Biologics

Trade Groups Comment on Externally Controlled Trials

The Biotechnology Innovation Organization (BIO) says the scope of an FDA draft guidance on considerations for the design and conduct of externally controlled trials for drugs and biologics “is rather narrow, excluding aggregate-level data as well as hybrid designs using external controls to augment a randomized trial.” The BIO comment letter says the association believes that an extension of the scope to cover summary-level data and hybrid designs would significantly increase the practical utility of the guidance. It offers comments on data access and FDA/stakeholder engagement, multiple data sources, ethical considerations for randomized trial design, sensitivity analyses to address residual uncontrolled confounding, index date, target trial emulation, prospective data collection, and additional considerations, along with a table of line-by-line recommended changes.

Pharmaceutical Research and Manufacturers of America (PhRMA) says it supports FDA’s efforts over decades to recognize the potential value of external controls. PhRMA says it recommends:

FDA focus the final guidance more on the potential benefits of external controls and how trials using external controls may be used to support regulatory decision-making;
FDA explicitly note that the use of external controls is particularly common in the development of drugs for rare diseases;
broadening the draft guidance to address considerations when using external controls for trials not intended to be the primary basis of evidence supporting approval;
broaden the scope of the final guidance to include considerations for using external control data to supplement a control arm in a clinical trial, use of multiple control arms, and use of summary level data to establish an external control, study design, and analytic methods;
cross-reference applicable real-world evidence guidance throughout; and
FDA acknowledge in the final guidance that multiple data sources may be integrated to serve as the external control, including a potential combination of clinical data and real-world data.

PhRMA also provides specific comments and a table with line-by-line recommendations.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome