DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 - EDT

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 8:00am - 9:00am60 mins

Conference Registration and Continental Breakfast

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 9:00am - 9:15am15 mins

Chairman’s Welcome and Opening Remarks

Tom Croce - Vice President, Patient Advocacy, bluebird bio

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 9:15am - 10:00am45 mins

PATIENT FIRESIDE CHAT ● Strengthening Patient Relationships Authentically to Enhance the Therapeutic Development Process

Ella Balasa - Cystic Fibrosis Patient Advocate, Self-Employed
Amanda Moore - CEO, Angelman Syndrome Foundation

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 10:00am - 10:45am45 mins

INNOVATIVE FUNDING SPOTLIGHT ● The Search for Novel Funding — Prepare for and Expand Successful Partnering

Chris Garabedian - Chairman and CEO, Xontogeny

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 10:45am - 11:15am30 mins

Networking and Refreshment Break

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 11:15am - 12:00pm45 mins

Understand the Rare Disease Patient Journey — Actionable Insights from Discovery to Access

Tom Croce - Vice President, Patient Advocacy, bluebird bio
Wendy Erler - Vice President, Head Patient Advocacy, Alexion Pharmaceuticals

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 12:00pm - 1:00pm60 mins

Networking Luncheon

CUSTOMIZE YOUR LEARNING EXPERIENCE ★ CHOOSE BETWEEN 2 TAILORED BREAKOUT SESSIONS

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 1:00pm - 1:45pm45 mins

A. Patient-Driven Access

Patients as Partners in Innovation

Shannon Murray Resetich - Global Franchise Head, Rare Diseases, Sanofi

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 1:00pm - 1:45pm45 mins

B. Reimbursement, Value & Access

Discover Patient Support Program Needs for Patients with Rare Diseases

Sean McDade - Founder and CEO, PeopleMetrics

CUSTOMIZE YOUR LEARNING EXPERIENCE ★ CHOOSE BETWEEN 2 TAILORED BREAKOUT SESSIONS

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 1:45pm - 2:30pm45 mins

C. New Launch & Commercialization

Clinical Management of Patients with Complex Conditions

Nick Calla - Senior Vice President, Industry Relations, Orsini Specialty Pharmacy

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 1:45pm - 2:30pm45 mins

D. Partnering, Investment & Portfolio Strategy

Evaluate New and Emerging Markets to Stay on the Cutting Edge

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 2:30pm - 3:00pm30 mins

Networking and Refreshment Break

CUSTOMIZE YOUR LEARNING EXPERIENCE ★ CHOOSE BETWEEN 2 TAILORED BREAKOUT SESSIONS

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 3:00pm - 3:45pm45 mins

A. Patient-Driven Access

Redefining Patient Lexicon through Person First Approach

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 3:00pm - 3:45pm45 mins

B. Reimbursement, Value & Access

TBD

Brad Sippy - Founder & CEO, Tremeau Pharmaceuticals

CUSTOMIZE YOUR LEARNING EXPERIENCE ★ CHOOSE BETWEEN 2 TAILORED BREAKOUT SESSIONS

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 3:45pm - 4:30pm45 mins

C. New Launch & Commercialization

Deep Dive into Lessons Learned for Launching Rare Disease Products

Suzanne Morgan, PhD, MBA - Director, Patient Access Services & National Accounts, NS Pharma

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 3:45pm - 4:30pm45 mins

D. Partnering, Investment & Portfolio Strategy

Optimize Strategic Partnership Models and Investment for Orphan Drug Products

Imran Babar - Chief Business Officer, Cydan
Craig Martin - CEO, Global Genes

DAY 1 ✦ IN-PERSON ✦ Mon, Dec 6 4:30pm - 5:30pm60 mins

Networking Wine and Cheese Reception