In 1937, a pharmaceutical company wanted to make a kid-friendly, raspberry-flavored liquid version of the antibiotic sulfanilamide. Unfortunately, because of the chemical properties of this antibiotic, they couldn’t just dissolve it in water. Instead, they decided to use diethylene glycol, a liquid chemical with a sweet taste kids love. Diethylene glycol, commonly known as antifreeze, is a liquid normally put in cars to prevent the engine from freezing. Shortly after sales began, over 100 kids died from taking this medication. Congress responded by passing the Federal Food, Drug, and Cosmetic Act in 1938, giving the Food and Drug Administration (FDA) the power to test the safety of drugs before they came on the market.
The FDA has a long, rigorous approval process for bringing drugs to market. Drug companies extensively screen millions of compounds to find any with specific molecular activity. They test any active compounds they find in cells, and then in animals, both for safety and activity. Finally, they have to conduct three phases of clinical (human) trials:
Phase I: Is it safe? The drug is tested on 20-80 healthy people to make sure it doesn’t cause any major health problems. This is also when they figure out the right dose to give patients.
Phase II: Does it work? The drug is tested on 100-300 people with the disease it is intended to treat. Only half the patients receive the drug, while the other half get a placebo (sugar pill). Then, the clinicians compare the number of patients treated with drug who get better versus the number of patients with placebo who get better. Common side effects will become evident at this stage. If more people get better taking the drug than taking the placebo (without any debilitating side effects), they can move on to the next phase.
Phase III: Is it really safe? Now comes the large-scale clinical trial, where 1,000-3,000 people get either the drug or a placebo. Treating a large number of people allows for identification of less common side effects.
At this point, the drug can be approved and sold, though only with a doctor’s prescription.
But wait, there’s more!
Phase IV: Are there any extremely rare side effects? Many drugs will continue to be studied after they are on the market in case the previous trials missed anything. Let’s say a drug kills one in 1,000,000 people. You’re unlikely to see that if you’ve only tested the drug on 4,000 people. The FDA can and frequently does take drugs off the market if they fail a Phase IV trial.
This entire process typically takes about 14 years and $1 billion to get a single drug to market. That is why drugs are so expensive. Although a company could manufacture a month’s worth of pills for $1, they are trying to recoup the money they spent getting the drug approved in the first place. They even have a time limit on this. When a company gets a drug patent, they have exclusive rights to manufacture that drug. But after 25 years, the patent expires and another company can make a “generic” without having to go through the same FDA approval process. Generics are the exact same chemicals, but companies can sell them for much less. Sometimes a big company will buy up a smaller company with a patented drug that has already gone through clinical trials, thus skipping the initial expense. Some pharmaceutical companies will also make deals with other companies to prevent them from making generics after their patent expires, which would drive down the price.
Because of the long, expensive road to approval, these companies wouldn’t spend $1 billion unless they thought they could make that money back. It’s important to keep in mind that a pharmaceutical company is just like any other company. Their goal is to make money by producing and selling a quality product. The FDA only officially approves each drug for specific medical applications, though doctors are not legally limited to FDA-approved uses for each drug. This is also why you see so many commercials for prescription drugs. Pharmaceutical companies want as many people as possible to buy their drugs. Even though you can’t go to the store and buy them yourself, the companies want you to bug your doctors to prescribe their drugs. Direct-to-consumer advertising of pharmaceuticals has been around for less than 20 years, and is only legal in the US and New Zealand, but these companies continue marketing this way because it works.
So if drug companies are trying to convince you that you need their drugs so they can make money, how do you know which drugs you really need to take? The most important part of drug treatment (all treatment, actually) is having the correct diagnosis. Once diagnosed, your doctor will often be able to recommend the most appropriate treatment based on your age, specific symptoms, lab test results, and a host of other factors. So start with the doctor, not the drug.