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Cost Effectiveness of Transplant, Conventional Chemotherapy, and Novel Agents in Multiple Myeloma: A Systematic Review

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Abstract

Background

Treatments for multiple myeloma (MM) have been rapidly evolving. Newly developed treatment regimens are likely to be more effective but also cost more than conventional therapies.

Objective

We conducted a systematic review to compare the cost effectiveness of different classes of MM treatment.

Methods

We searched the PubMed, MEDLINE, Web of Science, and EMBASE databases for studies published during 1990–2018 comparing the cost effectiveness of transplant, chemotherapeutic and novel MM treatments. Titles and abstracts were independently reviewed for eligibility by two investigators. The quality of the included studies was evaluated using the 16-item, validated Quality of Health Economics Studies instrument.

Results

Twenty-four publications were included in the systematic review and summarized according to treatment regimen and line. For first-line treatment, transplant was the most cost-effective option for transplant-eligible MM patients [the incremental cost-effectiveness ratio (ICER) was $4053–€45,460 per quality-adjusted life-year (QALY) gained, and $3848–$72,852 per life-year gained (LYG)], and the ICER for novel agents compared with conventional chemotherapy was $59,076 per QALY and $220,681 per LYG. For second-line treatment, in comparisons of novel agent-based regimens, ICERs were inconsistent. However, bortezomib-based regimens, lenalidomide plus dexamethasone, and pomalidomide plus dexamethasone were each cost effective compared with dexamethasone alone (ICERs showed cost saving, £30,153 per QALY gained, and €39,911 per LYG, respectively).

Conclusions

For transplant-eligible MM patients, transplant is a cost-effective first-line treatment. More cost-effectiveness analyses comparing novel agents in the first-line treatment regimen are warranted to determine which agent or regimen is the most cost effective. In the second-line setting, it is unclear which novel agent-based regimen is most cost effective, but bortezomib-based regimens, lenalidomide plus dexamethasone, and pomalidomide plus dexamethasone were each cost effective compared with dexamethasone alone.

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Acknowledgements

The authors acknowledge the assistance with the literature search provided by Greg Pratt in the Research Medical Library, and the editorial services provided by Sarah Bronson in Scientific Publications at The University of Texas MD Anderson Cancer Center.

Author information

Authors and Affiliations

  1. Division of Cancer Prevention and Population Sciences, Department of Health Services Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

    Shuangshuang Fu

  2. Division of Management, Policy, and Community Health, School of Public Health, The University of Texas Health Science Center at Houston, 1200 Pressler St., Houston, TX, 77030, USA

    Chi-Fang Wu & David R. Lairson

  3. Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

    Michael Wang

Authors
  1. Shuangshuang Fu
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  2. Chi-Fang Wu
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  3. Michael Wang
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  4. David R. Lairson
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Contributions

SF: study concept and design, data collection and interpretation, and manuscript drafting and revision. CFW: data collection and interpretation, and manuscript drafting and revision. MW: data interpretation, and manuscript drafting and revision. DRL: study concept and design, data interpretation, and manuscript drafting and revision.

Corresponding author

Correspondence to David R. Lairson.

Ethics declarations

Conflict of interest

Shuangshuang Fu, Chi-Fang Wu, Michael Wang and David R. Lairson have no conflicts of interest to declare.

Sources of funding

None.

Appendices

Appendix 1: Search strategy for the four databases

1. Search strategy for Medline

1

MULTIPLE MYELOMA/

2

multiple myeloma*.ti,kf,ab.

3

(plasma cell* adj3 myeloma*).ti,kf,ab.

4

Kahler* disease.ti,kf,ab.

5

or/1-4

6

limit 5 to english language

7

limit 6 to yr=“1990 -Current”

8

exp “COSTS AND COST ANALYSIS”/

9

ECONOMICS, PHARMACEUTICAL/

10

MODELS, ECONOMIC/

11

ECONOMICS, MEDICAL/

12

(cost*3 adj5 (benefit* or analy* or control* or measur* or averag* or estimat* or evaluat* or annual* or minimiz* or minimis* or minimali* or utilit* or effectiveness or containment)).ti,kf.

