The relationship between cost and the recommendation, refusal, and discontinuation of treatment for chronic myeloid leukemia and multiple myeloma in Japan: a cross-sectional exploratory survey

Figures & data

Table 1. Characteristics of treating physicians.

Physician characteristics	Treating patients with CML n = 105	Treating transplant-ineligible patients with MM n = 107	p Value^a
Specialty, n (%)
Hematologist	77 (73.3)	89 (83.1)	.082
Hematologic oncologist	17 (16.2)	18 (16.8)	.901
Oncologist	11 (10.4)	0 (0.0)	<.001
Age, n (%)
20–29 y	3 (2.9)	1 (0.9)	.304
30–39 y	42 (40.0)	27 (25.2)	.022
40–49 y	26 (24.8)	29 (27.1)	.697
50–59 y	26 (24.8)	37 (34.6)	.118
60–69 y	7 (6.7)	12 (11.2)	.246
≥70 y	1 (1.0)	1 (0.9)	.989
Certification, n (%)
Certified by JSH	78 (74.3)	96 (89.7)	.003
JSH member but not certified	22 (21.0)	11 (10.3)	.032
Not a JSH member	5 (4.8)	0 (0)	.022
Type of hospital, n (%)
General	26 (24.8)	38 (35.5)	.089
National/public	27 (25.7)	42 (39.3)	.035
University	52 (49.5)	27 (25.2)	<.001
Number of beds, n (%)
≤99	0 (0)	0 (0)	–
100–199	7 (6.7)	5 (4.7)	.530
200–299	4 (3.8)	6 (5.6)	.537
300–399	6 (5.7)	9 (8.4)	.444
400–499	13 (12.4)	17 (15.9)	.464
≥500	75 (71.4)	70 (65.4)	.347
Patients treated in the past year, mean (SD)
MM	–	19.3 (10.9)	–
Transplant eligible	–	4.7 (4.3)	–
Transplant ineligible	–	14.6 (8.9)	–
CML	8.0 (10.3)	–	–

Abbreviations. CML, chronic myelogenous leukemia; JSH, Japanese Society of Hematology; MM, multiple myeloma.

^ap values are based on a chi-square test of the difference between MM and CML for each category.

Table 2. Patients not recommended for or who refused, discontinued, reduced, or skipped treatment due to cost; by line of therapy (Base: patients considered for treatment).

% (95% CI)	Chronic myelogenous leukemia					Transplant-ineligible multiple myeloma
	Overall	1st Line	2nd Line	3rd Line	4th Line	Overall	1st Line	2nd Line	3rd Line	4th Line
Patients, n	1057	513	227	162	155	1708	843	400	279	186
Non-optimal regimen or  no regimen recommended	6.53 (5.94, 7.11)	6.43 (5.19, 7.67)	7.05 (5.34, 8.76)	7.41 (5.61, 9.21)	5.16 (4.40, 5.92)	1.41 (0.96, 1.85)	1.30 (0.79, 1.82)	2.00 (0.88, 3.12)	0.36 (–0.15, 0.87)	2.15 (–0.53, 4.83)
Patient refused optimal  regimen	1.51 (1.26, 1.77)	0.97 (0.63, 1.32)	1.32 (0.89, 1.74)	3.09^b (1.52, 4.66)	1.94 (1.39, 2.48)	0.35 (0.17, 0.53)	0.47 (0.17, 0.77)	0.25 (–0.16, 0.66)	0.36 (–0.15, 0.87)	0.00 (0.00, 0.00)
Patients (on treatment), n	863	428	180	135	120	1550	765	367	256	162
Patient discontinued  treatment	1.97 (1.67, 2.27)	2.60 (1.28, 3.86)	1.11 (0.56, 1.67)	0.74 (0.36, 1.12)	2.50 (1.74, 3.26)	0.71 (0.36, 1.05)	0.26 (0.07, 0.45)	0.27 (–0.02, 0.69)	1.95^a^,c (–0.72, 4.62)	1.85^d (–0.29, 3.99)
Patient reduced treatment	4.17 (3.64, 4.70)	3.27 (1.83, 4.71)	5.56 (4.13, 6.99)	4.44 (3.37, 5.51)	5.00 (4.05, 5.95)	1.10 (0.71, 1.48)	1.44 (0.65, 2.22)	0.54 (0.00, 1.11)	1.17 (–0.02, 2.36)	0.62 (–0.34, 1.58)
Patient skipped treatment	3.48 (3.02, 3.93)	3.27 (1.72, 4.82)	2.22 (1.27, 3.18)	4.44 (3.24, 5.64)	5.00 (3.83, 6.17)	0.52 (0.17, 0.85)	0.39 (0.09, 0.69)	1.36^b^,e (–0.04, 3.11)	0.00 (0.00, 0.00)	0.00 (0.00, 0.00)

^ap ≤ 0.05 relative to value for overall respondents.

^bp ≤ 0.10 relative to value for overall respondents.

^cp ≤ 0.001 relative to value for 1st line therapy.

^dp ≤ 0.05 relative to value for 1st line therapy.

^ep ≤ 0.10 relative to value for 1st line therapy.

Figure 1. Percent of physicians reporting by number of patients not recommended or that refused, discontinued, reduced, or skipped treatment due to treatment cost.

Table 3. Physician attitudes concerning treatment cost.

