Abstract

IntroductionWell- or moderately differentiated gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are often slow-growing, and some patients with unresectable, asymptomatic, non-functioning tumors may face the choice between watchful waiting (WW), or somatostatin analogues (SSA) to delay progression. We developed a comprehensive multi-criteria decision analysis (MCDA) framework to help patients and physicians clarify their values and preferences, consider each decision criterion, and support communication and shared decision-making.MethodsThe framework was adapted from a generic MCDA framework (EVIDEM) with patient and clinician input. During a workshop, patients and clinicians expressed their individual values and preferences (criteria weights) and, on the basis of two scenarios (treatment vs WW; SSA-1 [lanreotide] vs SSA-2 [octreotide]) with evidence from a literature review, expressed how consideration of each criterion would impact their decision in favor of either option (score), and shared their knowledge and insights verbally and in writing.ResultsThe framework included benefit-risk criteria and modulating factors, such as disease severity, quality of evidence, costs, and constraints. Overall and progression-free survival being most important, criteria weights ranged widely, highlighting variations in individual values and the need to share them. Scoring and considering each criterion prompted a rich exchange of perspectives and uncovered individual assumptions and interpretations. At the group level, type of benefit, disease severity, effectiveness, and quality of evidence favored treatment; cost aspects favored WW (scenario 1). For scenario 2, most criteria did not favor either option.ConclusionsPatients and clinicians consider many aspects in decision-making. The MCDA framework provided a common interpretive frame to structure this complexity, support individual reflection, and share perspectives.FundingIpsen Pharma.Electronic supplementary materialThe online version of this article (10.1007/s12325-017-0653-1) contains supplementary material, which is available to authorized users.

Highlights

  • Well- or moderately differentiated gastroenteropancreatic neuroendocrine tumors (GEP-Neuroendocrine tumors (NETs)) are often slow-growing, and some patients with unresectable, asymptomatic, non-functioning tumors may face the choice between watchful waiting (WW), or somatostatin analogues (SSA) to delay progression

  • Of the 30criteria presented to the criteria workshop participants, 26 were considered relevant by more than 90% of the participants

  • This suggests that true understanding of the decision requires embracing its complexity, which has to be reflected in a framework that is intended to support the decision-making process

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Summary

Introduction

Well- or moderately differentiated gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are often slow-growing, and some patients with unresectable, asymptomatic, non-functioning tumors may face the choice between watchful waiting (WW), or somatostatin analogues (SSA) to delay progression. For unresectable disease, two randomized, double-blind, placebo-controlled trials, PROMID and CLARINET, demonstrated that the somatostatin analogues (SSAs) octreotide and lanreotide significantly prolong time to progression and progression-free survival (PFS) in patients with well- or moderately differentiated midgut or GEP-NETs, respectively [10, 11]. Both SSAs are administered every 4 weeks, octreotide via intramuscular injection, lanreotide via deep subcutaneous injection with the option of self-administration [12, 13]. The nature of the disease and the different options imply that patients’ preferences and circumstances are central to decision-making in this context, suggesting a systematic approach to help identify the relevant factors bearing on the decision and support shared decision-making

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