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  1. FDA Voices: Perspectives From FDA Leadership and Experts

Ensuring Innovation and Competition for Biologics Leads to More Timely Products for Patients

By: Janet Woodcock, M.D., Director, Center for Drug Evaluation and Research, and Peter Marks, M.D., Ph.D., Director, Center for Biologics, Evaluation and Research


Janet Woodcock, M.D.
Janet Woodcock, M.D.

Today, the FDA approved the agency’s 20th biosimilar product, Kanjinti (trastuzumab-anns), for the treatment of patients with certain types of breast or stomach cancer. It is the fifth FDA-approved biosimilar to Herceptin (trastuzumab), a remarkable development in and of itself, since about four years ago, there were no biosimilars approved in the U.S.

In 2010, Congress authorized an abbreviated licensure pathway through the Biologics Price Competition and Innovation Act (BPCI Act) for biological products that are demonstrated to be biosimilar to, or interchangeable with, an FDA-licensed biological reference product. One of the goals of the BPCI Act was to create greater competition in the medical marketplace that would increase treatment options for patients and lead to less expensive alternatives. This is critical, since biologics, which are generally complicated, large molecule drugs, have become a mainstay of modern medicine, play a critical role in the treatment of serious illnesses, and often present the only effective treatment option for some patients. Indeed, biologics today account for about a third of new therapies approved by the FDA.

Increasing competition in the market for biological products faces obstacles, such as current payment systems, litigation, and rebating practices. A robust market for safe, effective biosimilar products is crucial — more products on the market increases competition, which can lead to more access and reduced health care costs for patients and our nation’s health care system. Biologics, which represent almost 40 percent of all prescription drug spending, play an outsized role in rising drug prices, accounting for 70 percent of the growth in drug spending from 2010 to 2015. Biosimilars play a very important role in countering this trend, much as generics have helped make essential small molecule drug therapies available to patients at a reduced cost.

The FDA has now approved a total of 20 biosimilars for nine different reference products since 2015. This includes at least one biosimilar approved for Avastin, Enbrel, Epogen/Procrit, Herceptin, Humira, Neulasta, Neupogen, Remicade, and Rituxan — each among the top selling biological drug in the United States. Three biological reference products (Avastin, Epogen/Procrit, and Rituxan) have one approved biosimilar. Three (Enbrel, Neulasta, and Neupogen) have two approved biosimilars. Two (Humira and Remicade) have three approved biosimilars. And Herceptin, noted above, now has five approved biosimilars.

CBER Director, Peter Marks, M.D., Ph.D.
Peter Marks, M.D., Ph.D.

Multiple products in a class can also drive competition and innovation

We recognize that, while competition from biosimilar and interchangeable products is an essential part of ensuring that biological products are accessible to patients and evolve with science, competition among innovator products can provide patients with more choices and promote innovation. By facilitating innovation that leads to more approved products, we can promote more competition within classes of biological products. This can offer different therapeutic options for patients, which can drive innovation to benefit patients and opportunities for price competition. To that end, we’re advancing regulatory frameworks that provide clear development pathways to help bring safe and effective therapeutic options to market more efficiently. We’re also working to help promote technologies, like advanced manufacturing methods for vectors used in gene therapies, that could help increase supply of these critical tools, potentially lowering manufacturing costs and encouraging innovation by developers of these products. These scientific advancements and increased therapeutic options will ultimately benefit patients.

While the U.S. market for biosimilars is still maturing, we welcome the opportunity to briefly reflect on the success that our 20th biosimilar approval represents. We also know there is much more work to be done toward building the robust competitive environment we envision for biosimilars.

Making the road ahead as safe and smooth as possible

The FDA has been hard at work implementing the BPCI Act in a way that maintains a balance between encouraging and rewarding medical innovation and facilitating robust and timely market competition. The timely and efficient reviews of biosimilar applications can help consumers have access to more affordable medicines after certain time periods related to patents and exclusivities have lapsed. We also reiterated our commitment to ensuring that biosimilars are successful by launching our Biosimilars Action Plan last year. The plan lays out four key goals to improve the efficiency of the biosimilar and interchangeable product development and approval process. It is focused on increasing regulatory clarity through guidance, educating patients, clinicians and payors about these products, and reducing gaming of the FDA requirements or other attempts to unfairly delay competition.

We have also issued a variety of detailed guidance documents for industry designed to provide greater clarity to manufacturers on scientific and regulatory considerations for the development of biosimilars. At the same time, we have been working to prevent some companies from engaging in unscrupulous efforts to prevent biosimilar competition, such as by blocking would-be competitors’ access to their products for research purposes.

In short, we are doing everything we can to develop and maintain efficient and effective methods of regulating the development and approval of new biosimilars that are as safe and effective as their reference products.

Finding the right approach

But finding the most effective pathway to efficiently support innovation is challenging. Clear and predictable regulatory pathways grounded in the best available science are essential. We need to avoid inflexible standards that can’t evolve quickly enough to keep up with technological developments and that can be used to prevent or delay competition and innovation. This is particularly important for biological products, which are generally complex and variable and often depend on state-of-the-art technological developments.

Likewise, product-specific standards like those put forth by non-governmental organizations, such as those of the U.S. Pharmacopeia (USP), can serve an important role for “small molecule” drugs that are relatively easily standardized and characterized. Manufacturers follow USP standards called monographs for many FDA-approved drugs. However, when it comes to biological products (including biosimilars) — the requirement to adhere to a monograph may actually hinder progress while not offering additional quality assurances.

The FDA already has standards in place to ensure the safety, purity, and potency (safety and effectiveness) of biological products, including biosimilar and interchangeable products. Requiring compliance with a monograph issued by a non-governmental entity can make a sponsor conform to two standards when only one is needed — potentially extending review time and delaying the time it takes for approval. Further, the USP monograph can be used by sponsors to gain an advantage for their product over others, based on patented manufacturing processes or tests and — in some cases — outdated technology. This is an example of the kind of redundancy and unnecessary burden within a regulatory approval pathway that the FDA seeks to avoid. Details of the potential effects of the USP monograph system on the development of future biological products, including biosimilars, are available in this CDER Conversations article.

Supporting voluntary standards for biological products

The FDA supports development of voluntary standards by USP and other consensus standards organizations, as this is consistent with the FDA’s flexible approach and can meet the needs of evaluating complex and diverse biologics. Our goal includes making sure that patients can access and afford these treatments, which often can mean the difference between life and death. And our efforts are designed to do that while maintaining the FDA’s gold standard of safety and effectiveness.

We are pleased to see our 20th biosimilar approval. It inspires all of us at the FDA to embrace the important work that lies ahead to safely and quickly facilitate the delivery of medical products to patients.

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