Key Dates

Related Announcements

Issued by

National Cancer Institute (NCI)

Purpose

The National Cancer Institute (NCI) is seeking information on pediatric preclinical models [e.g., cell lines and patient-derived xenografts (PDXs)] for which genomic characterization will enhance their research value. This request for information (RFI) is part of a planning effort to identify pediatric preclinical models that can be genomically characterized through a sequencing center supported by NCI to perform genomic characterization of such models.

Background

The importance of pediatric preclinical testing data is heightened in the current environment by provisions of the FDA Reauthorization Act of 2017 (FDARA) that amended the Pediatric Research Equity Act (PREA) in section 505B of the Federal Food, Drug, and Cosmetic Act (FDCA) to add a new molecularly targeted pediatric investigation requirement for certain oncology medicine applications for agents whose targets may be relevant to growth or progression of a pediatric cancer. As many as 200 agents per year may require some type of evaluation for their relevance to childhood cancers.

Preclinical testing programs using genomically characterized pediatric cancer models and validated testing methods can contribute by providing reliable testing results to use for agent prioritization decisions so that progress in identifying more effective treatments for children with cancer can be accelerated. The selection of appropriate preclinical models for testing for targeted agents is predicated on detailed genomic characterization of the models.

NCI has identified a sequencing center that will be able to perform genomic characterization of up to 500 pediatric preclinical models. Genomic characterization services for preclinical models will include whole exome sequencing, RNA-seq, and DNA methylation array analysis. The data generated will be incorporated with existing genomic characterization data into a Pediatric Preclinical Data Commons that NCI is establishing as part of the Childhood Cancer Data Initiative to serve as a resource to the pediatric research community.

NCI will review the information received in response to this request for information. Based on this review, respondents may be invited to submit specimens for genomic characterization. There will be no obligation for respondents to accept this invitation. As noted below, an Institutional Certification must be provided before genomic characterization can proceed. Genomic characterization is anticipated to begin in December, 2020 and continue through the first quarter of 2021.

Information Requested

This RFI seeks information on childhood cancer cell lines and PDX models and other human-derived preclinical models for which genomic characterization may enhance the utility of the models.

• A document (preferably Excel file) with a table providing information on the clinical/demographic characteristics of the models proposed for testing and information on model creation and quality assurance is requested. Please follow the PDX models Minimal Information standard (PDX-MI) data elements to the extent possible.
• Include in the table the extent of genomic characterization that has been performed on the models to date.
• Indicate in the table the ability to provide 100 mg of frozen tissue for each model by January 31, 2021.

As the intent of performing genomic characterization is to make the results available to the research community, the Institutional Certification(s) using the current NIH template that demonstrates that it is permissible to share individual-level genomic data will need to be generated for all samples proposed. Institutional Certifications specify the data use limitations and data use limitation modifiers, as determined by the institution's IRB after reviewing the informed consent agreed to by the participants. If the Institutional Certification is not available, please provide a Provisional Certification and describe the anticipated data use limitations and associated modifiers separately. A completed Institutional Certification will be required before models can be submitted for genomic characterization. Guidance on obtaining an Institutional Certification is available at the NIH website.

How to Submit a Response

Responses will be accepted through October 30, 2020. Responses are to be sent by e-mail to Malcolm.Smith@nih.gov. Please include the Notice number (NOT-CA-20-108) in the subject line.

The submitted information will be reviewed by NCI staff. Every respondent will receive an e-mail confirmation acknowledging receipt of a successfully submitted response. Responses are entirely voluntary. No proprietary, classified, confidential, protected health information, or other sensitive information should be included in your response. All individual responses will remain confidential.

This request is for information and planning purposes. The NIH does not intend to make any awards based on responses to this RFI or to otherwise pay for the preparation of any information submitted or for the Government's use of such information.

Inquiries

Please direct all inquiries to:

Malcolm A. Smith, MD, PhD
National Cancer Institute (NCI)
Telephone: 240-276-6087
Email: Malcolm.Smith@nih.gov