Evaluating process utilities for the treatment burden of chemotherapy in multiple myeloma in Japan: a time trade-off valuation study

Figures & data

Table 1. Vignettes for treatment schedule.

Name of treatment	Descriptive contents of treatment schedules
No treatment	There is no treatment- or hospital-visit-related burden.
Ide-cel	After a single session of treatment by drip infusion, monthly hospital visits are necessary. You take oral drugs every day to relieve or prevent symptoms or complications. During a monthly hospital visit, follow-up examinations are performed for about 3 h.
DRd therapy	During a 4-week period, patients receive drug administration by drip infusion on 4 hospital visits. They take an oral drug every day for 21 days and another one on Day 2 of every week. They also take oral drugs every day to relieve or prevent symptoms or complications. The above 4-week schedule is repeated twice. During the next 4 weeks, you receive drug administration by drip infusion on 2 hospital visits. You take an oral drug every day for 21 days and another one for 6 days. You also take oral drugs every day to relieve or prevent symptoms or complications. The above 4-week schedule is repeated 4 times. During the subsequent 4 weeks, you receive drug administration by drip infusion on a single hospital visit. You take an oral drug every day for 21 days and another one for 7 days. You also take oral drugs every day to relieve or prevent symptoms or complications. The above 4-week schedule is repeated all the time. On every hospital visit, drip infusion and follow-up examinations are performed for about 3–4 h.^a
IsaPd therapy	During the first 4 weeks, you receive drug administration by drip infusion on 4 weekly hospital visits. You take an oral drug every day for 21 days and another one on Day 1 of every week. You also take oral drugs every day to relieve or prevent symptoms or complications. During the subsequent 4 weeks, you receive drug administration by drip infusion on two hospital visits. You take an oral drug every day for 21 days and another one on Day 1 of every week. You also take oral drugs every day to relieve or prevent symptoms or complications. The above 4-week schedule is repeated indefinitely. On every hospital visit, drip infusion and follow-up examinations are performed for about 3 to 4 h.
DKd therapy	During a 4-week period, you receive drug administration by drip infusion on 7 hospital visits. You take an oral drug for 7 days. You also take oral drugs every day to relieve or prevent symptoms or complications. The above 4-week schedule is repeated twice. During the subsequent 4 weeks, you receive drug administration by drip infusion on 6 hospital visits. You take an oral drug for 7 days. You also take oral drugs every day to relieve or prevent symptoms or complications. The above 4-week schedule is repeated indefinitely. On every hospital visit, drip infusion and follow-up examinations are performed for about 3–4 h.^a
Kd therapy	During a 3-week period, you receive drug administration by drip infusion on 6 hospital visits. You take an oral drug on Days 1 and 2 of every week. You also take oral drugs every day to relieve or prevent symptoms or complications. The above 4-week schedule is repeated indefinitely. On every hospital visit, drip infusion and follow-up examinations are performed for about 3–4 h.
EPd therapy	During a 4-week period, you receive drug administration by drip infusion on 4 hospital visits. You take an oral drug on Day 1 of every week and another one every day for 21 days. You also take oral drugs every day to relieve or prevent symptoms or complications. The above 4-week schedule is repeated twice. During the subsequent 4 weeks, you receive drug administration by drip infusion on two hospital visits.^b You take an oral drug on Day 1 of every week and another one every day for 21 days. You also take oral drugs every day to relieve or prevent symptoms or complications. The above 4-week schedule is repeated indefinitely. On every hospital visit, drip infusion and follow-up examinations are performed for about 3–4 h.
Pd therapy	You take an oral drug every day for 21 days, and discontinue it for 7 days. You also take another one on Day 1 of every week, as well as oral drugs every day to relieve or prevent symptoms or complications. The above 4-week schedule is repeated all the time. During a monthly hospital visit, follow-up examinations are performed for about 3 h.

Abbreviations. DKd, daratumumab, carfilzomib, and dexamethasone; DRd, daratumumab, lenalidomide, and dexamethasone; EPd, elotuzumab, pomalidomide, and dexamethasone; Ide-cel, idecabtagene vicleucel; IsaPd, isatuximab, pomalidomide, and dexamethasone; Kd, carfilzomib and dexamethasone; Pd, pomalidomide and dexamethasone.

^aIn practice, drip infusion alone takes about 5 h, but the time required for hospital visit was standardized to 3–4 h for all treatment.

^bIn practice, the number of hospital visits is 1, but the survey was conducted with the description of two visits.

Table 2. Vignettes for disease, symptoms, and influence on daily activity.

