Notice Number: NOT-HD-11-013
Release Date: April 11, 2011
Response Date: May 6, 2011
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (http://www.nichd.nih.gov)
The NICHD invites all interested stakeholders to comment on gaps in pediatric therapeutics, which includes drugs and biologics that need further study. For 2011, therapeutic areas of interest to the BPCA Program include Pediatric Rheumatology, Hematology, Renal disease, and Pulmonology.
The purpose of this RFI is to collect information from the broader research community about the current gaps in pediatric therapeutics. The knowledge of the nature of pediatric diseases, including natural history, pathophysiology, and disease progression is fundamental to evaluating the effectivenss and toxicity of drugs used in children and adolescents. Our objective is to communicate the opportunity for input into the BPCA Program to as many stakeholders as possible.
This is a time-sensitive Request for Information (RFI).
The Best Pharmaceuticals for Children Act, authorized an incentive program for on-patent drugs that meet certain criteria first authorized in the 1997 Food and Drug Administration Modernization Act (FDAMA). The BPCA legislation also contains provisions for off-patent drugs and general support for pediatric product development. The legislation, as it applies to the NIH, authorizes a research program through the Department of Health and Human Services (HHS), with implementation through the NIH, specifically to the NICHD. The NICHD is responsible for the development of a 1) priority list of needs in pediatric therapeutics, in consultation with the FDA and experts in pediatrics; 2) sponsorship of relevant pediatric clinical trials; and 3)submission of resulting data to FDA for pediatric labeling changes.
Since 2002, the NICHD has sought public comment and collaborated with other NIH Institutes and Centers and experts in pediatrics to identify drugs in need of further study and prioritize needs in pediatric therapeutics. The priority list procedure has been revised to emphasize knowledge gaps in therapeutic areas in contrast to knowledge gaps about specific drug products. Specifically, the legislation authorizes that:
Public outreach is part of fulfilling NIH’s authority and responsibility to establish a program for pediatric drug testing and development as outlined in the BPCA legislation. The BPCA Priority List consists of key therapeutic needs in the medical treatment of children and adolescents. The list is organized by Therapeutic Area, which can be a group of conditions, a subgroup of the population, or a setting of care. Each calendar year, a few Therapeutic Areas are selected for discussion and further prioritization.
From that list, clinical, preclinical, or epidemiology studies are funded based on the needs that can be feasibily accomplished within the BPCA Program. One of the NIH’s responsibility under the BPCA 2007 legislation is to submit Proposed Pediatric Study Requests (PPSR) which outline the design, study population and propsed endpoints for a clinical study conducted under the BPCA Program. The goal is then for these PPSRs to be developed into Written Requests (WR). Written Requests generally define pharmacokinetics (PK), the PK-pharmacodynamic relationship, efficacy, and safety and is a blueprint for studies of on-patent and off-patent drugs that will promote labeling.
In an effort to understand and determine where conceptual, technical and methodolocial limitations exist in the safety, effectiveness and dose of medications used in children, and to identify compelling biomedical research questions and existing capabilities which can be addressed and tapped into in an effort to overcome these limitations, NICHD seeks the information requested below.
As part of the continuing BPCA prioritization process, the NICHD requests information on the following topics related to needs in pediatric therapeutics and the challenges which can be addressed in basic, translational, and clinical research endeavaors.
Please provide examples, (including any relevant references) and address whether the response addresses specific translational or clinical challenges in a particular therapeutic area, or is broadly applicable.
For your reference, the current list of studies funded by the BPCA Program and the 2011 BPCA Priority List of Pediatric Needs is located on the BPCA website:
http://bpca.nichd.nih.gov/about/process/status.cfm . The therapeutic areas of focus for 2011 include: Pulmonary therapeutics (focus on pulmonary hypertension treatment strategies and cystic fibrosis therapeutics), Rheumatology therapeutics, Therapeutics in Chronic Kidney Disease, and Hematology therapeutics.
We would like to sincerely thank you for your consideration of and responses to this notice. Your knowledge, expertise, and opinions are vital to our mandate of improving how drugs are used in children.
Reponses will be accepted until May 6, 2011. Please respond to Dr. Perdita Taylor-Zapata at the contact information below and reference this RFI identifier, NOT-HD-11-013.
In addition, if you are interested in participating in a working group in any of the above therapeutic areas, please send your contact information to Dr. Taylor-Zapata, as directed below.
This Request for Information (RFI) is for planning purposes only and is not a solicitation for applications or an obligation on the part of the United States (U.S.) Government, or the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) to provide support for any ideas identified in response to it. Please note that the U.S. Government will not pay for the preparation of any information submitted or for its use of that information.
NICHD will use the information submitted in response to this RFI at its discretion and will not provide comments to any responder’s submission. However, responses to the RFI submitted may be reflected in future solicitation(s). NICHD may contact any responder for the sole purpose of enhancing NICHD’s understanding of your RFI submission. The information provided will be analyzed and may appear in reports. Respondents are advised that the Government is under no obligation to acknowledge receipt of the information received or provide feedback to respondents with respect to any information submitted. No proprietary, classified, confidential, or sensitive information should be included in your response. The Government reserves the right to use any non-proprietary technical information in any resultant solicitation(s).
For additional questions and information, please contact:
Perdita Taylor-Zapata, MD
Obstetric and Pediatric Pharmacology Branch (OPPB)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
National Institutes of Health (NIH), Department of Health and Human Services (DHHS)
6100 Executive Boulevard, Room 4A01, MSC 7510
Bethesda, MD 20892
Rockville, Maryland 20852 (for express/courier service)
Telephone: 301- 496-9584
Fax: 301- 480-2897
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