NIH – Translational Research in Maternal and Pediatric Pharmacology and Therapeutics (R01 Clinical Trial Optional)

September 18, 2020 by


The purpose of this funding opportunity announcement (FOA) is to encourage applications that will perform basic, translational and clinical research to (1) advance the understanding of underlying mechanisms of drug action; (2) discover and develop novel therapeutics; (3) enhance the usage of existing drugs or drug repurposing for safer and more effective treatment for pregnant women, lactating women, neonates and children. The overall goal is to improve drug safety and efficacy for maternal and pediatric precision therapeutics.


Pregnant women and children are populations with dynamic physiology and pharmacology at increased vulnerability to drug toxicities. Physiological changes during pregnancy may affect the functions of different organ systems and increase the risk of pregnancy related conditions/complications and aggravated pre-existing conditions in pregnant women which require pharmacotherapeutic intervention.

In pregnant women, pregnancy induced physiological changes can alter the pharmacokinetic processes of drug absorption, distribution, metabolism, and excretion throughout the entire pregnancy and also impact the drug pharmacodynamic properties. For example, the increase in glomerular filtration rate leading to increased drug clearance during pregnancy generally requires a higher dose of drugs in order to achieve the desired therapeutic effects. Sometimes, the empirically increased doses can lead to unexpected adverse effects to both the pregnant women and their developing fetus as most drugs that enter the maternal circulation can cross the placenta through passive diffusion and may cause harm to the developing fetus. In addition, drugs can also get into breast milk during lactation and may adversely impact the wellbeing of neonates and infants. Understanding of pharmacokinetic and pharmacodynamic properties during pregnancy and lactation is important for optimized treatments to reduce maternal morbidity.

In children, growth and development can also affect the pharmacokinetic processes of drug absorption, distribution, metabolism and excretion at a different developmental stage from neonates to adolescent. These changes can affect the drug safety and efficacy.

Moreover, many other factors such as inter-individual heterogeneity, pharmacogenomic and epigenetic characteristics, hormones, microbiomes, environment, etc.can also impact drug metabolism and response that affect drug efficacy and toxicity. Understanding underlying molecular mechanism of drug action and how the factors influencing drug disposition and response is of paramount importance to provide safer and more effective therapeutics in pregnant, lactating and pediatric patients and prevent potential harmful effects to the pregnant and lactating women and their unborn and breastfeeding babies and children.

In past decades, significant advances in understanding the molecular and genetic basis of diseases, and identification and validation of various disease biomarkers, together with the expansion of robust technologies and tools have enabled the identification of new molecular targets and pathways for many diseases and conditions. These discoveries have created new avenues for drug research and drug development leading to find novel treatment for many diseases, but mostly for adult and non-pregnant populations.

Despite progress made in clinical pharmacology studies in pregnant women and children in recent decades, knowledge deficits remain in the understanding of molecular mechanism of drug actions in pregnant and lactating women and in neonates and children. How pregnancy and pediatric ontogeny influence pharmacokinetics, pharmacodynamics and pharmacogenomics remains to be further explored. Moreover, drug discovery and development in pregnant and lactating women and in neonates are almost lacking. Clearly, there is a great need of continued basic, translational and clinical research to advance our knowledge in safe and effective precision therapeutics to address unmet and emerging clinical needs for these special populations.

Key Dates:

Open Date (Earliest Submission Date): November 10, 2020
Letter of Intent Due Date(s): 30 days prior to application receipt date
Application Due Date(s): December 10, 2020, April 9, 2021, August 10, 2021, December 10, 2021, April 11, 2022, August 10, 2022, December 9, 2022

URL for more information:

companion R21:

Filed Under: Funding Opportunities