Life Science Compliance Update

June 30, 2016

Alliance for Health Reform Holds Biosimilar Panel

The Alliance for Health Reform recently held a briefing on biologic drugs and the emerging market for biosimilar products in the United States. The briefing allowed for a discussion of regulatory and policy questions that continue as the Food and Drug Administration (FDA) continues to review and approve applications for biosimilars. As of right now, the FDA has approved two biosimilars, but only one of those two approvals is available on the market.

The panel discussion included experts such as: Leah Christl, the associate director for therapeutic biologics at the FDA; Barbara Finck, the chief medical officer at Coherus Biosciences; Brian Lehman, the manager of pharmacy benefits and policy at the Ohio Public Employees Retirement System (OPERS); Leigh Purvis, director of health services research at AARP; and Angus Worthing, rheumatologist and member of ACR Government Affairs Committee, American College of Rheumatology.

Panelists represented a wide range of stakeholders and throughout the discussion, panelists talked about public acceptance of biosimilars, the costs of biosimilars (as well as potential for savings), and lingering regulatory issues such as standards for interchangeability and non-proprietary naming requirements.

Discussion Points

Interchangeability Standard

Leah Christl discussed what type of data will be necessary to demonstrate a biosimilar's interchangeability with its reference product. She noted that we are dealing with uncharted waters here, as the United States is the only country to actually differentiate between biosimilarity and interchangeability. She stated that interchangeability is a different standard, but not necessarily a higher standard. She, however, stopped short of giving specific data elements that will be required, noting that no guidance has yet been published.

She offered the suggestion to look to the statute, to see the types of information that might be necessary. She further noted that the information did not have to necessarily be clinical data, and can include elements from post-market analysis. The agency will consider the "totality" of the circumstances, avoiding a "one size fits all" approach.

FDA Regulatory Efforts

While it may seem like it is taking a long time for the FDA to do much of anything about biosimilars, it is important to remember that there are several pieces to the puzzle that need to be put together. According to Dr. Worthing, full transparency is key: it will allow patients to know what they are taking and will provide physicians with the information they need to know about what they're prescribing. He believes that "this can be accomplished by using distinct names for biosimilars, having clear information on FDA drug labels, and implementing consumer-oriented pharmacy dispensing practices."

Biosimilar Development

Barbara Finck spoke on biosimilar development, acknowledging that biosimilars have the same amino acid "backbone" as their reference drug, but as they are made of living cells, they have small differences as a result of manufacturing. Dr. Finck equated it to an expert forger making copies of the Mona Lisa, each copy would be very good, but there would still be small differences between the copy and the original; Dr. Christl equated it to brewing beer: the fermentation process can render each batch slightly different that the one before it, even though they always start with the same ingredients.

Public Acceptance of Biosimilars

The panelists generally discussed how important it is for the biosimilar market to be thriving: Leigh Purvis noted that it may be important for patients to fully understand that there is nothing to fear about biosimilars, and that they would (on average) save patients 25% to 30% as opposed to the reference product. Biosimilars, therefore, may be a solution to the contentious drug pricing topic frequently discussed these days.

Dr. Finck also agreed that biosimilars are safe and effective: she referenced stringent FDA statistical standards and the European experience. Biologics and biosimilars are expected to be covered under Part D.


In the end, Dr. Worthing noted, "It's easy to get lost in the weeds of regulatory issues, but biosimilars naming, labeling and interchangeability issues have real and profound safety and health consequences for patients."

June 08, 2016

FDA Announces Information Collection Regarding Biosimilar Suffixes

The Food and Drug Administration (FDA) has announced an information collection seeking sponsors of past and present biologic applications submit a proposed suffix composed of four lowercase letters for use as the distinguishing identifier included in the name designated by the FDA.

The information collection is pursuant to FDA biologics naming guidance issued in August 2015, "Guidance for Industry on Nonproprietary Naming of Biologic Products." The guidance outlines the FDA's intention for biologics to "designate a nonproprietary name that includes a core name and a distinguishing suffix," this information collection seeks proposed four letter suffixes from sponsors of biological product applications.

