http://www.raredr.com/news/gg-summit-agenda
What to Expect at the Global Genes Rare Disease Patient Advocacy Summit

RDR Staff

The Global Genes Patient Advocacy Summit is next week and we at RDR are thrilled to be attending and sharing some of content with our audience. Last year we interviewed dozens of patient advocates and we plan to interview even more this year.
 
The two day summit will include a who’s who of the rare disease community and will provide audiences with tools to improve how advocacy groups and patients can work with the science, clinical, pharmaceutical, and regulatory communities to advance their programs.
 
Below are some of the key presentations, ‘how to’ presentation, and track sessions being presented over the 2 days.
 
For a complete schedule of the summit, click here.
 

THURSDAY, SEPTEMBER 22, 2016

KEY PRESENTATIONS
Welcome & Meeting Kick Off
Amanda Knitter, Director of Patient Advocacy, Global Genes
 
Opening Keynote
Rare Disease in the 21st Century - The Look Ahead - Times are Changing and You Need a Plan
 
Medical Scientific Advisory Board Presents:
Game Changing Science, Technology and Clinical Care - What Patients Should Expect!
Speakers: Peter Goodhand,  Anthony Philippakis, Alvin Shih, Ryan Taft 
 
Update from the National Teen Advocacy Launch
Speakers: Shira Strongin 
 
Value, Pricing and Costs of Rare Disease Therapies
One Minute Video Presentation- Pfizer. Followed by: A look at drug pricing, the challenges and the real impact on patients. Panel discussion.
Speakers: Anne Bruns, Debra Miller, Doug Paul 
 
‘HOW TO’ PRESENTATIONS
Storytelling: Press and Public Speaking, 2) Advocating for Insurance Coverage: Medically Necessary Foods
Speakers: Nicole Dreyer-Gavin, Jennifer Payne, Chris Smith 
 
Effectively Engaging Teens in Rare Disease Advocacy, 2) Foundations: How to Write and Apply for a Grant
Speakers: Lisa Schoyer,  Shira Strongin 
 
Designing a Trust for Rare Families, 2) How to Design a Scientific Conference
Speakers: Timothy O'Connor,  Lisa Schill 
 
Working with Schools: Advocating for your Child's Individualized Education Plan
Speakers: Piper Paul 
 
‘TRACKS’
CARE & SUPPORT
Caring for Yourself as Caregiver
Speakers: Kathleen Kelly, C. Grace Whiting 
 
PATIENTS ROLE IN DRUG DISCOVERY: PRE-CLINICAL TOOLS
Natural History Clinical Trials. Brought to you by PRA Health Sciences.
Speakers: Mousumi Bose, Melissa Bryce Gamble 
 
WHAT YOU NEED TO BUILD AS A DISEASE COMMUNITY
Building an Engaged Patient Community
Speakers: Sue Khan, Blake Shewey
 
SCIENCE & TECHNOLOGY
Using Sequencing to Understand Rare Idiopathic Disorders
Speakers: Caroline Loewy,  Jeanette McCarthy 
 
CARE & SUPPORT
Transitioning Care: Pediatric to Adult Healthcare Providers
Speakers: Amy Miller 
 
PATIENTS ROLE IN DRUG DISCOVERY: PRE-CLINICAL TOOLS
Developing and Validating Biomarkers
Speakers: Isabelle Lousada, Douglas Macdonald 
 
WHAT YOU NEED TO BUILD A DISEASE COMMUNITY
Partnering with Pharmaceutical Companies in a Changing Landscape: A Discussion on How the Relationship is Evolving Including a Real World Case Study from IFOPA
Speakers: Betsy Bogard, Susan Stein 
 
SCIENCE & TECHNOLOGY
Gene Sequencing: Not Just For Diagnosis
Speakers: Tamar Grossman, PhD,  Luke Hickey 
 
CARE & SUPPORT
Building a Clinical Care Team and Why it Matters
Speakers: Kelly Ranallo,  Jodi Vento 
 
PATIENTS ROLE IN DRUG DISCOVERY: PRE-CLINICAL TOOLS
First Steps in Research and Drug Development: Animal Models for Rare Disease Research
Speakers: Ethan Perlstein 
 
WHAT YOU NEED TO BUILD A DISEASE COMMUNITY
Shared Data Sets: Importance of Having Data and Making it Available to Share
Speakers: Scott Sutherland,  Jennifer Thornton 
 
SCIENCE & TECHNOLOGY
New Approaches in Drug Development: RNA-Based Therapeutics
Speakers: Marc Riedl, MD 
 
