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Regulatory Update, August 2016

Monday, August 1, 2016

FDA Publishes Several Final Guidance Documents

Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices

In the June 21, 2016 Federal Register, the FDA announced the availability of the guidance titled “Leveraging Existing Clin­ical Data for Extrapolation to Pediatric Uses of Medical Devices.” This guidance explains the circumstances in which it may be appropriate to extrapolate existing medical device data to support pediatric device indications in several marketing ap­plications. This guidance also describes the FDA’s approach for determining whether extrapolation may be appropriate and the factors that should be considered within a statistical model for extrapolation. Extrap­olation may be appropriate when there are few differences in safety or effectiveness of the proposed device when used in adult as compared to the intended pediatric popu­lations and the adult data are of high qual­ity for borrowing.

The objectives of this final guidance are (1) to increase the availability of safe and effective pediatric devices by providing a roadmap for leveraging relevant existing clinical data for use in demonstrating a reasonable assurance of safety/effective­ness in PMAs and de novo requests, as well as for use in supporting approvals of HDEs; (2) to explain the circumstances in which it may be appropriate to leverage ex­isting clinical data to support pediatric de­vice indications and labeling; (3) to outline the approach the FDA uses to determine whether extrapolation is appropriate, and to what extent the data can be leveraged; and (4) to describe statistical methodology that can be used to leverage the data in a way that increases precision for pediatric inferences.

This will potentially streamline the pro­cess for establishing a pediatric intended use claim, and enhance and encourage pediatric device development programs. This guidance does not change the regula­tory threshold for valid scientific evidence. Sponsors should engage the FDA early in product development planning. For pur­poses of this document, “extrapolation” refers to the leveraging process whereby an indication for use of a device in a new pedi­atric patient population can be supported by existing clinical data from a studied patient population. That is, when existing data are relevant to a pediatric indication and determined to be valid scientific evi­dence, it may be appropriate to attempt to extrapolate such data to a pediatric use in support of demonstrating a reasonable as­surance of effectiveness or probable benefit and, occasionally, safety. The Docket No. is FDA-2015-D-1376.

Charging for Investigational Drugs under an IND Application

In the June 3, 2016 Federal Register, the FDA announced the availability of the guidance titled “Charging for Investiga­tional Drugs under an Investigational New Drug Application—Questions and An­swers.” The guidance document provides information for industry, researchers, phy­sicians, institutional review boards (IRBs) and patients about the implementation of the FDA’s regulation on charging for inves­tigational drugs under an investigational new drug application (IND) for the pur­pose of either clinical trials or expanded access for treatment use. The FDA received questions about its implementation of the charging regulation and is providing guid­ance in a question/answer format to ad­dress the most frequently asked questions. This guidance makes final a May 2013 final guidance. The Docket No. is FDA-2013-D-0447.

Expanded Access to Investigational Drugs for Treatment Use—Q&A

In the June 3, 2016 Federal Register, the FDA announced the availability of the guidance for industry titled “Expanded Access to Investigational Drugs for Treat­ment Use—Questions and Answers.” The guidance provides information for indus­try, researchers, physicians, institutional re­view boards (IRBs), and patients about the implementation of the FDA’s regulations on expanded access to investigational drugs for treatment use under an investigational new drug application (IND).

Expanded access refers to the use of an investigational drug when the primary purpose is to diagnose, monitor, or treat a patient rather than to obtain the kind of information about the drug that is gener­ally derived from clinical trials. Under the regulations, there are three categories of ex­panded access: (1) Expanded access for in­dividual patients, including for emergency use; (2) expanded access for intermediate-size patient populations (generally smaller than those typical of a treatment IND or treatment protocol—a treatment protocol is submitted as a protocol amendment to an existing IND by the sponsor of the existing IND); and (3) expanded access for wide­spread treatment use through a treatment IND or treatment protocol (designed for use in larger patient populations). The regu­lations are intended to facilitate, when ap­propriate, the availability of investigational new drugs outside of a clinical investigation to patients with serious or immediately life-threatening diseases or conditions who lack other therapeutic options. The Docket No. is FDA-2013-D-0446.

The Regulatory Update is excerpted from Re­search Practitioner, Volume 17 Number 04, July-August 2016.

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