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Upcoming Events / Rare Disease Research: A Prescription
  • Washington, DC

Rare Disease Research: A Prescription


Convene (14th Street, DC)
600 14th St NW
Washington, DC 20005


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Research into rare disease has led to new treatments and some breakthroughs. But are the policies and science aligned to produce treatments? We’ll speak with the lawmakers and patient advocates working to create this alignment.

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6:00 – 6:30 PM ET – Arrival + Registration
Drinks will be served. Masks strongly encouraged when not eating or drinking.

6:30 – 6:35 PM ET – Opening from STAT

Rick Berke, co-founder and executive editor, STAT

6:35 – 6:40 PM ET – Barth Syndrome: A Patient’s Story

Walker Burger, Barth syndrome study participant

6:40 – 6:45 PM ET – Welcome Remarks | SPONSOR SESSION

Del Lebel, head, US government affairs and policy, Alexion, AstraZeneca Rare Disease

6:45 – 7:00 PM ET – Rare Must Still Mean Diverse
Trials for drugs to treat rare diseases tend to be smaller and have a limited number of patients. New legislation aims to make sure they are racially and ethnically diverse despite that.

Congresswoman Robin Kelly, (D-IL.)
Rachel Cohrs, Washington correspondent, STAT (moderator)

7:00 – 7:10 PM ET – Policies That Can Help Science Help Patients | SPONSOR SESSION
Developing treatments for rare diseases comes with its own unique set of scientific and regulatory challenges. But there are policy solutions that can address those challenges, and bring treatments to people living with these conditions more efficiently and effectively.

Sharon Barr, Ph.D, senior vice president, head of research and product development, Alexion, AstraZeneca Rare Disease
Del Lebel, head, US government affairs and policy, Alexion, AstraZeneca Rare Disease

7:10 – 7:15 PM ET – Barth Syndrome: A Parent’s Story

Jay Scott Randell, D.M.D., Barth syndrome patient advocate

7:15 – 7:45 PM ET – Ultra Rare, Ultra Difficult
High prices have made rare disease drugs common and profitable. But some diseases are so rare that there is still no path to regulatory approval. We’ll examine the quandaries of one of the drug industry’s most challenging markets.

Reenie McCarthy, CEO, Stealth BioTherapeutics
Frank Sasinowski, J.D., director, Hyman, Phelps & McNamara
Ed Silverman, Pharmalot columnist, senior writer, STAT (moderator)

7:45 – 8:15 PM ET – Should the FDA Move Faster on ALS Medicines?
A congressional staffer who worked on a bill that gives patients with amyotrophic lateral sclerosis access to experimental drugs sits down with a patient advocate and a bioethicist to discuss the best next steps for speeding drugs to market.

Holly Fernandez Lynch, J.D., assistant professor of medical ethics and law, University of Pennsylvania
David Steury, legislative assistant for Congressman Mike Quigley (D-Ill.)
Tim Tobin, J.D., person living with ALS and advocate
Sarah Owermohle, Washington correspondent, STAT (moderator)

8:15 – 8:20 PM ET – Closing From STAT

Nicholas St. Fleur, general assignment reporter; associate editorial director of events, STAT

8:20 – 9:00 PM ET – Networking Reception
Drinks and light bites will be served. Masks strongly encouraged when not actively eating or drinking.

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