OPPTB Research Programs

The following information describes the branch's research programs and program areas.

Multiple knowledge gaps regarding the safe and effective use of pharmacological and therapeutic agents in children and in pregnant and lactating women have resulted in inadequate labeling and frequent off-label use of drugs in these populations. For instance:

  • Pregnant, lactating, and postpartum women undergo complex physiological changes impacting multiple organ systems, which may increase the risk of pregnancy-related conditions or complications and aggravate pre-existing conditions requiring pharmacotherapeutic intervention.
  • Time-dependent changes in drug-metabolizing enzymes, transporters, and other components of the drug metabolism pathway can alter therapeutic absorption, distribution, metabolism, and excretion (ADME) throughout the pregnancy and lactation periods.
  • Pediatric ontogeny can affect the pharmacokinetic processes of therapeutic ADME at every developmental stage, from the developing fetus to adolescence, affecting therapeutic effectiveness and safety.
  • Factors such as inter-individual heterogeneity, pharmacogenomic (PGx) and epigenetic characteristics, and environmental influences, among others, can influence drug effectiveness and safety in these populations.
  • Ethical and safety considerations, along with fewer available studies, historically, have limited the ability of pregnant and lactating women and pediatric populations to participate in traditional, randomized clinical trials.

Within this context, new approaches are required to assess and predict therapeutic effectiveness and safety. Further, research on novel therapeutics, innovative tools and models, and other technologies that promotes precision medicine and elucidates the underlying mechanisms of drug action are necessary to promote pharmacological and therapeutic discoveries for pediatric, obstetric, and lactating populations.

All OPPTB programs address these underlying knowledge and evidence gaps.

Program Officials: Antonello Pileggi and Perdita Taylor-Zapata (Pediatric Populations); Zhaoxia Ren (Obstetric Populations); Camille Fabiyi (Lactating Populations)

OPPTB supports a range of pharmacokinetic (PK), pharmacodynamic (PD), PGx, and other research to help ensure safety and effectiveness of medications and therapeutics for children and pregnant and lactating women in the prevention, treatment, and management of diseases and disorders.

Specific areas of research interest within pediatric and pregnant and lactating populations include:

  • Designing preclinical and nonclinical models, tools, and other technologies to assess or predict therapeutic safety or efficacy
  • Identifying and optimizing novel biological (e.g., genome editors, exosomes, tissue constructs) and chemical therapeutics
  • Repurposing and expanding the use of existing therapeutics
  • Developing formulations and drug delivery systems appropriate for use in populations of interest
  • Elucidating the impacts of ontogeny and physiological changes in therapeutic absorption, distribution, metabolism, and excretion (ADME), and on efficacy and safety
  • Supporting pharmacometric, PGx, and multi-omics studies to advance precision medicine
  • Identifying molecular targets and developing targeted medications for diseases that affect populations of interest
  • Documenting molecular or clinical biomarkers to support precision prescribing
  • Developing and testing implantable therapeutic constructs and devices, and diagnostic and point-of-care devices
  • Designing innovative clinical trials for special populations
  • Creating and evaluating novel drug safety prediction methodologies using real-world data

The branch funds and leads many projects and initiatives within this program area, including, but not limited to:

More recently, the branch issued a Request for Applications (RFA) to initiate a Transporter Elucidation Network, which will address key knowledge gaps in functional transport of nutrients and drugs to the developing fetus and infant through a focus on human placenta, lactating mammary gland, and developing gut and blood-brain barrier. The network will include centers funded by NICHD and the National Institute of Drug Abuse that will work both independently and in concert to generate knowledge and resources and advance the field. Visit RFA-HD-23-003: Elucidation and Validation of the role of Transporters in the Placenta, Lactating Mammary Gland, Developing Gut, and Blood Brain Barrier (UC2 Clinical Trial Not Allowed) for details.

Program Official: Katie Vance

Translational research, which seeks to transform basic science and clinical research discoveries into results that have direct clinical and health impacts, on pharmacology and therapeutics for fetal, neonatal, pediatric, pregnant, and lactating populations, also lags compared to behind research for adult and non-pregnant or non-lactating groups.

