Posts – Page 71 – Safe Biologics

ASBM Says Distinct USAN Names for Biosimilars are Needed

November 29, 2012

The Alliance for Safe Biologic Medicines (“ASBM“), a self-described “organization composed of diverse healthcare groups and individuals from patients to physicians, innovative medical biotechnology companies, and others who are working together to ensure patient safety is at the forefront of the biosimilars policy discussion,” urges FDA in a new paper to adopt unique non-proprietary names for all biological products licensed under the Public Health Service Act (“PHS Act”), and in particular biosimilar versions of reference products (even those that are interchangeable).

Read full article at FDA Law Blog

ASBM Paper on Biosimilar Naming Published in the Food and Drug Law Institute’s Policy Forum

November 28, 2012

Unique names key factor to ensure patient-safety in biosimilar pathway

WASHINGTON – The Food and Drug Law Institute (FDLI) Policy Forum today published a white paper on biosimilar naming authored by the Alliance for Safe Biologic Medicines (ASBM). “It’s All About the Name: What is the Imperative of Adopting Unique Names For Biologic and Biosimilar Therapeutics?” explains that in order to ensure patient-safety as biosimilars are approved in the U.S., they must be given distinct, non-proprietary names. The paper identifies key challenges and practical solutions to help facilitate a solution to ensure safety is the cornerstone of the biosimilars pathway.

In the paper, ASBM makes four policy recommendations:

All biologics should receive distinct non-proprietary names.
United States Pharmacopeia (USP) should work with FDA to adapt the product monograph system to accommodate the unique attributes of structurally-related, but distinct, biologic medicines.
The non-proprietary name of a reference product and product/s biosimilar to it should have a common, shared root but have distinct and differentiating suffixes.
Products designated interchangeable should have a distinct name from the reference product for which they are considered interchangeable to facilitate accurate attribution of adverse events.

“As an organization, ASBM has been working with our diverse membership and Advisory Board that represents patients, physicians, pharmacists, scientists, manufacturers and academics to support the FDA as they work to bring biosimilars to patients in the U.S.,” said Dr. Richard Dolinar, ASBM Chairman. “Naming has been a prominent biosimilar safety issue and we are honored to be featured in FDLI’s publication and hope that this paper helps identify the challenges and solutions we need to take into consideration.”

FDLI’s Food and Drug Policy Forum is a concise (approximately 10 page), twice-a-month, peer-reviewed, digital publication on current food and drug policy topics. Posed in the form of a question, each issue provides subscribers and purchasers with pertinent background information, relevant research, a discussion of central issues, relevant resources, and policy recommendations.

Read the paper here.

About the Alliance for Safe Biologic Medicines
The Alliance for Safe Biologic Medicines (ASBM) is an organization composed of diverse healthcare groups and individuals from patients to physicians, innovative medical biotechnology companies, and others who are working together to ensure patient safety is at the forefront of the biosimilars policy discussion. We serve as an authoritative resource center of information for the public, medical communities, the FDA and other state and federal policymakers during the implementation of the biosimilars pathway and beyond.

About the Food and Drug Law Institute
The Food and Drug Law Institute, founded in 1949, is a non-profit organization that provides a marketplace for discussing food and drug law issues through conferences, publications and member interaction. The scope of FDLI includes food, drugs, animal drugs, biologics, cosmetics, diagnostics, dietary supplements, medical devices and tobacco. As a not-for-profit 501(c)(3) organization, FDLI does not engage in advocacy activities. FDLI’s Mission is to provide education, training, and publications on topical food and drug law; act as a liaison to promote networking as a means to develop professional relationships and idea generation; and ensure an open, balanced marketplace of ideas to inform innovative public policy, law, and regulation.

Dr. Dolinar Quoted in November Issue of BioProcess International

November 15, 2012

In an article titled “Polysorbates, Immunogenicity, and the Totality of the Evidence” author Edward T. Maggio, quotes Dr. Dolinar’s testimony at the FDA pubic hearing, saying:

“Unwanted immunogenicity is the preeminent safety challenge associated with all biological therapeutics and can result in unexpected and sometimes severe adverse effects. Complicating matters, side-effects may only appear in patients after higher doses or prolonged duration of treatments and may be attributed to a number of patient, disease, or product related factors.”

Read the full article here.

ASBM Member Releases Position Statement on Substitution

November 7, 2012

The American Academy of Dermatology released a position statement on automatic substitution that calls for several thresholds to all be met before a biosimilar can be substituted. Read the statement here.

