Therapeutic Risk Management of Medicines

Stephen J. Mayall, Anjan Swapu Banerjee

• Publisher: Woodhead Publishing
• Year: 2014
• Print ISBN: 9781907568480
• eBook ISBN: 9781908818270
• Topic: Medicine
• Subtopic: Medical Theory, Practice & Reference

Therapeutic risk management of medicines is an authoritative and practical guide on developing, implementing and evaluating risk management plans for medicines globally. It explains how to assess risks and benefit-risk balance, design and roll out risk minimisation and pharmacovigilance activities, and interact effectively with key stakeholders.A more systematic approach for managing the risks of medicines arose following a number of high-profile drug safety incidents and a need for better access to effective but potentially risky treatments. Regulatory requirements have evolved rapidly over the past decade. Risk management plans (RMPs) are mandatory for new medicinal products in the EU and a Risk Evaluation and Mitigation Strategy (REMS) is needed for certain drugs in the US.This book is an easy-to-read resource that complements current regulatory guidance, by exploring key areas and practical implications in greater detail. It is structured into chapters encompassing a background to therapeutic risk management, strategies for developing RMPs, implementation of RMPs, and the continuing evolution of the risk management field.The topic is of critical importance not only to the pharmaceutical and biotechnology industries, but also regulators and healthcare policymakers.Some chapters feature contributions from selected industry experts. - An up-to-date practical guide on conceiving, designing, and implementing global therapeutic risk management plans for medicines - A number of useful frameworks are presented which add impact to RMPs (Risk Management Plans), together with regional specific information (European Union, United States, and Japan) - A comprehensive guide for performing risk management more effectively throughout a product's life-cycle

Part 1

Background to therapeutic risk management

Outline

1 Introduction

2 Basic principles of therapeutic risk management

3 The evolution of therapeutic risk management

4 Risk management in the European Union

5 Risk management in the United States

6 Overview of risk management around the world

1

Introduction

Abstract:

This book comprehensively reviews therapeutic risk management, a field that has expanded rapidly over the past decade. Although risk management has always existed for medicines, the introduction of new regulations has radically changed, formalised and structured the field. Risk management has become a critical factor for the success of a medicinal product, so it is essential for participants in the pharmaceutical industry and regulatory agencies to have an appropriate level of understanding. This book explains how to perform risk management effectively throughout a product’s life-cycle, whether assessing benefit–risk balance, developing risk management plans, implementing risk management activities, or interacting with regulatory authorities.

Key words

risk management plan; therapeutic risk management

1.1 The importance of therapeutic risk management

Our goal for this book is to provide an authoritative and practical guide to therapeutic risk management. It contains comprehensive information on planning, writing, implementing and evaluating risk management plans (RMPs) for medicines globally, including European Union RMPs (EU-RMPs) and Risk Evaluation and Mitigation Strategies (REMS). A number of useful frameworks are presented to help optimise the development of RMPs, together with specific advice covering differences between the European Union, the United States and other regions. This book complements current regulatory guidance, exploring key areas in more detail and assessing the future direction of the risk management field.

Therapeutic risk management has evolved rapidly over the past decade. Medicines have always required consideration of their safety risks as well as their benefits. However, regulations were previously not specifically focused on risk management and covered only routine methods for safety assessment and reducing risk. Any deviations beyond this approach for a product tended to be done on an ad hoc basis, rather than specifically guided by regulations.

Over a period of time there were several major, high-profile safety failures involving marketed medicines, which indicated that the existing approach, though important, was not sufficient. These incidents arose unpredictably, often had devastating public health consequences, and led to the withdrawal of the medicines from the market. In addition, there were many potentially useful therapies which had market authorisation either refused or delayed because their safety concerns were not adequately addressed.

Catalysed by these events, new regulations were enacted across the world to provide a more focused and structured approach to risk management, and these regulatory requirements continue to evolve. Consequently, therapeutic risk management has become a critically important topic for the pharmaceutical industry and the health authorities that regulate medicinal products. It has a strategic impact throughout the life-cycle of a product, is key to obtaining product approval, influences commercial success and, most importantly, protects patients by ensuring that a favourable balance exists between the benefits and risks of a medicine.

