Keeping Compliant with RACE Act FDA Legislation
An estimated 400,000 children worldwide develop cancer each year(1). Approximately one in four of these young patients cannot be cured via current standard therapies, and do not survive the disease.
Cancer is the leading cause of disease-related death in children, yet few effective therapies are available. The FDA created the RACE Act to increase the drug development pipeline for pediatric cancer. Since its implementation in 2020, pediatric cancer drug development and the number of clinical studies has already grown(2).
Oncology Preclinical Study Planning Toolkit
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Find the Right Model
Pediatric tumors have different genomic drivers and phenotypes than adult tumors, requiring unique preclinical models. Finding the right preclinical platform has been a major roadblock for oncology drug developers who need to test their assets for applicability to pediatric cancer.
The FDA RACE Act requires all drug developers to submit their study strategies for pediatric patients when seeking approval for oncology therapies.
Explore our extensive oncology offering, which can be applied to pediatric cancer drug development, with access to over 200 annotated, well-characterized, FDA RACE-compliant pediatric PDX models. Beyond RACE Act-compliant models, we also have experience running almost 600 fully-characterized in vivo, in vitro (including 3D), and in silico PDX models, for any oncology research.
How to Navigate Pediatric Cancer Drug Development
To help drug developers, who now must evaluate all oncology drugs for pediatric cancer before FDA submission, Charles River, as part of ITCC-P4, offers new and unique well-characterized pediatric PDX models. We can support your pediatric cancer drug development with our extensive expertise in oncology, from target ID to IND filing.
The ITCCP4 consortium was formed to foster pediatric cancer drug development with a growing number of well-annotated pediatric PDX models. Supported by an accompanying dataset consisting of molecular phenotyping and pharmacological characterization, these FDA RACE Act-compliant PDX models will reinforce the development of new drugs against children’s cancer.
Ensure Your Program is FDA RACE Act-Compliant
This webinar will address the problems of outstanding clinical trials for pediatric cancer drug development and provide alternatives for IND programs, highlighting pediatric PDX model collection.
Watch the webinar
Why Choose FDA RACE Act-compliant PDX Models
Charles River’s patient-derived xenografts (PDX) use tumor grafts established as models at low passage numbers (average of six passes removed from patient). They have not been grown in plastic or propagated as cell cultures.
Establishing xenograft tumor models from patient-derived tumor tissue (PDTT) at low passage is believed to conserve original tumor characteristics such as heterogeneous histology, clinical biomolecular signature, malignant phenotypes and genotypes, tumor architecture, and tumor vasculature. Based on this prevalent hypothesis, patient-derived xenografts are believed to offer relevant predictive insights into clinical outcomes when evaluating the efficacy of novel cancer therapies. As characteristics differ between adult and child cancer, pediatric PDX models are the most appropriate to showcase predictive insight for pediatric cancer drug development.
| Charles River Oncology Pediatric Offering | Traditional Market Offering |
|---|---|
| Unique FDA-compliant pediatric models | No pediatric model developed for RACE Act |
| Models already characterized and defined growth characteristics | Cells lines and tumor models not well established |
| Guidance from a dedicated Scientific Advisory Experts (SAS) team | Guidance at a cost |
| Support from Discovery to market (Safety and CDMO support) with consistency between expected results | Must rely on multiple partners to get to market |
Let our translationally relevant xenograft collection and scientific expertise aid you in your pediatric drug development.
Discover Our Pediatric Cancer Drug Development Models
PDX Pediatric Models
The constantly growing panel of PDX comprises:
- Brain tumors (40)
- Various sarcoma subtypes (92)
- Neuroblastoma (44)
- Other tumor types, including a growing number of liquid cancer models
CDX Pediatric Models
For your pediatric cancer drug development, we also developed pediatric CDX models; a panel of cell line derived models, which is available in addition to our RACE Act-compliant PDX portfolio. CDX models:
- Are pharmacologically and molecularly well characterized
- Support in vivo proof of concept studies and enable a direct translation from in vitro results to in vivo.
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Our Pediatric CDX Models include:
Tumor Type Cell Line All CCRF-CEM, CCRF-CEM-VCR, JURKAT, MOLT-3, MOLT-4, RCH-ACV AML Kasumi-1, MOLM-13, MV4-11, MV4-11 EF1A-iRFP713, Molm-13 EF1A-iRFP713, THP-1 Brain tumor D283 Med, Daoy Colon cancer DLD-1, DLD-1 CMV iRFP713 CML EM-2 Liver cancer HEP-3B, Hep G2 Lymphoma SU-DHL-1, Daudi, RAJI, RAMOS, Raji_NC Neuroblastoma CHP-212, IMR-32, KELLY, SH-EP, SH-SY5Y, SK-N-AS, SK-N-BE(2), SK-N-MC Renal cancer HEK-293 Sarcoma A-673, RD-ES, 678, Saos-2, U-2-OS, A-204, RH-30, TE671 Teratoma PA-1
Browse our Cancer Model Database
Support your in vitro, in vivo, and ex vivo studies with a user-friendly search, new model data (including HLA typing, growth curves, and tumor images), and multi-parameter search options for all tumor models (PDX and CDX) and cell lines.
Visit our database
Frequently Asked Questions (FAQs) About Pediatric Cancer Drug Development Related to the FDA RACE Act
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What is the RACE Act?
The RACE Act (Research to Accelerate Cures and Equity for Children Act) was approved by the U.S. Congress in 2017 and took effect on August 18, 2020.
The RACE Act is dedicated to pediatric cancer drug development. The act requires all new oncology therapeutics under consideration for approval by the U.S. Food and Drug Administration (FDA) to be evaluated for safety and efficacy in pediatric cancers if the treatment is directed at a molecular target relevant to pediatric cancer, including therapeutics with an orphan drug designation.
Alongside the FDA published a list of >200 relevant targets offering guidance for relevance in pediatric cancer.
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In our strategic drug development plan, when should our oncology drug be tested for the pediatric target?
Once the target is defined, it is worthwhile to check for feasibility in the context of pediatric cancer. This will define the future development of the drug. For additional details or to learn more about pediatric cancer drug development, contact us. We have a dedicated team to answer regulatory questions related to discovery and safety, that will be more than happy to guide you.
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What does well-characterized pediatric PDX models mean?
All models are accompanied by donor patient metadata, molecular data (WES, RNAseq, and methylation data), as well as pharmacology data (standard of care testing in a single mouse trial format), which is convenient for pediatric cancer drug development as pediatric tumors have different genomic drivers and phenotypes than adult tumors.
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Why are the pediatric models not available online in the Charles River Cancer Model Database?
The collection of RACE-compliant PDX Models can be found in the ITCCP4 database. Through the collaborative effort across multiple partners, born from the common desire to increase pediatric cancer drug development, it has been possible to populate this unique collection. To maintain and expand this PDX panel, the non-profit organization ITCCP4 gGmbH was founded on behalf of all stakeholders.