13

(cost*3 adj3 (benefit* or analy* or control* or measur* or averag* or estimat* or evaluat* or annual* or minimiz* or minimis* or minimali* or utilit* or effectiveness or containment)).ab. /freq=3

14

((cost*3 or economic* or pharmacoeconomic* or pharmaco-economic*) and myeloma*).ti.

15

((econom* or cost*3 or financ* or expenditure* or spend* or spent) adj5 (impact* or model* or evaluat* or analy* or burden)).ti,kf.

16

((econom* or cost*3 or financ* or expenditure* or spend* or spent) adj3 (impact* or model* or evaluat* or analy* or burden)).ab. /freq=2

17

QUALITY-ADJUSTED LIFE YEARS/

18

“quality adjusted life year*”.ti,kf,ab.

19

(ICER or QALY).ti,kf,ab.

20

((life adj year* adj2 gain*) or LYG).ti,kf,ab.

21

((“progression free life” adj2 year*) or PFLY).ti,kf,ab.

22

or/8-21

23

7 and 22

24

limit 23 to (editorial or letter)

25

 23 not 24

2. Search strategy for Embase

1

*MULTIPLE MYELOMA/

2

multiple myeloma*.ti,kw.

3

(plasma cell* adj3 myeloma*).ti,kw.

4

Kahler* disease.ti,kw.

5

(“multiple myeloma*” or “Kahler* disease” or (plasma cell* adj3 myeloma*)).ab. /freq=2

6

or/1-5

7

limit 6 to english language

8

limit 7 to yr=“1990 -Current”

9

exp ECONOMIC EVALUATION/

10

PHARMACOECONOMICS/

11

DRUG COST/

12

*HEALTH-CARE-COST/

13

*HEALTH-ECONOMICS/

14

(cost*3 adj5 (benefit* or analy* or control* or measur* or averag* or estimat* or evaluat* or annual* or minimiz* or minimis* or minimali* or utilit* or effectiveness or containment)).ti,kw.

15

(cost*3 adj3 (benefit* or analy* or control* or measur* or averag* or estimat* or evaluat* or annual* or minimiz* or minimis* or minimali* or utilit* or effectiveness or containment)).ab. /freq=2

16

((cost*3 or economic* or pharmacoeconomic* or pharmaco-economic*) and myeloma*).ti.

17

((econom* or cost*3 or financ* or expenditure* or spend* or spent) adj5 (impact* or model* or evaluat* or analy* or burden)).ti,kw.

18

((econom* or cost*3 or financ* or expenditure* or spend* or spent) adj3 (impact* or model* or evaluat* or analy* or burden)).ab. /freq=2

19

QUALITY-ADJUSTED LIFE YEAR/

20

“quality adjusted life year*”.ti,kw,ab.

21

(ICER or QALY).ti,kw,ab.

22

((life adj year* adj2 gain*) or LYG).ti,kw,ab.

23

((“progression free life” adj2 year*) or PFLY).ti,kw,ab.

24

or/9-23

25

8 and 24

26

limit 25 to (editorial or letter)

27

25 not 26

28

limit 27 to conference abstract

29

 27 not 28

3. Search strategy for PubMed excluding Medline

#11

#3 and #10

#10

#4 or #5 or #6 or #7 or #8 or #9

#9

“progression free life year”[tiab] OR “progression free life years”[tiab] OR PFLY[tiab]

#8

“life year gain”[tiab] OR “life year gains”[tiab] OR “life year gained”[tiab] OR LYG[tiab]

#7

“quality adjusted life year”[tiab] OR “quality adjusted life years”[tiab] or ICER[tiab] OR QALY[tiab]

