Description:
369 Development of Pediatric Cancer Drug Delivery Devices
Fast-Track proposals will be accepted.
Direct-to-Phase II will not be accepted.
Number of anticipated awards: 2-4
Budget (total costs, per award):
Phase I: $300,000 for 9 months;
Phase II: $2,000,000 for 2 years
Page 100
PROPOSALS THAT EXCEED THE BUDGET OR PROJECT DURATION LISTED ABOVE MAY NOT BE FUNDED.
Summary
Drug delivery systems are continually advancing and greatly assist the capabilities of cancer therapies and cancer survival. Yet, the pediatric population has not benefited, to the same extent as adult populations, from these advances in cancer therapy delivery device design and technology. A tailored dosage system for delivering cancer therapeutics to pediatrics is required. In addition to biological response differences, pediatrics vary anatomical with age and therefore require cancer therapy delivery devices and ports to be tailored for compatibility with anatomical dimensions of the patient. This topic is focused on drug delivery devices as opposed to drug delivery materials and vehicles. For example, in the case of central nervous system tumors—implantable drug delivery ports, pumps and ommaya reservoirs required for drug delivery to the cerebrospinal fluid often pose many issues for pediatric patients. Issues of device displacement, catheter migration and catheter fracture have all been reported and are primarily due to anatomical miss-compliance. Additionally, complications such as infection and insufficient wound healing are common because of devices with large profiles designed for use in adults. Furthermore, developmental and behavioral characteristics of young children should also be considered when designing a tailored device for pediatric patients – this includes changes in anatomy size for long-term implantable devices and mobility needs of children at different stages of development.
There is a need for versatile, efficient cancer therapy delivery devices that meet the needs of pediatric populations. This solicitation aims to aid the development of appropriate cancer therapy delivery devices that reflect pediatric patient specific designs and dosage parameters for pediatrics.
Project Goals
The purpose of this announcement is to assist the development of cancer drug delivery systems compatible to the needs of pediatric patients. This topic includes pediatric focused therapeutic targets within acceptable dosages suitable for pediatric patients and/or drug delivery systems designed to suit the needs of pediatric anatomical dimensions. Successful applicants will develop technologies to aid the administration of cancer therapies to pediatric patients, taking into account pediatric specific issues which include but are not limited to: dosage limitations, size restraints, comfort level and mobility. Adaption of currently available delivery devices for the pediatric population is also encouraged. One example is in the treatment of pediatric retinoblastoma where there have been some recent advances in the development of an episcleral device for delivering localized therapy to the retina and choroid, which has been tested in rabbits and is now proposed for testing in pediatric patients. This solicitation is not limited to cancer type or site, yet, justification of the need for pediatric-specific design parameters is encouraged. The offeror is required to outline and indicate the clinical question and unmet clinical need that the pediatric drug delivery device will address. This solicitation is not intended for drug formulation or nano-delivery systems; instead it is focused on delivery mechanisms and devices. In Phase I, offerors should demonstrate the proof of concept for the device proposed. Phase II projects will validate the device in the clinical setting.
Expected Activities and Deliverables
Phase I Activities and Expected Deliverables
• Select cancer type(s), site(s) and cancer drugs for the development of delivery device with adequate justification
• Design and develop a prototype of a drug delivery device that is
• Suitable for the anatomical restrictions of pediatric patients.
• Suitable for the dosage limitations of pediatric patients.
• Demonstrate preliminary proof-of-concept of the device in a suitable animal model.
• Develop the required specifications necessary to make the device clinic ready.
• Demonstrate understanding of the requirements to file a regulatory application for the device
Phase II Activities and Expected Deliverables
Page 101
• Build a device according to the specifications developed in Phase I.
• Optimize the device design and performance for a clinical setting, and
• Show the feasibility of this novel approach/technique that will fit in with current clinical workflow.
• Demonstrate the safety and efficacy of the device in relevant animal models as required by FDA.
• Develop and execute an appropriate regulatory strategy. If warranted, provide sufficient data to submit a regulatory application to obtain approval for clinical application.
• For offerors that have completed advanced pre-clinical work, NCI will support pilot human trials.
Page 102
NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES (NCATS)
The mission of the National Center for Advancing Translational Sciences is to catalyze the generation of innovative methods and technologies that will enhance the development, testing, and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions. For additional information, please visit our home page at http://www.ncats.nih.gov.
It is strongly suggested that potential offerors not exceed the total costs (direct costs, facilities and administrative (F&A)/indirect costs, and fee) listed under each topic area.