CARE & SUPPORT
Navigating Insurance and State Resources
Speakers: Sue DeFrancesco,  Marie Lamont 
 
PATIENTS ROLE IN DRUG DISCOVERY: PRE-CLINICAL TOOLS
Biobanking: A Necessity- The What, When, Why, and How-To's
Speakers: Deborah Requesens 
 
WHAT YOU NEED TO BUILD A DISEASE COMMUNITY
Building Your Scientific Team
Speakers: Christina Waters,  Monica Weldon 
 
SCIENCE & TECHNOLOGY
ARM Presents: Gene Therapy 101- CRISPR/cas9 and Gene Editing for Rare Disease
Speakers: Morrie Ruffin 
 

FRIDAY, SEPTEMBER 23, 2016

KEY PRESENTATIONS
A Call to ACTION for #CuresNOW: Change Policy, Save Lives
Speakers: Melissa Hogan 
 
Advancing Medical Research and Practice: How Will 3D Bioprinting Improve Patients' Lives?
Speakers: Eric M David 

Precision Medicine Initiative: Significance for the Rare Disease Community
How Patients Can Best Engage
Speakers: Emily Kramer-Golinkoff,  Daniel Levine, Matt Might 
 
‘HOW TO’ PRESENTATIONS
How to Find & Read a Scientific Paper, 2) School to Work Transition Programs
Speakers: Ethan Perlstein 
 
How to Recruit and Work With a Scientific Advisory Board, 2) Foundations: How to Give a Research Grant (i.e. To Early Stage Investigators)
Speakers: Mark Dant, Steve Grossman 
 
How to Interpret Clinical Research Findings, 2) Building a Comprehensive Care Center of Excellence
Speakers: Kelly Ranallo,  Christina Waters 
 
TRACKS

CAPACITY BUILDING
Developing Volunteer Leaders to Expand Your Organization's Reach and Services in the Community
Speakers: Eveline Honig, Louise Vetter 
 
PATIENTS ROLE IN DRUG DISCOVERY: RESEARCH AND CLINICAL TRIALS
How to Talk and Engage With Researchers and What Questions to Ask. Brought to you by PRA Health Sciences.
Speakers: Margie Frazier,  Gay Grossman 
 
PATIENT ACTIVISM
Understanding Expanded Access/Compassionate Use
Speakers: Kristina Broadbelt, John Lagus,  Steven Walker 
 
SCIENCE & TECHNOLOGY
Genetic Counselors: An Under-Utilized Resource and Tool for Patient Advocates
Speakers:Willonie Mendonca, Erica Ramos  
 
CAPACITY BUILDING
Growth and Transition: Adding Paid Staff to a Volunteer-Run Nonprofit
Speakers: Karen Pence,  Angie Rowe 
 
PATIENTS ROLE IN DRUG DISCOVERY: RESEARCH AND CLINICAL TRIALS
Understanding Clinical Trials
Speakers: Kent Theolke 
 
PATIENT ACTIVISM
Regulatory Advocacy: Patient Organization Case Studies on Working with the FDA
Speakers: Isabelle Lousada,  Jennifer Payne 
 
SCIENCE & TECHNOLOGY
The Genomics Revolution: Reliability and Access
Speakers: Hal Hoffman,  Ryan Taft 
 
CAPACITY BUILDING
Effective Social Media Engagement for Your Rare Disease Community
Speakers: Stephanie Fischer,  Melissa Hogan 
 
PATIENTS ROLE IN DRUG DISCOVERY: RESEARCH AND CLINICAL TRIALS
The Patient's Role in Designing Clinical Trials
Speakers: Jane Larkindale, Jamie Roberts,  Jeffrey W Sherman 
 
PATIENT ACTIVISM
Advocating at the State Level- Best Practices
Speakers: Sharon King,  Gina Szajnuk 
 
SCIENCE & TECHNOLOGY
Gathering Data from Wearable Devices; The Importance in Clinical Research and Care
Speakers: Donald Jones 
 
CAPACITY BUILDING
Designing a Sustainable Development Program for Long-Term Success
Speakers: Kathryn Seebold 
 
PATIENTS ROLE IN DRUG DISCOVERY: RESEARCH AND CLINICAL TRIALS
The Future of Clinical Research: New Clinical Trial Designs
 
PATIENT ACTIVISM
Advocating at the Federal Level- Best Practices
Speakers: Jennifer Bernstein, Jenny Nieto Carey 
 
SCIENCE & TECHNOLOGY
Big Data and Computer-Enhanced Medicine
Speakers: Anthony Chang,  Spyro Mousses 
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