Economic burden of relapsed or refractory multiple myeloma: Results from an international trial

Robinson, Don; Orłowski, Robert Z.; Stokes, Michael B.; He, Jianming; Huse, Samuel; Chitnis, A.S.; Kranenburg, Britte; Lam, Annette

doi:10.1111/ejh.12876

Cited by 15 publications

(8 citation statements)

References 31 publications

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“…Real-world considerations such as the tolerability, convenience, and practicality of therapy are not captured within clinical trial reports or using conventional endpoints; however, they may also contribute to discrepancies between clinical trial and real-world outcomes. For example, treatment with a novel agent or regimen in the real-world setting may be associated with substantial patient time and economic burden 91 , 92 , including direct and indirect costs associated with medications and attending regular clinic visits. The burden of treatment, such as the route of administration and travel to the treatment center 10 , 93 , may not be captured by standard clinical trial endpoints but may substantially affect the feasibility of long-term treatment in the real-world setting as patients may succumb to treatment fatigue outside of the ‘motivating’ environment of clinical trial participation.…”

Section: Understanding Disparities Between Different Clinical Trials mentioning

confidence: 99%

Interpreting clinical trial data in multiple myeloma: translating findings to the real-world setting

Richardson

Miguel

Moreau³

et al. 2018

Blood Cancer Journal

197

174

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Substantial improvements in survival have been seen in multiple myeloma (MM) over recent years, associated with the introduction and widespread use of multiple novel agents and regimens, as well as the emerging treatment paradigm of continuous or long-term therapy. However, these therapies and approaches may have limitations in the community setting, associated with toxicity burden, patient burden, and other factors including cost. Consequently, despite improvements in efficacy in the rigorously controlled clinical trials setting, the same results are not always achieved in real-world practice. Furthermore, the large number of different treatment options and regimens under investigation in various MM settings precludes the feasibility of obtaining head-to-head clinical trial data, and there is a temptation to use cross-trial comparisons to evaluate data across regimens. However, multiple aspects, including patient-related, disease-related, and treatment-related factors, can influence clinical trial outcomes and lead to differences between studies that may confound direct comparisons between data. In this review, we explore the various factors requiring attention when evaluating clinical trial data across available agents/regimens, as well as other considerations that may impact the translation of these findings into everyday MM management. We also investigate discrepancies between clinical trial efficacy and real-world effectiveness through a literature review of non-clinical trial data in relapsed/refractory MM on novel agent−based regimens and evaluate these data in the context of phase 3 trial results for recently approved and commonly used regimens. We thereby demonstrate the complexity of interpreting data across clinical studies in MM, as well as between clinical studies and routine-care analyses, with the aim to help clinicians consider all the necessary issues when tailoring individual patients’ treatment approaches.

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Section: Understanding Disparities Between Different Clinical Trials mentioning

confidence: 99%

Interpreting clinical trial data in multiple myeloma: translating findings to the real-world setting

Richardson

Miguel

Moreau³

et al. 2018

Blood Cancer Journal

197

174

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Section: Introductionmentioning

confidence: 99%

“…MM is also related to increased healthcare utilisation and high healthcare costs [5,6]. A recent study found that 42% of MM patients reported at least one disease-driven hospital visit during the previous six months, 71% declared at least one specialty visit during the three months prior, and 9% received emergency care in the previous three months [6]. Healthcare costs per MM patient have increased steadily between 2000 and 2014 for all healthcare services including treatment-related drug costs, the main contributor being outpatient visits [7].…”

Section: Introductionmentioning

confidence: 99%

Unplanned admissions for patients with myeloma in the UK: Low frequency but high costs

Kolovos

Nador

Kishore

et al. 2019

Journal of Bone Oncology

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Background Multiple myeloma (MM) is associated with high healthcare resource utilisation and increasing hospitalisation rates. The aim of this study was to characterise the hospital use by patients with MM in the English National Health Service (NHS). Methods Routinely-collected aggregate data about all NHS-funded hospital admissions of patients with MM were analysed. Data were obtained from the English Hospital Episodes Statistics on admissions between 1 April 2014 and 31 March 2018. Results A total of 754,345 admissions were reported over four years, equivalent to a mean of 188,586 admissions per year. Of the 41,845 patients admitted during this period, 42% were women and 58% men. From the total admissions, 90% were elective and 10% unplanned. Mean annual estimated costs over the period were £46 million for elective and £56 million for unplanned admissions. The number of elective admissions increased by 4.5% with costs increasing 1.5% per year; for unplanned admissions, these figures were 4.1% and 9.0%, respectively. Conclusions MM is associated with a significant number of hospital admissions and NHS costs. The majority of the hospital admissions are elective, but the highest burden in terms of costs relates to unplanned admissions, with numbers increasing over time.

