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Key Sessions

Seema Verma

Keynote Address from CMS Administrator Seema Verma

Centers for Medicare and Medicaid Services (CMS)

Jeff Marrazzo

Delivering Gene Therapies: A New Era in Medicine

Spark Therapeutics

Dec 12
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8:00am - 9:00am

Morning Coffee & Registration

More
Showing of Streams
10:30am - 11:15am

Networking Refreshment Break

More
Showing of Streams
3:15pm - 3:20pm
Conference Concludes

Conference Concludes

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8:00am - 9:00am 60 mins
Morning Coffee & Registration
9:00am - 9:30am 30 mins
Keynote Address
Keynote Address from CMS Administrator Seema Verma
  • Seema Verma - Administrator, Centers for Medicare and Medicaid Services (CMS)
9:30am - 10:00am 30 mins
Info
Keynote Address
Status update on the Administration’s HHS Pricing Blueprint
  • Eric Hargan - Deputy Secretary, Health and Human Services (HHS)

HHS has made addressing drug pricing a top priority. This session will cover how to address the challenges pharma is facing, including high list prices set by manufacturers, seniors and government programs overpaying for drugs due to lack of the latest negotiation tools, rising out-of-pocket costs, 


10:00am - 10:30am 30 mins
Info
Keynote Address
Delivering Gene Therapies: A New Era in Medicine
  • Keynote Speaker Jeff Marrazzo - CEO, Spark Therapeutics

Gene therapy, with the potential to deliver long-term, life-altering benefits in a single dose, is now a reality for appropriate patients suffering from certain previously untreatable genetic diseases. FDA Commissioner Scott Gottlieb recently said that he expects FDA to approve 40 gene therapies by 2022. A new era in medicine is at our doorstep, and we need a 21st Century health care system that can support and encourage these innovations. In this presentation, Jeff Marrazzo, CEO of Spark Therapeutics, a fully integrated, commercial gene therapy company, will share ideas on how we deliver on one of modern science's biggest opportunities to dramatically transform health care: turning the bits and bytes of the human genome into medicine. He will share a case study of Spark Therapeutics' work to define the value, set the price and secure reimbursement for the first FDA-approved gene therapy for an inherited disease in the United States - a major milestone for everyone involved, and an important watershed for patients and families living with genetic diseases. Ensuring patients and families have access to these new approaches to medicine requires changes to our health care system that recognize the unique nature of one-time gene therapies. He will speak to the urgency for new kinds of payment and reimbursement models that create the path to make one-time gene therapies an option for patients and families living with genetic diseases.

10:30am - 11:15am 45 mins
Networking Refreshment Break
11:15am - 12:00pm 45 mins
Info
Track A - Pathways to access, reimbursement and regulatory considerations
Addressing the Opioid Epidemic
  • Douglas Throckmorton - Deputy Director for Regulatory Programs, CDER, FDA

The FDA response to challenges in public health - managing opioids and shortages

11:15am - 12:00pm 45 mins
Info
Track B - MedDevice and Tech Perpectives Powered by Medtech Insight - Regulatory Landscape
The Growth of the Device Frontier: The path forward for drugs, device and combination products
  • Darin Oppenheimer - Executive Director, Drug Device Center for Excellence, Merck
  • Christina Kuhn - Associate, Covington & Burling LLP
  • Nicole Fisher - Founder and CEO, HHR Strategies

Personalized health and patient demand is driving the drug and device market forward. This panel will cover the many scientific advances in device and address the developmental and regulatory challenges facing biopharma.

 

12:00pm - 12:45pm 45 mins
Info
Track A - Pathways to access, reimbursement and regulatory considerations
Expanded Access and Right to Try
  • Moderator Jess Rabourn - CEO, WideTrial
  • Panelist Naomi Lopez-Bauman - Healthcare Policy Director, Goldwater Institute
  • Panelist Christopher Beardmore - CEO & Co-founder, Anova Enterprises, LLC

This session will examine the modern approaches to integrating access programs into the drug development process, including:

  • Current requirements of expanded access uses
  • Ethical and Legal issues to be considered
  • Commercial feasibility, cost, design and regulatory considerations.


