Arthritis Foundation Statement
on Follow-on Biologics
The Arthritis Foundation supports legislation to establish an efficient pathway for the approval of safe and effective follow-on biologics administered by the Food and Drug Administration (FDA).
Background:
Biotech medicines — more commonly known as "biologics" — involve proteins that are made from living cells, which involve many complex molecular pathways and folding or assembling of the final protein which affect how the protein functions. Biologics are defined as "a virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, or analogous product ... applicable to the prevention, treatment or cure of a disease or condition of human beings." (PHS Act §351(i), 42 U.S.C §262(i)).
The terms biosimilars, generic biologics, biogenerics, and follow-on biologics are often used informally to refer to certain products produced through biotechnology processes that are essentially "copies" of the original product. The concept of a "follow-on" product is that an applicant (manufacturer) could obtain approval for its product through the submission of an abbreviated application to the Food and Drug Administration (FDA). An "abbreviated application" means that the approval of a follow-on product is dependent on the FDA's conclusion regarding the safety and efficacy of a previously approved original product. The abbreviated application also may contain additional, essential data, other than the underlying clinical data supporting the approved product, to establish that the follow-product is safe and effective. An abbreviated pathway for approval of generic pharmaceuticals exists under Hatch-Waxman, but no similar legal authority exists for FDA to approve generic biologics.
Unlike small molecule therapies (pharmaceutical pills), which are easier to duplicate, the characteristics of a biotechnology manufactured protein make these medicines more complex to replicate in every stage of development and use. Additionally, the manufacturing process for biologics is unique to each biological product, and is not generally disclosed as part of the published patent, making it more challenging for the original biological product to be copied.
Arthritis and Biologics:
The innovation of biological therapies has transformed the treatment and care of many different forms of arthritis, including rheumatoid arthritis, ankylosing spondylitis, psoriatic arthritis, and juvenile arthritis. The current biological therapies profoundly reduce the pain and inhibit the progression of joint damage for these chronic and debilitating diseases, which affect an estimated 4 million people in the US. These complex therapies prevent disability from rheumatoid arthritis and other rheumatic conditions. For many patients with arthritis, these products are life-altering therapies; however, for some patients, access to these therapies is limited or non-existent due to their cost and availability. And for others, no treatment currently exists to treat their specific condition.
Arthritis Foundation Priorities for Follow-on Biologics Legislation:
The Arthritis Foundation supports legislation to establish an efficient pathway for the approval of safe and effective follow-on biologics within the Food and Drug Administration (FDA). We ask Congress to consider the following points as it creates a bill that will encourage innovation of new therapies while making safe, effective and cheaper biologics available to people with arthritis and other rheumatic diseases.
- The pathway should always be guided by the best current available science and technology to ensure all products are safe. Patient safety and efficacy must be the primary criteria in any approval process.
- Biologics are not the same as small molecule pills and the pathway needs to recognize and acknowledge the differences.
- The scientific principles that guide FDA's comparability policy are still valid and can be adopted for follow-on biologics. The FDA should be allowed to review any follow-on biologic using the same guidelines it uses for approved products. FDA must retain the essential discretion to require any additional data that are needed to assure the safety and efficacy of the follow-on biologic, including clinical trials if necessary.
- FDA should be given the authority using the current state of scientific knowledge to make the determination if a follow-on product is interchangeable (interchangeable defined as safely substituted for one another) with the approved product.
- Due to the especially unique nature of biologics and to minimize potential adverse health outcomes, the decision for which product should be prescribed is best made between the physician and the patient. Automatic substitution without the consent of the treating physician should not be permitted.
- Appropriate post-marketing studies and surveillance should be established by the FDA and adhered to by follow-on biologic manufacturers.
- The pathway should ensure a clear timeline to both manufacturers of approved biologics and follow-on biologics for when follow-on biologics can enter the market.
- An efficient pathway for follow-on biologics must strive to ensure continuing innovation and the development of new biological products.
- The decision making process regarding FDA approval of follow-on biologics should be as transparent to the public as possible with appropriate input from external stakeholders including patient organizations.
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04/2009
