Stakeholder Perspectives on Navigating Evidentiary and Decision Uncertainty in Precision Oncology

Abstract

(1) Background: Precision oncology has the potential to improve patient health and wellbeing through targeted prevention and treatment. Owing to uncertain clinical and economic outcomes, reimbursement has been limited. The objective of this pan-Canadian qualitative study was to investigate barriers to precision oncology implementation from the perspectives of health system stakeholders. (2) Methods: We conducted 32 semi-structured interviews with health technology decision makers (n = 14) and clinicians (n = 18) experienced with precision oncology. Participants were recruited using a purposive sampling technique. Interviews were analyzed using thematic analysis. Recruitment continued until two qualitative analysts reached agreement that thematic saturation was reached. (3) Results: While cautiously optimistic about the potential for enhanced therapeutic alignment, participants identified multiple decisional challenges under conditions of evidentiary uncertainty. Decision makers voiced concern over resource requirements alongside small benefitting patient populations and limited evidence supporting patient and health system impacts. Clinicians were comparatively tolerant of evidentiary uncertainty guiding clinical decision-making practices. Clinicians applied a broader definition of patient benefit, focusing on the ability to assist patients making informed clinical decisions. (4) Conclusions: Sustainable precision oncology must balance demand with evidence demonstrating benefit. We show that clinicians and decision makers vary in their tolerance for evolving knowledge, suggesting a need to establish evidentiary standards supporting precision oncology reimbursement decisions.

Stakeholders' expectations of precision medicine: A qualitative study to identify areas of (mis)alignment

“…Similar attempts have been made to discover differences between various stakeholder groups in terms of perceptions and expectations regarding patient engagement in medicines development, 18 prognostic-based genomic testing, 16 barriers to optimal implementation of PM, 19 or the optimal components of value frameworks. 11 Results of these studies are in line with those of our stakeholder expectation matrix, highlighting the need for improved communication and collaboration between all stakeholders if harmonization of PM implementation is desired.…”

Response to redefining health technology assessment: a comment on “the new definition of health technology assessment: a milestone in international collaboration”

“…Third, the new definition has been embraced by the HTA community. Since its publication, the new definition has been increasingly used as a reference, such as by Mukherjee (6) on the relevance of the new definition in the case of COVID-19 and beyond; by Pollard et al (7) to show that it reflects the need for continuous evidence generation informing both clinical and reimbursement decisions in precision oncology; and several papers (co) authored by Culyer as well (8;9). Indeed, the manuscripts announcing the new definition of HTA have been extensively cited.…”