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Where

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Fri

Oct

8:00am

12pm - 1pm

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12pm - 1pm

How to Build Schedule Blocks

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12pm - 1pm

How to Build Schedule Blocks

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12pm - 1pm

How to Build Schedule Blocks

C. Doe

M-CERSI Workshop:
Drug Development in Pediatric Heart Failure

Friday, October 27, 2017 | 8:00 a.m. - 5:00 p.m.

FDA's White Oak Campus

10903 New Hampshire Avenue

Building #31 - Great Room

Silver Spring, MD 20903

The cost to register for this event is determined by participant type.

This public workshop will also be available to view via webcast at no cost, but registration is still required.

Please email aanonsen@umd.edu with any questions.

Mark your calendars for Drug Development in Pediatric Heart Failure: Extrapolation, Clinical Trial Design, and Endpoints, a one-day public workshop jointly sponsored by the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI) and the Food and Drug Administration (FDA).

Scheduled for Friday, October 27, 2017 from 8:00 a.m. to 5:00 p.m., "Drug Development in Pediatric Heart Failure: Extrapolation, Clinical Trial Design, and Endpoints" will address challenges related to evaluation of products in pediatric heart failure including population to study, endpoints, extrapolation of adult efficacy data, innovative trial designs and statistical methods.

The workshop will provide an opportunity for relevant stakeholders, including representatives from academia, government, non-profit organizations, and industry, to discuss similarity of heart failure between adults and pediatric patients, trial design considerations, and use of innovative statistical and modeling approaches. Specifically, the workshop will include:

- Presentations on clinical manifestation and management of heart failure in pediatric patients, as compared to adults

- Discussion of alternative approaches to establishing efficacy including extrapolation of adult efficacy data, pharmacokinetic/pharmacodynamic endpoints, clinical endpoints, and innovative trial designs.

Stay tuned for more details about featured speakers and presentations.

Friday,

October 27

8:00 a.m.

Welcome/Introductory Remarks:
Setting the Scene

Lynne Yao, M.D.

Food and Drug Administration (FDA)

8:10 - 8:25 a.m.

Introductory Remarks

Robert Temple, M.D.
Food and Drug Administration (FDA)

8:20 - 8:35 a.m.

Opening presentation: General landscape of extrapolation of efficacy in pediatric drug development

Lily Mulugeta, Pharm.D.
Food and Drug Administration (FDA)

8:35 - 8:50 a.m.

Drug Development in Pediatric Heart Failure—EMA Perspective

Dobromir Penkov, M.D.
European Medicines Agency

PEDIATRIC HEART FAILURE:

CURRENT STATE OF KNOWLEDGE

8:50 - 9:20 a.m.

"Disease" Natural History in Adults and Children, and "Grouping" of Different Causes of Pediatric HF for Clinical Trials - Clinical Perspective

Joseph Rossano, M.D.

Children's Hospital of Philadelphia (CHOP)

9:20 - 9:50 a.m.

Management of Pediatric Heart Failure - Clinical Perspective

Daphne Hsu, M.D.
Montefiore Medical Center

9:50 - 10:10 a.m.

BREAK

10:10 - 10:40 a.m.

Role of Research Networks in Understanding the Natural History of the Disease and in Facilitating Clinical Trials— the Pediatric Heart Network perspective

Kristin Burns, M.D.

National Heart, Lung, and Blood Institute

10:40 - 10:45 a.m.

Patient Perspective

Callie Tansill-Suddath

10:45 - 11:30

Q&A and Moderated Panel Discussion

Moderators: Shari Targum, Lily Mulugeta

Skip (Robert) Nelson, M.D.

Food and Drug Administration

Joseph Rossano, M.D.

Children's Hospital of Philadelphia

Daphne Hsu, M.D.
Montefiore Medical Center

Kristin Burns, M.D.

National Heart, Lung, and Blood Institute

Steven Lipshultz, M.D.

Wayne State

Callie Tansill-Suddath

Preston Dunnmon

Food and Drug Administration

Dobromir Penkov, M.D.

European Medicines Agency

Gilbert Burckart
Food and Drug Administration

11:30 - 12:30 p.m.

LUNCH

TRIAL DESIGN CONSIDERATIONS IN PEDIATRIC HEART FAILURE

12:30 - 1:00 p.m.

Biomarkers and Endpoint Selection and the Role of Registries

Steven Lipshultz, M.D.

Wayne State

1:00 - 1:20 p.m.

Methodological and Statistical Considerations for Using External Controls in Clinical Trials

Ruthanna Davi, Ph.D.

Medidata

1:20 - 1:40 p.m.

The Use of Quantitative Tools Including Systems Pharmacology for Study Planning and Design Optimization

Jeffrey Barrett, Ph.D.

Sanofi

1:40 - 2:00 p.m.

The Role of Bayesian Decision-Making in Pediatric Drug Development: Case Examples

Andrew Thomson
Statistician
Office of Biostatistics & Methodology Support, European Medicines Agency

CASE EXAMPLES IN PEDIATRIC HEART FAILURE

2:00 - 2:30 p.m.

Current Approach When Limited Confidence in Inference from Existing Adult Efficiency Data: Case Example (Entresto)

Fabian Chen, M.D.

Novartis

2:30 - 2:45 p.m.

BREAK

2:45 - 3:15 p.m.

Considerations for Trial Design When There is Greater Level of Certainty That Adult Data Can Be Considered for Extrapolation to Pediatric Patients: Challenges and Opportunities; Case Example (Ivabradine)

Lisa Bollinger, M.D.

Amgen (Product developed and pediatric studies conducted by Servier)

3:15 - 4:15 p.m.

Moderated Panel Discussion

Moderator: Lynne Yao, M.D.

Robert Temple, M.D.
Food and Drug Administration (FDA)

Christoph Hornik

NICHD Pediatric Trials Network

Joga Gobburu, Ph.D.

University of Maryland

Andrew Thompson

European Medicines Agency

Jeffrey Barrett

Sanofi

Fabian Chen

Novartis

Lisa Bollinger

Amgen

Norman Stockbridge, M.D.

Dobromir Penkov

European Medicines Agency

Christoph Hornik, M.D.
NICHD Pediatric Trials Network

4:15 - 4:30 p.m.

Closing Remarks & Future Direction

Lynne Yao, M.D.

Food and Drug Administration (FDA)