This document provides an overview of biologics and biosimilars. It defines biologics as complex medications made from living cells that treat diseases in novel ways. Biosimilars are highly similar but not identical copies of biologics that are approved through an abbreviated pathway. The FDA will determine standards for interchangeability and require rigorous testing to ensure biosimilars are safely effective for each condition and patient group. Important issues for patients include ensuring safety, transparency, and choice in biologic treatment.

1. UNITED SPINAL ASSOCIATION
AUGUST, 2014
Biologics & Biosimilars:
An Overview
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2. What is a biologic?
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3. Biologics Are Complex
 Biologics are innovative medications that aren’t made, they are grown
in living cells.
 Biologics are complex molecules– up to 1,000 times larger than
conventional medicines.
 Manufacturing a biologic is a complex process that takes several
months from start to finish.
 It’s important to remember that biologic medicines can never be
exactly duplicated by two different manufacturers in the way that
simpler medications can.
Sources: EuropaBio, NDT Plus, Biosimilars, Nat. Biotechnol, Biopharm Int.
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4. Biologics Have Revolutionized Healthcare
 Although relatively new, biologics are already used by more
than 350 million patients worldwide.
 Biologic medicines revolutionized how diseases and
conditions such as rheumatoid arthritis, Crohn’s disease,
hemophilia, multiple sclerosis, and many other disorders are
treated.
 Patterned after proteins the body itself produces, biologics can
treat many serious diseases in ways conventional medicines
cannot.
Source: PhRMA
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5. What is a biosimilar?
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6. Biosimilars
 Biosimilars are products that are “highly similar” to (but not exact
copies of) the reference biologic product.
 As a part of the Affordable Care Act (ACA), Congress provided the Food
& Drug Administration (FDA) authority to review and approve
biosimilar versions of FDA approved biologic medicines. The FDA is in
the process of developing guidelines to approve biosimilars.
 While there are a small number of biosimilars approved in several
other countries, there are currently none approved for use in the United
States.
 The FDA received its first application for approval of a biosimilar in
July 2014.
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7. What Biosimilars Are…And Are Not
 As the name suggests, biosimilars are similar to a
particular biologic, they are not the same.
 All biosimilars differ from the innovative product and
from each other.
 They are not generic biologics.
Source: Annals of Oncology
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8. Why There Can Be No “Generic Biologics”
 The active ingredient of a biosimilar can, at best,
only resemble that of the original biologic.
 How an innovator company makes its biologic can
never be duplicated down to the last detail; a
biosimilar is made using different cells and different
processes.
Source: Letter from FDA to Congress
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9. Ensuring Safety And Effectiveness
 The variations between an innovator biologic and a
biosimilar could potentially trigger an attack against
a biosimilar by the body’s defenses.
 This could cause unwanted and/or unsafe symptoms or render
the biosimilar, and possibly the original biologic, less effective.
 To ensure comparable safety and effectiveness,
biosimilars require more thorough testing than
generic medicines.
Source: EMA, Pharmaceutical Sciences, CDER, Biosimilar News
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10. Issues for Patients to Consider
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11. Access to Biologics and Patient Welfare
 It is important to provide access to biologic
treatments for patients who need them, and
biosimilars play a part in meeting this goal.
 An equally and perhaps more important
consideration is to ensure patient safety and welfare
in all stages of development, approval, and
monitoring of biologics, including biosimilars.
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12. Important Issues for Patients
As federal agencies and the states implement the new biosimilars law, they are
making important decisions about the regulation of biosimilars that are crucial to
assuring patient safety.
 Interchangeability: This year, the FDA will be decide what standards must be
met for a biosimilar medicine to be judged “interchangeable” with the original
biologic medicine. Strict standards are needed to ensure patient safety.
 Rigorous Testing For Each Condition: The FDA will be determining whether
drug companies must conduct rigorous clinical testing to prove that a biosimilar
works safely in each distinct group of patients with that disease. Failure to perform
adequate testing in each group of patients and disease type may put patients at risk.
 Naming: Using a distinguishable but related non-proprietary name for biosimilar
medications (i.e., distinguishable from the original biologic) would enable medical
professionals and the FDA to better track these products and more easily access any
adverse reactions that may occur in patients who use biologics, including
biosimilars.
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13. Important Issues for Patients, (Continued)
As federal agencies and the states implement the new biosimilars law, they are
making important decisions about the regulation of biosimilars that are crucial
to assuring patient safety.
 FDA Transparency: Improved transparency throughout the application and
approval process of biosimilars is important to assure appropriate oversight and to
ensure that all voices, particularly those of patients, are heard.
 Substitution/Notification: Because even an interchangeable biosimilar will not
be identical to the original biologic medicine, most doctor and patient groups feel it
is important for a prescribing doctor to be notified after a pharmacist substitutes a
biosimilar for a prescribed biologic medicine. A biosimilar that is not
interchangeable should only be used if prescribed by an appropriate health
professional.
 Recordkeeping: Ensuring pharmacists maintain records of substitutions for
significant periods of time would allow the medical community to track long-term
treatment outcomes of biosimilars.
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14. What United Spinal Association Has Said About
Biosimilars
 “The FDA is expected to publish proposed guidance for public comment this year
related to key aspects of implementation of the biosimilars law. We need Congress
to hold oversight hearings on this important process and we urge the FDA to release
guidance as soon as possible that ensure patient safety, patient access to the right
biologic medicines, patient choice and full transparency.”
 “Choice should be at the center of any decision to substitute or switch therapies and
should only be decided by the patient and provider. Patient choice needs to be
preserved and regulatory decisions must be based on sound science.”
 “Biosimilar regulations must put patient safety first. Policymakers and regulators
must address appropriate patient safety and efficacy concerns as they relate to
decisions around interchangeability, clinical indications, labeling, naming and
substitution.”
 Particular attention must be given to assure that rigorous clinical testing proves that a biosimilar
works safely in each and every condition or disease for which it is approved to be prescribed, as
well as in each distinct group of patients with that disease.
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