Terminology and definitions : The fundamentals
This article provides a glossary of key terms that are used around the regulatory considerations and practice considerations associated with biosimilars
James Stevenson PharmD FASHP FFIP Department of Clinical Pharmacy University of Michigan College of Pharmacy , Ann Arbor , MI , USA
Over the past decade , biologics have become increasingly important in the pharmaceutical armamentarium . In 2017 , biologics represented 2 % of all US prescriptions , but 37 % of net drug spending . 1 Since 2014 , biologics have accounted for nearly all of the growth in net drug spending . Total US biologic sales in 2018 were estimated to have been $ 125 billion , an increase of 50 % since 2014 . 2
In response to the growing costs of these agents and the negative impact that the high expense has had on patient access , major regulatory bodies such as the European Medicines Agency and the Food and Drug Administration ( FDA ) have established regulatory guidance to facilitate the approval of biosimilars . 3
With the patents for many important biologics at or approaching expiry , the development of biosimilars was expected to be an important strategy to reduce overall health care expenditures and to improve access to these therapies .
Biologics differ from traditional small molecule pharmaceuticals in a number of important ways . These include the complexity and characterization of the drugs themselves as well as the manufacturing processes . These differences necessitate a different approach to the regulatory approval process for their safe and effective introduction into health care practice . This has resulted in the introduction of a number of terms and concepts that might be unfamiliar to pharmacists and other healthcare professionals .
The purpose of this article is to define and summarize the key terms that are used in the regulatory considerations and practice considerations associated with these agents for easy reference .
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Key terms and definitions Biologic product According to the FDA , biologic products are a diverse category of products and are generally large , complex molecules . These products may be produced through biotechnology in a living system such as a microorganism , plant cell , or animal cell , and are often more difficult to characterize than small molecule drugs . There are many types of biologic products in the US , including therapeutic proteins , monoclonal antibodies , blood and blood components , gene therapies , and vaccines . Biologics have inherent variations that can result from the manufacturing process . These variations can present challenges in characterizing and manufacturing that often do not exist for small molecule drugs . Part of the FDA review is to assess the manufacturing process and the strategy to control within-product variation . 4 , 5
Biosimilar Sometimes referred to as a ‘ follow-on biologic ’, a biosimilar is defined by the FDA as a biologic product that is highly similar to , and has no clinically meaningful differences from , an existing FDA-approved reference product . The biosimilar must be highly similar to the reference product with regard to structure , function , and purity , except for minor differences in clinically inactive components . It must also be demonstrated to have no clinically meaningful differences compared to the reference product in terms of safety , efficacy , or potency , as evidenced by pharmacokinetic , pharmacodynamic , immunogenicity , and clinical studies . 6 , 7 A biosimilar is approved by the FDA through an abbreviated regulatory process that is referred to as 351 ( k ) of the Public Health Service Act .
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