The guidance included information collection by requesting that applicants propose a suffix composed of four lowercase letters for use as the distinguishing identifier included in the proper name designated by the FDA at the time of licensure for biological products licensed under the Public Health Service Act. The guidance recommended that applicants submit up to 10 proposed suffixes, in the order of the applicant's preference, in addition to including supporting analyses demonstrating that the proposed suffixes met the factors described in the guidance.

The FDA estimated that they will receive a total of forty requests annually for the proposed proper name for biological products submitted under section 351(a) of the PHS Act, and 6 requests annually for the proposed proper name for biosimilar products and interchangeable products submitted under section 351(k) of the PHS Act.

Previous Response

There were a variety of responses submitted in response to an August 2015 Federal Register notice requesting public comment on the proposed collection of information. According to the FDA, most comments were supportive of the proposal to designate a suffix. Many suggested that a meaningful, distinguishable suffix may help to improve pharmacovigilance, enhance safety, and facilitate identification between biological products. Some comments supported the use of a random suffix to avoid creating an unfair advantage for specific manufacturers. One comment, however, noted that the FDA's estimate of six hours to submit proposed suffixes, which was supposedly based on the FDA's experience, is based only on the time needed to prepare the submission itself after multiple suffixes have been selected. The comment went on to note that because the FDA suggests that each respondent submit three suggested suffixes for consideration, the time needed to do an analysis of each suffix would exceed 720 hours per suffix or 2,160 hours total for the three suffixes, based on their own company experience.

The FDA believes those figures to be high and retain their original estimate of 6 hours, which is based on their familiarity with the average amount of time required by similar submissions to the FDA. The FDA does, however, revise their estimate upward to account for burdens associated with creating and submitting up to ten proposed suffixes for designation.

Industry Request and Opinion

This information collection follows a recent request by seventy different nonprofits and stakeholders, spearheaded by the Alliance for Safe Biologics, that the FDA use meaningful suffixes for biosimilar non-proprietary names, such as the one used with the first biosimilar approval for Zarxio. The group believes that meaningful suffixes are preferable to the random suffixes that are described in the aforementioned draft guidance, and what the agency used for its second approval for Inflectra.

The Alliance for Safe Biologics and other groups sent a letter to Leah Christi, Ph.D., associate director of therapeutic biologics and biosimilars at the FDA, saying, "Meaningful suffixes are easier for patients, providers and pharmacists to both recognize and remember, thus facilitating accurate association between adverse events and specific products." The group also noted that a survey of 400 biologic prescribers suggested they prefer meaningful suffixes over random by a six to one margin, while a survey of 401 United States pharmacists showed a preference for manufacturer-based suffixes over random.

The Biosimilars Forum, BIO and Pharmaceutical Research and Manufacturers of America (PhRMA) have also previously called for the FDA to use "meaningful" and "distinguishable" suffixes linked to the license holder's name.

Comment Submission

Comments on the information collection are due thirty days following publication and may be submitted via fax or email to the Office of Information and Regulatory Affairs, OMB, Attn: FDA Desk Officer, FAX: (202) 395-7285, email: All comments should be identified with the OMB control number 0910-NEW and title, "Nonproprietary Naming of Biological Products." Docket No. FDA-2013-D-1543 should also be referenced.

April 06, 2016

FDA: Biosimilars Labeling Guidance

After several delays the FDA has finally released its draft guidance that says a biosimilar's label does not have to be identical to that of its reference product, adding that differences may be appropriate to inform safe and effective use of the product. FDA also says it may be appropriate to include information about reference product uses not approved for the biosimilar for safety reasons, and additionally calls for a "biosimilarity statement" as part of the prescribing information highlights. FDA did not resolve how interchangeable products should be labeled, but the agency said it is considering the types of information that would support a demonstration that a biosimilar is interchangeable with a reference product. FDA indicating that labeling recommendations will be included in a future guidance.

Comments to the draft guidance are due June 3. They can be submitted to using Docket Number FDA-2016-D-0643.

