Intended for healthcare professionals

Rapid response to:

Feature Drug Regulation

What makes an orphan drug?

BMJ 2010; 341 doi: https://doi.org/10.1136/bmj.c6459 (Published 17 November 2010) Cite this as: BMJ 2010;341:c6459

Rapid Response:

Cost consequence of Colcrys is not that astounding

Hawkes and Cohen cited that more than 50-fold price increase (from $0.09 to $4.85 per pill) of colchicine (Colcrys, URL Pharma) in the US would add an estimated $50 million per year to Medicaid.1 2 To investigate the real cost consequence, we conducted a time-series analysis using the Medicaid State Drug Utilization Data.3

Results (further details on bmj.com) show that the average reimbursement for non-URL colchicine increased from $0.27 (all in 2009 dollars) per unit in 2000-2009 to $0.35 in the first quarter of 2010, while the URL's rose from $0.29 in 2000-2008 to $3.79 in 2009 and 2010 (figure). The average price (excluding co-pay) of non-URL colchicine increased 30%, and that of URL's were raised by a factor of approximately 12. This would add an extra $21 million per year to the state Medicaid programs if all patients on the single-ingredient colchicine treatment in 2007 switched to Colcrys at the monopoly price of $4.85 per pill as reported.1 2

Despite the fewer incremental costs and the US Food and Drug Administration's safety concern about the unapproved colchicine,2 4 the higher price still imposes a heavy financial burden on patients and/or their insurers. It seems to be a "side effect" of the orphan drug legislation. Changes and solutions are required. Currently, the patient assistance initiatives implemented by the manufacturer may be helpful to some extent.4 More importantly, other manufacturers should seek generic approval as soon as possible in order to stimulate competition and benefit patients.

Yan-Jun Zhang PhD student

yjzhang.yjz@gmail.com

Jeff J Guo associate professor, College of Pharmacy, University of Cincinnati Academic Health Centre, Cincinnati, OH 45267-0004, USA

Christina M L Kelton professor, College of Business, University of Cincinnati, Cincinnati, OH 45221, USA

Competing interests: None declared.

1 Hawkes N, Cohen D. What makes an orphan drug? BMJ 2010;341:c6459. (16 November.)

2 Kesselheim AS, Solomon DH. Incentives for drug development--the curious case of colchicine. N Engl J Med 2010;362:2045-7.

3 US Centers for Medicare & Medicaid Services. State Drug Utilization Data. http://www.cms.gov/MedicaidDrugRebateProgram/SDUD/list.asp.

4 US Food and Drug Administration. FDA orders halt to marketing of unapproved single-ingredient oral colchicine. 30 September 2010. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm227796.htm.

Average reimbursement per unit/pill ($, adjusted to 2009 value) by US Medicaid for URL Pharma's and non-URL companies' colchicine, 2000-2010 first quarter

Total amount reimbursed ($ million, 2009 value) for URL and Non-URL colchicine by US Medicaid, 2000-2010 first quarter

No. of prescriptions (in thousands) reimbursed for URL and Non-URL colchicine by US Medicaid, 2000-2010 first quarter

No. of units (in millions) reimbursed for URL and non-URL colchicine by US Medicaid, 2000-2010 first quarter

Supplementary table:

Company	Year	Total Units (M)	Total Rx (000)	Total Reimbursement (US$M)	Units/Rx	$/Rx	$/Unit
Non-URL	2000	9.35 	202.492	2.27 	46.2 	11.19 	0.24 
Non-URL	2001	10.81 	239.993	2.69 	45.0 	11.19 	0.25 
Non-URL	2002	11.76 	266.852	2.84 	44.1 	10.64 	0.24 
Non-URL	2003	13.44 	307.155	3.13 	43.8 	10.18 	0.23 
Non-URL	2004	14.45 	332.926	3.28 	43.4 	9.84 	0.23 
Non-URL	2005	16.20 	376.622	3.65 	43.0 	9.70 	0.23 
Non-URL	2006	4.92 	110.316	1.09 	44.6 	9.90 	0.22 
Non-URL	2007	4.43 	100.14	1.01 	44.2 	10.08 	0.23 
Non-URL	2008	4.61 	104.548	1.09 	44.1 	10.42 	0.24 
Non-URL	2009	4.30 	99.67	1.06 	43.1 	10.64 	0.25 
Non-URL	2010	0.81 	19.204	0.29 	42.2 	15.00 	0.36 
URL	2000	0.71 	15.221	0.18 	46.3 	11.70 	0.25 
URL	2001	1.11 	23.718	0.27 	46.9 	11.50 	0.25 
URL	2002	1.47 	33.266	0.35 	44.3 	10.41 	0.23 
URL	2003	1.60 	36.245	0.35 	44.2 	9.74 	0.22 
URL	2004	1.72 	39.869	0.43 	43.2 	10.70 	0.25 
URL	2005	1.70 	40.016	0.46 	42.4 	11.62 	0.27 
URL	2006	0.34 	7.734	0.11 	43.8 	14.29 	0.33 
URL	2007	0.07 	1.522	0.02 	43.1 	13.30 	0.31 
URL	2008	0.01 	0.149	0.00 	40.2 	10.62 	0.26 
URL	2009	0.00 	0.031	0.01 	41.5 	169.83 	4.09 
URL	2010	0.00 	0.054	0.01 	42.0 	153.78 	3.66

Competing interests:
None declared

Competing interests: Company Year Total Units (M) Total Rx (000) Total Reimbursement (US$M) Units/Rx $/Rx $/UnitNon-URL 2000 9.35 202.492 2.27 46.2 11.19 0.24 Non-URL 2001 10.81 239.993 2.69 45.0 11.19 0.25 Non-URL 2002 11.76 266.852 2.84 44.1 10.64 0.24 Non-URL 2003 13.44 307.155 3.13 43.8 10.18 0.23 Non-URL 2004 14.45 332.926 3.28 43.4 9.84 0.23 Non-URL 2005 16.20 376.622 3.65 43.0 9.70 0.23 Non-URL 2006 4.92 110.316 1.09 44.6 9.90 0.22 Non-URL 2007 4.43 100.14 1.01 44.2 10.08 0.23 Non-URL 2008 4.61 104.548 1.09 44.1 10.42 0.24 Non-URL 2009 4.30 99.67 1.06 43.1 10.64 0.25 Non-URL 2010 0.81 19.204 0.29 42.2 15.00 0.36 URL 2000 0.71 15.221 0.18 46.3 11.70 0.25 URL 2001 1.11 23.718 0.27 46.9 11.50 0.25 URL 2002 1.47 33.266 0.35 44.3 10.41 0.23 URL 2003 1.60 36.245 0.35 44.2 9.74 0.22 URL 2004 1.72 39.869 0.43 43.2 10.70 0.25 URL 2005 1.70 40.016 0.46 42.4 11.62 0.27 URL 2006 0.34 7.734 0.11 43.8 14.29 0.33 URL 2007 0.07 1.522 0.02 43.1 13.30 0.31 URL 2008 0.01 0.149 0.00 40.2 10.62 0.26 URL 2009 0.00 0.031 0.01 41.5 169.83 4.09 URL 2010 0.00 0.054 0.01 42.0 153.78 3.66

03 January 2011
Zhang
PhD student
Jeff J. Guo and Christina M L Kelton
College of Pharmacy, University of Cincinnati Academic Health Centre