Biologics

Biologics is all around us and is already a big part of our lives, providing breakthrough products and technologies to combat diseases. America’s biopharmaceutical research companies are using biological processes to develop 907 medicines and vaccines targeting more than 100 diseases, according to a new report from PhRMA.

What are “Biologics”?

Biological products include a wide range of products, such as vaccines, blood and blood components, allergenics, somatic cells, gene therapy, tissues, and recombinant therapeutic proteins. Biologics can be composed of sugars, proteins, or nucleic acids or complex combinations of these substances, or may be living entities such as cells and tissues. Biologics are isolated from a variety of natural sources – human, animal, or microorganism – and may be produced by biotechnology methods and other cutting-edge technologies. Gene-based and cellular biologics, for example, often are at the forefront of biomedical research, and may be used to treat a variety of medical conditions for which no other treatments are available.

How do Biological Products differ from Conventional Drugs?

• A biologic is manufactured in a living system such as a microorganism, plant, or animal cells. Most biologics are very large, complex molecules, or mixtures of molecules. Many biologics are produced using recombinant DNA technology while a pharmaceutical drug is typically manufactured through chemical synthesis, which means that it is made by combining specific chemical ingredients in an ordered process.
• Drugs generally have well-defined chemical structures, and a finished drug can usually be analyzed to determine all its various components. By contrast it is difficult, and sometimes impossible, to characterize a complex biologic by testing methods available in the laboratory, and some of the components of a finished biologic may be unknown.
• Therefore, for biologics, “the product is the process.” Because the finished product cannot be fully characterized in the laboratory, manufacturers must ensure product consistency, quality, and purity by ensuring that the manufacturing process remains substantially the same over time. By contrast, a drug manufacturer can change the manufacturing process extensively and analyze the finished product to establish that it is the same as before the manufacturing change.
• The living systems used to produce biologics can be sensitive to very minor changes in the manufacturing process. Small process differences can significantly affect the nature of the finished biologic and, most importantly, the way it functions in the body. To ensure that a manufacturing process remains the same over time, biologics manufacturers must tightly control the source and nature of starting materials, and consistently employ hundreds of process controls that assure predictable manufacturing outcomes.
• Process controls for biologics are established separately for each unique manufacturing process/product, and are not applicable to a manufacturing process/product created by another manufacturer. These process controls may also be confidential to the original manufacturer. Therefore, it would be difficult or impossible for a second manufacturer to make the “same” biologic without intimate knowledge of and experience with the innovator’s process.

Biologics: New Hope to Treat Diseases

Biological products often represent the cutting-edge of biomedical research and, in time, may offer the most effective means to treat a variety of medical illnesses and conditions that presently have no other treatments available. The mapping of the human genome coupled with a greater understanding of the molecular workings of disease, have improved the options to treat diseases in new and innovative ways. Biologics are structurally different from most existing treatments and allow for precise targeting, changing the treatment options available for many diseases. For instance, monoclonal antibodies (mAbs) are proteins that help the immune system identify and bind to foreign substances. Thirty years after initial development, these therapies help treat some of the most costly and challenging diseases. In many cases biologics are the first treatment available for a disease or offer a different way to treat a given disease.

Impressive progress against many cancers has been made with biologics, and biologic medicines being researched today are looked to for advances against such diseases as Alzheimer’s and Parkinson’s. In the last decade alone, biologics have accounted for one third of annual approvals, according to PhRMA’s New Drug Approval reports.

The 907 biologic medicines and vaccines in development promise to push the frontiers of science and bring new treatments to patients for our most challenging diseases. The medicines and vaccines – all in human clinical trials or under review by the US Food and Drug Administration (FDA) – include 338 monoclonal antibodies, 250 vaccines, 93 recombinant proteins, 60 cell therapies, 46 gene therapies, and 30 antisense medicines. America’s biopharmaceutical research companies using biological processes to develop 907 medicines and vaccines targeting more than 100 diseases, including

• 338 for cancer that target several different types of solid tumors, leukemia, and lymphoma.
• 176 for a variety of infectious diseases, including 134 vaccines
• 71 for autoimmune diseases, such as lupus, multiple sclerosis, and rheumatoid arthritis
• 58 treatments for cardiovascular diseases, such as congestive heart failure, and stroke.
• Other diseases include diabetes, digestive disorders, genetic disorders, neurologic, and respiratory disorders

Biologics remain a budding research field that holds tremendous promise for the treatment and prevention of numerous illnesses. They are extremely complex medications to produce and require several years of research and development before a product is approved for patient use. Despite the obstacles, US biotechnology firms account for 80 percent of the world’s research & development in biotechnology. Biologics hold great promise for the treatment and prevention of numerous illnesses; they hold a world of possibilities.

Center for Biologics Evaluation and Research

CBER is the Center within FDA that regulates biological products for human use under applicable federal laws, including the Public Health Service Act and the Federal Food, Drug and Cosmetic Act. CBER protects and advances the public health by ensuring that biological products are safe and effective and available to those who need them. CBER also provides the public with information to promote the safe and appropriate use of biological products.

References

1. What Are “Biologics”. http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CBER/ucm133077.htm. Accessed June 24, 2013.
2. Industry (Biologics). http://www.fda.gov/BiologicsBloodVaccines/ResourcesforYou/Industry/default.htm. Accessed June 25, 2013.
3. Medicines in Development for Biologics. http://www.phrma.org/medicines-development-biologics. Accessed June 25, 2013.
4. About the Center for Biologics Evaluation and Research. http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CBER/default.htm. Accessed June 24, 2013.
5. How do Drugs and Biologics Differ? http://www.bio.org/articles/how-do-drugs-and-biologics-differ. Accessed June 24, 2013.
6. Medicines in development Biologics.2013 report. http://108.166.83.204/sites/default/files/pdf/biologics2013.pdf. Accessed June 24, 2013.