Kerri Fitzgerald

DocwireNews

The U.S. Food and Drug Administration (FDA) issued a draft guidance that recommends limiting the use of placebo in double-blinded cancer clinical trials.&nbsp;&nbsp;&ldquo;Because investigators and patients in these&nbsp;trials do not know what treatment patients are receiving, this&nbsp;can decrease the likelihood of&nbsp;biased observations, decrease differential patient drop out, and allow for unbiased assessment of&nbsp;outcome measures, particularly when the assessment includes subjectivity, such as for quality of life measures,&rdquo; the guidance reads. The agency noted that the use of placebo in these kinds of trials presents both practical and ethical concerns. &ldquo;If possible, an active control is often preferred over placebo, and one option has been to&nbsp;conduct an open-label trial with a physician&rsquo;s choice of one of a few standard therapies as the&nbsp;comparator,&rdquo; according to the guideline.&nbsp;<blockquote class="twitter-tweet" data-width="500" data-dnt="true">
Thanks for sharing this <a href="https://twitter.com/US_FDA?ref_src=twsrc%5Etfw">@US_FDA</a> guidance, <a href="https://twitter.com/JTrentMDPhD?ref_src=twsrc%5Etfw">@JTrentMDPhD</a>. Agree that placebo should be used very cautiously as comparators in <a href="https://twitter.com/hashtag/sarcoma?src=hash&amp;ref_src=twsrc%5Etfw">#sarcoma</a> or <a href="https://twitter.com/hashtag/GIST?src=hash&amp;ref_src=twsrc%5Etfw">#GIST</a> trials. I like the thoughts here on the importance of unblinding patient treatments to minimize harm. <a href="https://t.co/SFOMRoCI4z">https://t.co/SFOMRoCI4z</a>
&mdash; Dale Shepard MD, PhD (@ShepardDale) <a href="https://twitter.com/ShepardDale/status/1034424918333681664?ref_src=twsrc%5Etfw">August 28, 2018</a>
</blockquote><script async src="https://platform.twitter.com/widgets.js" charset="utf-8"></script><blockquote class="twitter-tweet" data-width="500" data-dnt="true">
. <a href="https://twitter.com/US_FDA?ref_src=twsrc%5Etfw">@US_FDA</a> issues guidance clarifying that <a href="https://twitter.com/hashtag/cancer?src=hash&amp;ref_src=twsrc%5Etfw">#cancer</a> <a href="https://twitter.com/hashtag/clinicaltrials?src=hash&amp;ref_src=twsrc%5Etfw">#clinicaltrials</a> only need a placebo-control in select circumstances <a href="https://t.co/pJvT7gywE1">https://t.co/pJvT7gywE1</a>
&mdash; Robin Whitsell (@robinwhitsell) <a href="https://twitter.com/robinwhitsell/status/1033114238619934722?ref_src=twsrc%5Etfw">August 24, 2018</a>
</blockquote><script async src="https://platform.twitter.com/widgets.js" charset="utf-8"></script>The FDA presented cases when&nbsp;placebo-controlled trials could be considered:&nbsp;<ul>
<li data-leveltext="&#61623;" data-font="Symbol" data-listid="4" aria-setsize="-1" data-aria-posinset="1" data-aria-level="1">Trial sponsors should provide the rationale for the trial design. This justification is particularly important in the setting of a sham surgical procedure or when invasive methods are required for the&nbsp;administration of the placebo.&nbsp;</li>
<li data-leveltext="&#61623;" data-font="Symbol" data-listid="4" aria-setsize="-1" data-aria-posinset="2" data-aria-level="1">The FDA does not require patient-level maintenance of blinding at the time of disease recurrence or progression. Unless no other appropriate treatments are available,&nbsp;the agency recommends&nbsp;unblinding&nbsp;a patient when&nbsp;disease recurs or progresses.&nbsp;</li>
<li data-leveltext="&#61623;" data-font="Symbol" data-listid="4" aria-setsize="-1" data-aria-posinset="3" data-aria-level="1">When the patient experiences an adverse event that appears to be drug-related, the FDA recommends&nbsp;unblinding&nbsp;the patient and investigator if treating the event would involve one or more drugs or an invasive procedure. The patient should not be removed from the trial after being unblinded.&nbsp;</li>
<li data-leveltext="&#61623;" data-font="Symbol" data-listid="4" aria-setsize="-1" data-aria-posinset="4" data-aria-level="1">A detailed description in the protocol and statistical analysis plan of the proposal should be provided for both blinding (including whether the physiologic effects or adverse events associated with the investigational drug product will prevent effective blinding) and&nbsp;unblinding&nbsp;(including information regarding situations in&nbsp;which&nbsp;unblinding&nbsp;should occur).&nbsp;</li>
<li data-leveltext="&#61623;" data-font="Symbol" data-listid="4" aria-setsize="-1" data-aria-posinset="5" data-aria-level="1">If blinding is continued, the informed consent document should specify the risks and potential disadvantages of this approach, and the protocol should also include justification for the potential added risk.&nbsp;</li>
</ul>Placebo-designed trials may be useful in&nbsp;maintenance therapy, add-on trial designs, or in trials of adjuvant therapies (where standard of care is surveillance), according to the FDA.&nbsp;<a href="https://proddocwire.wpengine.com/hematology-oncology/fda-guidance-on-next-generation-sequencing-2/">FDA releases guidance on next-generation sequencing.</a>&nbsp;<a href="https://proddocwire.wpengine.com/hematology-oncology/fda-withdraws-draft-guidance-on-biosimilars/">FDA withdraws its draft guidance on biosimilars.</a>&nbsp;Source:<a href="https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM617931.pdf">&nbsp;</a><a href="https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM617931.pdf">FDA&nbsp;</a>

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The U.S. Food and Drug Administration (FDA) issued a draft guidance that recommends limiting the use of placebo in double-blinded cancer clinical trials.   ...

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