OBJECTIVES: Overall survival (OS) data is the traditional oncology endpoint desired by payers, however it does not provide insight into the quality of patient survival, and the need for long-term data collection may delay patients’ access to new medicines. Despite this, payers and health technology assessment (HTA) bodies still have a preference for survival-related endpoints The aim of this research was to identify and review published literature relating to novel patient-relevant endpoints in oncology, using prostate cancer as an example, and determine HTA bodies’ acceptance of these endpoints.

METHODS: A search of HTA websites and a targeted literature review were conducted. Literature published within the last 3 years and reporting on novel patient-relevant endpoints utilized in clinical trials in prostate cancer were included.

RESULTS: Several novel patient-relevant endpoints were identified that capture patient experiences in prostate cancer. This included time to deterioration in the Brief Pain Inventory-Short Form (BPI-SF), which was considered positively in published HTA appraisals, although a lack of a standard definition has been noted across the literature. Positive HTA opinions were also identified for the endpoint of time to symptomatic skeletal-related event (SSE), although concerns were noted regarding the relevance of this endpoint in hormone-resistant prostate cancer populations.

CONCLUSIONS: The necessity for patient-relevant endpoints that provide an indication of a patient’s quality of survival which can be measured within a shorter time period than OS data has become increasingly evident. The literature demonstrated that novel patient-relevant endpoints are currently used within prostate cancer trials and positively reviewed by HTA, although the need for standard definitions was highlighted and the relevance of each to different patient subgroups needs to be assessed. There are potential benefits in exploring the use of similar endpoints within clinical trials for oncology indications and assessing HTA opinion on the value of novel endpoints relative to OS.