Biosimilars Frequently Asked Questions

What are biologics?

Biologics are large, complex medicines, many of which are protein-based, that are derived from living material. The proteins in biological products have a three-dimensional structure that plays a critical role in the medicine’s safety and efficacy. This three-dimensional structure is determined by the order of the amino acids, the folding or bending of the amino acid chain, and the interactions between different parts of a single chain or between multiple chains joined together.

What are biologics used to treat?

Many biologics have been approved to treat a wide range of diseases and conditions including cancer, rheumatoid arthritis, and multiple sclerosis. Some 630 additional biotech medicines are currently in development to treat more than 100 diseases, including 250 for various cancers.

How are biologics different from small molecule pharmaceuticals?

Physically, biologics are much larger and more complex than traditional small molecule drugs. Another key difference between biologics and small molecule drugs is in their manufacturing. Small molecule drugs are made by adding and mixing together known chemicals using a series of controlled and predictable reactions-basic organic chemistry. The small molecule manufacturer controls the reactions that synthesize the molecule. Biologics, on the other hand, are grown and harvested from living cells- genetic engineering. The biologics manufacturer controls the environment of the cells but the cells complete all of the steps in the synthesis of the molecule. A unique cell line is genetically engineered to produce a specific therapeutic protein - the biologic product. Often, it takes many months to manufacture even small quantities of biological products.

Because biologic products derive from living matter, it is impossible for two biologic products made by different, unrelated manufacturers to be the same. In other words, different cells and different processes produce variants, not copies, of the biologic product. These subtle differences in structure can have a significant impact on the product’s clinical safety and therapeutic effect.

Why does the approval process for biosimilars need to be different from the approval process we use for small molecule generic drugs?

Biologics are very different from small molecule drugs because they are derived from living material. They are structurally larger and more complex than small molecule drugs and the manufacture of biologics requires the careful control of living cells. Living things are by nature heterogeneous, and the manufacturing process, which actually requires growing and harvesting the product, is quite different, too. In fact, because biologics derive from living matter, it is impossible using current science to apply the same standards of approval for generic drugs to biosimilars.

Why don’t patent protections provide the same certainty for biologics that they do for small molecule drugs?

Generic copies of small molecule drugs must meet standards of sameness in order to rely on the innovator product’s data and clinical trials to receive FDA approval. For small molecule drugs, if the innovator product’s patent life has not expired, then competitors cannot legally bring generic versions of the product to market.

Biologics are large, complex, molecules. This complexity, and because they derive from living matter, makes it impossible using current science for two biologic products to meet standards of sameness-that is, the two products would not be identical. Instead, biologics can only meet standards of similarity. Adhering to a standard of similarity for follow-on biologic products means it is possible for a competitor to attempt to rely on an innovator product’s data and clinical trials to receive FDA approval without infringing on the innovator product’s patent. In other words, the competitor product is similar enough to receive FDA approval but just different enough to avoid patent infringement-making it possible for the competitor to attempt to work around patents and meaning innovator biologic products could face follow-on competition much earlier than small molecule drugs.

In order to ensure adequate intellectual property protections for biologics, both patents and data protection are critical. Based on economic analysis and the unique aspects of biologics, at least 14 years of data exclusivity or data protection is needed before other companies can use the innovator company’s data - which was developed over many years - to seek approval for a biosimilar version. A base exclusivity period of at least 14 years is needed to provide more certainty for continued investment in R&D in this sector for development of new biologic medicines for unmet medical needs.

What is data exclusivity?

Data exclusivity or data protection refers to a defined period of time, beginning upon FDA approval, during which the FDA may not rely on the data submitted by an innovator, or its findings about the innovator’s product, to approve another company’s product based on limited (“abbreviated”) data packages. The data exclusivity period is unrelated to the duration of the patents that cover the medicine or its use.

Why should biosimilars be required to include clinical data to support their application for approval?

Generic versions of small molecule drugs must meet exacting standards of sameness in order to receive FDA approval. As a result, we usually can be assured that the generic version of a small molecule drug has the same clinical profile as the innovator or brand name product. For biologics, however, it is impossible for two products to be proven identical or to meet this sameness standard using current science. Instead, follow-on biologics-biosimilars-can only meet standards of similarity. Biosimilars are therefore variants, rather than copies, of the original product and thus data demonstrating their safe and effective use in patients is needed.

Why is it so important that policy-makers strike the right balance between fostering innovation and creating a pathway for the approval of biosimilars?

Policy makers need to create an environment that provides adequate intellectual property protections to ensure continued R&D investment. Due to the unique characteristics of biologics, including the risk and uncertainty in development, economic analysis of the time needed to recoup investment, and the high capital costs and time involved in building specialized facilities, both patent protection and a data exclusivity period of at least 14 years is needed. Venture capitalists and other sources of capital for firms in this sector, in particular, would be more likely to shift their investments to less risky ventures with a higher potential for a return on their investment without such
protection.

What principles do we want our congressional delegation to support in legislation?

America’s biopharmaceutical research companies support the establishment of a science-based biosimilars regulatory approval pathway with substantial incentives for innovation that ensures patient safety and does not hinder the development of future medicines. We believe Rep. Anna Eshoo’s (D-CA) bill (H.R.1548) better meets these principles than Rep. Waxman’s (D-CA) bill (H.R. 1427).

Rep. Eshoo’s bill provides innovator companies 12 years (plus 2 years for new indications) of data protection for biologics and a more balanced process for protecting patent rights. This will provide researchers and investors with some measure of certainty which is needed to make long-term research and development decisions and support future medical innovation. Moreover, the legislation also contains measures designed to assure that only high quality, safe and effective biosimilars will be approved, including requiring analytical studies, animal studies, and clinical trials.

Why don’t we support Chairman Waxman or Senator Schumer’s legislation?

Chairman Waxman’s and Senator Schumer’s (D-NY) legislation (H.R.1427 / S.726) only provide at most 5 years of data protection for biologics, and a less fair way of addressing patent protection. This means manufacturers of biologics would face considerably less certainty over the timing of follow-on biologic market entry. This could dramatically hinder future innovation as it could greatly impact research and development investment needed to generate new biologic medicines and new uses for biologics to treat some of the most challenging diseases facing patients today.

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