PATIENT PREFERENCES FOR TREATMENT BENEFITS IN SOLID METASTATIC CANCERS: A SYSTEMATIC LITERATURE REVIEW
Author(s)
Alexandra Jager1, Pauline C. Herscu, MSc2, Laura Martin-Mungapen, MSc3, Stephane Deparis, PhD4, Tommi Tervonen, PhD5, Boris Gorsh, PharmD6;
1Kielo Research, Senior Research Associate, Zug, Switzerland, 2Daiichi Sankyo EU GmbH, Munich, Germany, 3Daiichi Sankyo AG, Zurich, Switzerland, 4Kielo Research, Bordeaux, France, 5Kielo Research, Zug, Switzerland, 6Daiichi Sankyo Inc, Basking Ridge, NJ, USA
1Kielo Research, Senior Research Associate, Zug, Switzerland, 2Daiichi Sankyo EU GmbH, Munich, Germany, 3Daiichi Sankyo AG, Zurich, Switzerland, 4Kielo Research, Bordeaux, France, 5Kielo Research, Zug, Switzerland, 6Daiichi Sankyo Inc, Basking Ridge, NJ, USA
OBJECTIVES: Despite widespread acceptance of clinical endpoints like Progression-Free Survival (PFS) and Overall Survival (OS) by regulatory and Health Technology Assessment (HTA) bodies, limited evidence exists on how these endpoints are considered in patient preference studies. We aimed to understand the most frequently included treatment attributes, particularly benefit attributes (e.g. PFS, OS), in preference studies for metastatic solid tumors, and how they were defined.
METHODS: We searched PubMed and the Cochrane Library for studies published until February 2025. Two investigators screened abstracts and full texts, a third adjudicated disputes. Attributes and study characteristics were summarized descriptively.
RESULTS: 686 studies were screened; N=38 were synthesized. The most studied cancer types were breast (N = 9/38 [24%]), prostate (N = 8/38 [21%]) and lung (N = 7/38 [18%]) cancer (counting only studies where these cancers were the sole focus.) On average, studies included 6.2 attributes, comprising adverse event (2.7), benefit (2.1), process - how care is delivered, e.g. administration mode - (1.1) or cost (0.3) ones. Sixty-three percent of studies included PFS, 63% quality of life (QoL) benefits, 58% OS, and 24% other clinical benefit endpoints (e.g. Objective Response Rate (ORR)). Across cancer types, OS was most frequently included in lung (86%), PFS in breast (78%), and QoL in breast (78%) and prostate (75%) cancer studies. Benefit attributes were defined using a time scale (53%) (e.g., months/years), categorical variables (24%) (e.g., “worse” to “improved” QoL) and percentages (23%). Overall, there was a lack of standardization across benefit attribute names, definitions, and measurements.
CONCLUSIONS: Despite widespread use, definitions and operationalization of OS and PFS in patient preference studies varied widely, complicating comparability and interpretability. This combined heterogeneity across cancer indications and operationalization of benefit attributes restricts evidence transferability of patient preference evidence across settings.
METHODS: We searched PubMed and the Cochrane Library for studies published until February 2025. Two investigators screened abstracts and full texts, a third adjudicated disputes. Attributes and study characteristics were summarized descriptively.
RESULTS: 686 studies were screened; N=38 were synthesized. The most studied cancer types were breast (N = 9/38 [24%]), prostate (N = 8/38 [21%]) and lung (N = 7/38 [18%]) cancer (counting only studies where these cancers were the sole focus.) On average, studies included 6.2 attributes, comprising adverse event (2.7), benefit (2.1), process - how care is delivered, e.g. administration mode - (1.1) or cost (0.3) ones. Sixty-three percent of studies included PFS, 63% quality of life (QoL) benefits, 58% OS, and 24% other clinical benefit endpoints (e.g. Objective Response Rate (ORR)). Across cancer types, OS was most frequently included in lung (86%), PFS in breast (78%), and QoL in breast (78%) and prostate (75%) cancer studies. Benefit attributes were defined using a time scale (53%) (e.g., months/years), categorical variables (24%) (e.g., “worse” to “improved” QoL) and percentages (23%). Overall, there was a lack of standardization across benefit attribute names, definitions, and measurements.
CONCLUSIONS: Despite widespread use, definitions and operationalization of OS and PFS in patient preference studies varied widely, complicating comparability and interpretability. This combined heterogeneity across cancer indications and operationalization of benefit attributes restricts evidence transferability of patient preference evidence across settings.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR134
Topic
Patient-Centered Research
Disease
SDC: Oncology