NIH/NCI – Improving Outcomes for Pediatric, Adolescent and Young Adult Cancer Survivors (U01 Clinical Trial Required)

January 17, 2019 by

This Funding Opportunity Announcement (FOA) is associated with the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act of 2018 which authorizes the National Cancer Institute (NCI) to fund research to improve the care and quality of life of cancer survivors (including children and AYAs). This FOA is also associated with the Beau Biden Cancer MoonshotSM Initiative that is intended to accelerate cancer research in general.

The purpose of this FOA is to stimulate the development, testing, and/or scaling of innovative, feasible, and effective interventions to address adverse physical and psychosocial effects in survivors of pediatric and/or adolescent/young adult (AYA) cancer.  These interventions may be targeted to the patient or to the patient-caregiver dyad, and may include multilevel interventions delivered by providers, teams, communities, and/or care delivery systems.

This FOA requests applications from investigators to develop and test interventions that:

  • prevent, mitigate or manage adverse physical, psychosocial, and behavioral outcomes in pediatric and/or AYA cancer survivors; or
  • improve healthcare delivery.

Interventions addressing disparities in outcomes and/or access to needed care, or to address the needs of minority or medically underserved pediatric and/or AYA populations are of high priority.


Cancer is the leading cause of disease-related death past infancy among children in the United States (U.S.). For 2018 an estimated 10,590 new cases of cancer are expected to be diagnosed among children ages 0 to 14 years and 70,000 among AYAs between ages 15 and 39. Although survival rates vary depending on the type of cancer and other factors (e.g., age at diagnosis, race, socioeconomic status), major treatment advances in recent decades have resulted in more than 80% of children with cancer surviving 5 years or more.

The NCI Surveillance, Epidemiology, and End Results Program (SEER) estimates that there are more than 630,000 cancer survivors between the ages 0 to 39 in the United States, and studies suggest that over 60% of these survivors will suffer adverse physical, psychosocial, or behavioral outcomes months or years after completion of treatment. These disease and treatment-related adverse outcomes can significantly impair the quality of survival and the development of effective strategies to prevent, detect, mitigate, and manage these adverse effects is of high priority.  Adverse outcomes can vary in type (physical, behavioral, or psychosocial health effects) and severity (reduced quality of life or lifespan). Common adverse outcomes include premature/accelerated aging, cardiotoxicity, endocrine dysfunction, reproductive health issues, secondary cancers, financial toxicity, neurocognitive deficits, and other psychosocial issues. Interventions to address adverse outcomes within this population must also consider and surmount barriers to care as survivors transition from pediatric to adult care delivery settings.

Despite a growing awareness of the prevalence and severity of these adverse outcomes, efforts to prevent, mitigate, and manage them are hindered by a variety of factors, including barriers to receiving follow-up care and a paucity of evidence-based, tailored interventions to address the burden of morbidity.

Recognizing a need for pediatric and/or AYA survivorship research, Congress passed The Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act in May 2018.  The STAR Act was developed in collaboration with many childhood cancer organizations and is a comprehensive, bipartisan childhood cancer bill designed to advance pediatric and adolescent/young adult (AYA) cancer research and treatments, improve cancer surveillance, and enhance resources for survivors and their families. In addition to supporting improvements in cancer registry infrastructure and biospecimen research, the STAR Act authorizes the NCI to improve the care and quality of life of cancer survivors (including children and AYAs) by supporting the research solicited through this initiative.

Objectives and Main Requirements for this FOA

To enact STAR Act funding and address several of these research domains, this Funding Opportunity Announcement (FOA) invites cooperative agreement (U01) applications to develop interventions to prevent, mitigate, and manage adverse, long-term disease- and treatment-related outcomes and to improve healthcare delivery for survivors of pediatric and/or AYA cancers across their lifespans. The development of interventions that address health disparities and/or the needs and preferences of minority or other medically underserved populations will be of high priority in all research areas.

Overall Goals of this FOA

This FOA aims to stimulate the development, testing, and/or scaling-up of innovative, feasible and effective interventions to address the adverse physical, behavioral, and psychosocial effects in survivors of pediatric and/or AYA cancers in the following research areas:

  • Prevention, mitigation, and/or management of adverse outcomes; and
  • Improvement of healthcare delivery.

