Moore, David J., Dr., Adi, Y., Connock, M. and Bayliss, Sue (2009) Clinical effectiveness and cost-effectiveness of pegvisomant for the treatment of acromegaly: a systematic review and economic evaluation. BMC Endocrine Disorders, Vol.9 (Article 20). doi:10.1186/1472-6823-9-20

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Abstract

Background: Acromegaly, an orphan disease usually caused by a benign pituitary tumour, is characterised by hyper-secretion of growth hormone (GH) and insulin-like growth factor I (IGF-1). It is associated with reduced life expectancy, cardiovascular problems, a variety of insidiously progressing detrimental symptoms and metabolic malfunction. Treatments include surgery, radiotherapy and pharmacotherapy. Pegvisomant (PEG) is a genetically engineered GH analogue licensed as a third or fourth line option when other treatments have failed to normalise IGF-1 levels.
Methods: Evidence about effectiveness and cost-effectiveness of PEG was systematically reviewed. Data were extracted from published studies and used for a narrative synthesis of evidence. A decision analytical economic model was identified and modified to assess the cost-effectiveness of PEG.
Results: One RCT and 17 non-randomised studies were reviewed for effectiveness. PEG substantially reduced and rapidly normalised IGF-1 levels in the majority of patients, approximately doubled GH levels, and improved some of the signs and symptoms of the disease. Tumour size was unaffected at least in the short term. PEG had a generally safe adverse event profile but a few patients were withdrawn from treatment because of raised liver enzymes. An economic model was identified and adapted to estimate the lower limit for the cost-effectiveness of PEG treatment versus standard care. Over a 20 year time horizon the incremental cost-effectiveness ratio was £81,000/QALY and £212,000/LYG. To reduce this to £30K/QALY would require a reduction in drug cost by about one third.
Conclusion: PEG is highly effective for improving patients' IGF-1 level. Signs and symptoms of disease improve but evidence is lacking about long term effects on improved signs and symptoms of disease, quality of life, patient compliance and safety. Economic evaluation indicated that if current standards (UK) for determining cost-effectiveness of therapies were to be applied to PEG it would be considered not to represent good value for money.

Item Type:	Journal Article
Subjects:	R Medicine > RC Internal medicine R Medicine > RM Therapeutics. Pharmacology
Divisions:	Faculty of Medicine > Warwick Medical School > Health Sciences > Population, Evidence & Technologies (PET) > Warwick Evidence Faculty of Medicine > Warwick Medical School
Library of Congress Subject Headings (LCSH):	Acromegaly -- Treatment -- Great Britain, Somatotropin -- Synthesis, Pituitary gland -- Tumors, Drugs -- Effectiveness -- Great Britain, Drugs -- Prices -- Great Britain
Journal or Publication Title:	BMC Endocrine Disorders
Publisher:	BioMed Central Ltd.
ISSN:	1472-6823
Official Date:	8 October 2009
Dates:	Date Event 8 October 2009 Published
Volume:	Vol.9
Number:	Article 20
DOI:	10.1186/1472-6823-9-20
Status:	Peer Reviewed
Access rights to Published version:	Open Access
Funder:	West Midlands Primary Care Trusts' Levy (Great Britain) (WMPCTL), Pfizer Inc.

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