|
 The FDA uses the term more often, and for smaller advances, than
people use it colloquially, and this may lead patients to have
unwarranted confidence in new drug claims.
As the researchers on the new study describe it, the FDA Safety and
Innovation Act, passed in 2012, allows the FDA to give breakthrough
designation to any drug treating a serious or life-threatening
condition that "may demonstrate a substantial improvement over
existing therapies" for one clinical endpoint, or outcome, in
preliminary evidence.
“These clinical endpoints can be surrogate outcomes and don't have
to be a direct outcome of the disease,” said coauthor Tamar
Krishnamurti, a research scientist at Carnegie Mellon University in
Pittsburgh.
“For example, cholesterol can be a surrogate outcome for measuring
the effectiveness of a drug to treat heart disease,” she told
Reuters Health by email.
Many people with heart disease have high cholesterol levels, but not
all people with heart disease do - and many people with high
cholesterol levels have no heart disease at all, she said.
The researchers recruited almost 600 people online through Amazon’s
Mechanical Turk and randomly assigned them to read one of five short
descriptions of a recently approved drug for metastatic lung cancer
with ‘breakthrough’ designation.
One vignette only described the facts about the drug – including the
size of the drug trial, and the findings that about half of the
patients had their tumors shrink while on the drug, that the effects
lasted for seven months, and that side effects included diarrhea,
nausea and vomiting – without using the term breakthrough.
A second vignette used these facts and included the term
“promising,” while a third used the word “breakthrough.”
“While 'breakthrough' is an official designation that may lead to
accelerated drug approval, 'promising' is just a descriptor term
that FDA has used in about half of its press releases to describe
breakthrough drugs,” Krishnamurti said.
The fourth vignette included language required for FDA labeling,
clarifying that continued approval of the drug may be contingent on
further trials confirming its effectiveness.
The fifth vignette changed “may be contingent on” to “is contingent
on.”
The participants judged the drug’s benefits, harms and strength of
evidence based on the vignette they were assigned.
While 11 percent of respondents who read the facts-only description
rated the drug as very effective or completely effective, about 25
percent of those who saw the ‘promising’ or ‘effective’ descriptions
said the same. More of the second and third group of respondents
also believed the evidence supporting the new drug was strong or
very strong.
[to top of second column] |
And only 10 percent to 16 percent of people who read the fourth and
fifth vignettes, which added further explanations and contingencies,
were likely to believe incorrectly that the drug was proven to save
lives, compared to 31 percent of those who read the "breakthrough"
vignette, according to a report in JAMA Internal Medicine.
At the end of the survey, respondents read two descriptions of a
drug and were asked which they would take for a life-threatening
condition. One description included the word breakthrough and the
other did not. More than 90 percent of people chose the breakthrough
drug.
“Given that ‘breakthrough’ is an official designation mandated by
Congress, FDA is required to refer to this category of drugs as
such,” but the FDA is not required to use terms like “promising,”
Krishnamurti said.
“Breakthrough” is an aspirational term, chosen for the 2012 act to
help expedite the new drug approval process, which many criticize as
slow although there is research indicating that the process is not
slow, as Drs. Joseph S. Ross and Rita F. Redberg note in an
editorial published with the study.
The term breakthrough “may cause people to have unwarranted
confidence about these drugs, which could prevent them from making a
fully informed decision about whether to take the drug or not,”
Krishnamurti said.
Simple, clearly worded disclosures can help mitigate some of the
incorrect judgments associated with these designations, she said.
“To protect patients from spurious hopes for miracle cures, Congress
and the FDA should abandon the adoption of terminology like
breakthrough and focus on strengthening the evidentiary requirements
for meaningful clinical data to ensure the promise of new drugs and
devices,” Ross and Redberg write.
SOURCE: http://bit.ly/1NOENUw JAMA Internal Medicine, online
September 21, 2015.
[© 2015 Thomson Reuters. All rights
reserved.] Copyright 2015 Reuters. All rights reserved. This material may not be published,
broadcast, rewritten or redistributed. |
