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FDA works to advance real-world data collection in pregnancy and lactation

Sara Head, PhD, FDA Office of Women's Health

Regulatory NewsRegulatory News
Posted 16 May 2022 | By Mary Ellen Schneider 

FDA works to advance real-world data collection in pregnancy and lactation

3016 The US Food and Drug Administration (FDA) is working on ways to facilitate greater use of real-world data (RWD) to help understand the safety and efficacy of medications used during pregnancy and lactation.
Despite the widespread use of prescription and over-the-counter medications by pregnant and lactating women, there is often little or incomplete information to guide decisions about starting and stopping medications or changing doses.
“Although the lack of scientific data prevents us from understanding the true risk for most medications in pregnancy, there is research that suggests women overestimate the risk of taking medication and decide against use when their own research yields conflicting information on safety,” Sara Head, PhD, of the FDA Office of Women’s Health, said during a public workshop hosted by Johns Hopkins Center of Excellent in Regulatory Science and Innovation (JH-CERSI) and the FDA Office of Women’s Health.
The workshop comes as the FDA has been signaling how it would use RWD in making regulatory decisions. In 2021, the agency issued draft guidance documents on RWD use with clinical registries, RWD use with electronic health records and claims data, and how RWD could be incorporated into clinical study designs (RELATED: FDA issues draft guidance on study design using real-world data, Regulatory Focus 09 December 2021; FDA offers draft guidance for registries as RWD, Regulatory Focus 29 November 2021; FDA issues draft guidance on RWD sourced from EHRs, claims data, Regulatory Focus 29 September 2021).
Guidance on pregnancy & lactation
“It’s important to note that we have long been relying on RWD to inform the safety of drugs in pregnancy and lactation, long before it was called RWD. We can think of RWD as pharmacovigilance and observational studies,” said Leyla Sahin, MD, acting deputy director for safety in the division of pediatrics and maternal health in the Office of New Drugs in FDA’s Center for Drug Evaluation and Research (CDER).
The agency addressed many issues related to RWD in pregnancy and lactation in two draft guidances published in 2019, according to Sahin (RELATED: Studying Drugs in Pregnancy and Nursing Women: FDA Issues Draft Guidances, Regulatory Focus 08 May 2019).
The Postapproval Pregnancy Safety Studies draft guidance is currently being revised based on public comments. The guidance includes discussion of pharmacovigilance, pregnancy registries, electronic health record data and surveillance programs. It also covers complementary studies, which can provide data to augment information collected through pregnancy registries.
The draft guidance addresses considerations for when exposure in pregnancy is expected to be uncommon, but there is still a need to collect pregnancy safety data, such as for a rare disease. In these situations, an adequately powered pregnancy registry or database study may not be feasible, and the guidance offers an alternative study method called a “pregnancy surveillance program.”
“Since this guidance was published, we have encountered confusion related to this terminology,” Sahin said. “And so, the terminology is currently under discussion, and we have now moved to using the terminology, ‘descriptive pregnancy safety study.’”
This study method involves systematic collection of pregnancy-specific data, includes prospective and retrospective data collection, and may be conducted as part of a disease registry.
The Clinical Lactation Studies draft guidance, published at the same time, is also undergoing revisions based on public comments. It recommends conducting lactation studies for drugs expected to be used by lactating individuals and discusses study design and analysis considerations. “Lactation studies, even those that are conducted as opportunistic studies, may not be considered RWD per se, but do have an RWD component in that the milk samples are collected in infants under real world conditions,” Sahin said. “RWD, such as use data and safety data, can be collected to support data that are generated from lactation studies.”
PRGLAC initiatives
FDA was also involved in the national Task Force on Research Specific to Pregnant and Lactating Women (PRGLAC) to address gaps in knowledge and research regarding safe and effective therapies for pregnant and lactating women. The task force submitted two reports to Congress in 2018 and 2020.
The recommendations relevant to RWD include:
  • Increase the quantity, quality and timeliness of research involving therapeutic products used by pregnant and lactating women.
  • Optimize pregnancy registries to move away from single-product, single-company pregnancy registries and expand the use of disease-based registries.
  • Implement a proactive approach to protocol development and study design to systematically develop a plan for timely collection of data, including pregnancy safety data.
  • Design health record systems to link mother and infant records and leverage large studies and databases. 
“RWD plays an important role in filling the data gap as most pregnancy and lactation-related safety data are collected in the post-approval setting,” Sahin said. “We can’t rely on a single study to optimize safety assessment during pregnancy. We need multiple data streams.”
PDUFA VII commitments
Pregnancy safety is also a component of FDA’s commitments in the proposed reauthorization of the Prescription Drug User Fee Act (PDUFA VII) for Fiscal Years 2023-2027. Under the plan, FDA would develop a framework describing how data from different types of post-market pregnancy safety studies might be optimally used. By 30 September 2023, the agency would hold a public workshop on post marketing safety studies in pregnant women and publish a report describing the proposed framework by 30 September 2024.
Under the plan, the agency would conduct five demonstration projects evaluating various study designs, both when exposure in pregnancy is common and when it is rare. The commitment goals set 30 September 2027 as the deadline to update the proposed framework and develop guidance or policies to implement a standardized process for determining the necessity for and type of pregnancy post-marketing studies.


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