MarketsandMarkets Digital Event on Orphan Drugs and Rare Diseases( Time Zone - EASTERN STANDARD TIME (EST)

9 - 10 July 2020

EVENT OVERVIEW

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 9th -10th July 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

  • C-Level Executives, VPs/Directors, Heads, Professors, Associate professors (Biology, Biochemistry), Researchers from Pharma/Therapeutics / Biopharma and Universities- Rare diseases department
  • Patient Advocacy groups

  • 100+ C-level executives, Directors/VPs, Heads experts working on rare disease attending the event
  • 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

08:30 - 08:55

Welcome note from MarketsandMarkets

08:55 - 09:00

Opening remarks from the Chairman

Christine Von Raesfeld

Christine Von Raesfeld, Founder/CEO, People with Empathy

09:00 - 09:10

THE GROWTH OF ORPHAN DRUGS FOR RARE DISEASES

Keynote Presentation - Rare Diseases Research from 1983-2020 – Opportunities Then and Now

Stephen Groft

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

09:10 - 09:35

A patient-centric research paradigm for rare disease drug development

Scott Schliebner

Scott Schliebner , Senior Vice President, Center for Rare Diseases , PRA Health Sciences

09:35 - 10:05

EveryBODY is Unique: a patient perspective in the world of pharmacogenomics.

Christine Von Raesfeld

Christine Von Raesfeld, Founder/CEO, People with Empathy

10:05 - 10:30

Case Studies from Engaged Rare Disease Communities

Dan Donovan

Dan Donovan , Founder & CEO , rareLife solutions

10:30 - 10:45

CLASSIFICATION BY TYPES OF RARE DISEASES

Rare Ocular Diseases and how to maximize a Rare Disease Business model in a big pharma

Stavros Ntogiakos

Stavros Ntogiakos, Regional Head of Rare Ocular Disease, Novartis

10:45 - 11:10

Occam’s razor and rare diseases

Dr. Michal Konstacky MD, PhD, MBA

Dr. Michal Konstacky MD, PhD, MBA , Global Medical Lead SHP 647 , Shire Pharmaceuticals

11:10 - 11:35

Enhancing Access to Developmental Medicine

Neil Watson

Neil Watson , Director, Global Business Development , Clinigen Group

11:35 - 12:05

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

Jonathan Morton PhD

Jonathan Morton PhD , Agency lead, Comradis Limited

12:05 - 12:35

Closing remarks from chairman

Christine Von Raesfeld

Christine Von Raesfeld, Founder/CEO, People with Empathy

12:35 - 12:40

One-to-One Networking Break

12:40 - 01:10

Online Registration

08:30 - 08:55

Welcome note from MarketsandMarkets

08:55 - 09:00

Opening remarks from the Chairman

Christine Von Raesfeld

Christine Von Raesfeld, Founder/CEO, People with Empathy

09:00 - 09:10

CHALLENGES AND OPPORTUNITIES IN TREATMENT OF RARE DISEASES

Keynote Presentation - Designing Strategic Early Access Programs for High Cost Therapeutics “Focusing on the challenges and solutions from a sponsor perspective”

Karen Frascello

Karen Frascello , Director, Global Medical Affairs, Early Access , Alnylam Pharmaceuticals

09:10 - 09:35

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)

09:35 - 10:00

The Value of Real-World Data from Global Expanded Access Programs for Rare Diseases

John Lagus

John Lagus, Managing Director of Business Development, Tanner Pharma Group

10:00 - 10:25

Neurometabolic Syndromes: Potential & Promise in Therapy Development

Gregory R Stewart

Gregory R Stewart, PhD- Senior Vice President, Vector Delivery and Optimization, Axovant Sciences

10:25 - 10:50

PROGRESS TOWARDS FIGHTING RARE DISEASES

Presentation by Evelina Paberžė- Chief Operating Officer, HRA Pharma Rare Diseases

Evelina Paberze

Evelina Paberze, Chief Operating Officer, HRA Pharma Rare Diseases

10:50 - 11:15

Rare diseases and digital health

Pedro Lendinez Ortega

Pedro Lendinez Ortega , Key Account Manager, en Recordati Rare Diseases

11:15 - 11:40

Closing remarks from the Chairman

Christine Von Raesfeld

Christine Von Raesfeld, Founder/CEO, People with Empathy

11:40 - 11:45

One-to-one Networking Meetings

11:45 - 12:15

End of Day 2

12:15 - 12:15

SPEAKERS

Scott Schliebner

Scott Schliebner

Senior Vice President, Center for Rare Diseases , PRA Health Sciences

Karen Frascello

Karen Frascello

Director, Global Medical Affairs, Early Access , Alnylam Pharmaceuticals

Dan Donovan

Dan Donovan

Founder & CEO , rareLife solutions

Dr. Michal Konstacky MD, PhD, MBA

Dr. Michal Konstacky MD, PhD, MBA

Global Medical Lead SHP 647 , Shire Pharmaceuticals

Stavros Ntogiakos

Stavros Ntogiakos

Regional Head of Rare Ocular Disease, Novartis

Pedro Lendinez Ortega

Pedro Lendinez Ortega

Key Account Manager, en Recordati Rare Diseases

Stephen Groft

Stephen Groft

Senior Advisor to the Director NCATS, , NIH

John Lagus

John Lagus

Managing Director of Business Development, Tanner Pharma Group

Evelina Paberze

Evelina Paberze

Chief Operating Officer, HRA Pharma Rare Diseases

Gregory R Stewart

Gregory R Stewart

PhD- Senior Vice President, Vector Delivery and Optimization, Axovant Sciences

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)

Neil Watson

Neil Watson

Director, Global Business Development , Clinigen Group

Jonathan Morton PhD

Jonathan Morton PhD

Agency lead, Comradis Limited

Christine Von Raesfeld

Christine Von Raesfeld

Founder/CEO, People with Empathy

SPONSORS

PARTNERS