Physician attitude	Those treating patients with CML, % n = 105		Those treating transplant-ineligible patients with MM, % n = 107
	Agree/somewhat agree	Disagree/somewhat disagree	Agree/somewhat agree	Disagree/somewhat disagree
I always consider the balance between drug cost and efficacy when choosing a regimen for patients, including newly diagnosed cases	59.0	28.6	54.7	28.3
I consider the balance between drug cost and efficacy when choosing a regimen for later lines of treatment	46.7	43.8	53.8	19.8
I do not suggest/choose expensive regimens for patients who are unlikely to be able to afford their treatment	39.0	52.4	32.1	34.0
I proactively prescribe oral drugs that can be prescribed for long-term to help reduce the treatment cost burden on patients	53.3	40.0	30.2	30.2
I proactively reduce dose or skip treatment to reduce the treatment cost burden on patients	21.9	58.1	20.8	49.1
I prescribe drugs without thinking about the price margin	35.2	58.1	34.0	30.2
I prescribe drugs without thinking about the revenue of my department	48.6	43.8	34.0	34.0
I sometimes hesitate to prescribe expensive drugs based on their impact on the finance of medical insurance	57.1	36.2	48.1	31.1
Patients that are concerned about their cost burden when newly diagnosed are increasing	45.7	51.4	42.5	23.6
Patients that are concerned about their cost burden when choosing a regimen for later lines of treatment are increasing	46.7	50.5	45.3	17.9
More newly diagnosed patients vs those on later lines of treatment consult about dose reduction or skipping treatments because of the treatment cost burden	27.6	61.9	24.5	35.8
More patients on later lines of treatment vs those newly diagnosed consult about dose reduction or treatment suspension because of the treatment cost burden	31.4	59.0	28.3	30.2

Abbreviations. CML, chronic myelogenous leukemia; MM, multiple myeloma.

Table 4. Characteristics of patients not recommended treatment or who discontinued treatment owing to treatment cost as reported by physicians; by patient characteristics.

n (%)	All physicians N = 212	Physicians treating patients with CML n = 105	Physicians treating transplant-ineligible patients with MM n = 107
Age, y
≥65	36 (17.0)	23 (21.9)	13 (12.1)
≥75	102 (48.1)	36 (34.3)^b	66 (61.7)^b
Household composition
Lives with spouse and other family members/relatives	50 (23.6)	25 (23.8)	25 (23.4)
Lives with spouse only	6 (2.8)	3 (2.9)	3 (2.8)
Single, lives alone	60 (28.3)	28 (26.7)	32 (29.9)
Single mother/single father	10 (4.7)	6 (5.7)	4 (3.7)
Elderly-only household	67 (31.6)	22 (21.0)^b	45 (42.1)^c
Employment
Full-time	17 (8.0)	11 (10.5)	6 (5.6)
Part-time, contract/temporary	27 (12.7)	14 (13.3)	13 (12.1)
Self-employed	20 (9.4)	10 (9.5)	10 (9.3)
Unemployed	63 (29.7)	22 (21.0)^c	41 (38.8)
Pensioner	65 (30.7)	23 (21.9)^c	42 (39.3)
Welfare recipient	18 (8.5)	7 (6.7)	11 (10.3)
Other social factors
Foreign nationality	21 (9.9)	12 (11.4)	9 (8.4)
LGBT	3 (1.4)	3 (2.9)	0 (0.0)
Symptoms
Has subjective symptoms	48 (22.6)	15 (14.3)	33 (30.8)
Does not have subjective symptoms	93 (43.9)	50 (47.6)^c	43 (40.2)
Efficacy of treatment
MR4 or better	30 (28.6)	30 (28.6)	–
MMR to < MR4	29 (27.6)	29 (27.6)	–
PCyR to < MMR	7 (6.7)	7 (6.7)	–
sCR, CR	13 (12.1)	–	13 (12.1)
VGPR	24 (22.4)	–	24 (22.4)
PR	38 (35.5)	–	38 (35.5)
Duration of treatment, y
<1	36 (17.0)	17 (16.2)	19 (17.8)
1–<3	67 (31.6)	24 (22.9)	43 (40.2)
3–<5	33 (15.6)	15 (14.3)	18 (16.8)
≥5	26 (12.3)	19 (18.1)	7 (6.5)
Travel time for hospital visit, h
<1	50 (23.6)	25 (23.8)	25 (23.4)
1 to <2	65 (30.7)	25 (23.8)	40 (37.4)
≥2	34 (16.0)	19 (18.1)	15 (14.0)
Internet use for gathering information on disease/treatment
Seem to use the Internet actively	14 (6.6)	12 (11.4)	2 (1.9)^c
Seem to use the Internet to some degree	34 (16.0)	19 (18.1)	15 (14.0)
Do not seem to use the Internet	66 (31.1)	23 (21.9)^c	43 (40.2)

Abbreviations. CML, chronic myelogenous leukemia; CR, complete response; LGBT, lesbian, gay, bisexual, transgender; MM, multiple myeloma; MMR, major molecular response; MR4, molecular response 4; PCyR, partial cytogenetic response; PR, partial response; sCR, stringent complete response; VGPR, very good partial response.

^ap ≤ 0.001 relative to value for all physicians.

^bp ≤ 0.05 relative to value for all physicians.

^cp ≤ 0.10 relative to value for all physicians.