March 21-23, 2023
Delivered as a Hybrid EventPhiladelphia Marriott Downtownkeyboard_arrow_right
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DAY 2 ✦ IN-PERSON ✦ Mar 22 - EST (Eastern Standard Time, GMT-5)
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DAY 2 ✦ IN-PERSON ✦ Mar 22 - EST (Eastern Standard Time, GMT-5)
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8:00am - 8:30am30 mins
Continental Breakfast and Networking
PAP- Patient Assistance and Access Programs & Rare Disease Innovation and Partnership Summit
8:30am - 10:30am120 mins
Workshop A
Advocacy Insights to Enhance Patient Care
- Crystal Nisly - Certified Pharmacy Technician, Medication Assistance Caseworker, Orange County Free Clinic
- Julie Burns - Senior Manager, Patient Advocacy Relations, Genentech, A Member of The Roche Group
- Jill Pollander - VP, Patient Services, National Organization for Rare Disorders (NORD)
8:30am - 10:30am120 mins
Workshop B
Partnership and Development to Optimize Access
- Amanda Moore - CEO, Angelman Syndrome Foundation
- Anne Bruns - Senior Director, Global Clinical Patient Advocacy, PTC Therapeutics
10:30am - 11:00am30 mins
Networking and Refreshment Break
11:00am - 11:05am5 mins
Access and Reimbursement Strategies
Track Chair Opening Remarks
11:00am - 11:05am5 mins
Investing and Partnering
Track Chair Opening Remarks
- Nick Calla - Senior Vice President, Industry Relations, Orsini Healthcare
11:00am - 11:05am5 mins
Drug Development and Clinical Innovation
Track Chair Opening Remarks
11:05am - 11:35am30 mins
Access and Reimbursement Strategies
The Role of Patient Support Programs for Patients with Rare Diseases
- Anthony Sigrest - Head, Digital Patient Engagement and Innovation, Takeda
- Julie Kilcoyne - Senior Director, Healthcare Law, Blueprint Medicines
- Jacqui Nolet - Program Manager, Market Access Operations, Blueprint Medicines
11:05am - 11:35am30 mins
Investing and Partnering
Forecasting & Analytics- In the Era of Cell & Gene Therapies
- Sanobar Syed - Associate Director, Market Insights, BeiGene
11:05am - 11:35am30 mins
Drug Development and Clinical Innovation
Further Insights on FDA Guidance—Patient Focused Drug Development Series
11:35am - 12:05pm30 mins
Access and Reimbursement Strategies
Elevate Rare Disease Patient Experience Measurement from Foundational to Elite
- Sean McDade - Founder and CEO, PeopleMetrics
11:35am - 12:05pm30 mins
Investing and Partnering
Overcome Investment Challenges in the Rare Disease Space
- Jeff Livingstone - CEO, Igia Pharmaceuticals
- Imran Babar - Chief Business Officer, Sudo BioSciences
- Devin Rosenthal - Vice President, NovaQuest Capital Management
11:35am - 12:05pm30 mins
Drug Development and Clinical Innovation
Back to the Basics—Understanding the Drug Development Process
- Casey McPherson - Founder and President, To Cure A Rose Foundation
12:05pm - 12:35pm30 mins
Access and Reimbursement Strategies
Compare and Contrast—Global Access Pathways
- Swami Subramanian - Head, US/Global Pricing and Contracting, Regeneron
12:05pm - 12:35pm30 mins
Investing and Partnering
Managing Alliances and Partnerships
- Amanda Moore - CEO, Angelman Syndrome Foundation
- Tricha Shivas - Chief Strategy Officer, Foundation for Sarcoidosis Research
- Carrie Howell - Executive Director, Dup15q Alliance
- Derrell Porter - Founder & CEO, Cellevolve Bio
12:05pm - 12:35pm30 mins
Drug Development and Clinical Innovation
Shifting the Focus—Patient Centered Outcome Measures
- Kari Rosbeck - President & CEO, TSC Alliance
12:35pm - 1:50pm75 mins
Selections from The Disorder Channel Networking Luncheon
1:50pm - 2:20pm30 mins
Access and Reimbursement Strategies
Leverage Real World Evidence (RWE) for Improved Outcomes and Access
- Rachele Hendricks-Sturrup - Research Director, Real World Evidence, Duke-Robert J. Margolis, MD, Center for Health Policy
1:50pm - 2:20pm30 mins
Investing and Partnering
Optimize Investment Opportunities in Orphan Drugs
- Imran Babar - Chief Business Officer, Sudo BioSciences
- Everett Crosland - Chief Commercial Officer, Cognito Therapeutics
- Heather Petty - Director, Program Development, Xontogeny
1:50pm - 2:20pm30 mins
Drug Development and Clinical Innovation
Behind the Scenes—Life before the Clinical Trial
- Shane Hegarty - Chief Scientific Officer and Co-Founder, Axonis Therapeutics
2:20pm - 3:05pm45 mins
Access and Reimbursement Strategies
Boosting the Appeal of Clinical Trials- Making Participation of Greater Interest to Patients
- Wes Michael - President, Rare Patient Voice
- Richie Kahn - Co Founder and Principal, Canary Advisors
2:20pm - 3:05pm45 mins
Investing and Partnering
Developing Treatments for Rare Diseases on a Shoestring—The Batten Disease CLN1 Experience
- Ana Puhl - Associate Director, Collaborations Pharmaceuticals Inc.
2:20pm - 3:05pm45 mins
Drug Development and Clinical Innovation
Increasing Innovation in Clinical Trial Design
- Joe Grieco - VP, Clinical Development, Tris Pharmaceuticals
3:05pm - 3:35pm30 mins
Networking and Refreshment Break
3:35pm - 4:20pm45 mins
Access and Reimbursement Strategies
Setting Yourself Up for Success—Best Practices for a Successful Product Launch
- Andrew Udell - President, North America, Calliditas Therapeutics
- Catherine St. John - Senior Corporate Counsel, Comerical Law, Sanofi
- Kari Loeser - Vice President & Chief Compliance Officer, Cytokinetics
- Gwen Hyland - Head of Global Commercial Excellence, Rare Diseases, Alexion Pharmaceuticals: AstraZeneca Rare Disease
3:35pm - 4:20pm45 mins
Investing and Partnering
Exploring the Role of Specialty Pharmacy in Rare Disease
- Nick Calla - Senior Vice President, Industry Relations, Orsini Healthcare
3:35pm - 4:20pm45 mins
Drug Development and Clinical Innovation
Uncovering Access to Genomic Sequencing
- Jared Stephens - VP, Strategy & Business Development, Nanoscope Therapeutics
4:20pm - 5:05pm45 mins
Access and Reimbursement Strategies
Exploring Expanded Access Programs
- Jason Bradt - Executive Medical Director, Clincal Research, Pharming Healthcare Inc
- Lee Sutton - VP, Business Development for Expanded Access Programs, WEP Clinical
4:20pm - 5:05pm45 mins
Investing and Partnering
Dedicated Meeting Time
4:20pm - 5:05pm45 mins
Drug Development and Clinical Innovation
Using AI to Accelerate the Drug Discovery and Development Process for Rare Cancers
- Panna Sharma - CEO, President, Lantern Pharma, Inc.
5:05pm - 6:05pm60 mins
Close of Day and Networking Reception for Rare