Ronald A. Rader, Biotechnology Information Institute09.05.13
The Biotechnology Information Institute (of which I am president) has filed a Citizen Petition requesting that FDA assign both unique and biosimilar/(bio)generic-type (or class) names (and/or other identifiers) to approved biologic products and their active agents, along with disclosures of sufficient public information to enable an adequate understanding of product identity (what the products/agents are, which is largely dependent on their composition, bioprocessing and quality-related (CMC) aspects). The petition also requests information regarding nomenclature and product identity (CMC) aspects be disclosed prior to approval and discussed as part public advisory committee meetings. Further information on the petition is available at www.biopharmacopeia.com.
Biopharma products are each unique and differentiated on the basis of their composition, bioprocessing and quality-related (CMC) aspects, with any significant CMC changes defining a new product and thus requiring a new approval, either supplemental or full BLA. This petition requests FDA assign names (and/or other identifiers) to products and agents upon impending approval. The petition also requests: unique names/identifiers and related information disclosures concerning supplemental BLA changes in products/agents (tracking of product drift); and that any nomenclature for names/identifiers be coherent, science/product/entity-based and unencumbered by legacy regulatory requirements.
Despite biopharmaceuticals being the most complex of all consumer products, these products lack both unique and generic-type descriptive or otherwise useful authoritative non-proprietary (publically-usable) names! Currently there are no non-proprietary names (no nomenclature systems) for approved biologics that reflect the nature of the products (and active agents), what they are, with current non-proprietary names highly constrained by regulatory requirements. The official (established/compendial/USAN) non-proprietary names assigned by FDA, widely used for marketing and prescriptions, are insufficiently descriptive (short, for writing prescriptions), are not linked to any product/agent descriptions (are totally undefined), and do not change as the products/agents change (track product drift). Further, these names are fully ambiguous, with the same names, e.g., rituximab, used as both the name for the product and the active agent, with those encountering the names having to guess by context whether a name is referring to a product or agent. This may work well enough for generic small molecule drugs, but not for biopharmaceuticals — which are defined by their unique bioprocessing — particularly in the context of hundreds of biosimilars set to enter the U.S. market.
The petition has nothing to do with official names for these products. These have been the subject of years of yet-to-be-resolved controversies over what type official names FDA should assign to biosimilars, e.g., unique or (bio)generic names. Otherwise, biosimilar/(bio)generic or class names for biologic products and active agents simply do not exist yet.
It is very common for FDA review information related to product composition, bioprocessing and CMC aspects to have been 100% redacted (censored), as illustrated with multiple products in an appendix to the petition. In fact, the few descriptive sentences included in product inserts are often more informative than the cumulative product approval-related documentation available from FDA! The petition seeks more substantive and useful product/agent-related disclosures in review-related public documentations. Currently, lacking both useable names/ identifiers and the most basic authoritative descriptive information, biopharmaceuticals risk being tagged as “Frankenpharma-ceuticals,” as has happened with GMO food products.
The petition also requests that FDA’s proposed names and product-defining information, including CMC aspects, be discussed at public advisory committee meetings. Neither the public documents disclosed prior to these meetings nor the meetings themselves now include discussion of CMC-related aspects, which are much more a determinant and critical for an understanding of product safety than the minutiae of clinical studies, which now dominate these meetings.
No matter what names/identifiers FDA assigns to biologics, expert input and representation of diverse interests (uses/user communities) should be an integral part of developing names — the candidates for selection by FDA and other names for other uses/user communities. Regrettably, the U.S. biopharma industry lacks its own dedicated trade association that normally would be involved hands-on with such issues. The petitioner has proposed the U.S. Biopharmacopeia™ Registry of Biopharmaceutical Products project to propose candidate unique and non-unique names for biopharmaceutical products/ agents suitable for selective adoption by diverse user communities, including regulatory agencies, formularies, reference sources, public use, etc.; and post a public website providing this information. The Biopharmacopeia project might function similar to the CTFA/PCPA (cosmetics trade association) Dictionary project, which for nearly 40 years has involved an industry-based committee proposing cosmetic ingredient names to FDA, with these almost always adopted for official product labeling.