Item	Descriptive contents
Disease	You have a life-threatening disease related to a chronic disease. If you do not receive treatment, the disease may worsen. However, the number of treatment options for cure is limited.
Symptoms	There is no fatigue, or, even if it is present, its degree may be mild. Pain of the backbone or breastbone is present, sometimes requiring analgesic drugs. Appetite is slightly reduced. Numbness of the legs is sometimes noted.
Influence on daily activity	Due to symptoms of the disease or knowledge about the disease,  – Anxiety or depression is noted.  – You can work, but the kind of work is restricted to light work. As infectious diseases or fracture may occur, many everyday activities, such as housekeeping, exercise, and social activities with other persons, are restricted.

Table 3. Social background information.

Characteristics^a	Respondents (n = 319)	General population^b
Age, mean (SD), years	44.0 (13.57)	46.4
Residence
Saitama Prefecture	32 (10.0%)
Chiba Prefecture	38 (11.9%)
Tokyo	208 (65.2%)
Kanagawa Prefecture	41 (12.9%)
Prefectures other than the ones above	0 (0.0%)
Other than Japan	0 (0.0%)
Female	160 (50.2%)	(51.3%)
Final educational background
Completion of graduate school	7 (2.2%)	(2.2%)
Graduation from university (bachelor’s degree)	158 (49.5%)	(22.1%)
Graduation from college (associate’s degree)	38 (11.9%)	(7.8%)
Graduation from technical school	48 (15.0%)	(12.7%)
Graduation from senior high school	67 (21.0%)	(39.7%)
Graduation from junior high school	0 (0.0%)	(14.8%)
Others	1 (0.3%)
Employment status
Working
Regular staff/employees	163 (51.1%)	(31.6%)
Part-time staff/employees	34 (10.7%)	(9.4%)
Part-time workers	9 (2.8%)	(4.3%)
Temporary workers from worker dispatching offices	8 (2.5%)	(1.3%)
Contract employees	16 (5.0%)	(2.7%)
Commission	2 (0.6%)	(1.1%)
Company executives	3 (0.9%)	(3.0%)
Self-employed workers (employees: present)	4 (1.3%)	(1.1%)
Self-employed workers (employees: absent)	17 (5.3%)	(3.6%)
Helping with the family business	0 (0.0%)	(1.3%)
Side job	0 (0.0%)	(0.1%)
Others	3 (0.9%)	(0.8%)
Not working
Looking for a job	2 (0.6%)	(1.5%)
Attending school	17 (5.3%)	(5.4%)
Housekeeping	41 (12.9%)	(12.0%)
Others (e.g. retired)	0 (0.0%)	(20.5%)
Annual household income, JPY
<2,000,000	8 (2.5%)	(19.0%)
2,000,000 to <3,000,000	21 (6.6%)	(13.6%)
3,000,000 to <4,000,000	46 (14.4%)	(12.8%)
4,000,000 to <5,000,000	33 (10.3%)	(10.5%)
5,000,000 to <6,000,000	42 (13.2%)	(8.7%)
6,000,000 to <7,000,000	36 (11.3%)	(8.0%)
7,000,000 to <8,000,000	24 (7.5%)	(6.2%)
8,000,000 to <9,000,000	21 (6.6%)	(4.9%)
9,000,000 to <10,000,000	21 (6.6%)	(4.1%)
≥10,000,000	56 (17.6%)	(12.1%)
I don’t want to answer/don’t know.	11 (3.4%)

Abbreviations. JPY: Japanese yen; SD. standard deviation.

Percentages may not add up to 100% owing to rounding.

^aData are n (%) unless otherwise indicated.

^bSources^18–20.

Table 4. Clinical background information.

Characteristic^a	Respondents (n = 319)	General population^b
Status of hospital visit
Not consulting the hospital	224 (70.2%)	(59.6%)
I was previously diagnosed with the following diseases
Multiple myeloma	0 (0.0%)
Hematologic cancer	0 (0.0%)
I have not been diagnosed with these diseases	319 (100.0%)
I don’t know	0 (0.0%)
I know persons diagnosed with the following diseases
Multiple myeloma	5 (1.6%)
Hematologic cancer	40 (12.5%)
I don’t know any person diagnosed with these diseases	269 (84.3%)
I don’t know	7 (2.2%)
I have received chemotherapy
Yes	1 (0.3%)
No	318 (99.7%)
I don’t know	0 (0.0%)
I know persons who have received chemotherapy
Yes	167 (52.4%)
No	143 (44.8%)
I don’t know	9 (2.8%)
EQ-5D-5L index score, mean (SD)	0.97 (0.07)	0.91
EQ-5D-5L index score, ceiling	247 (77.4%)	(57.3%)
VAS, mean (SD)	86.1 (10.9)	77.5

Abbreviations. COPD, chronic obstructive pulmonary disease; SD, standard deviation; VAS, visual analog scale.