Our own interest in therapeutic risk management arose from globally advising numerous companies within the pharmaceutical industry. Experience from working for a large pharmaceutical company as a global pharmacovigilance and risk management physician, teaching the subject to postgraduates and practitioners world–wide and the active practice of, and prescribing in, clinical medicine have also been valuable. We have gained extensive knowledge from developing and/or implementing over fifty risk management plans on behalf of clients during the past decade. These projects have covered a variety of therapeutic areas, geographical regions and types of product. We have often been approached to assist with medicines that have potentially problematic safety issues, requiring innovative solutions to address safety concerns and enable marketing authorisation. Our clients have included a wide selection of organisations, ranging from the ‘Top 10’ pharmaceutical companies, through mid-size pharma corporations, to smaller biotechnology (‘biotech’) firms.

In addition, a few chapters have contributions from selected industry experts.

1.2 Relevant audiences for and structure of this book

This book should be useful to a variety of readers who wish to deepen their understanding of therapeutic risk management. It is an important topic, not only for employees within the pharmaceutical industry, but also for those in regulatory agencies, as well as policymakers and, increasingly, healthcare payers. Prescribers and other healthcare professionals are exposed to the ‘front line’ of risk management due to their interactions with patients and would benefit from an enhanced understanding of the field, improving the decisions they take. Academic researchers, undergraduates and postgraduate students in disciplines such as pharmacy, pharmacology and health economics should also have satisfactory knowledge of this important and still emerging area. Finally, risk management practitioners in other industries should be interested in transferable learnings.

Risk management is performed by increasingly multidisciplinary teams within the pharmaceutical industry and significantly impacts many other areas within companies. Therefore, a wide variety of company personnel should benefit from better knowledge of this topic, including those from safety, risk management, pharmacovigilance, epidemiology, regulatory, medical affairs, clinical development, non-clinical development, commercial and health outcomes functions within these companies, as well as those in senior management.

This book has a modular structure. Readers who have never previously encountered risk management can develop their understanding of the subject from the early chapters. More experienced readers may wish to refresh and extend their knowledge, or to browse specific topics of interest. The chapters are broadly grouped into four different themes:

■ Chapters 1–6 provide a comprehensive background to therapeutic risk management – They initially introduce the basic concepts of risk management and describe its evolution, highlighting some major drug safety incidents. Risk management is an ongoing process throughout the life-cycle of a medicinal product, so appropriate activities for each stage are described. There are variations between the EU, the US and other regions due to factors such as different legislation and healthcare systems. Therefore, the key features and regulations in different regions are explained and compared.

■ Chapters 7–10 focus on developing an RMP – In the EU and certain other countries, a risk management plan (RMP) is essential for the approval of each novel medicinal product. Since companies usually intend to market their therapies in Europe, an EU-RMP is normally required as part of the global strategy. Detailed advice is provided on how to develop a suitable document, including creating a safety specification, pharmacovigilance planning, evaluating the need for risk minimisation and the selection of appropriate risk minimisation activities. The relevant steps for developing a REMS, which is required for some products in the US, are also covered.

■ Chapters 11–13 mainly cover the implementation of RMPs – These chapters explain how a company should perform risk management to develop RMP documents and roll out associated activities successfully. Appropriate company structures and processes are also described. A suitable approach to evaluate the effectiveness of risk management should be planned and subsequently performed. Various types of evaluation techniques are discussed and an integrated model is proposed. Many of the steps in risk management require contact between the company and regulatory authorities, so key processes are described and advice is provided on how to interact effectively.

■ Chapters 14–18 describe the continuing evolution of risk management – The final chapters of this book explore important topics that are still emerging. Development RMPs are increasingly widespread and focus on managing risks earlier in the product life-cycle to optimise safety prior to market authorisation. There is a growing need to consider both the benefits and risks of a therapy together. Progress on suitable approaches to assess the benefit–risk balance of a medicine and publicly communicate it is described. Further types of risk management used within the pharmaceutical industry and other key industries are summarised, including important transferable learnings. Finally, the future outlook for therapeutic risk management is considered.

Key points

This book is a practical guide to therapeutic risk management, which has become critical to the success and safer use of medicinal products.

It should ...

Table of contents

• Cover image
• Title page
• Table of Contents
• Copyright
• List of figures and tables
• List of abbreviations
• Acknowledgements
• Foreword
• About the authors
• Additional contributors
• Part 1: Background to therapeutic risk management
• Part 2: Developing a risk management plan
• Part 3: Implementation of risk management plans
• Part 4: Continuing evolution of risk management
• Index