#6

((cost[tiab] or costs[tiab] or economic*[tiab]) and myeloma*[ti] and (benefit*[tiab] or analys*[tiab] or analyz*[tiab] or control*[tiab] or measur*[tiab] or averag*[tiab] or estimat*[tiab] or evaluat*[tiab] or annual*[tiab] or minimiz*[tiab] or minimis*[tiab] or minimali*[tiab] or utilit*[tiab] or effectiveness[tiab] or containment[tiab]))

#5

((“cost effective”[tiab] or “cost benefit”[tiab] or “cost utility”[tiab] or “economic evaluation”[tiab] or cost[ti] or costs[ti] or economic*[ti] or pharmacoeconomic*[ti] or pharmaco-economic*[ti]) and myeloma*[ti])

#4

((econom*[tiab] or cost[tiab] or costs[tiab] or financ*[tiab] or expenditure*[tiab] or spend*[tiab] or spent[tiab]) and myeloma*[ti] and (impact*[tiab] or model*[tiab] or evaluat*[tiab] or analy*[tiab] or burden[tiab]))

#3

#1 and #2

#2

myeloma*

#1

publisher[sb]

4. Search strategy for Web of Science

# 15

(#13 not #14) ANDLANGUAGE: (English)

# 14

(#13) ANDLANGUAGE: (English) ANDDOCUMENT TYPES: (Meeting Abstract)

# 13

(#12 and py=1990-2018) ANDLANGUAGE: (English)

# 12

(#4 and #11) ANDLANGUAGE: (English)

# 11

(#5 or #6 or #7 or #8 or #9 or #10) ANDLANGUAGE: (English)

# 10

(ts=((“progression free life” NEAR/2 year*) or PFLY)) ANDLANGUAGE: (English)

# 9

(ts=((life adj year* NEAR/2 gain*) or LYG)) ANDLANGUAGE: (English)

# 8

(ts=(“quality adjusted life year” or “quality adjusted life years” or ICER or QALY)) ANDLANGUAGE: (English)

# 7

(ts=((econom* or cost* or financ* or expenditure* or spend* or spent) NEAR/5 (impact* or model* or evaluat* or analy* or burden))) ANDLANGUAGE: (English)

# 6

(ti=((cost* or economic* or pharmacoeconomic* or pharmaco-economic*) and myeloma*)) ANDLANGUAGE: (English)

# 5

(ts=(cost* NEAR/5 (benefit* or analy* or control* or measur* or averag* or estimat* or evaluat* or annual* or minimiz* or minimis* or minimali* or utilit* or effectiveness or containment))) ANDLANGUAGE: (English)

# 4

#2 not #3

# 3

(#2) ANDDOCUMENT TYPES: (Editorial Material OR Letter)

# 2

(#1) ANDLANGUAGE: (English)

# 1

ts=(multiple myeloma*)

Appendix 2: QHES assessment for each individual study

Study

Q1

Q2

Q3

Q4

Q5

Q6

Q7

Q8

Q9

Q10

Q11

Q12

Q13

Q14

Q15

Q16

Henon et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

0

Trippoli et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

0

Sampson et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

1

1

0

Kouroukis et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Mehta et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

0

Hornberger et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Moller et al.

1

0

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Brown et al.

1

0

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Corso et al.

1

0

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Fragoulakis et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Garrison et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Kim et al.

1

0

1

NA

0

1

0

1

1

1

1

0

1

0

1

1

Pandya et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

0

Shah et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

1

1

0

Blommestein et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Borg et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

0

Jakubowiak et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Usmani et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Chen et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Jakubowiak et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Pelligra et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Prinja et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

0

Carlson et al.

1

1

1

NA

1

1

1

1

1

1

1

1

1

0

1

1

Gueneau et al.

0

0

1

NA

1

1

1

1

1

1

1

1

1

0

1

0

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Fu, S., Wu, CF., Wang, M. et al. Cost Effectiveness of Transplant, Conventional Chemotherapy, and Novel Agents in Multiple Myeloma: A Systematic Review. PharmacoEconomics 37, 1421–1449 (2019). https://doi.org/10.1007/s40273-019-00828-y

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