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.
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.
Abby Bronson, SVP, Research Strategy, Parent Project Muscular Dystrophy
Jane Larkindale, Ph.D., VP, Research Development, Friedreich’s Ataxia Research Alliance
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
Janet Lambert, CEO, 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