Novel tools, methodologies, and other resources are essential to accelerating the translational process, particularly those with broad applicability to the scientific and clinical communities who work with pregnant and lactating women and children, and with those with physical or intellectual disabilities in these groups.

In 2023, OPPTB issued funding announcements (PAR-23-130: R01 Clinical Trial Optional, and PAR-23-131: R21 Clinical Trial Optional) to support Translational Research in Maternal and Pediatric Pharmacology and Therapeutics projects.

Grants funded through these announcements will:

  • Advance precision medicine in pregnant/lactating women, children, and adolescents through the development of novel, generalizable tools, models, and other technologies that could have a direct clinical or health impact.
  • Enhance the understanding of the underlying mechanisms of drug action, including the role of pediatric ontogeny and the dynamic physiological changes that occur during pregnancy, lactation, or the post-partum period.
  • Discover and develop novel therapeutics or enhance the usage of existing drugs or drug repurposing for safer and more effective medications in pregnant, lactating, and postpartum women, neonates, and children.

Program Official: Antonello Pileggi

Regenerative medicine focuses on restoring tissue or organ function impaired by genetics, injury, toxicity, or other causes. Progress in regenerative processes, such as stem cell biology, tissue engineering, and biomaterials development, during the last three decades has created new opportunities to improve prognoses and quality of life for individuals, including those whose treatment options are limited or exhausted.

The branch supports research in regenerative medicine to create and enhance medical devices to they are safe and effective for children and pregnant and lactating women.

Three-Dimensional (3D) Printing of Implantable Devices

The use of 3D printing technology in regenerative medicine allows for “bioprinting”—the creation of biological, cellular, and tissue-based products. Bioprinting enables innovative approaches to regenerative/reparative medicine processed that were previously cumbersome. Such constructs are highly desirable, particularly for restoring function of body structures that require adaptation and remodeling, such as devices for pediatric populations, as well as those for pelvic and reproductive organs in obstetric and gynecologic populations.

Topic areas for this research include:

  • Using bioprinted implants to address specific pediatric clinical problems, in which the device needs to adapt to the child’s growth to reduce, minimize, or prevent the need for repeated corrective interventions
  • Developing and testing 3D-printed biodegradable scaffolds appropriate to specific clinical problems
  • Generating regulatory evidence for using bioprinted tissue constructs to treat gynecologic, obstetric, and pediatric diseases and disorders
  • Conducting small-scale safety and feasibility pilot clinical studies (e.g., first-in human studies)

In 2021, the branch co-led the Advancing Bioprinting and Regenerative Medicine Solutions for Obstetric, Gynecologic, and Pediatric Applications Workshop, as well as a crowdsourcing effort to further expand discussions and knowledge in this area.Follow-up efforts, with a focus on bioprinting and regenerative medicine, are ongoing. Most recently, the branch released RFA-HD-23-004: Bioprinted Tissue Constructs for Obstetric, Gynecologic, and Pediatric Applications (R01 Clinical Trial Optional) to address these areas.

Public-Private Partnership (PPP) to Support Pediatric Medical Device (PMD) Development and Commercialization

The current, persistent lack of access to medical device options specifically designed and approved for pediatric populations demands that all sectors work together in new ways to meet this urgent need. NICHD—through OPPTB—and the National Institute of Biomedical Imaging and Bioengineering (NIBIB) are working with the Foundation for the National Institutes of Health PMD Design Phase external link to establish a PPP that consolidates the national PMD ecosystem and to develop strategies that reduce risk in development and commercialization of pediatric medical devices. 

NICHD and NIBIB are using an Other Transaction Agreement (OTA) structure to facilitate the establishment of this PPP, which will include agencies, regulators, and public health officials at all levels of government, life sciences companies, non-governmental organizations, academic biomedical research institutions, and other groups and entities that may not normally be involved with pediatric device research. Additional information about the PPP efforts are available at:

  • OTA-23-001: Research Opportunity Announcement (ROA): Designing a Public-Private Partnership to Support Pediatric Medical Device Development and Commercialization (PDF 217 KB)
  • NOT-HD-23-018: Notice of Information: NICHD to issue a sole source award to support the Designing a Public-Private Partnership to Support Pediatric Medical Device Development and Commercialization Project
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