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From bench to bedside, biologics require excellence at every stage

From bench to bedside, biologics require excellence at every stage

The Nobel Prize in Medicine will never be awarded for excellence in the formulation and manufacture of biologic medicines. That’s understandable.

Yet making effective and safe biologic products, including biosimilars, is incredibly important for patients and valuable for our health care system. Quality manufacturing is essential to our ability to provide consistent products for patients, and to provide them in a way that is scalable for the thousands — sometimes millions — of doses that will be needed.

Patients who need these medicines deserve the product the Food and Drug Administration approved, whether that’s the reference biologic or a biosimilar, not an adulterated or inadequate version of it. FDA regulations and guidance along with regulatory pathways are designed to assure this transpires as planned.


What I learned in 16 years working for the FDA and in my first year at Amgen, a company that develops and manufactures biologics, including biosimilars, is that immense skill, experience, vigilance, and commitment are essential for ensuring the quality of a biologic medicine each time a new batch is made. It’s an important understanding and one that regulators and industry should be communicating to the public.

The FDA recently proposed a rating system for manufacturing facilities with the goal of providing greater transparency and promoting manufacturing quality. While the FDA said that the immediate impetus for the rating system was to address drug shortages, the proposal is another example of FDA’s continued commitment to ensuring quality and transparency among manufacturers, and appears to be a promising step for the industry.

Biologic medicines present unique and complex challenges, from both technical and regulatory perspectives. There are many potential points of failure in a biologic manufacturing process, such as the quality of the raw materials, sterility of the facilities, and deviations from process parameters. Minor differences in manufacturing processes can have a significant effect on product quality. And even if a formulation works in animal and human trials, there are often difficulties in scaling production of a biologic medicine to supply all of the patients who could benefit from it.

These challenges are substantially greater for biologics than for chemically synthesized small-molecule drugs. Biologic medicines are more complex and require mastery of manufacturing processes involving living cells that are inherently more variable than components involved in chemical synthesis. These medicines often achieve their therapeutic results through intricate mechanisms of action driven by critical quality attributes — features associated with the product that can affect safety, potency, pharmacokinetics, and overall quality. So these medicines must be consistently manufactured to ensure the expected safety and efficacy.

Quality relies on tens of thousands of individuals who work in and contribute to biologic product development and manufacturing. They perform their jobs with commitment to a single quality standard that defines manufacturing excellence for all biologics.

The FDA’s Current Good Manufacturing Practices, as well as similar guidelines from the World Health Organization, are the foundation of a robust quality system for biologic medicines in the U.S. When such practices are followed, in tandem with internal audits and inspections by regulatory authorities, a manufacturer can establish a framework to identify, scientifically evaluate, and control potential risks to product quality. Beyond the obvious benefit to patients, quality manufacturing is also a key to fostering a healthy, sustainable marketplace for all biologic medicines, including biosimilars.

It is important for prescribers and patients to be informed that systems are in place to support the development of safe, effective, and high-quality biologics. Without this confidence and the continued implementation of existing robust, scientifically appropriate regulatory standards, our loftiest aspirations for reference biologics and biosimilars won’t come to fruition.

It’s crucial for all manufacturers of biologic medicines around the globe to be deeply committed to quality. By adhering to rigorous global standards, the industry can help build public confidence and continue to earn trust among regulators, patients, and prescribers. This public trust cannot be taken for granted.

Biologics have revolutionized treatment and provided substantial gains in both morbidity and mortality. If prescribers and patients are to derive their full value, we must continue to ensure that all finished biologic medicines are subject to the appropriate oversight necessary for manufacturing and quality control.

Quality and trust go together. As an industry, biologic manufacturers must deliver on quality. Public trust of sophisticated biological products will follow. Better public understanding of the challenges and successes involved in delivering biologic medicines to patients through patient and provider educational initiatives can help build that trust. The continued collective vigilance of both regulatory authorities and industry, and the commitment to appropriate manufacturing and quality controls, can help prescribers and patients have confidence in the transformative medicines that we develop and manufacture.

As we begin a new year and a new decade, the industry must renew its collective commitment to share that story with the public and further strengthen confidence in these important medicines.

Leah Christl, Ph.D., is executive director, global regulatory and R&D policy at Amgen. She previously served as the associate director of therapeutic biologics in the FDA’s Office of New Drugs in the Center for Drug Evaluation and Research, and director of the Therapeutic Biologics and Biosimilars Staff.

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