3rd Medical Affairs & Rare Disease

East Coast

November 5-6, 2020

Online Livestream

Event Overview

The new coronavirus has paralyzed our world, but older diseases remain as dangerous as ever. Rare disease patient needs have become even more challenging in an age of social distancing and fears of contagion.

To help serve the patients who are under the most risk and are least able to wait for society to reopen, Dynamic Global Events invites you to participate in the 3rd annual Medical Affairs & Rare Disease conference, this year held entirely streaming online. At a time when people are forced to stay apart, this forum helps you unite multi-stakeholder teams across functions, organizations, and therapeutic areas, in order to solve problems and identify rare disease solutions.

The Inn at Penn, a Hilton Hotel

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3600 Sansom St,
Philadelphia, PA 19104
(215) 222-0200

Hotel Website

Special Event Rate
Book Today!

About the Chairperson

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Dr. Matthew T. Roe has been a faculty member at Duke University School of Medine and the Duke Clinical Research Institute (DCRI) since 1999. As a senior investigator at the DCRI, he has been the principal investigator for numerous phase II-IV cardiovascular clinical trials and is currently a co-principal investigator for the ADAPTABLE trial—the first large-scale pragmatic trials being conducted in the PCORnet network. He has also served in leadership roles for several observational registries focusing upon the treatment and outcomes of patients with cardiovascular disease and has served as the Director of the DCRI Clinical Research Fellowship since 2010.

Featured Speakers

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Craig Lipset

Head of Clinical Innovation
Pfizer

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Jeff Kingsley

Chief Executive Officer
IACT Health 

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Dawn Anderson

Managing Director, Life Sciences Strategy and Operations, R&D Practice
Deloitte

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Ross Friedberg

General Counsel & Chief Privacy Officer
Doctor on Demand

Top Reasons to Attend

  1. Maps for adapting rare disease development and outreach to a post-COVID world
  2. Detailed strategies for ensuring patient centricity in rare disease clinical trials
  3. Tools for spotting and avoiding the most urgent mistakes that can strike a rare disease program
  4. Insights for building and learning from cross-disciplinary teams
  5. New visions for the medical affairs must-haves for both small and large pharma

Attendee Profile

This conference is designed for senior leaders and executives in pharmaceutical, biotech, and medical device companies responsible for:

  • Rare Disease
  • Orphan Drugs
  • Gene Therapy
  • Medical Affairs
  • Medical Messaging
  • MSL
  • Clinical Affairs
  • Patient Affairs
  • Patient Outreach
  • Patient Engagement
  • Patient Advocacy
  • Medical Publication
  • Planning
  • eClinical
  • Health Economics & Outcomes Research

Thursday

November 5th, 2020 | Day 1

CONFERENCE TIMES ARE EASTERN STANDARD TIME

9:00 AM - 9:15 AM

Chairperson’s Opening Remarks

Ioannis Tomazos - Senior Director, Global HEOR
ALEXION PHARMACEUTICALS

9:15 AM - 10:00 AM

Medical Affairs as the Strategic Driver Within an Organization

Isabel Firmino - Vice President, Head of Global Medical Affairs Therapeutic Areas
ALEXION PHARMACEUTICALS

10:00 AM - 10:45 AM

Spotlight on the Medical Communications Skills Needed for Rare Disease

Ana Bozas - Associate Director, Medical Communications
AKCEA THERAPEUTICS

10:45 AM - 11:00 AM

Networking Break

11:00 AM - 11:45 AM

Collaborative Public-Private Partnerships for Real World Evidence Collection and Expanded Access

Wendy White - Co-Founder
RAREITI

Natalie Douglas - Co-Founder
RAREITI

11:45 AM - 12:30 PM

Throw Away the Textbook and Listen to Your Patient: Monitoring a Disease of Exclusion

The biggest mistake providers make is following antiquated diagnostic criteria that neglects the real diversity of the phenotype.  For autoinflammatory disorders like Still’s Disease, current research lacks adequate tools for monitoring or quantifying both primary and secondary efficacy treatment endpoints.

  • Review advances in serology, targeted treatments, and key systemic findings
  • Target both pediatric and adult rheumatology and immunologists
  • Create earlier options for detection, research, and collaboration

Shalla Newton - Co-Founder
STILL’S DISEASE, THE 411

David Maher - Co-Founder
STILL’S DISEASE, THE 411

12:30 PM - 1:00 PM

Luncheon

1:00 PM - 1:45 PM

Doesn’t the Rare Disease Patient Matter? Reviewing Recent Experiences With NEJM, FDA, and NICE

Rare is different. We know that. We hear time and again how important the voice of the patient is in drug development and commercialization. We buy into it. We believe it. We work to make it happen. But then, when it comes to some important decision makers, sometimes it feels as though there is no consideration for the rare disease patient in the determinations they render. This presentation will review 3 very recent experiences in which the plight of the rare disease patient was not considered.

