Many patients and physicians assume
that the safety and effectiveness of newly approved drugs is well understood by
the federal Food and Drug Administration (FDA). The article describes a study
of the clinical trial evidence supporting FDA drug approvals, and the authors
conclude that the quality of that clinical trial evidence varies widely. The
team evaluated the strength of clinical trial evidence supporting FDA approval
decisions for new drugs by characterizing key features of efficacy trials, such
as trial size, design duration, and end points. FDA approved 188 novel
therapeutic agents for 206 indications on the basis of 448 pivotal efficacy
trials and that the quality of the clinical trial evidence supporting approval
of these drugs varies widely across indications.
Most drugs were approved based on
two efficacy trials, but for 74 indications (36.8%), drugs were approved based
on only one efficacy trial. Additionally, for 91 indications (45.3%), drugs
were approved based solely on trials using surrogate endpoints as their primary
outcome. There was also significant reliance on surrogate endpoints, defined as
“any endpoint using a biomarker expected to predict clinical benefit.” Trials
using surrogate endpoints as their primary outcome formed the exclusive basis
of approval for 91 indications (45%) among the study sample. The team also found
that only 40% of drug approvals involved a clinical trial that compared a new
drug to existing treatment offerings. This is an important step for determining
whether the new drug is a better option than existing, older drugs. Survey data shows that patients expect
drugs approved by the FDA to be both safe and effective.
These results are unsurprising
because the FDCA (Federal Food, Drug, and Cosmetic Act) specifically authorizes
FDA to approve drugs based on clinical trial evidence as such varied quality.
Congress has made a policy decisions that FDA should not require that drug
approval supported by two trials studying clinical endpoints. The authors
suggest that FDA adopt a “life-cycle” approach for drug safety and
effectiveness, communicating a therapies risk and benefit information in an
updated summary that distinguishes between the types of evidence relied on for
initial and continuing approval. It also requires adequate and robust
post-market surveillance systems that allow for re-assessments of drug efficacy
and safety after launch.
This study is in hopes to educate
the public about what kinds of evidence actually support many drug approvals.
Its important for patients and practitioners to understand the quality of
evidence that supports drug approvals because the quality of that evidence goes
to the amount of certainty we should have about the safety and effectiveness of
a drugs. This study also increases knowledge about the relative strength of the
evidence supporting many drug approvals, it can also enable a more informed
debate about whether Congress and FDA have struck the right balance between
allowing access to effective drugs for patients who need them, and preventing
harms from access to ineffective and unsafe drugs.
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