HTA Readiness for Tumor-Agnostic Therapies: Lessons From Analogues Across Major Markets
Author(s)
Nikhil Taxak, PhD1, Thomas Gilboy, BA (Hons)2, Keshav Nagaraja, BEc, MBA2, Shrinivas Mukku, PhD2.
1Access Infinity Ltd, Hyderabad, India, 2Access Infinity Ltd, London, United Kingdom.
1Access Infinity Ltd, Hyderabad, India, 2Access Infinity Ltd, London, United Kingdom.
OBJECTIVES: Over the past decade, oncology therapy area has witnessed remarkable growth in the approval of drugs spanning multiple tumor types. Since Jan 2020, 9 molecules have been approved as tumor-agnostic drugs across global markets. This trend is reflected in the evolving landscape of oncology clinical trials with 10-12% of phases 2, 2/3 and 3 trials being tumor-agnostic as of date. Given the current trajectory, we explored how these therapies have been perceived by the global payers specifically on their willingness to attribute value to evidence at launch and to engage in constructive steps.
METHODS: Secondary research was conducted to identify approved tumor-agnostic drugs in addition to the data obtained from Nuro, Access Infinity’s proprietary data platform. HTA outcomes and payer perceptions were analysed for pembrolizumab, entrectinib, larotrectinib and selpercatinib across 6 key global markets (Australia, Canada, England, France, Germany and Scotland) to determine commonalities and differences in payer reviews and constructive suggestions provided to the manufacturers by the payers.
RESULTS: The evidence package for these tumor-agnostic drugs comprised of pooled results from multiple single arm trials. Overall, neutral to unfavorable HTA outcomes were seen while there were similarities in terms of challenges such as the lack of comparative data, the population (size and selection) and endpoints used to demonstrate value across in-scope markets. Constructive suggestions from the payers included providing some sort of comparative analysis based on indirect treatment comparison, real-world and registry data.
CONCLUSIONS: Tumor-agnostic therapies have faced payer scrutiny owing to evidence issues however, there were some signals that payers are willing to engage with manufacturers to provide patient access even without a robust evidence package. It is imperative for the manufacturers and the global market access teams to adopt a tumor-agnostic approach in evidence generation, such as pooled analysis of tumor types.
METHODS: Secondary research was conducted to identify approved tumor-agnostic drugs in addition to the data obtained from Nuro, Access Infinity’s proprietary data platform. HTA outcomes and payer perceptions were analysed for pembrolizumab, entrectinib, larotrectinib and selpercatinib across 6 key global markets (Australia, Canada, England, France, Germany and Scotland) to determine commonalities and differences in payer reviews and constructive suggestions provided to the manufacturers by the payers.
RESULTS: The evidence package for these tumor-agnostic drugs comprised of pooled results from multiple single arm trials. Overall, neutral to unfavorable HTA outcomes were seen while there were similarities in terms of challenges such as the lack of comparative data, the population (size and selection) and endpoints used to demonstrate value across in-scope markets. Constructive suggestions from the payers included providing some sort of comparative analysis based on indirect treatment comparison, real-world and registry data.
CONCLUSIONS: Tumor-agnostic therapies have faced payer scrutiny owing to evidence issues however, there were some signals that payers are willing to engage with manufacturers to provide patient access even without a robust evidence package. It is imperative for the manufacturers and the global market access teams to adopt a tumor-agnostic approach in evidence generation, such as pooled analysis of tumor types.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA189
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Systems & Structure
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology