Discovery of the Genetic Basis of Childhood Cancers and of Structural Birth Defects: Gabriella Miller Kids First Pediatric Research Program (X01 Clinical Trial Not Allowed) | PAR 23 035

FAQs: NIH PAR-23-035 (Kids First Pediatric Research Program, X01)

What is PAR-23-035?

PAR-23-035 is an NIH funding opportunity under the Gabriella Miller Kids First Pediatric Research Program. Its purpose is to expand the Kids First Data Resource by generating high-quality genomic (and related) datasets from existing pediatric cohorts, especially cohorts involving childhood cancers and/or structural birth defects.

What is the main goal of this opportunity?

The central goal is to add new, well-curated sequencing datasets (plus linked clinical and phenotypic information) into the Kids First Data Resource Center so they can be broadly used by the research community. The emphasis is on building a shared resource rather than producing data intended only for a single lab or institution.

What does the X01 activity code mean for this opportunity?

The opportunity is labeled X01, which signals that the primary "award" is research services and data generation (such as sequencing performed at a Kids First Program-supported sequencing center), rather than a traditional grant structured around a large, flexible research budget.

Are clinical trials allowed under this funding opportunity?

No. The notice is explicitly labeled "Clinical Trial Not Allowed," meaning it is not intended to support interventional clinical studies. It is designed for sequencing and related data generation using pre-existing cohorts and specimens.

What kinds of cohorts are a good fit?

This opportunity is aimed at investigators who already have well-characterized collections of samples from children with cancer and/or structural birth defects. The cohorts should be suitable for generating genomic data and depositing it, along with clinical or phenotypic descriptors, into the Kids First ecosystem for broad use.

What pediatric conditions are in scope?

The scientific focus is on childhood cancers and structural birth defects. The program is interested in cohorts that can help explain the genetic basis of these conditions, including potential biological links between congenital anomalies and later increased cancer risk.

Does the program support studying germline genetics, somatic genetics, or both?

Both are in scope, where appropriate. Applications may focus on inherited (germline) variants that predispose to cancer, tumor-specific (somatic) changes that drive pediatric malignancies, genetic causes and mechanisms of congenital anomalies, or connections between birth defects and cancer risk.

What types of sequencing or assays does the program prioritize?

Whole genome sequencing is the main emphasis. However, the announcement allows additional or alternative approaches when scientifically justified, including whole exome sequencing, transcriptome sequencing (for example, gene expression and fusion detection), and epigenomic assays (such as those performed on tumor tissue or other affected tissues).

Can applicants propose approaches other than whole genome sequencing?

Yes, as long as the alternative (or additional) assays are scientifically justified and are the best fit for the disease question and sample types. The opportunity explicitly makes room for approaches like whole exome sequencing, transcriptome sequencing, and epigenomic assays.

Where will sequencing be performed?

The intent is that sequencing is conducted at a Kids First Program-supported sequencing center, using the applicants existing samples from qualifying pediatric cohorts.

What happens to the data generated through an awarded project?

The expected outcome is a curated, shareable resource. Generated sequence data, along with associated clinical and phenotypic descriptors, will be incorporated into the Kids First Data Resource Center for access by the broader research community.

Is data sharing a required part of the program?

Yes. A central feature of the opportunity is structured data sharing through the Kids First Data Resource Center. The program is designed to enable broad access, secondary analyses, cross-cohort comparisons, aggregation for rare disease and rare cancer discovery, and method development that is difficult with siloed datasets.

Why does the opportunity emphasize clinical and phenotypic information?

The program is building a data resource that is useful across many studies. Linking genomic data to clinical or phenotypic descriptors supports harmonization across cohorts and increases the value of the dataset for downstream analyses and reuse by the wider community.

Is there a stated priority around racial and ethnic diversity?

Yes. A major cross-cutting priority is improving racial and ethnic representation in pediatric genomic resources. The announcement specifically encourages cohorts from underrepresented racial and ethnic groups or projects that measurably increase representation within Kids First-associated research.

What kinds of diversity-focused projects are especially responsive?

Projects that address representation gaps by design, rather than as an afterthought, are particularly aligned with the intent described. The opportunity encourages applications that focus on underrepresented racial and ethnic groups or substantially increase representation in Kids First datasets.

Who is eligible to apply?

Eligibility is broad. It includes universities, nonprofits, hospitals, small businesses, and various U.S. government entities. It also explicitly includes mission-driven and community-linked institutions such as Historically Black Colleges and Universities (HBCUs), Hispanic-serving institutions, Tribally Controlled Colleges and Universities, Alaska Native and Native Hawaiian Serving Institutions, and Asian American Native American Pacific Islander Serving Institutions, as well as faith-based or community-based organizations.

Are organizations in U.S. territories or regional organizations eligible?

Yes. The opportunity explicitly includes U.S. territories or possessions and regional organizations among eligible applicants.

Are non-U.S. (foreign) entities eligible to apply?

Yes. The eligibility description explicitly includes non-U.S. (foreign) entities, consistent with the goal of capturing diverse pediatric cohorts wherever they exist.

Does the public summary list an award ceiling or the expected number of awards?

No. In the provided fields, the public summary does not state an award ceiling or an expected number of awards.

What is the CFDA number and category for this opportunity?

The opportunity is described as a discretionary NIH grant in the health category with CFDA 93.310.

What are the dates listed in the public summary?

The posting was created February 6, 2023, and the original closing date listed is March 21, 2023.

What should applicants focus on to be competitive, based on the description provided?

The description suggests applicants should center their proposal on: (1) a well-defined, well-characterized pediatric cohort with existing specimens; (2) clear scientific questions that genomic and related assays can answer; (3) a strong rationale for the sequencing and assay strategy (whole genome sequencing emphasized, alternatives allowed with justification); and (4) readiness to provide samples and metadata in a form suitable for harmonization and broad sharing through the Kids First Data Resource Center.

What does it mean that this opportunity is meant to grow the Kids First Data Resource?

It means the program is aiming to expand the breadth and utility of Kids First by adding new pediatric diseases, disorders, populations, and dataset types. Proposals that bring in new conditions, new populations, or new kinds of pediatric datasets are aligned with the stated intent.