A New Era in Advancing Care for Pregnant and Breastfeeding Patients

One major challenge facing the clinical research ecosystem is not whether, but how to study drug safety and efficacy in pregnant and breastfeeding populations. A growing number of organizations—including the ICH E21 Expert Working Group (EWG) and TransCelerate’s Interpretation of Pharmacovigilance Guidances & Regulations  (IGR PV) initiative—are diligently seeking ethical and responsible solutions to protect these specific populations. 

The lack of data from clinical trials on the use of medicines in pregnant and breastfeeding individuals can lead to gaps in understanding the best care for these patients. Unfortunately, excluding pregnant and breastfeeding women from clinical trials prevents prescribers and patients from accessing evidence-based information essential to making informed medication benefit-risk assessments and appropriate medical decisions.  

This crucial issue was explored in a previous article in TransCelerate’s IGR PV blog series, but in this final installment, we will:

• recap some of our work with the ICH E21 EWG (Inclusion of pregnant and breastfeeding Individuals in clinical trials) and the IGR PV pregnancy and breastfeeding team;
• discuss freely available solutions created for the industry’s use; and
• share what is on the horizon for both groups.

Improving intricate issues

Many challenges make it difficult to gather safety information that can help guide medication benefit-risk assessments for pregnant and breastfeeding populations. Among the most common are:

• Significant differences in countries’ regulatory guidances, making it challenging to include pregnant women in global studies, even among those aiming to advance inclusivity.
• Inconsistencies around post-marketing study designs. Although registries and enhanced PV are options for safety monitoring, there is presently no global standard for collecting evidence in pregnant and breastfeeding populations that can generate evidence-based information in a timely manner.

Therefore, when the IGR PV team was assembled in 2017, its first order of business was to review global drug safety regulations and guidelines for key topics. To bolster regulatory harmonization, the team created a comprehensive landscape assessment that uncovered numerous ambiguities and discrepancies. Several articles were subsequently published, summarizing key findings and calling for the harmonization of PV regulations for post-marketing studies. 

Similar to the IGR PV team, the ICH E21 EWG also takes a patient-centric approach to improving data capture to support the use of medications during pregnancy and breastfeeding. When the ICH E21 EWG was formed in 2022, it used the IGR PV team’s landscape assessment and its companion document titled Points to Consider Concerning the Use of Medicines in Pregnancy Throughout the Product Lifecycle (Based on the Regulatory Guidance Across the Globe) to shape and inform its final concept paper.

In a way, it can be said that the IGR PV team’s landscape assessment helped set the stage and corroborate the need for the larger scope of work still in progress with the ICH E21 EWG.

Enabling better patient care

After several years of commitment, the IGR PV pregnancy and breastfeeding team will complete its work at the end of 2024. However, before its completion, it plans to publish one more article: Post-approval Activities Providing Data on the Safety of Medication Use During Pregnancy and Lactation—A TransCelerate Perspective.

The ICH E21 EWG will carry the torch into 2025, pressing forward with efforts to promote global harmonization in how regulations treat the inclusion of pregnant and breastfeeding individuals in clinical trials. The ICH 21 EWG hopes to get a sign-off on its technical document in early 2025, with an initial draft ready for public consultation later in the year.

Now that the group is past the “concept” phase and starting to dive into the details, it is beginning to have healthy debates about how to achieve its objectives. The ICH E21 EWG includes many different organizations with various perspectives from all over the world. Although everyone has patient safety at the center of their work, there are various ways to capture medication safety data on pregnant and breastfeeding populations. Despite the acknowledged complexities, the enthusiasm for fostering alignment remains strong.

Ultimately, both the ICH E21 EWG and the IGR PV team want to enable better patient care for pregnant and breastfeeding populations. It’s been an immense honor for us to work with so many amazing people dedicated to encouraging innovation and cooperation within the biopharmaceutical ecosystem—all with the primary objective to enhance both the safety and efficacy of medications for this patient population in a more efficient manner.

To learn more about the ICH E21 EWG, visit the ICH efficacy guidelines page.

To access TransCelerate’s entire suite of IGR PV solutions and resources—including those for regulations governing the use of medicines in pregnancy and breastfeeding—visit our IGR PV solutions page.

About the authors:

• Maria Fernanda, MD, is the Associate Vice President – Oncology, Safety, at Eli Lilly and Company. She focused much of her professional life on Obstetrics and Gynecology before joining the Industry. With vast experience in drug safety, she actively participates in the work of TransCelerate’s IGR PV Topic 7 team and the ICH E21 Expert Working Group.
• Dorina Bischof, MD (OB-GYN), is Senior Vice President, Global Head of Patient Safety and Pharmacovigilance at Novartis. Since November 2022, she is the rapporteur for the ICH E21 Expert Working Group.