Affiliations: [a] Amgen Inc., Thousand Oaks, CA, USA | [b] Bayer HealthCare LLC, Berkeley, CA, USA | [c] F. Hoffmann - La Roche Ltd., Basel, Switzerland
Correspondence:
[*]
Corresponding author: Gustavo Grampp, Amgen Inc., Thousand Oaks, CA, USA. E-mail:ginog@amgen.com
Abstract: Biotherapeutic products (BTPs), also known as biotherapeutic medicines,
contain structurally complex active substances produced by living organisms.
Due to their complexity and method of manufacture BTPs require distinct
regulatory approval standards relative to chemically-synthesized small
molecule medicines. This is also relevant for licensing copied versions of a
BTP, or similar biotherapeutic products (SBPs) made by a different
manufacturer where regulatory concepts developed for generics should not
have been applied. In all these licensing scenarios regulators need to
evaluate the results of comparability exercises, including sensitive
head-to-head analytical, pre-clinical and clinical comparisons with the
original product as a basis for approval.
SBPs do not contain chemically identical active substances, and may have
slightly different benefit-risk profiles, therefore it is necessary to
monitor post-approval safety on a product-specific basis. Policymakers may
therefore emphasize the need for product-specific identification in patient
records and safety reports using either a unique trade name or a
distinguishable non-proprietary naming system. The unique nature of BTPs
also informs the nature and degree of interchangeability between the
originator and SBPs versions. Many policymakers also emphasize that
switching between SBPs should only occur with the involvement of the
prescriber. It is recommended that pharmacy substitution would only be
appropriate when there is a robust framework for a competent authority to
assess product-specific evidence of interchangeability. Another challenge is
posed by the historical existence in some jurisdictions of copy BTPs that
were not assessed according to current regulatory standards. To address this
situation the World Health Organization has proposed a regulatory assessment
framework wherein the status of such products can be normalized via the
orderly submission and review of supplementary data.