Ronald. A. Rader is president, Biotechnology Information Institute, and the author/publisher of BIOPHARMA: Biopharma-ceutical Products in the U.S. and European Markets (biopharma.com). For further information or to lend your support to the petition, contact Mr. Rader at biotech@biopharma.com.
Biopharma products are each unique and differentiated on the basis of their composition, bioprocessing and quality-related (CMC) aspects, with any significant CMC changes defining a new product and thus requiring a new approval, either supplemental or full BLA. This petition requests FDA assign names (and/or other identifiers) to products and agents upon impending approval. The petition also requests: unique names/identifiers and related information disclosures concerning supplemental BLA changes in products/agents (tracking of product drift); and that any nomenclature for names/identifiers be coherent, science/product/entity-based and unencumbered by legacy regulatory requirements.
Despite biopharmaceuticals being the most complex of all consumer products, these products lack both unique and generic-type descriptive or otherwise useful authoritative non-proprietary (publically-usable) names! Currently there are no non-proprietary names (no nomenclature systems) for approved biologics that reflect the nature of the products (and active agents), what they are, with current non-proprietary names highly constrained by regulatory requirements. The official (established/compendial/USAN) non-proprietary names assigned by FDA, widely used for marketing and prescriptions, are insufficiently descriptive (short, for writing prescriptions), are not linked to any product/agent descriptions (are totally undefined), and do not change as the products/agents change (track product drift). Further, these names are fully ambiguous, with the same names, e.g., rituximab, used as both the name for the product and the active agent, with those encountering the names having to guess by context whether a name is referring to a product or agent. This may work well enough for generic small molecule drugs, but not for biopharmaceuticals — which are defined by their unique bioprocessing — particularly in the context of hundreds of biosimilars set to enter the U.S. market.
The petition has nothing to do with official names for these products. These have been the subject of years of yet-to-be-resolved controversies over what type official names FDA should assign to biosimilars, e.g., unique or (bio)generic names. Otherwise, biosimilar/(bio)generic or class names for biologic products and active agents simply do not exist yet.
It is very common for FDA review information related to product composition, bioprocessing and CMC aspects to have been 100% redacted (censored), as illustrated with multiple products in an appendix to the petition. In fact, the few descriptive sentences included in product inserts are often more informative than the cumulative product approval-related documentation available from FDA! The petition seeks more substantive and useful product/agent-related disclosures in review-related public documentations. Currently, lacking both useable names/ identifiers and the most basic authoritative descriptive information, biopharmaceuticals risk being tagged as “Frankenpharma-ceuticals,” as has happened with GMO food products.
The petition also requests that FDA’s proposed names and product-defining information, including CMC aspects, be discussed at public advisory committee meetings. Neither the public documents disclosed prior to these meetings nor the meetings themselves now include discussion of CMC-related aspects, which are much more a determinant and critical for an understanding of product safety than the minutiae of clinical studies, which now dominate these meetings.
No matter what names/identifiers FDA assigns to biologics, expert input and representation of diverse interests (uses/user communities) should be an integral part of developing names — the candidates for selection by FDA and other names for other uses/user communities. Regrettably, the U.S. biopharma industry lacks its own dedicated trade association that normally would be involved hands-on with such issues. The petitioner has proposed the U.S. Biopharmacopeia™ Registry of Biopharmaceutical Products project to propose candidate unique and non-unique names for biopharmaceutical products/ agents suitable for selective adoption by diverse user communities, including regulatory agencies, formularies, reference sources, public use, etc.; and post a public website providing this information. The Biopharmacopeia project might function similar to the CTFA/PCPA (cosmetics trade association) Dictionary project, which for nearly 40 years has involved an industry-based committee proposing cosmetic ingredient names to FDA, with these almost always adopted for official product labeling.
Ronald. A. Rader is president, Biotechnology Information Institute, and the author/publisher of BIOPHARMA: Biopharma-ceutical Products in the U.S. and European Markets (biopharma.com). For further information or to lend your support to the petition, contact Mr. Rader at biotech@biopharma.com.
