The cost-effectiveness of biopharmaceuticals

Figures & data

Figure 1 Growth of cost-utility literature, 1976–2009.

Table 1 Examples of published cost-utility analyses

Intervention	Cost-effectiveness ($US/QALY)*	Author
Treatment of multiple sclerosis with interferon β-1b	$500,00–$1,800,000	Parkin, et al.15
Methotrexate with infliximab in treating rheumatoid arthritis	$12,000	Wong, et al.16
Survival with epoetin-alfa in women with stage IV breast cancer	$17,000	Martin, et al.17
Ranibizumab for the treatment of subfoveal neovascular macular degeneration	$54,000–$130,000	Brown, et al.18
First line bevacizumab in combination with irrotecan and 5-FU/LV in treating metastatic colorectal cancer	$130,000–$180,000	Tappenden, et al.19
Omalizumab in patients with severe persistent allergic asthma	$43,000	Brown, et al.20
Abatacept plus oral disease modifying antirheumatic drugs in patients with moderate to severely active rheumatoid arthritis	$46,000–$51,000	Vera-Llonch, et al.21

* in 2008 USD.

Table 2 Distribution of interventions studied in cost-utility analyses, 1976–2009

Intervention*	N	%
Pharmaceuticals	1114	46.7
Surgical	339	14.2
Care delivery	296	12.4
Medical procedures	295	12.4
Screening	263	11.0
Biopharmaceuticals	258	10.8
Diagnostic	206	8.6
Medical devices	205	8.6
Health education	201	8.4
Immunizations	148	6.2
Other	120	5.0

* Because an article could be assigned to more than one intervention, categories are not mutually exclusive and the sum of percentages will exceed 100%. Source: Tufts CEA Registry, www.cearegistry.org.

Table 3 Characteristics of CUAs for biopharmaceuticals and conventional pharmaceuticals

	Biopharmaceuticals n = 258 (%)	Conventional pharmaceuticals n = 845 (%)	All interventions
Country
United States	79 (30.6)	358 (42.4)	1049 (44.0)
United Kingdom	62 (24.0)	155 (18.3)	428 (18.0)
Canada	19 (7.4)	65 (7.7)	165 (6.9)
Netherlands	6 (2.3)	35 (4.1)	147 (6.2)
Sweden	20 (7.8)	42 (5.0)	97 (4.1)
Other/Multinational	72 (27.9)	190 (22.5)	497 (20.9)
Sponsor
Industry	133 (51.6)	392 (46.4)	679 (28.5)
Non-industry	64 (24.8)	294 (34.8)	1146 (48.1)
Non-disclosed	61 (23.6)	159 (18.8)	558 (23.4)
Prevention stage
Primary	2 (0.8)	77 (9.1)	389 (16.3)
Secondary	18 (7.0)	176 (20.8)	474 (19.9)
Tertiary	238 (92.2)	592 (70.0)	1520 (63.8)
Study theme
Public health	2 (0.8)	50 (5.9)	350 (14.7)
Men's health	1 (0.4)	30 (3.6)	62 (2.6)
Women's health	11 (4.3)	108 (12.3)	245 (10.3)
Elderly	5 (1.9)	28 (3.3)	99 (4.2)
Children	15 (5.8)	24 (2.8)	147 (6.2)
None/NS*	224 (86.8)	605 (71.6)	1480 (62.1)
Top ten journals of publication (%)
	Pharmacoeconomics (7.4)	Pharmacoeconomics (9.9)	Pharmacoeconomics (4.6)
	Value Health (6.2)	Value Health (5.2)	Value Health (3.4)
	Rheumatology (4.3)	Curr Med Res Opin (4.7)	Int J Technol Assess Health Care (2.9)
	Curr Med Res Opin (3.9)	Ann Intern Med (2.4)	Ann Intern Med (2.6)
	Int J Technol Assess Health Care (3.5)	Osteoporos Int (2.4)	Curr Med Res Opin (2.4)
	Aliment Pharmacol Ther (2.7)	Clin Ther (2.0)	Vaccine (2.0)
	Ann Intern Med (2.7)	Eur J Health Econ (1.7)	BMJ (1.8)
	Clin Ther (2.3)	Med Decis Making (1.5)	JAMA (1.5)
	J Clin Oncol (2.3)	Breast Cancer Res Treat (1.4)	Med Decis Making (1.5)
	Eur J Health Econ (1.9)	Cancer (1.3)	J Clin Oncol (1.1)
	Other (62.8)	Other (67.5)	Other (76.2)

* NS = Not specified

Table 4 Methodological quality of cost-utility literature

Methodological criteria	Biopharmaceuticals n = 258	Non-biopharmaceutical interventions n = 2125	p value
Stated study perspective	88.0%	81.9%	0.007
Costs and QALYs discounted	86.4%	84.7%	0.447
Stated time horizon	91.9%	87.8%	0.057
Stated year of currency	83.5%	83.1%	0.417
Correctly calculated ICERa	85.7%	73.1%	<0.001
Average quality scoreb	4.55	4.33	<0.001

Note:

a ICER = Incremental cost-effectiveness ratio ($/QALY);

b Based on the subjective reviewer rating.

