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Fool’s gold or pot of gold? The challenges of demonstrating the value of tumour agnostic therapies

By 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.

This move towards TA R&D is a natural continuation of the PD-1/PD-L1 success story that has driven the clinical community towards a more genomic approach to treating cancer. Looking to the future, we can expect to see this trend continue as clinicians move away from the current histology-dependent approach towards a biomarker-driven paradigm.

To date, Vitrakvi (larotrectinib) & Rozlytrek (entrectinib) are the only ‘pure’ TA products to receive conditional EMA approval and accelerated FDA approval for NTRK positive tumours.

Whilst Keytruda was the first therapy to be approved for pan-tumour use in 2017, its Microsatellite Instability-High or Mismatch Repair-Deficient indication differs from the NTRK TA therapies.

Instead of targeting a specific genetic defect, Keytruda targets tumours that have a high degree of genetic instability. Of course, this is only one of Keytruda’s numerous indications, which is a further point of differentiation from the NTRK TA therapies. As Vitrakvi and Rozlytrek lead with their TA indications, the challenges faced by these therapies are wholly different to Keytruda.

Vitrakvi and Rozlytrek target NTRK gene mutations and fusions in patients with locally advanced or metastatic disease and no satisfactory treatment alternatives. NTRK alterations generally have low expression in more common cancer types such as lung and breast cancer but higher expression in ultra-rare cancer types. This phenomenon is common across TA therapies and further emphasises their potential utility in patients with rare conditions and high unmet need.

Manufacturers look set to try to capitalise on this with a further ~ten tumour-agnostic therapies anticipated to launch in the coming years targeting the same or various other mutations. Targets include the receptor tyrosine kinases, anaplastic lymphoma kinase (ALK), c-ROS oncogene 1 (ROS1), and rearranged during transfection (RET), to name a few.

In the coming years we expect to see worldwide sales of Vitrakvi and Rozlytrek to grow as the products become more readily available around the world. Current forecasts suggest that by 2026 sales will reach $596m for Vitrakvi and $402m for Rozlytrek, accounting for 2.7% of Bayer’s sales and 1% of Roche’s respectively. As familiarity and acceptance of TA therapies grows we will likely see an increase in sales beyond these estimates as the products are used earlier in the treatment paradigm

How to demonstrate value to regulators and payers?

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.

To find out what they had to say, click here to download our latest Free Thinking whitepaper.

George Forsyth is a Market Access Consultant at Research Partnership

2nd July 2021

From: Research

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