Drugs

Guide on Scientific Info on Unapproved Uses

David McFarland

— 2 min read
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FDA has released a revised https://www.fda.gov/regulatory-information/search-fda-guidance-documents/communications-firms-health-care-providers-regarding-scientific-information-unapproved-uses entitled “Communications From Firms to Health Care Providers Regarding Scientific Information on Unapproved Uses of Approved/Cleared Medical Products — Questions and Answers.” The documents is intended to provide the agency’s current thinking on common questions about companies’ communications to healthcare providers (HCPs) on “scientific information on unapproved uses” (SIUU), which is a new term added to FDA’s lexicon.

The revised draft guidance supersedes a 2014 draft guidance entitled “Distributing Scientific and Medical Publications on Unapproved New Uses — Recommended Practices." The agency says it was revised based on stakeholder feedback, including comments received on the 2014 guidance.

The revised guidance covers the following SIUU communications:

  • Published scientific or medical journal articles (reprints)
  • Clinical practice guidelines (CPGs)
  • Scientific or medical reference texts (reference texts)
  • Materials from independent clinical practice resources
  • Firm-generated presentations of scientific information from an accompanying published reprint

The guidance addresses what FDA says companies should consider in determining whether a source publication can serve as the basis for an SIUU communication. “Source publications that serve as the basis for SIUU communications should describe studies or analyses that are scientifically sound and provide clinically relevant information,” the agency draft says. “For human and animal drugs, randomized, double-blind, concurrently controlled superiority trials are usually regarded as the most rigorous design and informative to clinical practice, and therefore the most likely to provide scientifically sound and clinically relevant information; however, other well-designed and well-conducted trials are also able to generate scientifically sound and clinically relevant information.”

Regarding medical devices, the draft guidance points to the types of “valid scientific evidence” described in 21 CFR 860.7, which “may include well-controlled investigations, partially controlled studies, studies and objective trials without matched controls, well-documented case histories conducted by qualified experts, and reports of significant human experience with a marketed device.”

The document also notes that real-world data and associated real-world evidence about medical products “may be scientifically sound and clinically relevant depending on the characteristics of the data and the nature of the analyses. Other types of well-designed and well-conducted studies and analyses can also be informative to HCPs, but any study or analysis described in a source publication should be evaluated in light of its limitations to determine whether the study or analysis is scientifically sound and provides clinically relevant information.”

FDA also says that studies without an adequate comparison or control group, isolated case reports, and other reports that lack enough detail to permit scientific evaluation would typically not be scientifically sound or clinically relevant. It says data from “early stages of medical product development can produce results that are not borne out in later studies,” and therefore are “unlikely to be sufficiently reliable by themselves to allow for a determination of clinical relevance.”

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