3rd Edition MarketsandMarkets

Orphan Drugs and Rare Diseases Virtual Conference [Time Zone - Central European Time (CET)]

15th -16th March 2022

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.

The 3rd Annual MarketsandMarkets Orphan Drugs and Rare Diseases Conference Virtual Event is scheduled to be held on 15th -16th March 2022 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

  • Current Orphan Drugs landscape and implications of COVID19
  • Study worldwide patterns in uncommon infections and its advances in the remedial and demonstrative market
  • Finding innovative and alternative ways in funding the development of Orphan Drugs
  • How is FDA managing orphan drugs during the pandemic?
  • The growth of orphan drugs for rare diseases
  • Challenges and opportunities in treatment
  • Improving clinical trials for rare diseases

Academia & CEOs, Owners, Business Heads, VPs, Directors, Heads, Managers 

From the Departments of: 

  • Personalized & Innovative Medicine
  • External R&D Innovation
  • Regenerative Medicine
  • Rare and Ultra-Rare Diseases
  • Cell and Gene Therapy
  • Molecular Geneticist
  • Translational Science
  • Patient Advocacy Groups
  • Program Management
  • Public, Regulatory, Medical   Affairs
  • Patient Engagement
  • Market Access
  • Clinical Research Organizations
  • Health Economics Outcomes Research
  • Investments and Funding
  • Business Planning and Operations
  • Specialty Pharmacies

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 - ODD and Market Exclusivity

 Dairine Dempsey

Dairine Dempsey, Non-Executive Director, Omnispirant Therapeutics

14:00 - 14:30

Recent insights into Patient Access to Orphan Drugs in Switzerland

Andreas Uttenweiler

Andreas Uttenweiler, Head of Patient Value Access & Public Affairs, Takeda Pharmaceuticals

14:30 - 15:00

Clinical trials in the Covid pandemic. Executing our phase 2b global studies for IMR-687

Rahul Ballal

Rahul Ballal, President and Chief Executive Officer, IMARA Inc

15:00 - 15:30

Pre and Post COVID – what has changed in the rare disease clinical research landscape?

Davy Yeung

Davy Yeung,  Managing Director and Vice President of Operations, Tailored Clinical Research Solutions (TCRS)

15:30 - 16:00

Clinical research during crisis: Covid-19 and the war in Ukraine

Mariusz Olejniczak

Mariusz Olejniczak, Chief Executive Officer, WPD Pharmaceuticals Inc

16:00 - 16:30

Closing remarks from Chairman

16:30 - 16:35

  Networking Break | One-on-One Meetings End of Day 1

16:35 - 16:35

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

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.

14:00 - 14:30

Interrogating preclinical models of rare disease.

Shailesh Agarwal

Shailesh Agarwal, Assistant Professor of Surgery, Harvard Medical School, Partner, Mainspring Venture Partners

14:30 - 15:00

FINDING FABRY

Subir Roy

Subir Roy, Associate Director for Global Medical Affairs |Project Lead, MIT-Takeda Program on Fabry

15:00 - 15:30

Advocacy Engagement: Establishing Relationships in the Rare Disease Community

June Sanson

June Sanson, Senior Director, Patient Advocacy and Industry Relations, Acadia Pharmaceuticals Inc.

15:30 - 16:00

Closing remarks from the Chairman

16:00 - 16:05

Networking Break | One-on-One Meetings End of Conference

16:05 - 16:05

CONFERENCE SPEAKERS

ADVISORS

June Sanson

June Sanson

Senior Director, Patient Advocacy and Industry Relations, Acadia Pharmaceuticals Inc.

Rudiger Schulze

Rudiger Schulze

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

 Dairine Dempsey

Dairine Dempsey

Non-Executive Director, Omnispirant Therapeutics

SPEAKERS

June Sanson

June Sanson

Senior Director, Patient Advocacy and Industry Relations, Acadia Pharmaceuticals Inc.

Rudiger Schulze

Rudiger Schulze

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

 Dairine Dempsey

Dairine Dempsey

Non-Executive Director, Omnispirant Therapeutics

Mariusz Olejniczak

Mariusz Olejniczak

Chief Executive Officer, WPD Pharmaceuticals Inc

Andreas Uttenweiler

Andreas Uttenweiler

Head of Patient Value Access & Public Affairs, Takeda Pharmaceuticals

Shailesh Agarwal

Shailesh Agarwal

Assistant Professor of Surgery, Harvard Medical School, Partner, Mainspring Venture Partners

Subir Roy

Subir Roy

Associate Director for Global Medical Affairs |Project Lead, MIT-Takeda Program on Fabry

Davy Yeung

Davy Yeung

 Managing Director and Vice President of Operations, Tailored Clinical Research Solutions (TCRS)

SPONSORS