White paper: Fool’s gold or pot of gold?The challenges of demonstrating the value of Tumour Agnostic therapies

George Forsyth

Targeted therapies are considered the future of medicine and tumour agnostic (TA) therapies are no exception. TA therapies work by targeting a single genetic aberration that can be found across multiple tumour types. Expression of these genetic defects is often associated with rare cancer subtypes in which patients have few therapeutic options available to them.

In the past, the small number of patients in each histology with the relevant genetic profile made them an unappealing target for manufacturers due to limited market potential. Now, the ability to target these patients across multiple histologies in larger volumes using biomarker testing provides an attractive opportunity for pharmaceutical companies.

Whitepaper - Fools gold or pot of gold
Whitepaper - Fools gold or pot of gold

Targeted therapies are considered the future of medicine and tumour agnostic (TA) therapies are no exception. TA therapies work by targeting a single genetic aberration that can be found across multiple tumour types. Expression of these genetic defects is often associated with rare cancer subtypes in which patients have few therapeutic options available to them.

In the past, the small number of patients in each histology with the relevant genetic profile made them an unappealing target for manufacturers due to limited market potential. Now, the ability to target these patients across multiple histologies in larger volumes using biomarker testing provides an attractive opportunity for pharmaceutical companies.

In order to secure regulatory approval, manufacturers need to clearly demonstrate TA products are safe and efficacious. However, demonstration of safety and efficacy to regulators is not the key challenge facing manufacturers wishing to achieve market access. The key challenge is how to demonstrate therapeutic value to payers relative to standard of care therapies across multiple histologies, as acceptance by regulatory authorities does not automatically translate into acceptance by national payers. Manufacturers need to demonstrate value to both.

Earlier this year we spoke to over 20 payers across Europe to discuss the challenges of assessing therapeutic value. Download our latest Free Thinking whitepaper to find out what they had to say

Download the white paper

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