Draft guidance draws mixed response from industry stakeholders
Late late week, the FDA published draft guidance recommending that reference biologic products and biosimilars have nonproprietary names that share a core drug substance name and an FDA-designated suffix unique for each product.
For interchangeable biological products, the FDA is seeking public comment on whether the nonproprietary name for such a product should include a distinct suffix or share the same suffix as its reference product.
“To create market competition among biological products and lower costs, the Affordable Care Act created a new approval pathway for products that are biosimilar to and interchangeable with FDA-licensed biological products. The FDA is committed to encouraging the development of these biosimilar and interchangeable products. Biological products derived from living organisms can treat patients with cancer, chronic kidney diseases and auto-immune diseases, such as rheumatoid arthritis and inflammatory bowel disease,” Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, and Karen Midthun, director of FDA’s Center for Biologics Evaluation and Research, wrote in a blog post on the draft guidance. “Earlier this year, the agency approved the first biosimilar, and other products are in development. But one key issue is how to name biological products to ensure safe use and foster acceptance of these new products.”
According to Woodcock and Midthun, the proposed naming convention addresses two key issues: inadvertent substitution of biologics not determined to be interchangeable by the FDA — to prevent medication errors — and safety monitoring of biological products once they’re on the market, to make it easier to track usage of biologics in all care settings, such as outpatient, hospital and pharmacy.
“FDA is also considering, and has requested public input on, the benefits and challenges of other naming approaches, such as a suffix derived from the name of the license holder,” they added.
However, Bertrand Liang, chairman of The Biosimilars Council, a division of the Generic Pharmaceutical Association (GPhA), said the FDA’s recommendations on naming conventions for biosimilars “warrant serious scrutiny” because they could impede patient access to new, more affordable medicines and raise safety concerns.
“We believe that biologics and biosimilars should be required to have the same International Nonproprietary Name (INN) with no added ‘FDA-designated suffix.’ Adverse events and product recalls for small-molecule and biologic drugs already are successfully identified using the national drug code (NDC) and lot number and company name, and there is no compelling evidence that biosimilars should be handled differently,” Liang stated upon the FDA’s release of the draft guidance.
He noted that there’s already a precedent for shared names that hasn’t resulted in any patient safety issues and that shared INNs without suffixes are used in the European Union, Canada, Australia and Japan. “Adding a random collection of letters to the product’s nonproprietary name confers no additional safety benefit and, in fact, would require the health care professional to be armed at all times with a code-breaking reference,” he said.
The Academy of Managed Care Pharmacy (AMCP) also expressed disappointment with the FDA’s draft guidance and proposed rule for biosimilar product names to bear a nonproprietary name with an FDA-designated suffix.
“The current naming convention using the same nonproprietary name has proven safe and effective globally for small molecule drugs and for biological products in Europe, and therefore it should be the standard in the United States,” commented AMCP chief executive officer Edith Rosato. “AMCP is concerned that any departure from the currently accepted nonproprietary naming system will create confusion among health care practitioners and patients, have negative effects on the ability to ensure safe dispensing and tracking, and result in lower market adoption and cost savings.”
Meanwhile, the Biologics Prescribers Collaborative (BPC), an organization of groups representing doctors who regularly prescribe biologics, hailed the FDA’s naming proposal, noting that the draft guidance reflects the fact that biosimilars are not generics.
“Prescribers who work every day with biologics — and their patients — are the winners from FDA’s decision. The agency recognized that distinguishable names are essential for pharmacovigilance, patient safety and transparency,” BPC stated. “BPC has strongly advocated for distinguishable names because it is consistent with the science, reflecting that no two biological products can be the same. As biosimilars come to market, it is crucial for physicians to recognize that while highly similar, there will be differences between a biosimilar and the originator product as well as among all biosimilars of the originator.”
The FDA’s recommendations also address the issue of substitution, according to BPC. “Moreover, as FDA recognized, it is critical that physicians also understand that while a product may be approved as biosimilar, it does not mean it is interchangeable with the reference product, meaning that it has not been shown safe to switch back-and-forth between the two,” BPC said.
The Biosimilars Forum, a coalition of 11 biopharmaceutical companies formed this spring, welcomed the FDA’s proposal as well, pointing out that designating official names and proper names for certain biological products helps clarify policy issues on biosimilars.
“We applaud the FDA for reinforcing within the proposed regulation the approach that each biosimilar is a unique drug. We believe this important distinction supports the forum’s position on the CMS J Codes for biosimilars; as single source drugs, biosimilars require separate J Codes,” the Biosimilars Forum said in a statement. “The forum is concerned that the CMS proposed rule may limit the future treatment options that health care professionals would be able to offer their patients and, thus, thousands of patients who are expected to benefit from biosimilars may have limited access to these therapies.”
In their blog post, the FDA’s Woodcock and Midthun added that another issue is what to do with previously approved biological products that have nonproprietary names without a suffix.
“Applying the naming convention to these products would encourage routine use of designated suffixes in ordering, prescribing, dispensing and recordkeeping practices and avoid inaccurate perceptions of the safety and effectiveness of biological products based on their licensure pathway,” they said.
In March, the FDA announced that Zarxio (filgrastim), a cancer treatment from Sandoz Inc., became the first biosimilar product approved in the United States. The product is a biosimilar of Neupogen, an oncology product from Amgen Inc. In announcing the approval, the FDA noted that to a biosimilar must show that it has no clinically meaningful difference in terms of safety and efficacy from the reference product. Zarxio was approved for the same indications as Neupogen.
The Biologics Price Competition and Innovation Act created an abbreviated licensing pathway for biologics shown to be “biosimilar” to or “interchangeable” with an FDA-licensed biological product (the reference product). The abbreviated pathway is intended to allow reliance on certain scientific knowledge about the safety and effectiveness of the reference product and permits a biosimilar product to be licensed based on less than a full complement of product-specific preclinical and clinical data.