Sometimes it seems like we hear news of a "breakthrough" drug nearly every day. Yet true breakthroughs are rare.
How can you decide whether the new drug you’re hearing about is truly important? It’s not easy. For starters, be skeptical about dramatic headlines. Don’t accept terms like "breakthrough" at face value. While the early part of a story may make it sound as though a drug is amazing, later you may learn that the findings are preliminary, or that side effects are unacceptable.
Read on for ways to better understand the news you hear about the next big drug discovery.
Start with the study
What type of study is it? The most powerful drug trials are randomized controlled trials (RCTs), which randomly assign a similar pool of study subjects to receive either the new drug or a placebo. Neither the study subjects nor the researchers know what each participant is taking during the trial. Researchers then compare how the two groups fared. RCTs can provide a true sense of whether a drug works for people with similar characteristics.
By contrast, observational studies might collect information on thousands of people to discover whether there’s a link between a treatment and a disease. For example, such a study might find that those taking vitamin C have a lower incidence of diabetes. That doesn’t mean that vitamin C caused the lowered risk. Maybe people taking the vitamin are more health-conscious, eat better, and exercise more. Perhaps those healthy habits — not vitamin C — lead to the lower rate of diabetes. Studies like these can find a link, or association, between a treatment and a desired outcome, but cannot determine that the treatment actually caused the benefit. And that makes them far less powerful than RCTs.
Ask how the study relates to you
Do the outcomes reported really matter to you? For example, cholesterol-lowering medications should do more than just lower total cholesterol. The overall goal is to lower the risk of heart attack, stroke, or other complications of cardiovascular disease, including death. So, while lowering cholesterol may be a good thing, it’s not the only thing: look for changes in measures you care most about rather than a surrogate measure.
Are the study subjects similar to you? Heck, are the study subjects even human? It’s not rare to read about a breakthrough drug, only to realize it’s only been studied in mice, or rats, or zebrafish. Even if the subjects are human, they may be so different from you in age range, general health, and other measures that study results don’t apply to you. Some groups of people may not be well represented as participants. Studies may exclude people who do or don’t have certain conditions, take certain drugs, or are too old, too young, or too sick.
Then consider actual benefits, possible risks, and costs
How are results reported? Imagine a study that found a new drug could reduce heart attacks by 15%. Sounds great, right? But it may not be so impressive if you know the actual numbers. Let’s say heart attacks occur in 10% of study subjects taking a placebo and 8.5% of those taking a new drug. That’s a 15% reduction in relative risk. Yet the absolute reduction in heart attacks was only 1.5%, which may not be much of a benefit in real terms. It’s accurate to express changes in terms of relative risk or absolute risk, but only reporting relative rates can be misleading. The best news reports include both.
Are the findings meaningful? Studies often report changes that, while statistically significant, aren’t big enough to matter. For example, let’s say a pain medication lowered average pain by 2 points on a 0 to 100 scale. That might be statistically significant, but people taking the drug are unlikely to notice any meaningful improvement.
Are the benefits worth the risks and its cost? Even if a drug performs well in a trial, if it causes serious side effects in half the study subjects or costs $100,000 a year, it isn’t going to be a big breakthrough for most people.
A timely example
Aducanumab (Aduhelm) was approved by the FDA in June 2021 to treat Alzheimer’s disease. Here are some notable headlines just after the approval was announced:
A new day for Alzheimer’s disease treatment after FDA approval of Biogen’s Aduhelm
FDA approves Aduhelm, first new Alzheimer's disease treatment since 2003
FDA Approves Breakthrough Drug for Alzheimer’s Disease
Yet, in the hundreds of news stories that covered the initial announcement, some important details were not always made clear:
- The studies leading to approval did not convincingly demonstrate a benefit for the symptoms of Alzheimer’s disease. Instead, the drug was approved because it appeared to reduce the amount of a protein in the brain (called amyloid) thought to contribute to disease development.
- The study subjects had early stages of dementia. Those with more advanced cases were excluded from participating, because it was thought they would have little chance of improving.
- Aducanumab treatment is expensive: estimates are that it will cost $56,000 a year, plus the cost of frequent brain MRIs. In trials, it caused potentially serious side effects in more than a third of participants, such as brain swelling or bleeding into the brain.
- The approval could soon be reversed if additional studies required by the FDA find no compelling evidence of benefit for patients’ symptoms.
The bottom line
The next time you read or hear a news story about a new drug, keep an open mind. Maybe it’s a big step forward — or maybe not. Don’t be taken in by dramatic headlines or soundbites. Look for important details and ask questions. Remember: the "breakthrough" you’re hearing about today may never actually make it to market, or may even cause more harm than good.
Follow me on Twitter @RobShmerling
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