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Biologics and Biosimilars: Background and Key Issues Biologics and Biosimilars: Background and Key Issues (name redacted) Specialist in Biomedical Policy September 7, 2016 Congressional Research Service 7-.... www.crs.gov R44620 Biologics and Biosimilars: Background and Key Issues Summary A biological product, or biologic, is a preparation, such as a drug or a vaccine, that is made from living organisms. Compared with conventional chemical drugs, biologics are relatively large and complex molecules. They may be composed of proteins (and/or their constituent amino acids), carbohydrates (such as sugars), nucleic acids (such as DNA), or combinations of these substances. Biologics may also be cells or tissues used in transplantation. A biosimilar, sometimes referred to as a follow-on biologic, is a therapeutic drug that is similar but not structurally identical to the brand-name biologic made by a pharmaceutical or biotechnology company. In contrast to the relatively simple structure and manufacture of chemical drugs, biosimilars, with their more complex nature and method of manufacture, will not be identical to the brand-name product, but may instead be shown to be highly similar. The Food and Drug Administration (FDA) regulates both biologics and chemical drugs. Biologics and biosimilars frequently require special handling (such as refrigeration) and processing to avoid contamination by microbes or other unwanted substances. Also, they are usually administered to patients via injection or infused directly into the bloodstream. For these reasons, biologics often are referred to as specialty drugs. The cost of specialty drugs, including biologics, can be extremely high. In April 2006, the European Medicines Agency (EMA) authorized for marketing in Europe the first biosimilar product, Omnitrope, a human growth hormone. The EMA has authorized a total of 21 biosimilars for the European market. The introduction of biosimilars in Europe has reduced prices for biologics overall, in some cases by 33% compared with the original price of the brand- name product. For one drug in Portugal, the price reduction was 61%. In contrast, the pathway to marketing biosimilars in the United States has had several barriers. FDA approved Omnitrope in June 2006, following an April 2006 court ruling that the FDA must move forward with consideration of the application. At the time Omnitrope was approved, FDA indicated that this action “does not establish a pathway” for approval of other follow-on biologic drugs and stated that Congress must change the law before the agency can approve copies of nearly all other biotech products. Four years later, in March 2010, Congress established a new regulatory authority for FDA by creating an abbreviated licensure pathway for biological products demonstrated to be “highly similar” (biosimilar) to or “interchangeable” with an FDA-licensed biological product. The new authority was accomplished via the Biologics Price Competition and Innovation Act (BPCIA) of 2009, enacted as Title VII of the Affordable Care Act (ACA, P.L. 111-148). In addition, Congress authorized FDA to collect associated fees via the Biosimilar User Fee Act of 2012 (BsUFA, P.L. 112-144). FDA has approved three biosimilars for marketing in the United States: Zarxio (filgrastim-sndz) in March 2015, Inflectra (infliximab-dyyb) in April 2016, and Erelzi, (etanercept-szzs) in August 2016. The entry of such products on the U.S. market may result in price reductions similar to those that have occurred in Europe. Congressional Research Service Biologics and Biosimilars: Background and Key Issues Contents Introduction ..................................................................................................................................... 1 The Need for Biosimilars Legislation ............................................................................................. 3 New Pathway for Biosimilars .......................................................................................................... 6 Other Topics Related to Biosimilars ................................................................................................ 9 Naming ...................................................................................................................................... 9 Labeling ................................................................................................................................... 11 Transition ................................................................................................................................ 12 Interchangeability and Substitution ......................................................................................... 13 Reauthorization of the Biosimilar User Fee Act (BsUFA) ...................................................... 14 Tables Table 1. Relative Size of Chemical and Biologic Drugs ................................................................. 1 Table 2. Biosimilars Authorized for Marketing in Europe by the EMA ......................................... 5 Table 3. Biosimilars Approved for Marketing in the United States by FDA................................... 8 Table 4. FY2017 Fee Rates Under BsUFA .................................................................................... 16 Table B-1. Comparison of Biologic Drug Prices ........................................................................... 20 Table B-2. Examples of Average Biologic Drug Prices for Top-Selling Drugs in 2015 ............... 21 Table C-1. The Top Three Selling Drugs, 1996-2015 ................................................................... 22 Table D-1. CDER Purple Book: Licensed Biological Products .................................................... 23 Table D-2. CBER Purple Book: Licensed Biological Products .................................................... 24 Appendixes Appendix A. Major Laws on Biologics Regulation ...................................................................... 17 Appendix B. Comparison of Biologic Drug Prices ....................................................................... 20 Appendix C. Top Selling Drugs .................................................................................................... 22 Appendix D. The Purple Book ...................................................................................................... 23 Contacts Author Contact Information .......................................................................................................... 25 Congressional Research Service Biologics and Biosimilars: Background and Key Issues Introduction A biologic or biological product is a preparation, such as a therapeutic drug or a vaccine, made from living organisms, either human, animal, yeast, or microorganisms. Biologics are composed of proteins (and/or their constituent amino acids), carbohydrates (such as sugars), nucleic acids (such as DNA), or combinations of these substances. Biologics may also be cells or tissues used in transplantation. A biosimilar, sometimes referred to as a follow-on biologic, is a therapeutic drug that is similar but not structurally identical to the brand-name biologic made by a pharmaceutical or biotechnology company. The brand-name product is sometimes referred to as the innovator or reference product. In contrast to biologics, most commonly used drugs—over-the-counter drugs and most prescription drugs—are synthesized via a chemical process. A generic drug is chemically identical to its reference brand-name drug. The molecular structure of a commonly used chemical drug is much smaller than a biologic and therefore less complicated and more easily defined. For example, Table 1 shows that the chemical drug aspirin contains nine carbon atoms, eight hydrogen atoms, and four oxygen atoms while the biologic drug Remicade contains over 6,000 carbon atoms, almost 10,000 hydrogen atoms, and about 2,000 oxygen atoms. Inflectra, which is biosimilar to Remicade, was approved by FDA in April 2016. Table 1. Relative Size of Chemical and Biologic Drugs Drug (nonproprietary name) Molecular formula chemical drugs aspirin C9H8O4 Tylenol (acetaminophen) C8H9NO2 Sovaldi (sofosbuvir) C22H29FN3O9P small biologic drugs Lantus (insulin glargine) C267H404N72O78S6 Epogen (epoetin alfa) C809H1301N229O240S5 Neupogen, Zarxio (filgrastim) C845H1339N223O243S9 growth hormone (somatropin) C990H1528N262O300S7 large biologic drugs Enbrel, Erelzi (etanercept) C2224H3472N618O701S36 Remicade, Inflectra (infliximab) C6428H9912N1694O1987S46 Source: Drugs @FDA and Drugs.com. Notes: The nonproprietary name of a drug product is used in drug labeling, drug regulation, and scientific literature to identify a pharmaceutical substance or active pharmaceutical ingredient. C, carbon; H, hydrogen; O, oxygen; N, nitrogen; F, fluorine; P, phosphorus; S, sulfur. The Food and Drug Administration (FDA) regulates both biologics and chemical drugs. The Center for Biologics Evaluation and Research (CBER) within FDA regulates what are often referred to as traditional biologics, such as vaccines, blood and blood products, allergenic Congressional Research Service 1 Biologics and Biosimilars: Background and Key Issues extracts, and certain devices and test kits.1 CBER also regulates gene therapy products, cellular therapy products, human tissue used in transplantation, and the tissue used in xenotransplantation—the transplantation of non-human cells, tissues, or organs into a human.2 The FDA Center for Drug Evaluation (CDER) regulates prescription brand-name and generic drugs, over-the-counter
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