Preferences and Priorities for Relapsed Multiple Myeloma Treatments Among Patients and Caregivers in the United States

Auclair, Daniel; Mansfield, Carol; Fiala, Mark A.; Chari, Ajai; Cole, Craig E.; Kaufman, Jonathan L.; Orloff, Gregory; Siegel, David S.; Zonder, Jeffrey A.; Mange, Brennan; Yesil, Jennifer; Dalal, Mehul; Mıkhael, Joseph

doi:10.2147/ppa.s345906

Cited by 5 publications

(2 citation statements)

References 25 publications

(20 reference statements)

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“…About half of the studies ( n = 16, 47%) used in-depth or semi-structured interviews. Only two studies (6%) involved patient focus groups in developing the attributes [ 41 , 52 ]. We found that the attribute identification process was not sufficiently reported, which may have impacted the transparency and validity of some DCEs.…”

Section: Resultsmentioning

confidence: 99%

Patient Preferences in Targeted Pharmacotherapy for Cancers: A Systematic Review of Discrete Choice Experiments

Jiang

Ren

et al. 2022

PharmacoEconomics

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Background Targeted pharmacotherapy has been increasingly applied in cancer treatment due to its breakthroughs. However, the unmet needs of cancer patients are still significant, highlighting the urgency to investigate patient preferences. It is unclear how patients deliberate their choices between different aspects of targeted therapy, including cost, efficacy, and adverse events. Since discrete choice experiments (DCEs) have been widely applied to patient preference elicitation, we reviewed DCEs on targeted therapy for different cancers. We also synthesized evidence on the factors influencing patients' choices and their willingness-to-pay (WTP) for survival when treated by targeted therapy. Methods We searched databases, including PubMed, EMBASE and MEDLINE, up to August 16, 2022, supplemented by a reference screening. The attributes from the selected studies were categorized into three groups: outcomes, costs, and process. We also calculated the relative importance of attributes and WTP for survival whenever possible. The purpose, respondents, explanation, findings, significance (PREFS) checklist was used to evaluate the quality of the included DCE studies. Results The review identified 34 eligible studies from 13 countries covering 14 cancers, such as breast, ovarian, kidney, prostate, and skin cancers. It also reveals a rising trend of DCEs on this topic, as most studies were published after 2018. We found that patients placed higher weights on the outcome (e.g., overall survival) and cost attributes than on process attributes.

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Section: Resultsmentioning

confidence: 99%

Patient Preferences in Targeted Pharmacotherapy for Cancers: A Systematic Review of Discrete Choice Experiments

Jiang

Ren

et al. 2022

PharmacoEconomics

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“…The availability of newer treatment options has facilitated personalized management strategies to optimize outcomes but also has led to an increasingly complex treatment pathway (11,12). As a result, physicians need to balance efficacy and safety with assessment of patients' needs and related considerations, such as those associated with treatment administration procedures, when considering therapeutic options (13).…”

Section: Introductionmentioning

confidence: 99%

Treatment preferences of patients with relapsed or refractory multiple myeloma in the United States, United Kingdom, Italy, Germany, France, and Spain: results from a discrete choice experiment

Thomas,

Ailawadhi,

Popat

et al. 2023

Front. Med.

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IntroductionNewer treatment options for relapsed/refractory multiple myeloma (RRMM) with efficacy and safety profiles that differ from traditional therapies have facilitated personalized management strategies to optimize patient outcomes. In the context of such personalized management, understanding how treatment characteristics influence patients’ preferences is essential. This study assessed patients’ preferences for RRMM treatment attributes and determined trade-offs between potential benefits, administration procedures, and adverse effects.MethodsPatients’ preferences were evaluated using a discrete choice experiment (DCE). Patients with RRMM who reported failing two lines of anti-myeloma treatment (immunomodulatory agent and a proteasome inhibitor [PI]) or ≥ 3 lines (including ≥1 PI, immunomodulatory agent, or anti-CD38 monoclonal antibody), were recruited across the US, UK, Italy, Germany, France, and Spain. DCE attributes and levels were identified using a targeted literature review, a review of clinical data for relevant RRMM treatments, qualitative patient interviews, and input from clinical and myeloma patient experts. The DCE was administered within an online survey from February–June 2022. Preference data were analyzed using an error-component logit model and willingness to make trade-offs for potential benefits, and relative attribute importance scores were calculated.ResultsOverall, 296 patients from the US (n = 100), UK (n = 49), Italy (n = 45), Germany (n = 43), France (n = 39), and Spain (n = 20) participated in the DCE. Mean (standard deviation) age was 63.8 (8.0) years, 84% had a caregiver, and patients had a median of 3 (range: 2–8) prior lines of therapy. Efficacy attributes most influenced patients’ preferences, with increasing overall response rate (25–85%) and overall survival (6 months to 2 years) contributing to ~50% of treatment decision-making. Administration procedures were also considered important to patients. Avoiding individual side effects was considered relatively less important, with patients willing to tolerate increases in side effects for gains in efficacy. Patient characteristics such as rate of disease progression, sociodemographics, or clinical characteristics also influenced treatment preferences.ConclusionPatients with RRMM were willing to tolerate increased risk of side effects for higher efficacy. Preferences and risk tolerance varied between patients, with preference patterns differing by certain patient characteristics. This highlights the importance of shared decision-making for optimal treatment selection and patient outcomes.

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A patient survey indicates quality of life and progression-free survival as equally important outcome measures in multiple myeloma clinical trials

Fleischer,

Zapf,

Allgaier

et al. 2023

J Cancer Res Clin Oncol

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Preferences and Priorities for Relapsed Multiple Myeloma Treatments Among Patients and Caregivers in the United States

Cited by 5 publications

References 25 publications

Patient Preferences in Targeted Pharmacotherapy for Cancers: A Systematic Review of Discrete Choice Experiments

Patient Preferences in Targeted Pharmacotherapy for Cancers: A Systematic Review of Discrete Choice Experiments

Treatment preferences of patients with relapsed or refractory multiple myeloma in the United States, United Kingdom, Italy, Germany, France, and Spain: results from a discrete choice experiment

A patient survey indicates quality of life and progression-free survival as equally important outcome measures in multiple myeloma clinical trials

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