Oops, you're using an old version of your browser so some of the features on this page may not be displaying properly.

MINIMAL Requirements: Google Chrome 24+Mozilla Firefox 20+Internet Explorer 11Opera 15–18Apple Safari 7SeaMonkey 2.15-2.23

Previous Page Next Page

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 [5]. Since then, other such approvals have followed including those for NTRK inhibitors larotrectinib and entrectinib, dorstarlimab (an anti-PD-1 antibody) and the combination of dabrafenib and trametinib in for BRAF V600E gene mutated cancers.


  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. Looney AM, Nawaz K, Webster RM. Tumour-agnostic therapies. Nat Rev Drug Discov. 2020 Jun;19(6):383-384.
  5. 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.

This site uses cookies. Some of these cookies are essential, while others help us improve your experience by providing insights into how the site is being used.

For more detailed information on the cookies we use, please check our Privacy Policy.

Customise settings
  • Necessary cookies enable core functionality. The website cannot function properly without these cookies, and you can only disable them by changing your browser preferences.