Highlights
- •Tumour eradication and reduced risk of recurrence are important to patients.
- •Time-to-event end-points are commonly accepted in health technology assessment (HTA).
- •Pathological complete response is often not considered as patient-relevant in HTA.
- •Adoption of (neo)adjuvant end-points in HTA improves access to innovative therapies.
Abstract
Health technology assessment (HTA) of clinical and economic value of a new intervention
is an integral step in providing the access of patients to innovative cancer care
and treatment. Overall survival (OS) is the preferred criterion for demonstrating
the therapeutic efficacy in HTA given its direct clinical and patient relevance. However,
with often long life expectancy of patients with early cancer, analysis of OS becomes
less practical. Partially due to this reason, pathological complete response (pCR)
and time-to-event end-points like disease-free survival are frequently incorporated
into the pivotal clinical trials in the neoadjuvant and adjuvant settings. However,
there exists a discrepancy between different national HTA bodies regarding the acknowledgement
of patient relevance of these end-points. In this article, we analysed the perspectives
of patients on different aspects of end-points used in clinical trials in early cancer.
Gathered evidence strongly suggests that complete tumour eradication and reduced risk
of recurrence provide important psychological benefits thus signifying that pCR and
time-to-event end-points are directly relevant to patients. Additionally, we reviewed
opinions on patient relevance of neoadjuvant and adjuvant therapy end-points adopted
by HTA bodies during the recent evaluations. We found that improvements in end-points
used in the adjuvant setting were commonly considered as valuable to patients. In
contrast, opinions on patient relevance of neoadjuvant therapy end-points varied between
the national HTA bodies. Universal acknowledgement of patient relevance of therapeutic
end-points for early cancer by HTA bodies is necessary to balance the inequality in
uptake of innovative therapies into national healthcare systems.
Keywords
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Article info
Publication history
Published online: February 19, 2021
Accepted:
January 12,
2021
Received in revised form:
January 5,
2021
Received:
October 22,
2020
Identification
Copyright
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