The Patient Was…

The question usually starts something like this: If you were marooned on a deserted island…?

“…what one person would you like to be there with you?

                                    or

“…what one book would you like to have with you?

                                    or

“…what one food would you like to have available?

And so on.

The question I ponder, as a physician, is: What one medication would you like to have with you?

Let’s eliminate the useless ones in that setting, like the “little blue pill.” Do I need to explain?

The question may seem pointless, even silly, but it raises an important issue. Leaving aside the “deserted island” part of the question, are there medications so important to your health and well-being, that they could enhance your survival absent other medicines?  What is the single drug you would want if you could have only one?

For the purpose of this discussion, we’ll eliminate (AI would correct this word, I’m sure) those people with specific illnesses requiring specific medicines, like insulin-dependent patients with diabetes. Let’s consider only those adult individuals in apparently good health who have the option of a single medication available to them. What are sensible choices? Is there a best choice?

The two major ways I think about this question of selecting a single drug to have at my disposalare, first, what non-specific medical situations do I really want to avoid and, second, what specific types of illness are most likely to affect me?

In the first instance, pain immediately comes to mind. Pain can arise from a variety of different causes, including many illnesses and injuries. But if you want to cover the prospect of pain, what do you choose? If you choose something like a salicylate (the commonest is aspirin) or acetaminophen (Tylenol is the commonest brand) or a so-called NSAID (non-steroidal anti-inflammatory drug, like ibuprofen (Motrin or Advil are common brands), you are choosing relatively mild pain relievers. If you really fear severe pain, you have to move up to more powerful drugs, like opiates. Do you want to choose that kind of protection as your only available medication for an indeterminate period of time? It would not be my choice, even though the prospect of severe pain is a frightening one.

In the second instance, considering what types of serious illness are most likely to affect the greatest number of people, I think of those conditions that are the leading causes of disability and death. Heart disease is the leading cause of death and disability worldwide. If you broaden the category from just heart disease to cardiovascular disease, and thereby include stroke, the impact on death and disability increases further. Following heart and vascular disease as causes of mortality and disability are cancer and chronic respiratory disease. Note that non-communicable diseases, diseases that are not transmitted from one person to another (essentially non-infectious), lead the way as bad players in global health, including in the U.S.

Once you consider the leading causes of global illness, as described above, think about what is preventable. If you know how to prevent cancer with a medication, please tell the rest of us, because science hasn’t figured it out yet. Similarly for chronic respiratory disease. Smoking and air pollution make it worse, but preventive medication: no way.

Let’s focus on heart disease, the single leading cause of worldwide disability and death. The therapeutic area is rife with acronyms and initialisms: SGLT2i, BBs, ACEIs, ARBs, PSK9i, MRAs, and now GLP-1s. They all represent important medications in preventing and/or treating various forms of heart disease. But the leading cause of serious disease, within the cardiovascular category, is ischemic heart disease, disease caused by cholesterol-filled plaques blocking blood flow in the coronary arteries supplying blood to the heart muscle. This leads to heart attacks, meaning injury and death of heart muscle, as well as a common form of heart failure where the heart cannot pump adequate amounts of blood for the needs of the body tissues.

The development of ischemic heart disease with its consequence of heart muscle injury is a complicated process. In its simplest terms, the major components are formation of cholesterol- containing plaques in the walls of blood vessels and the subsequent rupture or cracking of the plaques leading to the formation of blood clots that actually block the blood flow through the arteries. This means the process could be affected by either preventing the growth of the plaques or by inhibiting the formation of blood clots.

And we do have some medications that affect those processes. Among these are two of the best known drug types: salicylates (aspirin) and so-called statins. Statins actually represent a group of similar-acting drugs that share part of a common generic name designation: atorvastatin (brand name Lipitor) and rosuvastatin (brand name Crestor) are common examples. Statins work in the body by lowering the amount of cholesterol in the blood stream, thereby reducing the formation of plaques in the walls of blood vessels. Aspirin works by preventing the early stages of blood clot formation in the arteries.

But we said at the beginning of this theoretical scenario that you could choose only one drug. And if the main disease you want to prevent is the one that causes the most disability and death globally, and you know that is ischemic heart disease, how do you choose between a statin and aspirin?

One way to think about the choice is to consider whether the drugs do something other than prevent or treat ischemic heart disease. In the case of aspirin, it helps prevent blood clots at low doses, but it also treats mild or moderate pain and reduces inflammation at higher doses. So, it has multiple uses. Of course, at higher doses the side effects increase, and these include bleeding which can be serious and even life-threatening.

Statins also have multiple properties. While reducing blood cholesterol is their major effect, they also decrease inflammation in the body. In addition, not only do statins reduce plaque formation by reducing cholesterol circulating in the blood stream, but they also stabilize existing plaques so rupture or cracking of the plaque is prevented and blood clot formation is thereby reduced.

Weighing all the available choices of drugs, and thinking of all possible contingencies and diseases, my choice for a single drug, if I had to make that difficult selection, would be a statin. I would be addressing the most likely cause of disability or death, with a medication proven to save lives, and with a minimum of serious side effects.

Fortunately, modern medicine has a multitude of medications for almost every disease, common or otherwise. Luckily, most of us have access to them all, as needed. When one or more are prescribed for you, take them as your physician directs. Along with proper lifestyle choices, medications are one of our great life supports.