Table 5 Distribution of weighted cost-utility ratios* by type of intervention

	No.	Median	Inter-quartile range	Cost-savinga (%)	Dominatedb (%)
All ratios	4608	$9,041	$2,439–$26,478	16.9	8.5
Biopharmaceuticals	538	$15,412	$3,2845–$45,287	15.2	5.6
Care delivery	493	$11,617	$3,239–$37,385	17.6	6.3
Conv. pharmaceuticals	1863	$7,094	$1,813–$23,323	18.6	9.6
Diagnostic	422	$8,846	$2,705–$29,775	13.7	19.0
Health ed. or behavior	362	$5,279	$1,662–$17,988	21.0	3.0
Immunizations	366	$8,852	$2,697–$21,931	18.0	4.1
Medical device	366	$14,236	$4,778–$48,761	23.2	8.2
Medical procedure	429	$13,877	$4,307–$44,995	21.0	17.2
Screening	617	$8,785	$2,763–$20,893	11.0	9.9
Surgical	574	$8,790	$1,937–$25,492	15.3	10.8

* Presented as $US/QALY (in 2008 USD)

a Interventions with improved health benefits and decreased costs

b Interventions with decreased health benefits and greater costs.

Table 6 Influence of factors on the favorability of biopharmaceutical cost-utility ratiosa

	Adjusted ORb	95% Confidence Interval	p value
Sponsorship
Industry	3.85	2.22–6.68	<0.001
Non-industry	1.58	0.89–2.18	0.118
Disease
Infectious disease	5.34	3.11–9.16	<0.001
Malignant neoplasms	0.50	0.26–0.96	0.037
Rheumatologic diseases	0.93	0.53–1.65	0.813
Endocrine disorders	1.07	0.56–2.04	0.832
Neurological conditions	0.39	0.17–0.89	0.025

Note: Reference categories are non-disclosed sponsorship and “other” diseases.

a Presented as $US/QALY (in 2008 USD)

b Adjusted Odds Ratio: Indicates how much a factor increases or decreases the probability the ratio of a biopharmaceutical is favorable (i.e., below the weighted median of all ratios included in the analysis$9,041 per QALY)) relative to the reference category. For example, ratios of biopharmaceuticals from CUAs sponsored by industry sources are 3.85 times more likely than CUAs that did not disclose the sponsorship to be below the overall weighted median.

Parkin D, Jacoby A, McNamee P, Miller P, Thomas S, Bates D. Treatment of multiple sclerosis with interferon beta: an appraisal of cost-effectiveness and quality of life. J Neurol Neurosurg Psychiatry 2000; 68:144 - 149; http://dx.doi.org/10.1136/jnnp.68.2.144; PMID: 10644777

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Wong JB, Singh G, Kavanaugh A. Estimating the cost-effectiveness of 54 weeks of infliximab for rheumatoid arthritis. Am J Med 2002; 113:400 - 408; http://dx.doi.org/10.1016/S0002-9343(02)01243-3; PMID: 12401535

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Martin SC, Gagnon DD, Zhang L, Bokemeyer C, Van Marwijk Kooy M, van Hout B. Cost-utility analysis of survival with epoetin-alfa versus placebo in stage IV breast cancer. Pharmacoeconomics 2003; 21:1153 - 1169; http://dx.doi.org/10.2165/00019053-200321160-00002; PMID: 14594437

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Brown MM, Brown GC, Brown HC, Peet J. A value-based medicine analysis of ranibizumab for the treatment of subfoveal neovascular macular degeneration. Ophthalmology 2008; 115:1039 - 1045

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Tappenden P, Jones R, Paisley S, Carroll C. The cost-effectiveness of bevacizumab in the first-line treatment of metastatic colorectal cancer in England and Wales. Eur J Cancer 2007; 43:2487 - 2494; http://dx.doi.org/10.1016/j.ejca.2007.08.017; PMID: 17910914

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Brown R, Turk F, Dale P, Bousquet J. Cost-effectiveness of omalizumab in patients with severe persistent allergic asthma. Allergy 2007; 62:149 - 153; http://dx.doi.org/10.1111/j.1398-9995.2006.01310.x; PMID: 17298423

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Vera-Llonch M, Massarotti E, Wolfe F, Shadick N, Westhovens R, Sofrygin O, et al. Cost-effectiveness of abatacept in patients with moderately to severely active rheumatoid arthritis and inadequate response to methotrexate. Rheumatology (Oxford) 2008; 47:535 - 541; http://dx.doi.org/10.1093/rheumatology/ken007; PMID: 18356179

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