Background:
The Interpretation of Pharmacovigilance Guidances & Regulations Initiative shares expertise to more efficiently and effectively meet the intent of ambiguous regulations/guidances and Health Authority operational expectations around the world. Secondarily, the initiative will take opportunities to raise Health Authority awareness of the real-life impact of ambiguous regulations/guidelines. While addressing these aims, the initiative will consider the highest priority to be the continued enhancement of patient safety.
Current Topics:
Given the relatively sparse regulatory guidance addressing pharmacovigilance compliance to help pharmaceutical companies deal with the growing volume and complexity of safety data potentially available from Digital Health ecosystems, there is a need to clarify regulatory information on digital health, including current terminology and definitions, relevant to PV to increase clarity and communication within stakeholders. Understanding the global landscape of existing/evolving regulation and guidance (including gaps) on digital health and the relevant context for pharmacovigilance is essential for this evaluation and to enable regulatory compliance (both current and future). This initiative will take a phased approach to first identify key elements of digital health ecosystem relevant to patient safety and PV to understand the current PV regulatory landscape for digital health in post-marketing settings. Based on the insights obtained and with a patient-centric approach, propose focus areas for current and future guidance, frameworks and solutions to facilitate use of digital health technologies’ outputs in post-market pharmacovigilance in compliance with HA expectations.
Status: In Progress
Guidance: A list of guidances and regulations will be provided with released solutions.
Agency: Multiple
Guidance Date: Multiple
Deliverables:
| Asset Type | Title | Description |
|---|---|---|
 | DHT PV Considerations Guide | A tool designed to facilitate the user’s identification and independent assessment of various critical considerations that may significantly affect pharmaceutical operations when introducing new DHTs in the ecosystem. Its core functionality revolves around a detailed questionnaire that explores certain key aspects of DHT, such as Purpose, Technology, Regulatory, Data, and Deployment. Users input relevant data into the tool, which then processes this information to deliver a multifaceted set of considerations. Note: This tool is currently incompatible with macOS. |
| Survey of Digital Health Use: PV Challenges & Opportunities | A summarization of Member Company survey results which provide benchmarking information that captures the current landscape of digital health in the pharmacovigilance field among survey participants, pinpointing opportunities for growth. It also highlights challenges with digital health use, providing insights to inform future deployments. |
| Digital Health Landscape Assessment | A robust analysis of digital health guidance, regulations, and industry position papers across key global markets, highlighting the role and impact of digital health in pharmacovigilance. |
 | Digital Health Glossary (PDF) Digital Health Glossary (Excel) | A catalogue of the common vocabulary and synonyms to aid in understanding terms directly related to digital health to increase clarity, communication, and better decision-making in this space. |
| Digital Health Reference Resource Library | A catalogue of identified regulations and guidelines to aid in understanding what is driving digital health in selected countries. |
In 2019, 4.2 million babies were born in the European Union. In the same year, the number of live births in the United States was around 3.75 million. While the mothers may have needed to take medicines during their pregnancies (drug utilization studies estimate that between 44% and 99% of pregnant women will take medication during pregnancy), only very few are approved for use during pregnancy and breastfeeding.
Understanding the global landscape of existing and evolving regulations & guidance on monitoring the safety of medicines taken during pregnancy and breastfeeding is a challenge for researchers, be it in industry or academia. Addressing this challenge is precisely what the Interpretation of PV Guidances & Regulations (Pregnancy and Breastfeeding Topic) working team is currently doing. Using a phased approach, the team has started to analyze the current regulatory ecosystem (and relevant initiatives) in focused territories, covering drug safety regulations and guidances addressing pregnancy and breastfeeding from early clinical development to post-marketing settings. Based on the insights obtained, in the second phase, the team will propose solutions to help pharmaceutical companies better understand and comply with ambiguous or inconsistent regulatory requirements or guidance. These solutions will also help companies meet Health Authorities’ expectations by more effectively and efficiently operationalizing the development of applicable processes and tools to monitor medication exposures and safety events in pregnant and breastfeeding women. Ultimately, this initiative aims to help women, their partners, and health care professionals make informed decisions when medical treatment is required.
Status: In Progress
Guidance: A list of guidances and regulations will be provided with released solutions.
Agency: Multiple
Guidance Date: Multiple
Deliverables:
| Asset Type | Title | Description |
|---|---|---|
| Regulations Governing Medicines for Maternal and Neonatal Health: A Landscape Assessment | This peer-reviewed publication summarizes key findings from a landscape assessment of regulations and guidances on the use of medicines in pregnancy and breastfeeding issued by health authorities in various territories (including the Americas, Europe, Africa, and Asia Pacific) and outlines relevant initiatives undertaken by health authorities, academic institutions, industry consortia, and public-private organizations. |
| Call to Action: Harmonization of Pharmacovigilance Regulations for Post-marketing Pregnancy and Breastfeeding Safety Studies | The article focuses on post-marketing pregnancy and breastfeeding safety studies, raising awareness of the industry perspective on challenges due to the lack of harmonization and gaps in current PV regulations. It is intended to increase awareness among the industry and health authorities and calls for global legislation harmonization. |
| Improving Safety and Efficacy of Medicines for Pregnant and Breastfeeding People | An infographic introducing: 1) Why medication use in pregnant people is so important. 2) The right of pregnant people and their partners to have access to medicines with evidence-based information on the safety and efficacy of their use during pregnancy & breastfeeding. 3) How the TransCelerate team is focusing on raising awareness of current regulatory requirements, the industry perspective on challenges, and considerations when developing medicinal products to be used for maternal health. |
| Points to Consider for Pregnancy throughout the Product Lifecycle (Based on the Regulatory Guidance across the Globe) | Considerations based on regulatory requirements, when including pregnant women in clinical trials through post-marketing studies. Aimed at raising awareness of current regulatory requirements, industry perspective on challenges, and considerations when developing medicinal products to be used for maternal health. |
| Initiatives & Regulatory Landscape Assessment Output | The Landscape Assessment summarizes the current regulatory ecosystem (and relevant initiatives) in selected territories, covering drug safety regulations and guidances addressing pregnancy and breastfeeding from early clinical development to post-marketing settings. |
| Regulatory Landscape Comparison by Regulation Topic | Comparison of in-scope guidances and regulations in the selected territories, against ICH and CIOMS pregnancy and breastfeeding provisions. |
Completed Topics:
These topics are complete. No new solutions or updates are planned.
