ICON Public Company (ICLR) Status Update summary

Key insights and background

• Pregnant individuals are significantly underrepresented in clinical trials, largely due to ethical concerns, historical precedent, and regulatory barriers, despite widespread medication use during pregnancy.

• Only a small fraction of drugs have safety data for use in pregnancy or lactation, leading to knowledge gaps and public health concerns.

• Surveys show high awareness of clinical research among women, but participation remains low, with misconceptions and lack of information as major barriers.

• Motivators for participation include desire for safety information, reimbursement, and trusted communication from healthcare providers.

Historical and regulatory evolution

• Exclusion of pregnant women from trials was driven by past tragedies (e.g., thalidomide) and reinforced by regulatory and ethical caution.

• Recent years have seen a shift, with advocacy groups and global regulators pushing for inclusion, recognizing the need for safety and efficacy data.

• Authorities like the FDA, EMA, WHO, and ICH have issued or are developing guidelines to support inclusion and data collection for pregnant and lactating individuals.

• The FDA and other agencies are piloting frameworks and demonstration projects to optimize post-marketing safety data and inform future guidance.

Challenges and consequences

• Pregnant individuals face limited treatment options, anxiety, and often make medication changes without professional guidance due to lack of reliable information.

• Exclusion from trials leads to off-label use, dosing uncertainties, and potential harm, while also stalling innovation in obstetrical therapeutics.

• Diversity in clinical trials remains a challenge, but recent FDA guidance emphasizes the need to include pregnant and lactating individuals as underrepresented populations.