The pharmaceutical industry operates on a massive scale, developing medicines that impact global health and drive significant economic activity. These drugs range from treatments for common chronic illnesses affecting millions to specialized therapies for rare conditions. This landscape is constantly shifting as new treatments emerge and our understanding of diseases evolves.
How “Top” Drugs Are Defined
When discussing “top” pharmaceutical drugs, the term can be defined in two distinct ways, each offering a different perspective on a medicine’s impact. Differentiating between these metrics is necessary for a clear picture of the industry.
One method of ranking is by global revenue, which identifies the “blockbuster” drugs that generate the highest sales worldwide. These are often newer, patent-protected medications, such as biologics, that come with high price tags. Their financial success is a result of effectively treating serious or widespread conditions and commanding significant market share before generic versions become available.
A second way to define a top drug is by prescription volume. This metric highlights the medications that are dispensed most frequently to patients. These are often established drugs for which affordable generic versions are available. They are the workhorse medications prescribed for common, chronic conditions like high blood pressure or high cholesterol, reflecting their widespread use in managing public health.
Leading Drugs by Global Revenue
The list of highest-grossing pharmaceuticals is dominated by innovative treatments for cancer, autoimmune diseases, and metabolic disorders. These drugs often feature complex mechanisms and address conditions with significant unmet needs, which justifies their high costs and drives substantial revenue for their manufacturers.
- Keytruda (pembrolizumab) is a medication for modern cancer therapy, with approvals for a wide range of malignancies. As a PD-1 inhibitor, it works by blocking a protein on immune cells that cancer cells use to hide. This action “unmasks” the cancer, allowing the body’s own immune system to recognize and attack the tumor.
- Humira (adalimumab) has been a leading treatment for autoimmune disorders like rheumatoid arthritis and Crohn’s disease, though its revenue is declining due to biosimilar competition. It is a TNF inhibitor that blocks a protein promoting inflammation. By neutralizing this protein, Humira reduces the inflammatory processes that cause pain and damage.
- Ozempic (semaglutide) was initially approved for type 2 diabetes, but its popularity has surged due to its weight loss effects. As a GLP-1 receptor agonist, it mimics a natural hormone to stimulate insulin release, suppress a hormone that raises blood sugar, and slow stomach emptying. This dual benefit for diabetes and weight management has driven immense demand.
- Eliquis (apixaban) is used for preventing and treating blood clots, such as those that can cause a stroke in patients with atrial fibrillation. It is a direct Factor Xa inhibitor, which selectively blocks a protein necessary for the blood clotting process. Its oral administration and predictable effect have made it a preferred choice over older anticoagulants.
- Comirnaty, the COVID-19 vaccine from Pfizer and BioNTech, also ranks high in global revenue, with sales that have fluctuated with pandemic trends. It uses mRNA technology to instruct the body’s cells to produce a harmless piece of the virus’s “spike protein.” The immune system then creates antibodies and T-cells, building a memory that protects against future infection.
Most Prescribed Medications by Volume
In contrast to high-revenue specialty drugs, the most prescribed medications are affordable, generic drugs that treat the most common health issues. These are foundational therapies that millions of people rely on daily to manage chronic conditions.
- Atorvastatin, known by its brand name Lipitor, is consistently one of the most prescribed drugs. It is used to treat high cholesterol and lower the risk of cardiovascular events like heart attacks. As a statin, it works by inhibiting a liver enzyme responsible for producing cholesterol, thereby reducing “bad” cholesterol in the bloodstream.
- Levothyroxine is a frequently prescribed medication used to treat hypothyroidism, a condition where the thyroid gland is underactive. This drug is a synthetic version of a primary thyroid hormone that regulates the body’s metabolism. Replacement therapy with levothyroxine restores normal hormone levels, relieving symptoms like fatigue and weight gain.
- Metformin is a first-line medication for the treatment of type 2 diabetes. It helps control blood sugar by decreasing glucose production in the liver and improving the body’s sensitivity to insulin. Unlike some other diabetes drugs, metformin rarely causes low blood sugar because it does not increase the pancreas’s insulin production.
- Lisinopril is an angiotensin-converting enzyme (ACE) inhibitor for treating high blood pressure and heart failure. ACE inhibitors work by blocking an enzyme that narrows blood vessels. This causes blood vessels to relax and widen, which lowers blood pressure and makes it easier for the heart to pump blood.
Emerging Drug Classes and Future Blockbusters
The pharmaceutical landscape is continually evolving, with new classes of drugs showing the potential to become the next generation of blockbusters. These emerging therapies often target novel biological pathways or use new technologies to address diseases in previously impossible ways.
Building on the success of drugs like Ozempic, the GLP-1 agonist class is expanding beyond diabetes and obesity. Researchers are exploring its potential for other conditions, including non-alcoholic fatty liver disease (NAFLD), neurodegenerative diseases like Alzheimer’s and Parkinson’s, and cardiovascular disease. The ability of these drugs to influence metabolism and inflammation opens up numerous therapeutic possibilities.
Another area of intense research is in new treatments for Alzheimer’s disease, specifically amyloid-targeting therapies. Drugs like Leqembi (lecanemab) are monoclonal antibodies designed to remove amyloid-beta plaques, the protein clumps that accumulate in the brains of Alzheimer’s patients. Given the number of people affected by Alzheimer’s, this class has enormous market potential if it can meaningfully slow cognitive decline.
The success of COVID-19 vaccines has also brought mRNA technology to the forefront of medical innovation. Beyond infectious diseases, researchers are using mRNA to develop personalized cancer vaccines designed to help a patient’s immune system attack tumors. This technology is also being explored for treating rare genetic disorders and autoimmune diseases, positioning it as a transformative force in medicine.