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

Rare Disease Research: A Prescription

Where

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

When

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About

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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AGENDA

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 – Welcome from STAT

Speakers to be announced

6:35 – 6:40 PM ET – Welcome Remarks | SPONSOR SESSION

Del Lebel, head, US Government affairs and policy, Alexion Pharmaceuticals

6:40 – 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-Ill.)
Rachel Cohrs, Washington corresponden, STAT (moderator)

7:00 – 7:10 PM ET – Fireside Chat | SPONSOR SESSION

Sharon Barr, senior vice president, head of research and product development, Alexion, AstraZeneca Rare Disease
Del Lebel
, head, US Government affairs and policy, Alexion Pharmaceuticals

7:10 – 7:20 PM ET – Patient Interstitial

Walker Burger, Barth syndrome study participant
Jay Scott Randell, D.M.D., Barth syndrome patient advocate
Ed Silverman, Pharmalot columnist, senior writer, STAT (moderator)

7:20 – 7:50 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, Director, Hyman, Phelps & McNamara
Ed Silverman, Pharmalot columnist, senior writer, STAT (moderator)

7:50 – 8:00 PM ET – Closing Remarks

8:00 – 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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Host

  • STAT