Notice Number: NOT-HD-16-011
Key Dates
Release Date: June 14, 2016
Response Dates: July 31, 2016 and August 31, 2016
None
Issued by
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Purpose
The NICHD invites all stakeholders to submit nominations for research to be prioritized in the development of the Best Pharmaceuticals for Children Act (BPCA) Program’s 2017 Priority List of Needs in Pediatric Therapeutics. The NICHD is gathering nominations for drugs and therapeutic areas (e.g., pediatric condition, subpopulation, or setting of care) that require further study in children. Suggested topics not related to a specific drug, biologic, or device (e.g., training or research collaborations) will be considered by the NICHD outside of this framework.
This is a time-sensitive Request for Information (RFI). There will be two opportunities for public input in 2016:
1. Written responses will be accepted through July 31, 2016. All responses will be compiled into a database that will be reviewed by a committee of stakeholder representatives. The review will result in a preliminary priority list.
2. There will be an annual stakeholders meeting in December 2016 to review the final priority list and provide updates on the BPCA drug development program.
The purpose of this RFI is to obtain knowledge and gather information regarding current gaps in pediatric therapeutics. The information from this RFI will be used for prioritization planning purposes only and shall not be construed as a solicitation for applications, abstracts, or quotations, or as an obligation on the part of the NICHD to provide any funds on the basis of responses.
Background
The NICHD, part of the National Institutes of Health (NIH), leads a pediatric drug development program to identify and prioritize needs in pediatric therapeutics under BPCA. Originally authorized in 2002, BPCA was reauthorized in 2007 and 2012 as part of the Food and Drug Administration Safety and Innovation Act (FDASIA). The legislation has two components:
1. Encouragement of pediatric drug testing through incentives to private companies to conduct research in drugs used in children.
2. Authorization of a research program through the Department of Health and Human Services (HHS), with responsibility for the research program implementation through the NIH, specifically the NICHD. The NICHD, in consultation with the U.S. Food and Drug Administration (FDA) and experts in pediatrics, is responsible for development of a priority list of needs in pediatric therapeutics, and the subsequent sponsoring of clinical trials of drugs that emanate from the priority list.
Nominations for the BPCA Priority List of Needs in Pediatric Therapeutics are solicited as 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; it 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.
For reference, the 2015 BPCA Priority List of Pediatric Needs is located on the BPCA website: https://bpca.nichd.nih.gov/prioritization/status/Pages/status.aspx.
Information Requested
The NICHD is interested in concrete and relevant ideas and approaches to addressing scientific and regulatory needs in therapeutic areas, drugs, biologics, and devices in an effort to best target clinical (or pre-clinical) studies sponsored through the BPCA Program. Nominations should focus on identifying the scientific opportunity and approaches, technology, and expertise useful for a project’s development rather than on a funding instrument or solicitation design.
The BPCA nomination form consists of 10 comment areas and was developed to solicit information pertinent to the specific evaluation criteria described below.
Please note that with the exception of individuals' email addresses and phone numbers, all responses will also be placed in a public access file. If you choose to omit contact information, your response will still be given full consideration.
Evaluation Criteria
All nominations will be reviewed and evaluated on six key criteria, as follows:
*Relevance to BPCA mission and goals
*No disqualifying ethical concerns
*Evidence: consideration of the level of evidence available and current gaps
*Impact: potential effect on children, society, and delivery of care
*Population: consideration of the different populations that may benefit from the research
*Feasibility: consideration of the resources available to conduct the study.
Nominations must meet at least five of the six criteria to be considered.
Prioritization Process
The NICHD has established a prioritization process through which all nominations will be considered and evaluated by leading pediatric experts and stakeholder representatives.
Gather Nominations (May-August 2016)
The NICHD is soliciting research nominations beginning in May 2016 by interacting with key stakeholder groups and requesting submission of the nomination form from the public and other stakeholders.
Prioritize Therapeutic Areas (December 2016)
The NICHD will convene key stakeholders to evaluate and prioritize the drug and Therapeutic Area nominations. From this assessment, the NICHD will finalize the top Therapeutic Areas for the next Priority List of Pediatric Needs.
Prioritize Pediatric Needs (Occurs Annually)
Each calendar year, the NICHD convenes at least one expert workgroup in a Prioritized Therapeutic Area. These workgroups provide more specific expertise in an area and assist the NICHD in evaluating the need for further drug studies within each of the identified therapeutic areas.
Responses
Submissions in response to this RFI will be considered by the NICHD, the FDA, and an ad hoc evaluation panel to be convened by the NICHD as part of this prioritization process. Nominations will be accepted through August 31, 2016. This nomination form is the only vehicle by which nominations for therapeutic area, drugs, biologics, and devices will be collected. The form corresponds to one nomination; a party may complete the form multiple times if interested in submitting more than one nomination.
The preferred format is online submission of the nomination using the following URL: https://www.surveymonkey.com/r/BPCAPriorityListNominations. As an alternative, a nomination form is also available as a PDF document by request to [email protected]. The completed PDF form can be sent via email or mail to the contact listed below.
Emails may be sent to [email protected] . Please indicate “BPCA Nomination” as the subject line of your email.
Letter responses should be addressed to the attention of Dr. Taylor-Zapata at the address below.
Responses to this RFI are voluntary. Do not include any proprietary, classified, confidential, trade secret, or sensitive information in your response. The responses will be reviewed by NIH staff, and individual feedback will not be provided to any responder. The Government will use the information submitted in response to this RFI at its discretion. The Government reserves the right to use any submitted information on public NIH websites, in reports, in summaries of the state of the science, in any possible resultant solicitation(s), grant(s), or cooperative agreement(s), or in the development of future funding opportunity announcements.
This RFI is for information and planning purposes only and shall not be construed as a solicitation, grant, or cooperative agreement, or as an obligation on the part of the Federal Government, the NIH, or individual NIH Institutes and Centers to provide support for any ideas identified in response to it. The Government will not pay for the preparation of any information submitted or for the Government's use of such information. No basis for claims against the U.S. Government shall arise as a result of a response to this request for information or from the Government's use of such information.
Inquiries
Please direct all inquiries to:
Perdita Taylor-Zapata, M.D.
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Telephone: 301- 496-9584
Email: [email protected]
and Human Services (HHS)
