Biologics, Biosimilars, and Biobetters

An Introduction for Pharmacists, Physicians and Other Health Practitioners

Iqbal Ramzan

• Publisher: Wiley
• Year: 2021
• Print ISBN: 9781119564652
• eBook ISBN: 9781119564669
• Edition: 1
• Topic: Medicine
• Subtopic: Biotechnology in Medicine

A comprehensive primer and reference, this book provides pharmacists and health practitioners the relevant science and policy concepts behind biologics, biosimilars, and biobetters from a practical and clinical perspective.

• Explains what pharmacists need to discuss the equivalence, efficacy, safety, and risks of biosimilars with physicians, health practitioners, and patients about
• Guides regulators on pragmatic approaches to dealing with these drugs in the context of rapidly evolving scientific and clinical evidence
• Balances scientific information on complex drugs with practical information, such as a checklist for pharmacists

1
Innovator Biologics, Biosimilars, and Biobetters: Terminology, Nomenclature, and Definitions

Iqbal Ramzan

Sydney Pharmacy School, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia

---

KEY POINTS

• Many different terms are used for innovator biologics, biosimilars, and biobetters internationally.
• Greater harmonization of terminology, definitions, and nomenclature across different regulatory jurisdictions and countries would assist health practitioners and patients in understanding the complex issues of biologic medicines.
• The salient language of innovator biologics, biosimilars, and biobetters are introduced in this chapter to set the context for the rest of the book, which deals with specific issues in greater detail pitched at pharmacists, doctors, and patients.

---

Abbreviations

Abbreviation	Full name
ABPI	Association of British Pharmaceutical Industry
AfPA	Alliance for Patient Access
BAP	Biosimilars Action Plan
BLA	Biologics License Application
BPC	Biologics Prescribers Collaborative
BPCI Act	Biologics Price Competition and Innovation Act
CAR‐T	Chimeric Antigen Receptor Therapy
CHMP	Committee for Medicinal Products for Human Use (EMA)
CIOMS	Council of International Organizations of Medical Sciences
CQAs	Critical Quality Attributes
CVMP	Committee for Medicinal Products for Veterinary Use
Da	Dalton
DNA	Deoxyribonucleic Acid
EMA	European Medicines Agency
EPAR	European Public Assessment Report
EU	European Union
FDA	Food and Drug Administration
GABi/GaBI	Generics and Biosimilars Initiative
LDN	Limited Distribution Network
mAbs	monoclonal Antibodies
NHS	National Health Service
NICE	National Institute for Clinical Excellence
NMS	Non‐Medical Switching
NOBs	Non‐Original Biologics
PBS	Pharmaceutical Benefits Scheme
P&T	Pharmacy & Therapeutics (Committee)
PTMs	Post‐Translational Modifications
QbD	Quality by Design
QUM	Quality Use of Medicine
RMP	Risk Management Plan
RPS	Reference Product Sponsor
RWE	Real‐World Evidence
SEBs	Subsequent‐Entry Biologics
SMD	Small Molecule Drug
UMC	Uppsala Monitoring Centre
UNESCO	United Nations Educational, Scientific and Cultural Organization
USA	United States of America
USD	US Dollar
WHO	World Health Organisation

1.1 Place of Biologics in Modern Therapeutics

Biologic therapies have entirely revolutionized the treatment of many debilitating, life‐changing chronic autoimmune diseases like rheumatic arthritis and plaque psoriasis as well as life‐threatening cancers for which no viable treatment option has existed previously. They also play a critical therapeutic role in many endocrine disorders and neurodegenerative conditions. Biologics are the fastest growing sector of the drug market¹ and are also the most expensive therapies. As a result, “highly similar” versions of innovator biologics, biosimilars have been introduced to provide cost‐effective biologic treatments.

The first innovator biologic was introduced ~40 years ago, and the first biosimilar was introduced in the European Union (EU) and United States (USA) in 2006 and 2015, respectively. Currently, there are over 300 biologics registered worldwide and the EU has over 60 approved biosimilars. In the United States, biosimilars are an emerging market, with 19 approved biosimilars. Biosimilar market access comparison between the United States and EU has shown that market access in the United States is less favorable. This is due to many factors including lack of incentives to prescribe biosimilars in the United States and small price discounts of biosimilars compared to innovator biologics.²

In many countries, including Australia and emerging pharmaceutical markets like Brazil, biosimilar use is actively encouraged as governments attempt to contain the costs of expensive innovator biologics. Assuming discounts on off‐patent innovator biologics and biosimilars of ~50%, it is predicted that by 2020 there will be annual savings of over €8–10 billion in the EU.³

Biologics are complex proteins or protein‐like molecules produced using biotechnology techniques in living cells. Their structural, functional, and manufacturing processes lead to clinical concerns/controversy about their efficacy and safety, including the potential for treatment failure and severe immunogenicity reactions. Pharmacists, doctors, and other health professionals, there...

Table of contents

• Cover
• Table of Contents
• Title Page
• Copyright Page
• List of Contributors
• Foreword
• Preface
• 1 Innovator Biologics, Biosimilars, and Biobetters: Terminology, Nomenclature, and Definitions
• 2 Approved Biologic Medicines and Biosimilars in Major Regulatory Jurisdictions
• 3 Status of Biologic Drugs in Modern Therapeutics‐Targeted Therapies vs. Small Molecule Drugs
• 4 Major Classes of Biotherapeutics
• 5 Drug Targets for Biologics
• 6 Pivotal Biology, Chemistry, Biochemistry, and Biophysical Concepts of Biologics and Biosimilars
• 7 Biosimilarity and Interchangeability of Biologic Drugs‐ General Principles, Biophysical Tests, and Clinical Requirements to Demonstrate Biosimilarity
• 8 Pharmacokinetics of Biologics
• 9 Pharmacogenomics of Biologics
• 10 International Regulatory Processes and Policies for Innovator Biologics, Biosimilars, and Biobetters
• 11 Pharmacovigilance of Innovator Biologics and Biosimilars
• 12 Pharmacoeconomics of Biologic Medicines and Biosimilars
• 13 New Emerging Biotherapies: Cutting‐Edge Research to Experimental Therapies
• 14 Optimizing Use of Biologic Medicines Using a Quality Use of Medicines Approach
• 15 Knowledge Areas and Competency Standards on Biologic Medicines for Pharmacists and Pharmacy Students
• 16 A Checklist for Pharmacists on Biologics and Biosimilars: Tips to Enhance Patient‐Centered Discussions
• Index
• End User License Agreement