Biologics: The New Face of Medicines
Today we are far luckier than our ancestors of only a generation ago. New hope has emerged for those suffering from certain traditionally hard-to-treat diseases, like inflammatory bowel disease, the inflammatory arthroses (e.g., rheumatoid arthritis), psoriasis, HIV, multiple sclerosis, and even cancer.
Canadians have been using biologics – in a more rudimentary form – for over a hundred years. Vaccines and insulin are examples of the earliest use of biologic agents, derived from animal sources. Vaccines, antibiotics, cancer therapies, genetic testing, and innovative diagnoses and treatments for diseases such as diabetes and cystic fibrosis are all derived from biotechnology processes.
Conventional medicines, defined by their chemical structures and formulas, are known as small molecule medicines. They directly affect multiple sites in the body, and usually impact various systems. This often leads to unwanted side effects. They generally have a short duration of action, necessitating chronic daily dosing.
In contrast, the new biological treatments get right to the core of the problem causing the disease. They last longer and have minimal side effects. Better still, these drugs can often "stop" the disease process in its tracks. Biologics can decrease hospitalization and surgery rates in many of the diseases they treat, and can positively impact return to work and productivity. They are certainly the major growth area in pharmaceuticals.
The Differences Matter
As with all drugs, Health Canada requires scientific evidence to show the biologic product is safe, efficacious, and of high quality. Relative to chemical drugs, more detailed chemistry and manufacturing information is submitted because slight variations in either process or facility design can result in a different final product.
Biologic agents are defined as substances that can be made only by a living system. They are large molecules relative to their chemical cousins – usually over 1,000 times larger – and they have more complex structures. Because they are proteins, the usual oral route can't work because the body would digest them, not unlike sources of protein gained from the diet. So biologics are taken by injection, subcutaneously (under the skin), or intravenously, directly into a vein.
The first biologics were made of proteins that were based on the cell line that helped produce them, often derived from mice. Though effective, they were fraught with side effects such as allergic reactions to their foreign proteins. Further development in this class led to products that contained less and less of these foreign proteins and finally the creation of protein therapeutics that are 100% human.
Recent scientific advances have identified key molecular drivers of disease. Since some autoimmune diseases have common underlying mechanisms, a single biologic drug can successfully treat multiple diseases. State-of-the-art technology has permitted the manufacture of tailor-made molecules to mimic human proteins.
The Cost of Better Health
Since these newer drugs are much more expensive, they are generally used after conventional therapies have failed, or cannot be taken due to the occurrence of or risk of harmful side effects. They are also indicated for patients who have signs that their disease will progress quickly. According to the Public Health Agency of Canada, biologics work much more quickly than other disease-modifying agents for arthritic conditions, and patients may notice an improvement in their arthritis within a few days to a week.
Biologics cost more for several reasons. First, they are much more complex to make, with a whole new research and manufacturing infrastructure needed. Trials involving these new therapies take longer, with more rigorous trial design and patient monitoring. As well, since the diseases targeted are relatively rare, fewer patients are treated than with conventional medicines.
The cost means affordability is an issue with both plan sponsors and members. Each year, treatments can cost $20,000 or more, creating problems particularly for small employers and employees with significant co-pays. Pooling mechanisms and caps on out-of-pocket expenses can help alleviate the strain.
Medicines have come a long way from simply targeting and improving the symptoms of a disease. Now, with the advent of new biologics, the disease process can be altered and sometimes even stopped. That's so much better than trying to fix illnesses by trial and error, wasting time and money. For many, biologics have helped shorten the long and winding path to a cure.
Categories: Disease Management