If a pharmaceutical company's drug harms or kills a patient, that's front-page news. But that's not the only way that patients can be harmed. A patient who isn't given a helpful medicine is also harmed, but unlike with the first example, this story doesn't make it onto the front page or any other page of the newspaper for that matter, because the victim and the harm caused are invisible. Who was the victim? We usually don't know. What drug would have helped them and how much? We usually don't know.
The 19th century French economist Frederic Bastiat taught economists to study both what is seen and what is unseen. (Read What Is Seen and What Is Not Seen.) For instance, if a broken window is replaced, the natural tendency is to focus on the benefit to the economy--and the glazier--of the payment for the window (the seen) while overlooking the negative result of the building's or car's owner not spending that same amount of money on something else (the unseen).
The "seen" in the pharmaceutical example is a specific drug harming a specific person. The "unseen" is a drug not helping a person. This unseen situation often results from a drug not being developed, perhaps because a company didn't see the economic payback, or because the drug was explicitly rejected by the FDA.
Sometimes, however, the unseen can be observed by careful observation. Consider two young boys with the same deadly disease-- Duchenne muscular dystrophy. Max Leclaire, 10, is currently in a clinical trial for a new drug that has miraculously reversed some of his debilitating symptoms. His brother Austin, 13, tried to enter the clinical trial but didn't meet the criteria.
Sarepta Therapeutics is developing eteplirsen. For the last year Max has received a once-weekly IV infusion in a double-blind clinical trial. After just four months, Max's parents realized that their son was getting the active drug because it has not only slowed the progression of the disease but has dramatically improved his symptoms. They just wish their second boy, who has steadily worsened, could have the same chance.
The juxtaposition of the two boys' conditions allows us to say with some confidence that Austin is being harmed by not receiving eteplirsen. Why won't Sarepta give eteplirsen to Austin? Essentially, the FDA won't allow it. According to the FDA, the proof of eteplirsen's efficacy and safety hasn't been established. Would Austin's parents, given everything they know about eteplirsen, willingly give it to Austin today? Definitely.
By being forced to follow the FDA's assessment of what an acceptable risk is (for all drugs, not just those in clinical trials) instead of his parents' own, Austin's situation gives us a rare opportunity to shine a light on a normally unseen problem and to see just how the FDA can harm patients.