FDA Announces a Public Workshop on Drug Development in Pediatric Heart Failure

FDA Announces a Public Workshop on Drug Development in Pediatric Heart Failure:  Extrapolation, Clinical Trial Design, and Endpoints 

The U.S. Food and Drug Administration (FDA) is announcing a one-day public workshop titled “Drug Development in Pediatric Heart Failure: Extrapolation, Clinical Trial Design, and Endpoints,” jointly sponsored by the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI) and FDA. The purpose of this public workshop is to provide an opportunity for stakeholders, including clinicians, academia, industry, and FDA, to address challenges related to the evaluation of products in pediatric heart failure including population to study, endpoints, and extrapolation of adult efficacy data. The Drug Development in Pediatric Heart Failure workshop will also provide a forum for discussion on the use of registry data, as well as alternative trial designs and statistical methods. 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.

Date, Time, and Location

Date:  Friday, October 27, 2017

Time:  8:00 a.m. to 5:00 p.m.  

Location:  FDA White Oak, Bldg. 31, Rm. 1503A,10903 New Hampshire Avenue, Silver Spring, MD 20993

Agenda

Review the workshop agenda at: https://oct27workshop.splashthat.com/.

Registration Information

The registration fee to attend this public workshop in person is as follows:

• Industry Representatives - $50
• Nonprofit Organizations and Academia Representatives (Other Than University of Maryland) - $50
• University of Maryland, College Park and Baltimore, Faculty, Staff, and Students - $0
• Federal Government Employees - $0

There is no registration fee for attending the workshop via the webcast, but registration is required. 

There will be no onsite registration.

Register for the workshop at: https://bioeumd.wufoo.com/forms/xsim9dm0hjrelx/. 

Contact for Further Information 

Jacquline Yancy, Center for Drug Evaluation and Research, Food and Drug Administration, Bldg. 22, Rm. 6319,10903 New Hampshire Avenue, Silver Spring, MD 20993, 301-796-7068,Jacquline.Yancy@fda.hhs.gov.

See the Federal Register announcement at: https://www.gpo.gov/fdsys/pkg/FR-2017-09-21/pdf/2017-20106.pdf.