March 21-23, 2023
Delivered as a Hybrid EventPhiladelphia Marriott Downtownkeyboard_arrow_right
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DAY 3 ✦ IN-PERSON ✦ Mar 23 - EST (Eastern Standard Time, GMT-5)
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DAY 3 ✦ IN-PERSON ✦ Mar 23 - EST (Eastern Standard Time, GMT-5)
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8:30am - 9:00am30 mins
Continental Breakfast and Networking
9:00am - 9:05am5 mins
Access and Reimbursement Strategies
Track Chair Opening Remarks
9:00am - 9:05am5 mins
Investing and Partnering
Track Chair Opening Remarks
- Nick Calla - Senior Vice President, Industry Relations, Orsini Healthcare
9:00am - 9:05am5 mins
Drug Development and Clinical Innovation
Track Chair Opening Remarks
- Terry Jo Bichell - Founder & Director, COMBINEDBrain
9:05am - 9:35am30 mins
Access and Reimbursement Strategies
Equity Spotlight—Working toward a Universal Healthcare System
- Jean-Pascal Roussy - Head, Global Public Affairs, Sanofi
9:05am - 9:35am30 mins
Investing and Partnering
Navigate How Biopharma Companies Evaluating Partnerships in the Rare Disease Space
- Devin Rosenthal - Vice President, NovaQuest Capital Management
- Andrew Leger - Head, US Business Development, Ferrer
- Sohini Mazumdar - Senior Director, Business Development, Agios Pharmaceuticals
9:05am - 9:35am30 mins
Drug Development and Clinical Innovation
What, How, Why—Conceptual Models
- Terry Jo Bichell - Founder & Director, COMBINEDBrain
9:35am - 10:35am60 mins
Access and Reimbursement Strategies
The Importance of Young Adult Involvement and Advocacy in Healthcare
- Eimile McKinnon - Young Adult Rare Representative, EveryLife Foundation for Rare Diseases
9:35am - 10:05am30 mins
Investing and Partnering
How to Promote Growth during Uncertain Times—When will the Biotech Pendulum Swing Again?
- John Maslowski - Chief Commercial Officer, Forge Biologics
9:35am - 10:05am30 mins
Drug Development and Clinical Innovation
TNX-2900 (Intranasal Oxytocin + Magnesium) in Development for the Treatment of Hyperphagia in Adolescents and Young Adults with Prader-Willi Syndrome
- Herbert Harris - Executive Vice President, Translational Medicine, Tonix Pharmaceuticals
10:05am - 10:35am30 mins
Investing and Partnering
Lessons Learned from Commercializing a Rare Disease Medicine
- Johanna Rossell - CCO, Enzyvant Therapeutics
10:05am - 10:35am30 mins
Drug Development and Clinical Innovation
Modernizing the System to Sustain Newborn Screening
- Amy Brower - Associate Project Director and Co-Principal Investigator, American College of Medical Genetics and Genomics
10:35am - 11:05am30 mins
Networking and Refreshment Break
11:05am - 11:35am30 mins
General Session
Advancing Rare Disease Research with Patient Centrality and Precompetitive Approaches- Screen4Care as a Case.
- Edith Gross - Senior Manager, Health and Social Sciences, EURORDIS-Rare Disease Europe
11:35am - 12:20pm45 mins
General Session
Rare Disease Awareness—Delivering Authentic Lived Experiences in a Challenging Regulatory Landscape
- Rebecca Stewart - CEO, Rare Revolution Magazine
- Nicola Miller - Creative Director & Editor-in-Chief, RARE Revolution Magazine
- Wendy Erler - VP Patient Experience, STAR, & Global Patient Advocacy, Alexion Pharmaceuticals: Astrazeneca Rare Disease
- Maria Picone - CEO & Co-Founder, TREND Community
12:20pm - 1:35pm75 mins
Networking Luncheon
1:35pm - 2:20pm45 mins
General Session
The 4 Ps of Advancing Precision Medicine – Patients, Providers, Partners and Pharma
- Chowdary Dondapati - Head, CDx Marketing, Precision Medicine (US), Bayer Oncology
2:20pm - 3:05pm45 mins
General Session
Strengthening Relationships—Putting the Patient in the Driver’s Seat
- Jennifer McNary - Co-Founder, Canary Advisors
- Chelsey Hathaway McCarthy - Executive Director, The DDX3X Foundation
- Elsa Lavita - Director, Patient Advocacy, MorphoSys
- Rayne Rodgers - Director, Patient Advocacy, Kinevant Sciences, Inc.