BIO Principles on Patient Safety in the Substitution of Biologic Products

October 29, 2012

In August, BIO released Principles on Substitution for Biologic Products. Read the Principles here.

ASBM Statement on Automatic Substitution

October 26, 2012

The Patient Protection and Affordable Care Act passed by Congress and signed into law by President Obama in March 2010, contained a provision establishing an abbreviated pathway for the approval of biosimilars (also referred to as followon biologics, or subsequent entry biologics) in the U.S.

In February 2012, the U.S. Food and Drug Administration (FDA) released a set of three draft guidance documents on biosimilar product development to assist industry in developing such products in the U.S. The guidance documents outlined the FDA’s current thinking on key scientific and regulatory factors involved in submitting applications for biosimilar products to the agency but did not provide any insight on how the agency would handle, “interchangeable biosimilars.” In fact, the regulatory agency sent an unambiguous signal on this topic when it stated in the draft guidance “[t]his document is not intended to provide an overview of FDA’s approach to determining interchangeability because FDA is continuing to consider the type of information sufficient to enable FDA to determine that a biological product is interchangeable.”

On May 24, 2012 ASBM Chairman Richard Dolinar, M.D., convened a working group of Advisory Board members to discuss the elements of a physician notification policy for interchangeable biosimilars that prioritizes patient safety and protects the relationship between physicians and their patients but also respects the sovereignty of pharmacists as healthcare providers. As ASBM continues to work with stakeholders to develop an official position statement on this very important topic there are a few key principles that ASBM believes must be reflected in any policy recommendation:

(1) Physicians have the authority to specify “do not substitute” for biological products and that specification overrides any policy – e.g. by payers or state law – that would have substitution be the standard or default practice;

(2) Physicians and pharmacists should work collaboratively to ensure that the treating physician is aware of the exact biologic – by manufacturer – given to a patient in order to facilitate patient care and accurate attribution of any adverse events that may occurs; and

(3) The timing of the notification process must not impose an undue burden on the pharmacist and need not be in advance of a substitution being made but must be timely enough to facilitate accurate record keeping and attribution of adverse events by the physician.

BIO Principles

October 25, 2012

BIO developed the bellow set of Biosimilar Principles to serve as recommendations for Congress for creating a biosimilar pathway:

As Congress explores the creation of any regulatory pathway for follow-on biologics, it is essential that Congress recognize and adopt the following key principles:

Ensure Patient Safety. Patients should not have to accept greater risks or uncertainties in using a follow-on product than an innovator’s product. Thus, Congress should:

Ensure that approval of follow-on biologics is based on the same rigorous standards of safety, purity, and potency applied by FDA for the approval of pioneer biotechnology products.
Recognize that clinical trial evidence and data are fundamental for evaluating and demonstrating the safety and effectiveness of a follow-on biologic, and must be conducted on a product-by-product basis. In particular, immunogenicity testing is necessary to avoid putting patients at risk of adverse effects from immune reactions.
Not preclude adequate post-market evaluation of follow-on biologics products. It is critical that follow-on biologics are properly evaluated through post-marketing surveillance and post-marketing clinical studies as needed.
Avoid specific constraints on the scientific conclusions FDA can reach in evaluating the similarity or comparability of follow-on biologics.
Ensure that follow-on biologics will be assigned a non-proprietary name readily distinguishable from that of the innovator’s version of the product. Assigning the same name to a product that is not the same would be confusing and misleading to patients, physicians, and pharmacists, could result in inadvertent substitution of the products, and would make it difficult to quickly trace and address adverse events that may be attributable to either the innovator or follow-on product.

Recognize Scientific Differences Between Drugs and Biologics. Biologics are much more complex than small molecule chemical drugs. Thus, Congress should:

Recognize that the methods used to show that one chemical drug is the same as another are different from and insufficient for biologics. Thus, versions of a biological product made by different manufacturers must be evaluated on a case-by-case basis, because they will differ from each other in certain respects. The methods used by innovators to demonstrate continued safety and effectiveness after a manufacturing process change are insufficient to demonstrate safety and effectiveness of a follow-on biologic made by a different manufacturer using a different process.
Recognize that, as innovator companies’ experiences with respect to pioneer biotechnology products have shown, and as FDA has long emphasized through its regulation and guidance, small product or manufacturing differences in biologics can result in significant safety and/or effectiveness differences.

