Table 4.

Identified key domains to address the challenge of endpoint selection

Challenges with Endpoint Selection
Key Domains	Approaching new endpoints	Patient preferences on endpoints	Position of HTA and other stakeholders	Long-term generation & secondary use of data	Endpoint challenges in RCTs
Description	Review of applicable endpoints at the commencement of each clinical development program and early agreement across all involved stakeholders if new endpoints are required in a particular setting	The view of patients should be taken into consideration for the determination of an endpoint as ‘patient-relevant’ in HTAs	Early collaboration should make the position of all involved stakeholders transparent and lead to resolution of differing views	Agreement on the need for the generation of long-term data and secondary use of data by all involved stakeholder groups	Review of and agreement on appropriate endpoints and feasibility of RCTs to generate comparative data on these endpoints between the involved stakeholders before commencement of the clinical program
Guiding Questions	Are new endpoints applicable to this disease area?	How to empower patients’ voice in the determination of patient-relevance of any specific endpoint?	Who should be involved in the definition of an endpoint? How can differing views on endpoint acceptability between stakeholders be resolved?	How can long-term data generation and secondary use of data be facilitated?	How can we ensure that endpoints match evolving treatment paradigms in an increasingly targeted oncology environment?