Changes in Companion Diagnostic Labelling: Implementation of FDA's April 2020 Guidance for Industry for In Vitro CDx Labeling for Specific Oncology Therapeutic Groups

Ther Innov Regul Sci. 2022 Sep;56(5):689-697. doi: 10.1007/s43441-022-00422-z. Epub 2022 Jun 10.


Advanced understanding of the molecular pathways of oncologic diseases has shifted therapeutic treatment development to focus on mechanism of actions targeting specific genomic alterations. These precision medicines are indicated for patient subsets defined by these specific mutations as determined by diagnostic devices approved by the Food and Drug Administration (FDA). The Intended Use section within the companion diagnostic (CDx) labeling has historically specified the therapeutic products for which they have been clinically validated. In April 2020, the FDA reiterated their position that therapeutic class labeling may be used, if appropriate, instead of named products. Labels for FDA approved in vitro CDxs were reviewed to evaluate the implementation of therapeutic class labeling. A total of 47 devices have been approved as of 2 January 2022, of which 3 labels were found to contain therapeutic class labeling: two devices targeting EGFR mutations for the treatment of non-small cell lung cancer (NSCLC), and one targeting BRAF V600E and BRAF/MEK inhibitor combinations for melanoma. Two devices received therapeutic class labeling upon initial approval, while the third implemented the language though a label revision. A total of 25 different indications were identified across the 47 CDx devices, of which 9 (34.6%) were associated with more than 1 CDx device. Implementation of therapeutic class labeling has been slow following the release of the FDA's April 2020 guidance; however, the potential to incorporate such language into existing and newly approved CDx labels exists. Precedence and manufacturer experience are expected to drive an increase in therapeutic class labeling.

Keywords: Companion diagnostic; Labeling; Oncology; Precision medicine; Therapeutic class.

Publication types

  • Review
  • Research Support, N.I.H., Extramural

MeSH terms

  • Carcinoma, Non-Small-Cell Lung*
  • Humans
  • Lung Neoplasms*
  • Precision Medicine
  • Proto-Oncogene Proteins B-raf / genetics
  • United States
  • United States Food and Drug Administration


  • Proto-Oncogene Proteins B-raf