Watch the Event Livestream!
The Policy Lunch Briefing featuring Francis Collins, the FDA’s Perspective Talk featuring Celia Witten, and the Externally-Led Patient-Focused discussion will all be available for streaming on Tuesday, May 21.
The talks from ARM’s Annual Dinner will not be available for livestreaming, however, they will be filmed and all videos will be posted to this website by the week of June 3. Please check back here at that time.
Tuesday, May 21, 2019
Open to ARM Members Only
9:00am – 9:50am
U.S. Regulatory Affairs Committee Meeting (Waterside 2)
10:00am – 10:50am
U.S Market Access & Value Committee Meeting (Waterside 2)
11:00am – 11:50am
Government Relations & Policy Committee Meeting (Waterside 2)
Patient Engagement Committee Meeting (Harbor Room)
Policy Lunch Briefing with NIH Director Dr. Francis Collins
12:00pm – 1:15pm
This briefing will address NIH’s critical scientific and policy work in the area of cell and gene therapy including planned changes in the RAC review for gene therapy trials and NIH support of research and development of gene therapies. The address will also allow time for audience members to ask questions during a dedicated Q&A session.
Francis Collins, M.D., Ph.D., Director, National Institutes of Health (NIH)
The View From the Hill
1:15pm – 2:15pm
This panel will consist of bipartisan, senior level healthcare staffers from the House and Senate who will give an insider’s perspective on chances for the reform of Medicare and Medicaid programs to allow for value-based payment models and other priority issues for the sector.
Alex Ruoff, Health Reporter, Bloomberg Government
Kathleen Laird, Senior Health Policy Advisor, U.S. Senator Tammy Baldwin (D-WI)
Jeff Lucas, Health Policy Advisor, U.S. Senator Bill Cassidy (R-LA)
Colleen Ramsey Nguyen, Senior Policy Advisor, U.S. Representative Ami Bera (D-CA)
Developments in Regenerative Medicine: FDA’s Perspective
2:15pm – 2:45pm
This briefing will provide an update on the agency’s regulatory framework for regenerative medicine products including implementation of the Regenerative Medicine Advanced Therapy (RMAT) designation, new agency guidance for product developers, and other initiatives.
Celia Witten, M.D., Ph.D., Deputy Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
Externally-Led Patient-Focused Drug Development Meetings and Their Role in the Product Review Process
2:45pm – 3:30pm
This panel will discuss how these meetings are organized and the systematic approach they provide to ensure that patients’ experiences, perspectives, needs, and priorities are meaningfully incorporated into drug development and evaluation.
Janet Lambert, CEO, Alliance for Regenerative Medicine
Abby Bronson, SVP, Research Strategy, Parent Project Muscular Dystrophy
Jane Larkindale, Ph.D., VP, Research Development, Friedreich’s Ataxia Research Alliance
Legislative Fly-In FAQ Session
3:30pm – 4:00pm
This session will allow those attending the Legislative Fly-In the following day an opportunity to review issues and talking points, and cover frequently asked questions related to the Hill visits. ARM staff will be available to answer questions and provide background information as needed.
After a short afternoon break, join us again for ARM’s 10th Annual Dinner. The Annual Dinner is one of ARM’s premier events celebrating major accomplishments over the past year in the regenerative medicine and advanced therapies field. This intimate dinner recognizes the key corporate, scientific and policy leaders in our sector – including members of Congress, patient advocates, disease-foundation executives and other industry champions.
6:00pm – 7:00pm
7:00pm – 9:00pm
Matthew Patterson, Chairman and CEO, Audentes Therapeutics; Chairman, Alliance for Regenerative Medicine
Cara O’Neill, M.D., Scientific Director and Executive Vice President, Cure Sanfilippo Foundation
Kristin Smedley, President, Curing Retinal Blindness Foundation
Legislator of the Year
Each year ARM presents its annual “Legislator of the Year” award to key lawmakers who have demonstrated recent and substantial leadership in advancing this sector.
U.S. Senator Bill Cassidy (R-LA)
U.S. Senator Mark Warner (D-VA)
Wednesday, May 22, 2019
Each participant will be assigned to a group based on state/districts and will meet with key members of Congress and Administration officials to describe the immense near-term potential of advanced therapies and the need for legislation that supports the development of life saving treatments.
Open to ARM Members Only, Registration Required
7:00am – 8:00am
Breakfast & Legislative Issues Briefing (Seaport 2)
9:00am – 3:00pm