Dan Donovan - CEO
RARE LIFE SOLUTIONS

1:45 PM - 2:30 PM

Creating Strategic Plans for Cross Functional Collaboration and Growth

Andy Overmyer - Head of Global Business Operations
AKCEA THERAPEUTICS

2:30 PM - 3:00 PM

Break

3:00 PM - 3:45 PM

Education, Advocacy, and Technology to Accelerate Rare Disease Progress

Joan Chambers - Senior Director, Marketing and Outreach
CENTER FOR INFORMATION & STUDY ON CLINICAL RESEARCH PARTICIPATION

Harsha Rajasimha - Founder
INDOUSRARE

Friday

November 6th, 2020 | Day 2

9:00 AM - 9:15 AM

Chairperson’s Recap of Day One

Ioannis Tomazos - Senior Director, Global HEOR
ALEXION PHARMACEUTICALS

9:15 AM - 10:00 AM

HEOR to Determine the Value of Rare Disease Therapeutics

  • Determine value of rare disease therapies
  • Use Real World Evidence to show orphan drug value and inform HTA evaluations 
  • Center the role of patients in demonstrating the impact of orphan drugs 

Ioannis Tomazos - Senior Director, Global HEOR
ALEXION PHARMACEUTICALS

10:00 AM - 10:45 AM

Knowledge Transfer Challenges in Rare Disease

Shweta Rane - Director, Global Scientific Communications
ALEXION PHARMACEUTICALS

10:45 AM - 11:00 AM

Networking Break

11:00 AM - 11:45 AM

Expanding and Changing Role of MSLs in Rare Disease

Keri Kasun - Senior MSL, Rare Disease, Spinal Muscular Atrophy
BIOGEN

Michelle Gilmor - Senior Medical Director, Rare Disease
REGENXBIO

11:45 AM - 12:30 PM

Form Patient Advisory Panels to Reduce the Patient Burden to Participate

Patricia Roselle - Global Head, Patient Network Management
SANOFI

12:30 PM - 1:15 PM

Luncheon

1:15 PM - 2:00 PM

Leverage Medical Claims Data to Improve Patient Outcomes

Robert Consalvo - Director of Strategic Commercial Engagement
H1 INSIGHTS

2:00 PM - 2:45 PM

PANEL: Inspire the Creation and Maintenance of Patient Advocacy Boards

  • Ensure regular interaction with the chronic illness patient and advocate community
  • Streamline communication with those doing the research and inspire them to do more
  • Expand genetic research efforts while keeping genetic information confidential and safe

MODERATOR:
Deborah Vick - Patient Advocate; Owner
CRAFTING4ACAUSE

Michele Johnson - Patient Advocate; Author
THE PAIN FREE PA

Robin Powers - Patient Advocate

Charlene Hafer-Macko - Associate Professor, Neurology
UNIVERSITY OF MARYLAND SCHOOL OF MEDICINE

FEATURED SPEAKERS

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Ana Bozas

Akcea Therapeutics

Associate Director, Medical Communications

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Isabel Firmino

Alexion Pharmaceuticals, Inc.

Vice President, Head of Global Medical Affairs Therapeutic Areas

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Shweta Rane

Alexion Pharmaceuticals, Inc.

Director of Global Scientific Communications

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Joan A. Chambers

The Center for Information & Study on Clinical Research Participation

Senior Director, Marketing & Outreach

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Robert Consalvo

H1 INSIGHTS

Director of Strategic Commercial Engagement

Andy Overmyer

Andy Overmyer

Akcea Therapeutics

Head of Global Business Operations

Wendy White

Wendy White

GLOBAL GENES

Chairman

Co-Founder, RAREITI

Natalie Douglas

Natalie Douglas

GLOBAL GENES

Vice Chairman

Co-Founder, RAREITI

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Harsha K. Rajasimha

Jeeva Informatics Solutions Inc.

Founder and CEO

Founder Chairman, Indo US Organization for Rare Diseases

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Israel Gutierrez

Geron Corporation

Vice President, Pharmacovigilance and Drug Safety

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Dan Donovan

RARE LIFE SOLUTIONS

CEO

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David Maher

STILL’S DISEASE, THE 411

Co-Founder

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Keri Kasun

BIOGEN

Senior MSL, Rare Disease, Spinal Muscular Atrophy

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Shalla Newton

STILL’S DISEASE, THE 411

Co-Founder

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Patricia Roselle

SANOFI

Global Head, Patient Network Management

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Ioannos Tomazos

ALEXION

Senior Director, Global HEOR

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Deborah Vick

CRAFTING4ACAUSE

Patient Advocate; Owner

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May Orfali

PYC THERAPEUTICS

CMO

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Michele Johnson

THE PAIN FREE PA

Patient Advocate; Author

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Charlene Hafer-Macko

UNIVERSITY OF MARYLAND SCHOOL OF MEDICINE

Associate Professor, Neurology

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Michelle Gilmor

REGENXBIO

Senior Medical Director, Rare Disease

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Robin Powers

Patient Advocate

CONFERENCE REGISTRATION


LIVE STREAM ATTENDEE

STANDARD

Register Anytime

$896

Register 2 Attendees, Get 1 Complimentary Registration*!

Interested in sending groups of 7 or more? Contact us directly for pricing.

EVENT SPONSORS


Scarritt Group - Official Media Partner

OFFICIAL LIVESTREAM PROVIDER


SPONSOR THIS EVENT

Contact Amy Chapman at (561) 571-7687 or achapman@dgeconfs.com

MEDIA PARTNERS


PM360 - Media Partner

PREMIER MEDIA PARTNER