-

Add to Calendar
Session Title Start Time End Time
Registration and Networking Breakfast
11/6/2019 8:00 AM 11/6/2019 8:30 AM
Welcome
11/6/2019 8:30 AM 11/6/2019 8:45 AM
Keynote
Dr. Lewis-Hall will discuss the patient journey and how we as an industry can improve the patient experience from working with regulators toward more patient-centric clinical endpoints in trials through developing better tools to engage patients in their own care to improve access and compliance to their treatments. 		11/6/2019 8:45 AM 11/6/2019 9:45 AM
Two Sides of the Coin: The Patient Experience and the Scientists that Study their Biology
Patients and scientists can experience the arduous trek from drug discovery to treatment from opposite ends. Scientists seek to understand the molecular and cellular basis of a disease to treat or cure a patient population. Meanwhile, patients know the reality of their symptoms and day-to-day limitations. How can we bridge the divide and give scientists the opportunity to step back and hear directly from patients about how their biology affects a person first-hand? 		11/6/2019 9:45 AM 11/6/2019 10:45 AM
Networking Break
11/6/2019 10:45 AM 11/6/2019 11:30 AM
The Million Dollar Question: What is a One-Time Cure Worth to Society?
Innovative treatments like a curative gene therapy are creating unprecedented access issues. How can we better communicate a breakthrough therapy’s value to payers and patient communities alike to secure access? What happens when the value of a therapy is indisputable but society still debates its costs? While the gift of health is priceless for sick patients, it’s clear that not everyone agrees that it’s worth the cost. 		11/6/2019 11:30 AM 11/6/2019 12:30 PM
Lunch & Workshop: Improving the Clinical Trial Experience for Rare Disease Patients
Participants will learn more about the patient journey through a clinical trial. Then they will apply the knowledge and engage in an “overcoming obstacles” activity designed to encourage collaboration among rare disease patients, trial sponsors, and advocates. You will identify obstacles to trial participation and work to develop creative solutions. 		11/6/2019 12:30 PM 11/6/2019 1:30 PM
Quantifying the Value of Health: Perspectives from a Patient, Payer, Provider, & Manufacturer
For a patient, the value of a therapy can mean living a normal life, even for part of the day. For a payer, value can mean improvement in health for a reasonable amount of money. For a manufacturer, it can mean broad access to the given patient population that will respond positively to a drug. And then there’s the growing role of Health Technology Assessments in seeking to create a standardized, data-driven, and evidence-based approach to measuring the value of a drug. How can we meet in the middle? 		11/6/2019 1:30 PM 11/6/2019 2:30 PM
Patient Keynote: Our Family’s Journey with A Rare Disease
For the past 25+ years, Mark Dant and the Ryan Foundation have partnered with numerous research scientists and universities to help innovative projects move toward treatment in lysosomal storage disease. They have been key advocates speaking to the FDA and in 2009 successfully championed the US House of Representatives to pass the Ryan Dant Health Care Opportunity Act, a bill designed to help those living on Medicaid assistance become gainfully employed. The Dant’s journey has been documented on CBS 60 Minutes, CNN, Biography Magazine, Readers Digest in 13 languages around the world, Golf Digest, the LA Times and numerous newspapers and news outlets across the US. 		11/6/2019 2:30 PM 11/6/2019 3:30 PM
Networking Reception
11/6/2019 3:30 PM 11/6/2019 4:30 PM