New Guidance Observational Studies to Support Drug Dev
Mar 21, 2024 12:53:12 GMT
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Post by icemandios on Mar 21, 2024 12:53:12 GMT
March 20, 2024 01:22 PM EDT
FDA+
When can observational studies support a drug's effectiveness? FDA explains in new draft guidance
Zachary Brennan
Senior Editor
The FDA on Wednesday released highly-anticipated draft guidance on when drug sponsors can use observational studies to help demonstrate a drug’s effectiveness, for the first time offering clarity on what data and explanations sponsors should provide the agency.
A major focus of the FDA’s new guidance is making sure data from observational studies are reliable and relevant. Data collected in observational studies have often offered hope that a therapy might be effective, only to be later contradicted by evidence from randomized controlled trials.
In the new 8-page draft guidance, the FDA discusses some of the risks it sees, including issues like confounding because of non-comparable patient groups, and bias based on patient selection and study outcome measurements.
The draft also spells out what exactly sponsors should submit to the FDA, with sections outlining what to submit on the study design, data sources and analytical approach.
“Sponsors should finalize the study protocol, including the research question of interest and rationale for the study design, before initiating study conduct,” the agency said in the draft. It also asks them to lay out potential alternative designs and explain why they ended up using their proposed approach.
In a 2020 paper published in JAMA, now-FDA Commissioner Robert Califf and several co-authors wrote about the potential benefits of observational studies, arguing that increasingly sophisticated medical records and data systems could allow researchers to parse patient data for insights and identify those worth studying further.
But Califf and his colleagues also highlighted the risks, saying that “aggregating information about diagnosis, comorbidities, treatment, and outcomes can lend a patina of technical excellence that obscures the influence of systematic bias.” And he warned that profit motives could skew the judgment of investigators who want to find data to support their hypothesis.
The new draft guidance is part of a suite of documents the FDA has released on real-world evidence. It builds off a 2018 framework in which the agency promised more clarity on how drug sponsors could use data collected outside of carefully controlled clinical trials in order to support new indications for approved drugs, or to satisfy post-approval study requirements.
FDA+
When can observational studies support a drug's effectiveness? FDA explains in new draft guidance
Zachary Brennan
Senior Editor
The FDA on Wednesday released highly-anticipated draft guidance on when drug sponsors can use observational studies to help demonstrate a drug’s effectiveness, for the first time offering clarity on what data and explanations sponsors should provide the agency.
A major focus of the FDA’s new guidance is making sure data from observational studies are reliable and relevant. Data collected in observational studies have often offered hope that a therapy might be effective, only to be later contradicted by evidence from randomized controlled trials.
In the new 8-page draft guidance, the FDA discusses some of the risks it sees, including issues like confounding because of non-comparable patient groups, and bias based on patient selection and study outcome measurements.
The draft also spells out what exactly sponsors should submit to the FDA, with sections outlining what to submit on the study design, data sources and analytical approach.
“Sponsors should finalize the study protocol, including the research question of interest and rationale for the study design, before initiating study conduct,” the agency said in the draft. It also asks them to lay out potential alternative designs and explain why they ended up using their proposed approach.
In a 2020 paper published in JAMA, now-FDA Commissioner Robert Califf and several co-authors wrote about the potential benefits of observational studies, arguing that increasingly sophisticated medical records and data systems could allow researchers to parse patient data for insights and identify those worth studying further.
But Califf and his colleagues also highlighted the risks, saying that “aggregating information about diagnosis, comorbidities, treatment, and outcomes can lend a patina of technical excellence that obscures the influence of systematic bias.” And he warned that profit motives could skew the judgment of investigators who want to find data to support their hypothesis.
The new draft guidance is part of a suite of documents the FDA has released on real-world evidence. It builds off a 2018 framework in which the agency promised more clarity on how drug sponsors could use data collected outside of carefully controlled clinical trials in order to support new indications for approved drugs, or to satisfy post-approval study requirements.
