Physicians may be overly optimistic about the benefits of newly approved drugs, especially when the therapies are labeled as a “breakthrough,” according to a survey of nearly 700 practicing clinicians.
The doctors tend to overestimate the efficacy of newer therapies and often believe treatments are supported by stronger evidence than they actually are, according to a JAMA study published Tuesday.
And misconceptions about what constitutes a breakthrough and how the drug approval process works may lead to certain therapies being over-prescribed and patients being let down by the overstated benefits, the authors state.
The Food and Drug Administration introduced a breakthrough designation for certain therapies in 2012 with the signing of the controversial Safety and Innovation Act (PDF). Drugs can get the label if "preliminary clinical evidence” shows the treatments substantially improved serious or life threatening conditions. The review and development process for those drugs could then be fast-tracked.
As of April 2016, the FDA has received more than 340 requests for breakthrough designation. Of those, 111 were granted it, 168 were rejected and 47 were withdrawn from consideration, according to approval reports.
If manufacturers do not conduct adequate post approval studies to prove the benefit, safety and efficacy of the drug, it can be quickly pulled from the market, the act states.
However, rapid approval has been criticized by patient safety advocates. They warn that real-world negative experiences are collected over years and that therapies might not be recalled until they harm patients. No “magical post-market surveillance system” exists to flag and pull troubling products from the market quickly, critics say.
A 2015 study found that use of language such as “breakthrough” and “promising” significantly impacted consumers' views about how a drug works.
In the JAMA study released Tuesday, researchers from the Brigham and Women's Hospital in Boston, the Dartmouth Institute for Health Policy and Clinical Practice in Lebanon, New Hampshire and the American Board of Internal Medicine in Philadelphia, evaluate the impact of the lingo on clinicians.
It seems to have the same impact as it does on consumers, found the survey of more than 680 internists and specialists.
When given a hypothetical scenario in which a patient had a serious medical condition and clinicians were given two options, only 6% chose the treatment option with “early promising results.” The other 94% selected the alternative drug described as “breakthrough.” That's an unexpected finding considering that both drugs were actually breakthrough therapies.
The clinicians were also quizzed on what breakthrough means and about the FDA approval process. Only nine people got all the answers correct and the majority only correctly answered one on a three-question quiz about the type of evidence the FDA requires to approve a drug.
A second five-question quiz asked about their understanding of the breakthrough designation. About 42% hadn't even heard of the term before taking the survey. Still, 58% were fairly certain that breakthrough drugs represent a major improvement over existing therapies.
The authors note the potential for inherent bias in the survey and say the results cannot be generalized. Still, the findings suggest “substantial deficits” in knowledge about FDA rapid approval programs, they state.