Presentation on theme: "“Biosimilars Needs Names: But What Type(s), Servicing What Uses and Whose Interests?”. Ronald A. Rader President Biotechnology Information Institute 1700."— Presentation transcript:
“Biosimilars Needs Names: But What Type(s), Servicing What Uses and Whose Interests?”. Ronald A. Rader President Biotechnology Information Institute 1700 Rockville Pike, Suite 400 Rockville, MD Phone: Web sites: BIOSIMILARS.com BIOPHARMA.com BIOPHARMACOPEIA.com
Background: Author and Publisher BIOPHARMA – only information resource/reference specializing in biopharmaceuticals (www.biopharma.com); now in 11 th yearwww.biopharma.com “The reference products reference” BIOSIMILARS.com – portal for biosimilars (and biobetters, biogenerics) pipeline information; marketed product reference; manufacturers and reg. document repository; licensing opportunities; news blog; glossary of terms; links to resources; etc. “Biosimilars Information for the Biopharmaceutical Industry” Biosimilars pipeline study – in press; the most comprehensive pipeline study; visit for further informationwww.biosimilars.com U.S. BIOPHARMACOPEIA Registry of Biopharmaceutical Products – visit proposed project forwww.biophamacopeia.com nomenclature development and a public reference database
The Biosimilars/Biobetters Pipeline (n =911) biosimilars = 427 ; biobetters = 367; ref. products = 117 Caution: Future major players (biggest cos.) pipelines not included (not yet disclosed; many will license-in)!
Every successful recombinant protein will likely have 5-10 or many more biosimilars, and ASAP! Product(s) Sales ($B) Biosimilars in the Pipeline Enbrel$ Avastin$6.987 Rituxan$ Remicade$6.526 Herceptin$ Humira$5.496 Lantus$4.835 Lucentis$3.112 Aranesp$3.004 Neupogen$ Neulasta$ Epogen/Procrit$ TNF inhibitors30 Interferon alfa53 Interferon beta20 mAbs/antibody-like107
Biosimilars in the Pipeline by Estimated Year of U.S. Launchability, now-2022
Economic Impact: U.S. Biosimilars Launchable Dates by Reference Products Sales ~$100 billion (as $millions on the Y axis), of current ref. product sales distributed by their earliest likely year of U.S. biosimilars launchability Now (2011/2) 2020
Biosimilars Are Biopharmaceuticals/biologics The most complex of all commercial products! Agents/products are differentiated (defined) on basis of primary structure plus bioprocessing PROCESS = PRODUCT Biopharmaceuticals can be named on based on: – Agent/product – structures; scientific basis – Regulatory – approvals define product; But with biologics, often no meaningful relationship between approvals and agent/product structure and characteristics, i.e. an agent/product or its approval may change, but the other remains unchanged) – Real world – common, popular; best avoided (if similar at all, then it’s biosimilar)
Product Nomenclature Is the No. 1 Determinant of Public and Professional Perception of Biosimilars! Will biosimilars be viewed as distinct high-end biopharmaceuticals, comparable to innovator products, or as low-end generics best avoided? It all depends on their names! Will biosimilars be named (and, thus, defined) based on generic relationships and dependencies (making them 2 nd -class, like generic drugs)? Will unique names for biosimilars confound or negate making appropriate and needed comparisons with reference products?
What is the Current Status of Biopharmaceutical/biologic Information? Biologics approvals – secret pacts between FDA and manufacturers regarding agent, product and processing specifications and their allowable variations; Products meeting BLAs are approved, not any published definitions FDA rarely, if ever, bothers to define products (disclose basics aspects of CMC), even in reg. documentation (rarely ever more than a few sentences included in product inserts) If process = product, we know little or often nothing about marketed biopharmaceutical (reference) products! And now biosimilars will be approved based on comparisons and analogies! More/better information needs to be disclosed in the context of biosimilars; How will anyone be able to make comparisons?
What Needs to be Named? Unique names are obviously needed, but exactly what is to be named - the active agent, the finished product or both?; and at what level of specificity (e.g., manufacturing processes and facilities)? Will names be associated with distinct active agents and products, or will FDA- assigned names that mostly vaguely refer to approvals (the status quo)? Are unique names needed for both active agents and finished products? [absolutely] Are biosimilar/generic relationship-based names also needed for both active agents and finished products [Yes] Are generation and version identifiers needed, including to track “product drift?” [absolutely; like with software and other products] When do agents/products become new agents/products requiring new names? [If can’t answer this, then any nomenclature system is useless!] Basic problem: Traditional nomenclature resources either have serious conflicts of interest, e.g., are sources for current drug nomenclature (all generic, in terms of biopharmaceuticals), are not interested or lack biopharmaceutical knowledge
Biosimilar/generic vs. Unique/Distinct Names Biosimilar/generic names greatly facilitate biosimilars marketing, cost-savings [and substitution/interchangeability, a future concern to be kept in mind] and information retrieval (products with similar stems alphabetize together and can be retrieved) Unique names greatly facilitate safety pharmacovigilance and tracking, but confound marketing and information retrieval (if stems differ, similar products likely not retrievable) Both types of names are needed, for different uses and users, but FDA can select only one
FDA Biosimilar ‘Established’ (Official; Compendial; Nonproprietary) Name Issues Currently totally chaotic, with FDA almost always adopting inherently generic USAN names (generally, adopting foreign (WHO/UN)- controlled INNs designed to be generic names) – never designed for biopharmaceuticals – totally inconsistent, e.g, different nomenclature adopted for each product class – often made ‘unique’ by adding suffixes, Greek letters, subscripts, etc. – names are arbitrary/chaotic, some designed to be meaningful, some express relationships, many are just totally meaningless (to be fully unique) – no predictability, no real system; little transparency/public input FDA’s own ‘proper names’ for biologics and its Unique Ingredient Identifier (UNII)/Substance Registration System and CAS and IUPAC are inherently generic, e.g., fully ignore glycoforms, only consider primary structure Trade names (trademarks) are generally unique, but are proprietary (need permission to use) and can be easily gamed, renamed, etc. Adding company names to USANs or other generic names would make every mention an advertisement NDCs – specific and unique, but numbers will not work as names
FDA Decisions Required How specific and/or generic will names be? Adopt a rational system or use arbitrary/meaningless names? Are both specific and/or generic names needed for active agents and/or products? Adopt USAN/INN for biosimilars (keep the status quo; a terrible option from an information management perspective)? What information (product definitions or descriptive information) will be associated with names (exactly what is being named)? Who will disclose this and when? How will agent/product changes be reflected (or not) in names? Will FDA give industry and other vested interests the opportunity to provide input to nomenclature development? Will FDA give us a nomenclature system suitable for the U.S. biopharmaceutical industry or adopt the status quo (jury-rigged USANs/INNs), rationalizing this as what the EU and others use?
U.S. BIOPHARMACOPEIA Registry of Biopharmaceutical Products (proposed) Funded by industry and/or FDA Proposes acceptable unique and biosimilar/generic names for both active agents and products for selective adoption by FDA, other regulators, publishers, formularies, public use, etc. model – CTFA Dictionary; since mid-1970s, cosmetics trade association-coordinated committee proposes ingredient names almost always adopted by FDA for cosmetic product labeling Online products registry – with authoritative biosimilars (and ref. products) nomenclature, associated descriptions, and manufacturer- submitted public and regulatory documentation
For Further Information: “Nomenclature of Biosimilars Will Be Highly controversial, BioProcess International, June 2011, p (at Web sites: