Tumour-Agnostic Treatment

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Tumour agnostic treatment is a genomically-informed treatment strategy that enriches for novel targets regardless of histological origin. This treatment typically fulfils the following criteria [1]:

  1. Tumours are enriched for ≥1 molecular alteration.
  2. Alterations are likely to predict response to a therapy.
  3. Alterations are found across a variety of cancers.

The following table summarises the key differences between development of a tumour-agnostic treatment (assessed in “basket trials”) versus traditional approval of cancer therapies [1-3].

Table 2: Tumour-Agnostic Treatment Versus Traditional Cancer Therapies

  Tumour-agnostic treatment Traditional cancer therapies
Basis of approval
  • Biomarker present across many tumour types.
  • Biomarkers of response, establishing the effects of context, and deciphering mechanisms of treatment resistance across a variety of tumour types. 
  • Consider heterogeneity of drug effects in different biomarker-positive tumour types.
  • Can be directed toward a specific genomic abnormality in a specific tumour type.
Patient population
  • Small patient cohorts with diverse tumour types and a common genomic event.
  • Large patient cohorts with tumours originating from a single anatomic site.
  • Tumours that commonly have heterogeneous genomic aberrations.

In May 2017, the first approval of a tumour-agnostic treatment (pembrolizumab for tumours deficient in mismatch repair or with high microsatellite instability) was received [4]. 


  1. Offin M, Liu D, Drilon A. Tumor-Agnostic Drug Development. J Clin Oncol 2018; 286-295.
  2. Remon J, Dienstmann R. Precision oncology: separating the wheat from the chaff. ESMO Open 2018; 3: e000446.
  3. Flaherty KT, Le DT, Lemery S. Tissue-Agnostic Drug Development. Am Soc Clin Oncol Educ Book 2017; 38: 222-230.
  4. Davis AA, McKee AE, Kibbe WA, Villaflor VM. Complexity of Delivering Precision Medicine: Opportunities and Challenges. Am Soc Clin Oncol Educ Book 2018; 38: 998-1007.

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Last update: 24 June 2019