A "Medical Breakthrough" Isn't What You Probably Think

How many times have we all heard some development in medical research described as a "medical breakthrough"?

Here are some examples:

• In 2016, multiple news services announced a breakthrough in treating strokes. Instead of using potentially deadly "clot busting" drugs, surgeons had mastered a technique  called embolectomy. The surgeon threads a catheter with a tiny mesh tip through an artery into the brain, and when the catheter is removed, the clot comes out. What the stories in the news failed to mention was that the procedure was introduced as a "last resort therapy," that had actually been performed, although with great success, on just one patient two years earlier.
• In 2015 the CBS news program Sixty Minutes ran a story on a clinical trial of using polio virus to treat the aggressive form of brain cancer glioblastoma with a screen reading "Cancer Breakthrough." Then the reporter revealed that the person who inspired the headline had died.

• In 2014, over 10,000 people died of Ebola in West Africa. In 2015, the respected medical journal Lancet reported that a vaccine had been tested for treating Ebola exposure. News organizations heralded a "medical breakthrough," but failed to note that 16 out of 21 people who were given the vaccine more than 10 days after exposure to the virus developed the generally fatal infection.

The dictionary definition of a breakthrough is “a sudden, dramatic, and important discovery or development." The FDA definition, however, is quite different. The FDA Fact Sheet states that a breakthrough is:

• A drug
• Intended to be used by itself or with other drugs to treat a life-threatening condition
• That offers a significant improvement over currently available treatments.

If the best currently available treatment is successful 50 percent of the time and carries a 20 percent risk of sudden death from the treatment itself, and the new treatment is successful 50 percent of the time and carries a 2 percent risk of sudden death, like the "clot catcher" treatment for stroke, then the new treatment is a breakthrough. If the best currently available treatment extends life three months and the new treatment extends life for a year, as seems to be the case with the polio vaccine for brain cancer, then the new treatment is a breakthrough. If the best available treatment results in a 90 percent death rate and the new treatment results in an 80 percent death rate, like the Ebola vaccine, then the new treatment is a breakthrough.

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A breakthrough treatment is a better treatment. It's almost never a miracle treatment, although it may seem to be a miracle at first. So why does the FDA bother to designate some treatments as breakthroughs?

The breakthrough designation just means that the FDA will fast-track its decision on approving further testing of the drug. When a drug receives breakthrough status, the FDA will rule in 60 days whether to approve or deny further testing. When research seems to have genuine potential for treating a life-threatening disease, the FDA generally goes out of its way to make sure the drug is tested sooner so, if it really works, and that's not a given, then it can be put on the market sooner.

What Your Doctor Probably Doesn't Know About Medical Breakthroughs

It isn't just reporters who don't understand the nature of medical breakthroughs. It's often also doctors themselves.

In April of 2016 a group physician researchers in private practice or associated with Brigham and Women's Hospital in Boston, the American Board of Internal Medicine, or the Dartmouth Institute for Health Policy and Clinical Practice published findings of a survey of doctors in the Journal of the American Medical Association. They randomly selected 300 primary care providers and 900 specialists who were certified by the American Board of Internal Medicine to participate in a survey. The doctors were presented were asked to choose to prescribe one of two new drugs, only one of which was described as a "breakthrough," after reading a mock press release just offering the facts about the drug, or describing the facts about the drug and calling it a breakthrough, or describing the facts about the drug, calling it a breakthrough, and noting that the FDA was rushing its approval.

In the study, 77 percent of physicians surveyed responded that they thought when the FDA gave a drug breakthrough status, it meant that there was high-quality evidence for that drug. In reality, breakthrough status doesn't mean that at all. Some breakthrough drugs are promising but ultimately don't pan out. The FDA approves them for further testing that fails to find that they are effective. A breakthrough also doesn't mean that a drug is a miracle. It just means that it is better than what is currently available.

Why does this common misunderstanding matter? The reason is very simple. People hoping to get into clinical trials that allow them to be treated with breakthrough drugs pass up opportunities to be treated with other drugs that may not work as well for everybody, but make work better for them. And in any clinical trial, about half of patients will be given a placebo so scientists can demonstrate that the active treatment works. You may stake everything on a breakthrough and get no treatment at all.

There are seven words that should set off an alarm whenever you see them describing a new drug or treatment:

• Breakthrough
• Cure
• Dramatic
• Hope
• Miracle
• Promising
• Victim

These words mean different things to different people. What is a cure, really? Does that mean you will never have the disease again? Or that it goes away for five years, as is the standard for a "cure" in breast cancer and prostate cancer treatment? 

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The term "dramatic" generally just draws attention to a story. Most people would prefer to decide for themselves just how much "hope" they have in a new treatment. After all, it's not just a matter of whether a new treatment works. It's also a matter of whether the treatment is available and affordable.

The term promising means “likely to be successful." It gives listeners a reason for establishing expectations. However, medical breakthroughs are evaluated in terms of how they work in groups of patients, not how they work for individual patients. There are always circumstances that are uniquely your own. These may make the drug work better for you, but they may also interfere with your treatment.

And who wants to be a victim?

No medical research is complete until it works for you. When you have a life-threatening disease, it's hard to accept that some treatments just don't always work. However, your survival and your recovery are never about any single treatment. They are always about the totality of your inner strengths, your social support, and your medical care. Don't pursue miracles at the expense of effective care.