It irks me to hear a new pharmaceutical product being advertised as “safe and effective”. Every pharmaceutical product seems to be safe and effective these days, and I’ve been hearing about “safe and effective” too many times over the past few years.
This short paper written by Peter Gotzsche summarizes what’s wrong with safe and effective.
https://www.bmj.com/content/bmj/380/bmj.p601.full.pdf
Peter Gotzsche DrMedSci, MD, MSc is a Danish internal medicine physician, and was co-founder of the Cochrane Collaboration, one of the most trusted evidence based organizations. I see him as an independent thinker and a scientist with courage to speak his mind, wrong or right. Even if one were to disagree with his conclusions, few would doubt his expertise in critically appraising the scientific literature. Voices like Dr. Gotzsche teach me a lot. His opinions are a refreshing break from the consensus in medicine that inundates daily practice, the groupthink in medicine.
Here is a quick summary, in my own words, of the three ways drugs are mischaracterized as described in the article.
Safe and effective:
No intervention can be only safe and effective. This claim implies that a drug is harmless. Actually all drugs with an effect must have harm. Otherwise the drug would be inert and unable to produce efficacy. Safe and effective thus suffers an error of omission; it only tells half the story.
Risk and benefit:
The problem with “risk and benefit” is that this statement implies that the drug has benefits but not harms, only risk of harms. Rather, stating it the other way would be more correct: Drugs have harm, with possible benefit. Harm, however infrequent or small, is essentially a certainty. Benefit is not. So Gotzsche advocates for the discussion of benefits and harm, rather than risks and benefits.
Benefit:risk ratio
The benefit:risk ratio is one I haven’t heard used. This can only useful if risk and benefit are on the same scale, which rarely happens. For example, consider a patient with a terminal illness on high dose opiates. The benefit of pain relief is not on the same scale as the harms of addiction, respiratory depression, and death. And for this patient’s case, high dose opiates sounds like a reasonable treatment option, despite opiates commonly perceived as having a poor benefit:risk ratio. The ratio leaves no room for value judgements. Simply stating the benefit:risk ratio of a drug is useless.
Before reading this article, I’ve always used risks vs benefits to describe drugs. I no longer use risks vs benefits, and instead use harms vs likelihood of benefits. Harms are nearly guaranteed while finding a benefit is like finding a needle in a haystack. Finding a condition in which a drug provides benefit requires the perfect combination of disease state, patient conditions, dose, and duration. Only then can the likelihood of benefits outweigh the harms.
Words matter. What patients want to know is not if the drug they are considering is safe and effective. What patients want to know is, what is the likelihood it will benefit me, and what are the harms? 1
Despite the ubiquitous use of “safe and effective” to describe medical products, it is lazy wording at best, and deceptive at worst. I can’t help but think that safe and effective is more of a marketing term than a scientific term.
A simple list of questions that I would consider if I were to consider an intervention:
What is the benefit?
For whom do the benefits outweigh the harms?
How big is the benefit?
What are the harms that we know of?
On average, do more people experience the benefit than are harmed?
There are many more questions to consider, but I think this is a good starting place.
Very good to bring awareness to this perspective. The "safe and effective" phrase is certainly biased in that it leaves out the potential for harm. This potential for harm also is, unsurprisingly, often left out of medical literature as well.