Percentages may not add up to 100% owing to rounding.

^aData are n (%) unless otherwise indicated.

^bSource²⁰.

Table 5. TTO utility values.

Health state^a	Mean	SD	Median	Range	95% CI	Floor, n (%)	Ceiling, n (%)
No treatment	0.758	0.336	0.900	−0.950–1.000	0.721–0.795	5 (1.6%)	118 (37.0%)
Ide-cel	0.729	0.343	0.850	−0.950–1.000	0.692–0.767	5 (1.6%)	100 (31.3%)
DRd therapy	0.501	0.461	0.600	−0.950–0.950	0.450–0.552	14 (4.4%)	38 (11.9%)
IsaPd therapy	0.553	0.428	0.700	−0.950–0.950	0.506–0.601	9 (2.8%)	46 (14.4%)
DKd therapy	0.507	0.446	0.600	−0.950–0.950	0.458–0.556	13 (4.1%)	38 (11.9%)
Kd therapy	0.552	0.428	0.700	−0.950–0.950	0.505–0.599	12 (3.8%)	39 (12.2%)
EPd therapy	0.547	0.425	0.700	−0.950–0.950	0.501–0.594	10 (3.1%)	45 (14.1%)
Pd therapy	0.722	0.350	0.850	−0.950–0.950	0.684–0.761	6 (1.9%)	85 (26.6%)

Abbreviations. CI, confidence interval; DKd, daratumumab, carfilzomib, and dexamethasone; DRd, daratumumab, lenalidomide, and dexamethasone; EPd, elotuzumab, pomalidomide, and dexamethasone; Ide-cel, idecabtagene vicleucel; IsaPd, isatuximab, pomalidomide, and dexamethasone; Kd, carfilzomib and dexamethasone; Pd, pomalidomide and dexamethasone; SD, standard deviation; TTO, time trade-off.

^aNumber of samples = 319.

Table 6. Comparison of TTO utility values.

Health state^a	Mean	SD	Median	Range	95% CI	p-Value^b
No treatment—Ide-cel	0.028	0.204	0.000	−1.400–0.950	0.006–0.051	.014
No treatment—DRd therapy	0.257	0.336	0.200	−0.400–1.950	0.220–0.294	<.001
No treatment—IsaPd therapy	0.204	0.291	0.100	−0.500–1.650	0.172–0.236	<.001
No treatment—DKd therapy	0.251	0.323	0.200	−0.500–1.900	0.215–0.286	<.001
No treatment—Kd therapy	0.206	0.285	0.100	−0.450–1.900	0.174–0.237	<.001
No treatment—EPd therapy	0.210	0.290	0.150	−0.500–1.650	0.178–0.242	<.001
No treatment—Pd therapy	0.036	0.247	0.000	−1.500–1.650	0.008–0.063	.010
Ide-cel—DRd therapy	0.229	0.310	0.150	−0.900–1.650	0.194–0.263	<.001
Ide-cel—IsaPd therapy	0.176	0.282	0.100	−0.900–1.650	0.145–0.207	<.001
Ide-cel—DKd therapy	0.223	0.304	0.150	−0.900–1.900	0.189–0.256	<.001
Ide-cel—Kd therapy	0.177	0.266	0.100	−0.900–1.700	0.148–0.207	<.001
Ide-cel—EPd therapy	0.182	0.267	0.100	−0.900–1.650	0.153–0.211	<.001
Ide-cel—Pd therapy	0.007	0.213	0.000	−1.100–1.650	−0.016–0.031	.537

Abbreviations. CI, confidence interval; DKd, daratumumab, carfilzomib, and dexamethasone; DRd, daratumumab, lenalidomide, and dexamethasone; EPd, elotuzumab, pomalidomide, and dexamethasone; Ide-cel, idecabtagene vicleucel; IsaPd, isatuximab, pomalidomide, and dexamethasone; Kd, carfilzomib and dexamethasone; Pd, pomalidomide and dexamethasone; SD, standard deviation; TTO, time trade-off.

^aNumber of samples = 319.

^bPaired t-test.

Household Statistics Office. Summary report of comprehensive survey of living conditions [Internet]; 2019 [cited 2022 Aug 30]. Available from: https://www.mhlw.go.jp/english/database/db-hss/dl/report_gaikyo_2019.pdf

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