and General Manager, Germany and Central / Eastern Europe , Ultragenyx Pharmaceutical Inc.

15:20 - 15:50

Closing remarks from the Chairman

15:50 - 15:55

End of Conference

15:55 - 15:55

SPEAKERS

Monica Vinhas de Souza, MD, PhD

Monica Vinhas de Souza, MD, PhD

Executive Member of the research and ethics committee, Hospital de Clínicas de Porto Alegre (HCPA)

Stephen Groft

Stephen Groft

Senior Advisor to the Director NCATS, , NIH

Sebastian Waldmeier

Sebastian Waldmeier

Brand Director Gene Therapy/Luxturna, Region Europe, Novartis

Kimon Doukoumetzidis

Kimon Doukoumetzidis

Region Europe Brand Director Gene Therapy – Luxturn, Novartis

 Dairine Dempsey

Dairine Dempsey

Non-Executive Director, Omnispirant Therapeutics

Alex Monaghan

Alex Monaghan

Senior Director, Business Partnerships , Within3

Rudiger Schulze

Rudiger Schulze

Vice President and General Manager, Germany and Central / Eastern Europe , Ultragenyx Pharmaceutical Inc.

 David Garzon Lafuente

David Garzon Lafuente

Head Iberia Rare Diseases, Chiesi

Hadley Mahon

Hadley Mahon

Head of Commercial & Partnerships , Mendelian

Christopher Rudolf

Christopher Rudolf

Founder & CEO, Volv Global

Mariusz Olejniczak

Mariusz Olejniczak

Chief Executive Officer, WPD Pharmaceuticals Inc

SPONSORS