12:00pm - 12:45pm 45 mins
Track B - MedDevice and Tech Perpectives Powered by Medtech Insight - Regulatory Landscape
Digital Heath Updates (connecting devices/combination devices/reimbursements/pre-cert)
  • Moderator Danny Al-Faruque - Senior Reporter, Medtech Insight - Pharma Intelligence
  • Bradley Merrill Thompson, RAC - Member, Epstein Becker Green
  • Adam Dakin - Managing Director, Dreamit HealthTech
12:45pm - 1:45pm 60 mins
Track A - Pathways to access, reimbursement and regulatory considerations
Networking Lunch
12:45pm - 1:45pm 60 mins
Track B - MedDevice and Tech Perpectives Powered by Medtech Insight - Regulatory Landscape
Networking Lunch
1:45pm - 2:30pm 45 mins
Info
Track A - Pathways to access, reimbursement and regulatory considerations
The FDA Biosimilars Action Plan—What to Expect?
  • Christine Simmon - Senior Vice President, Policy & Strategic Alliances Executive Director, Biosimilars Council

Policymakers and payers seek to contain healthcare costs and increase access to innovative medicines. Yet despite encouraging greater biosimilars use, physician interest is low. Patient demand muted. The first biosimilar was approved in the US almost four years ago, yet US utilization remains lower than anticipated. Earlier this year, FDA rescinded draft guidance to foster greater biosimilar development. Is the biopharma industry interested in promoting biosimilar use? Will manufacturers communicate ways to increase biosimilar uptake? Will FDA action make development easier? Should patient advocacy groups enter the conversation? This session will map-out how pending changes in the US health landscape may soon drive access to biosimilars in the US.

The FDA, in an unprecedented move, withdrew its pivotal draft guidance on establishing analytical similarity and announced a new effort to create a Biosimilars Action Plan to expedite approval of biosimilars. The essential elements of the new plan that the FDA will soon announce include a template approach to developing global dossier, analytical similarity testing for relevant attributes using least number of reference lots, removing bridging studies for non-US comparator, use of in silico models to use PK/PD data to support clinical efficacy, and providing a better clarity of the development requirements for biosimilars. The author has provided to FDA his recommendations and provides a current view of how the landscape of biosimilar products is about to change in the US, to allow the BPCIA to live up to its expectations to make biosimilars more accessible.

1:45pm - 2:30pm 45 mins
Info
Track B - MedDevice and Tech Perpectives Powered by Medtech Insight - Regulatory Landscape
Panel: EU-MDR: What is changing and how can we get a step ahead?
  • Panelist Hugo Xi, MD, MBA - Director, Medical Affairs, Abbott Quality & Regulatory, Abbott Laboratories
  • Panelist George Cusatis - Associate Director, Drug-Device Center of Excellence, Merck

EU-MDR regulations are here, but what is really changing and how can you stay ahead of them? This panel will discuss questions like the below and more:

  • What factors are people considering?
  • What changes are being made?
  • What role can notifying bodies play?
  • What has to be done to establish equivalence (espec. For second gen innovation products)? Etc.
2:30pm - 3:15pm 45 mins
Info
Track A - Pathways to access, reimbursement and regulatory considerations
CAR-T: Perspectives in value-based payment, medicare, and reimbursement

CAR-T was one of the first categories where CMS focused on new payment models. This panel will cover the latest on where things stand with these models, and what the future holds for CAR-T. 

2:30pm - 3:15pm 45 mins
Track B - MedDevice and Tech Perpectives Powered by Medtech Insight - Regulatory Landscape
Real World Evidence for Med Tech and Devices - The who, the what and the how.
  • Pamela Goldberg - President and CEO, MDIC
  • Heather Colvin - Director, MD Regulatory Affairs Evidence & Outcomes Policy, Johnson and Johnson
  • Marc Boutin - CEO, National Health Council
3:15pm - 3:20pm 5 mins
Conference Concludes