Scientific Scope

Applicants responding to this FOA should propose a clinical trial that aims to develop/refine and test an intervention to improve physical, psychosocial, or behavioral adverse effects or to improve healthcare delivery using any one of the following trial designs:

  • early phase studies to develop and preliminarily test a novel intervention;
  • phase II or phase III studies that focus on testing efficacy;
  • trials that examine intervention effectiveness in in real-world settings (e.g., in community settings, with community-based providers); and
  • dissemination and implementation studies (including hybrid effectiveness-implementation designs) examining the scale up and spread of empirically supported interventions in diverse healthcare settings.

Please note the following:

  • Interventions may be targeted to the patient or to the patient-caregiver dyad, and may include multilevel interventions delivered by providers, teams, communities, and/or care delivery systems.
  • Interventions proposed for development and testing should address established access barriers in this population, including geographic distance, literacy, functional deficits, and preferences to optimally engage.
  • Applications that leverage advances in digital and mobile health solutions and/or accelerate simultaneous integration of research-tested interventions into care delivery are strongly encouraged.
  • To be responsive to this FOA, applications must include meaningful proximal endpoints.
  • Studies that aim to define the mechanism of action (i.e., how and why an intervention works) should include considerations of the pragmatic implications as well (e.g., by describing how such explanatory knowledge can be used to amplify intervention effects or by planning allowance for an intervention to be modified for easier implementation, reduced intervention delivery cost, or greater acceptability).
  • Studies that test the effectiveness and/or dissemination/implementation of interventions in pragmatic trials should focus on interventions with demonstrated feasibility and efficacy.

Specific research directions for the two research areas of the FOA include, but are not limited to, the following topics.

Prevention/Mitigation of Adverse Outcomes:

  • Interventions to improve access and decision-making about the use of fertility services before and after treatment and/or to improve subsequent reproductive outcomes in pediatric and/or AYA survivors.
  • Test interventions designed to prevent or mitigate late effects (e.g., secondary malignancies; endocrine, cardiac or metabolic complications) or persistent/long-term effects (e.g., fatigue, cognitive dysfunction, functional impairment, frailty, and emotional distress) if implemented during or immediately following the completion of cancer-directed treatment.
  • Identify effective preventive strategies to mitigate adverse outcomes in known pediatric and/or AYA cancer survivor subgroups at higher risk for specific adverse effects or those who are most likely to receive the greatest benefit from early intervention.
  • Test rehabilitative and/or wellness interventions that aim to expand the reach, adoption, effectiveness, and/or maintenance of healthy lifestyle changes that have been shown to improve long-term outcomes for pediatric and AYA survivors. Such lifestyle changes may include addressing unhealthy body composition (overweight or underweight), poor diet quality, avoidance of tobacco, and excess alcohol use.
  • Identify interventions for caregivers/parents/spouses of pediatric or AYA cancer survivors that mitigate the adverse sequelae of their caregiving role, support the caregiver in meeting the demands of their role, and/or improve pediatric or AYA cancer survivor outcomes (e.g., physical and mental health, adherence to treatment).

Healthcare Delivery:

  • Understand the components, features, and comparative costs and value of models of care designed to optimize transitions in care. These aspects could include, for example, transitions between pediatric and adult care settings, specialty and primary care settings, or transitions based on geographic relocation.
  • Identify care delivery approaches that strengthen self-management, improve clinical outcomes, improve access, and achieve guideline concordant care, including appropriate surveillance for long-term and late treatment effects, and management of multimorbidity.
  • Evaluate the outcomes of risk-based healthcare delivery targeted to specific subgroups who warrant more intensive intervention and/or follow-up.
  • Test approaches to care coordination and communication that produce favorable effects on cancer control, late treatment effects, comorbidity, and health services outcomes. Approaches could include methods for digitizing and securely distributing health records and other provider-to-provider and provider-to-patient communications, for example. Outcomes might include cost, quality, and experiences of care or other relevant endpoints.

Interactions Among Awardees. Each U01 award will be based on an independent project. Nonetheless, all the U01 awardees will be expected to engage in trans-network interactions and participate in an annual investigator meeting to share knowledge, progress and findings with other awardees. It is anticipated that these interactions and sharing experiences may facilitate rapid translation of study results.

Non-responsive Applications. Applications with the following characteristics will be considered nonresponsive and will not be reviewed:

  • Applications that do not propose the development and testing of interventions addressing physical, psychosocial, and/or behavioral adverse effects in survivors of pediatric and/or AYA cancers in at least one of the two FOA research areas defined above.
  • Applications proposing interventions to address short-term, transient adverse effects.
  • Applications proposing drug development studies.

Deadlines:  March 15, 2019; January 3, 2020 (letters of intent due February 15, 2019; December 3, 2019)

Filed Under: Funding Opportunities