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

Abstract

Background and aims: To sustainably address challenges in implementing precision medicine (PM), coordinated efforts of different stakeholders are required. Understanding their expectations represents a first key step toward aligning on future actions and strategies. Here, we aimed to explore the expectations of different stakeholders from themselves and each other regarding PM.

Methods: This collaborative qualitative study was initiated by the global multistakeholder consortium From Testing to Targeted Treatments (FT3). Structured interviews were conducted with participants from five stakeholder groups: patients/patient advocates, healthcare providers (HCPs), researchers, policymakers/regulators/payers and industry representatives. A broad reach across geography, roles, experiences, and disease areas was sought. Results were analyzed by grounded theory methodology.

Results: All stakeholders stated that optimal implementation of PM can only be achieved through collaboration; industry representatives were the biggest promoters of collaboration. Stakeholders agreed that PM should be implemented focusing on the patient's best interest; HCPs were seen as important gatekeepers for PM by interacting directly with patients, and policymakers/payers were perceived as the most important drivers of access to PM. Areas of misalignment included the role of industry in clinical trial design and in access to PM (perceived as important by patients, HCPs and policymakers but not by industry representatives), and the stakeholders responsible for elaborating guidelines on PM use (patients indicated policymakers, while researchers indicated themselves). Priorities for optimal PM implementation and suggested actions included the need for enhancing high-level policy focus, improving genomic literacy, optimizing the health technology assessment for PM, advocating for equitable access, promoting collaboration between industry and other stakeholder groups and development of reliable research standards.

Conclusion: Stakeholder expectations revealed in this study suggested that no stakeholder group can drive change on its own; a global, multistakeholder collaborative approach that brings together current programs and best practices to support universal access to PM is needed.

Keywords: equitable access; patient advocacy; precision medicine; priorities; stakeholder alignment; stakeholder expectations.

Figure 1

Overview of the study process. FT3, From Testing to Targeted Treatments.

Figure 2

Overview of the interviewees according to stakeholder group and geography. EU, European Union; HCPs, healthcare professionals; N, number of stakeholders; UK, United Kingdom; USA, United States of America.

Figure 3

Stakeholder expectation matrix. HCPs, healthcare providers; MTBs, molecular tumor boards; PM, precision medicine; R&D, research, and development.

Figure 4

Access points for PM and barriers identified across the patient journey. HCPs, healthcare providers; MTBs, molecular tumor boards.

Figure 5

Priorities and suggested actions to overcome the barriers to optimal implementation of PM. HCPs, healthcare providers; MDTs, multidisciplinary teams; PM, precision medicine.