Maintain the Physician-Patient Relationship. Small molecule generic drugs can be designated as therapeutically equivalent and may be dispensed interchangeably with innovator products without physician knowledge. In contrast, the current state of science is not sufficient to establish interchangeability for complex follow-on biologics. Indeed, FDA recently stated that it “has not determined how interchangeability can be established for complex proteins.” Accordingly, Congress should ensure that patients are not given follow-on biologics unless expressly prescribed by a physician.

Preserve Incentives for Innovation. In order to preserve incentives to research, develop and manufacture new innovative therapies and cures, as well as new indications for such products, any statutory pathway for follow-on biologics must:

Include substantial non-patent data exclusivity, during which time follow-on manufacturers could not rely on FDA’s prior approval of pioneer biologics to support approval of their own products. Such data exclusivity is necessary because a follow-on biologic may be similar enough to a pioneer biologic for regulatory approval purposes, but different enough to avoid the innovator’s patents. Thus, non-patent exclusivity is necessary to maintain effective market protection. Further, the fledgling nature of the biologics industry, its heavy dependence on access to significant amounts of high-cost public and private investment capital, and the high risks and costs involved in the development of new biologic medicines all warrant a substantial period of exclusivity.
Respect intellectual property and other legal rights. Follow-on biologic products should not be approved until after all statutory protections, including data exclusivity and patent protections, are no longer available for the approved pioneer product. Any follow-on biologics pathway should fully respect existing trade secret protections for innovators’ data and not permit the use of protected data for the purpose of approving follow on products. It also must not abrogate or limit constitutional or statutory rights of patent holders to protect against infringement.
Provide adequate notice and process rights. Any follow-on biologics regulatory pathway should ensure that any patent challenge involving the follow-on biologic product will be litigated prior to marketing approval of the follow-on product, in order to protect the innovator’s intellectual property rights and avoid confusion in the medical, patient, and payer communities. Further, any follow-on biologics regulatory pathway should not create special patent litigation rules that favor follow-on biologics manufacturers.
Ensure Transparent Statutory and Regulatory Processes. Manufacturers of innovator products should be provided full and fair opportunities to engage Congress and other stakeholders in a meaningful public process. Establishing a balanced and rigorous statutory pathway for follow-on biologics requires deliberative evaluation of numerous complex scientific, legal, intellectual property and economic issues.

Further, any such pathway must require that FDA follow a transparent and public process in determining data requirements for the approval of specific follow-on biologics.

Continue to Prioritize FDA Review and Approval of New Therapies and Cures. Any applications for approval of follow-on biologics will raise novel and complex questions of science and law, requiring substantial time and additional resources to ensure a thorough regulatory review for safety, purity, and potency. In order to avoid slowing down FDA’s review and approval of new therapies and cures, many for currently untreatable and serious diseases, Congress must ensure that workload associated with these new applications does not harm FDA’s ability to efficiently review new drugs and biologics, and that new treatments continue to have the highest review priority.

More information on the Principles can be found on BIO’s website.

Inside Health Policy: Stakeholders Debate Biologic Product Drift’s Impact On Biosimilars

October 25, 2012

One biosimilar developer is pushing FDA to address how changes to innovator biologics over time could affect biosimilar products that reference them, advocating for the agency to use comparability standards as a basis for determining biosimilarity, allow biosimilar determinations based only on one version of an innovator product if drift occurs and investigate ways to slow down innovator activities that create drift. Other sources said, however, that biologic products naturally drift over time and manufacturing improvement changes should not be prevented by FDA.

Concerns over biologic drug product drift — product changes that can occur over time as a result of manufacturing changes — are resurfacing as industry groups continue to push FDA to provide more information on how it will determine biosimilar interchangeability.

Read the article on Inside Health Policy.

Inside Health Policy: Stakeholders Debate Biologic Product Drift’s Impact On Biosimilars

October 25, 2012

Read the article on Inside Health Policy.

Phoenix Forum

October 5, 2012

The Alliance for Safe Biologic Medicines (ASBM), the International Cancer Advocacy Network (ICAN) and AZBio hosted “Biosimilars Policy Forum: Ensuring Patient Safety” on September 27, 2012 in Phoenix, AZ, to discuss the complex challenges the Food and Drug Administration (FDA) faces as it seeks to establish a regulatory pathway that will lead to the approval of biosimilars in the U.S.