The Guidance

FDA's general principle is outlined in the guidance, recommending that, "in the biosimilar product labeling, applicants incorporate relevant data and information from the reference product labeling, with appropriate product-specific modifications." As a general matter, FDA believes the product should not include a description of these data because a clinical study supporting the licensure of the biosimilar product generally would not be designed to independently demonstrate the safety and efficacy of the product. However, labeling must meet the content and format requirements of the physician labeling rule and the pregnancy and lactation rule, regardless of the format of the reference product label.

The guidance outlines that relevant data and information from the reference product labeling that should be incorporated into the biosimilar product labeling depends on "whether the applicant is seeking approval for all conditions of use or fewer than all conditions of use of the reference product for the biosimilar product." FDA says that it anticipates "similar" text in biosimilar product labeling that is based on the reference product labeling, although it need not be identical and should reflect currently available information necessary for the safe and effective use of the biosimilar.

Furthermore, FDA outlines that the labeling for the biosimilar product should be "specific to the conditions of use sought for the biosimilar product and should be consistent with language previously approved for the reference product for those conditions of use." FDA explains circumstances where there may be a biosimilar licensed for fewer indications than the reference product. Additionally, FDA discusses inclusion of initial U.S. approval of the biosimilar and a biosimilarity statement which the FDA recommends including "on the line immediately beneath the initial U.S. approval date in highlights, that the product is biosimilar to the reference product."

The FDA also notes that in biosimilar product labeling, "the approach to product identification depends on the context of the information being presented." FDA recommends that the biosimilar product name be used in the labeling text that is "specific to that biosimilar product or refers solely to the biosimilar product." FDA further recommends that if the product has a proprietary name, "the proprietary name be used in these instances." If "a proprietary name is not available for the biosimilar product, the biosimilar product's proper name should be used". FDA outlines examples in the guidance, including when to use the biosimilar product name, the reference product name, core name, and more than one product name.

The guidance discusses FDA-approved patient labeling, including updated safety information and additional conditions of use. FDA also explains its thinking on the submission of initial and revised labeling. Regarding interchangeable biologics, FDA is still considering the issue and will make recommendations on the labeling of interchangeable products in future guidance.


According to a report, industry may not be too pleased with the guidance. Although Regulatory Focus quoted Duncan Emerton, senior director at FirstWord Pharma, as saying the guidance does not have any major surprises as it aligns with the way FDA labeled the first approved biosimilar in the US, Zarxio. Emerton says he's spoken to lots of physicians "and the general consensus has been a need for full disclosure (i.e. include all the relevant data for the biosimilar whenever possible)."

The details of the guidance are important, but it is also important that the document was released in the first place. "The big news about the draft guidance is that they got it out, and this alone will end some uncertainty for both the biosimilar applicant and the originator of the biologic, also known as the reference product sponsor," Siegmund Gutman, chair of Proskauer's life sciences patent practice told Bloomberg BNA.

Bloomberg BNA also reported that the Generic Pharmaceutical Association and its Biosimilars Council released a statement that it was a pleased with the guidance that "provides manufacturers with additional clarity needed to manufacture and distribute more affordable versions of biologic medicines for patients."

Meanwhile, Patients for Biologics Safety and Access and the American College of Rheumatology released statements indicating that the clinical trial data for the biosimilar should be included in the labeling and that the label should specify whether the supporting clinical data for each indication are derived from studies of the biosimilar or the reference biologic.

The guidance comes as an IMS Institute for Healthcare Informatics report highlights that the use of biosimilar drugs could save more than $100 billion in the United States and five European markets by 2020. The report reviewed markets in Britain, France, Germany, Italy, Spain, and the United States. It included eight commonly used brand-name medications that are scheduled to lose patent protection within the next five years and the projected savings that would result if corresponding biosimilars were introduced into the market. The report estimated that the United States and five European markets would save a combined $110 billion. However, the report noted the savings could not be realized unless health care providers are properly educated about biosimilars and drug makers adopt "smart market access strategies" to spur the drugs' use.


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