Guidance: See list of guidances considered in “A Framework on the Presentation of Safety Risks Throughout the Product Lifecycle” below.
Agency: Multiple
Date: Multiple
Approach:
- The topic team reviewed definitions of risks in key guidances (GVP and ICH).
- The topic team then provided clarity through a Framework that describes how to present risks in key safety documents throughout the product lifecycle in a manner that complies with the relevant guidances.
Value:
This topic’s objective was to support the presentation of risk information in accordance with Health Authority expectations, through developing a Framework that maps out regulatory requirements and operational considerations for presenting risks at the various product lifecycle stages.
Deliverables:
| Asset Type | Title | Description |
|---|---|---|
| A Framework on the Presentation of Safety Risks Throughout the Product Lifecycle | A Framework including a comparison of regulatory requirements for presentation of risks across the guidelines and phases of the product lifecycle, along with operational considerations on how to present risks in key regulatory/safety documents. |
 | Presentation of Risks Overview Video | An introduction and high level overview of what can be found in “A Framework on the Presentation of Safety Risks Throughout the Product Lifecycle.” |
Implementation Experience:
As solutions are used throughout the R&D ecosystem, TransCelerate receives feedback related to solution implementation experiences. Below is a featured example for this topic:
Anyone can submit an experience through the Engage with Us page and in select cases, we share the experiences on Our Impact page in a blinded way.
This topic’s objective was to support patient safety commitments by identifying various approaches to meet Health Authority expectations for safety issue notifications in an effective and efficient manner.
Guidance: See a list of guidances considered in the Safety Issue Notification Guide below
Agency: Australian Therapeutic Goods Administration, Health Canada, Ministry of Health New Zealand, Risk Management and Drug Information Department (Israel), Saudi Food and Drug Administration, SwissMedic, and Turkish Ministry of Health
Guidance Date: See a list of dates for the guidance considered in the Safety Issue Notification Guide below
Deliverables:
| Asset Type | Title | Description |
|---|---|---|
| Safety Issue Notification Guide | A guide to aide understanding of safety issue notification guidances for in-scope countries. |
| Webinar: Safety Issue Notification Guide | A webinar presentation where TransCelerate team members provide an overview of the Safety Issue Notification Guide. |
This topic’s objective was to enhance patient safety by facilitating adherence to unclear regulatory guidance calling for applicants to take a more holistic view of combination products.
Guidance: Postmarketing Safety for Combination Products Draft Guidance for Industry & FDA Staff
Agency: FDA
Guidance Date: March 2018
Deliverables:
| Asset Type | Title | Description |
|---|---|---|
| FDA Post Marketing Safety Reporting for Drug-Device Combinations Implementation Guide | An Implementation Guide to aide pharmaceutical manufacturers’ understanding of ambiguous areas of the PMSR for Combination Products Draft Guidance for Industry and FDA Staff (March 2018). |
| FDA Post Marketing Safety Reporting for Drug-Device Combinations Implementation Guide: Webinar | A webinar presentation where TransCelerate team members provide an overview of the FDA Post Marketing Safety Reporting for Drug-Device Combinations Implementation Guide. |
This topic’s objective was to interpret ambiguous portions of Reference Safety Information Guidance Q&A, engage with regulators and share findings on implementation challenges and approaches.
Guidance: CTFG Q&A Document – Reference Safety Information (Nov. 2017)
Agency: EMA
Q&A Date: November 2017
Deliverables:
| Asset Type | Title | Description |
|---|---|---|
| Call for Reharmonization: Clinical Trials: Global Pharmacovigilance Regulations | To meet these goals, we at TransCelerate implore representatives for regulatory authorities to work with industry sponsors, in the spirit of ICH, to identify potential ways to re-harmonize global pharmacovigilance processes and requirements. |
| Publication on the Evolving Remit of the Reference Safety Information: Observations on Implementation of the 2017 Clinical Trials Facilitation Group (CTFG) Question & Answer Document | This manuscript highlights industry challenges in operationalizing the requirements of the 2017 Clinical Trial Facilitation Group (CTFG) Question & Answer document. Surveys reveal actions taken in the creation, submission and implementation of the Reference Safety Information (RSI) to meet the requirements of the CTFG and EU national competent authorities whilst staying compliant with global regulatory requirements. |
This topic’s objective was to interpret ambiguous portions of the draft guidance, engage with regulators, and share findings on implementation challenges and approaches.
Guidance: The previous draft guidance for industry from the FDA on Safety Assessment for IND Safety Reporting is no longer publicly available.
Agency: FDA
Guidance Date: December 2015
Deliverables:
| Asset Type | Title | Description |
|---|---|---|
| Publication on TransCelerate Experience with FDA’s 2015 Safety Assessment Guidance | In this publication, TransCelerate member companies summarize their experience in operationalizing FDA’s clinical trial monitoring and reporting requirements with a key focus on FDA’s Draft 2015 Safety Assessment Guidance. |