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“…Específicamente, el 94,4% del coste total se asocia a costes directos derivados de servicios ambulatorios (49,1%), hospitalizaciones (32,7%) y prescripciones complementarias (18,2%) 28 . Dicha estimación se llevó a cabo en base a las tendencias en supervivencia y costes asociados al MM en Estados Unidos 9,29 . En consecuencia, estos resultados deben contemplarse con precaución por la dificultad de extrapolar dicha tendencia a otros países con sistemas sanitarios diferentes.…”

Section: Carga Económicaunclassified

Impacto clínico, asistencial, económico y social del abordaje ideal del Mieloma Múltiple. Proyecto SROI-MM

Merino¹,

Ivanova²,

Maravilla³

et al. 2019

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Resumen ejecutivo __________________________________________________ 3.1 Justificación y objetivos ________________________________________________ 3.2 Metodología ________________________________________________________ 3.3 Resultados __________________________________________________________ 3.4 Conclusiones ________________________________________________________ 4 Introducción al mieloma múltiple _____________________________________ 4.1 Descripción _________________________________________________________ 4.1.1 Definición ____________________________________________________________ 4.1.2 Diagnóstico ___________________________________________________________ 4.1.3 Pronóstico ___________________________________________________________ 4.1.4 Tratamiento __________________________________________________________ 4.2 Epidemiología _______________________________________________________ 4.3 Calidad de vida relacionada con la salud __________________________________ 4.4 Carga económica _____________________________________________________ 4.5 Retos del futuro y necesidades no cubiertas ______________________________ 5 Justificación ______________________________________________________ 6 Objetivos ________________________________________________________ 7 Metodología ______________________________________________________ 7.1 Reuniones de trabajo con expertos ______________________________________ 7.1.1 Participantes y funciones ________________________________________________ 7.1.1.1 Comité Asesor _________________________________________________ 7.1.1.2 Grupo Multidisciplinar de Trabajo _________________________________ 7.1.2 Dinámica de trabajo ____________________________________________________ 7.1.2.1 Primera reunión con Comité Asesor _______________________________ 7.1.2.2 Segunda reunión con Grupo Multidisciplinar de Trabajo _______________ 7.2 SROI: Retorno Social de la Inversión _____________________________________ 7.2.1 Inicios del método SROI _________________________________________________ 7.2.2 Introducción al método SROI _____________________________________________ 7.3 Otros aspectos metodológicos __________________________________________ 8 Propuestas para la mejora del abordaje del MM __________________________ 9 Retorno Social de la Inversión de las propuestas para la mejora del abordaje actual del MM ________________________________________________________ 9.1 Grupos de interés ____________________________________________________ INFORME FINAL 6 CLÍNICO, ASISTENCIAL ECONÓMICO Y SOCIAL DEL ABORDAJE IDEAL DEL MIELOMA MÚLTIPLE PROYECTO SROI-MM IMPACTO 9.2 Marco general de la inversión y el retorno ________________________________ 9.3 Propuestas del área de Diagnóstico______________________________________ 9.3.1 Propuesta 1. Información escrita sobre la enfermedad y su tratamiento (en caso de necesitarlo), dirigida a pacientes y familiares en el momento del diagnóstico ____ 9.3.2 Propuesta 2. Creación de equipos multidisciplinares con todas las especialidades implicadas, incluyendo atención primar...

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Economic burden of relapsed or refractory multiple myeloma: Results from an international trial

Abstract: Substantial RRMM indirect, social costs were observed. Better major tumor response may reduce hospital visits.

Cited by 15 publications

References 31 publications

Interpreting clinical trial data in multiple myeloma: translating findings to the real-world setting

Interpreting clinical trial data in multiple myeloma: translating findings to the real-world setting

Unplanned admissions for patients with myeloma in the UK: Low frequency but high costs

Impacto clínico, asistencial, económico y social del abordaje ideal del Mieloma Múltiple. Proyecto SROI-MM

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