Abstract
Purpose
Condition-specific preference-based measures can offer utility data where they would not otherwise be available or where generic measures may lack sensitivity, although they lack comparability across conditions. This study aimed to develop an algorithm for estimating utilities from the short bowel syndrome health-related quality of life scale (SBS-QoL™).
Methods
SBS-QoL™ items were selected based on factor and item performance analysis of a European SBS-QoL™ dataset and consultation with 3 SBS clinical experts. Six-dimension health states were developed using 8 SBS-QoL™ items (2 dimensions combined 2 SBS-QoL™ items). SBS health states were valued by a UK general population sample (N = 250) using the lead-time time trade-off method. Preference weights or ‘utility decrements’ for each severity level of each dimension were estimated by regression models and used to develop the scoring algorithm.
Results
Mean utilities for the SBS health states ranged from −0.46 (worst health state, very much affected on all dimensions) to 0.92 (best health state, not at all affected on all dimensions). The random effects model with maximum likelihood estimation regression had the best predictive ability and lowest root mean squared error and mean absolute error, and was used to develop the scoring algorithm.
Conclusions
The preference-weighted scoring algorithm for the SBS-QoL™ developed is able to estimate a wide range of utility values from patient-level SBS-QoL™ data. This allows estimation of SBS HRQL impact for the purpose of economic evaluation of SBS treatment benefits.
Similar content being viewed by others
References
O’Keefe, S. J. D., Buchman, A. L., Fishbein, T. M., Jeejeebhoy, K. N., Jeppesen, P. B., & Shaffer, J. (2006). Short bowel syndrome and intestinal failure: Consensus definitions and overview. Clinical Gastroenterology and Hepatology, 4, 6–10.
Buchman, A. L. (2004). The medical and surgical management of short bowel syndrome. Medscape General Medicine, 6, 12.
Nightingale, J., & Woodward, J. M. (2006). Guidelines for management of patients with a short bowel. Gut, 55, iv1–iv12.
Seetharam, P., & Rodrigues, G. (2011). Short bowel syndrome: A review of management options. Saudi Journal of Gastroenterology: Official Journal of the Saudi Gastroenterology Association, 17, 229.
Carlsson, E., Bosaeus, I., & Nordgren, S. (2003). Quality of life and concerns in patients with short bowel syndrome. Clinical Nutrition, 22, 445–452.
Winkler, M. F., Hagan, E., Wetle, T., Smith, C., Maillet, J. O., & Touger-Decker, R. (2010). An exploration of quality of life and the experience of living with home parenteral nutrition. JPEN. Journal of Parenteral and Enteral Nutrition, 34, 395–407.
Carlsson, E., Berglund, B., & Nordgren, S. (2001). Living with an ostomy and short bowel syndrome: Practical aspects and impact on daily life. Journal of Wound, Ostomy, & Continence Nursing, 28, 96–105.
Baxter, J. P., Fayers, P. M., & McKinlay, A. W. (2006). A review of the quality of life of adult patients treated with long-term parenteral nutrition. Clinical Nutrition, 25, 543–553.
Kalaitzakis, E., Carlsson, E., Josefsson, A., & Bosaeus, I. (2008). Quality of life in short-bowel syndrome: Impact of fatigue and gastrointestinal symptoms. Scandinavian Journal of Gastroenterology, 43, 1057–1065.
Brooks, R. (1996). EuroQol: The current state of play. Health Policy, 37, 53–72.
Dolan, P. (1997). Modeling valuations for EuroQol health states. Medical care, 35, 1095–1108.
Feeny, D., Furlong, W., Boyle, B., & Torrance, G. W. (1995). Multi-attribute health status classification systems: Health Utilities Index. Pharmacoeconomics, 7, 490–502.
Papaionnou, D., Brazier, J., & Parry, G. (2011). How valid and responsive are generic health status measures, such as the EQ-5D and SF-36, in schizophrenia? A systematic review. Value in Health, 14, 907–920.
Tosh, J., Brazier, J., Evans, P., & Longworth, L. (2012). A review of generic preference-based measures of health-related quality of life in visual disorders. Value in Health, 15, 118–127.
FDA. (2009). Guidance for industry patient-reported outcome measures: Use in medical product development to support labeling claims. Available at: http://www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf.
Lin, F., Longworth, L., & Pickard, S. (2012). Evaluation of content on EQ-5D as compared to disease-specific utility measures. Quality of Life Research, 22, 853–874.
Versteegh, M. M., Leunis, A., Uyl-de Groot, C. A., & Stolk, E. A. (2012). Condition-specific preference-based measures: Benefit or burden? Value in Health, 15(3), 504–513.
Marra, C. A., Woolcott, J. C., Kopec, J. A., Shojania, K., Offer, R., Brazier, J. E., et al. (2005). A comparison of generic, indirect utility measures (the HUI2, HUI3, SF-6D, and the EQ-5D) and disease-specific instruments (the RAQoL and the HAQ) in rheumatoid arthritis. Social Science and Medicine, 60(7), 1571–1582.
Brazier, J., Rowen, D., Mavranezouli, I., Tsuchiya, A., Young, T., & Yang, Y. (2012). Developing and testing methods for deriving preference-based measures of health from condition specific measures (and other patient based measures of outcome). Health Technology Assessment, 16, 1–114.
Yang, Y., Brazier, J. E., Tsuchiya, A., & Coyne, K. (2009). Estimating a preference-based index from the Over Active Bladder questionnaire. Value in Health, 12, 159–166.
Berghoefer, P., Fragkos, K., Baxter, J. P., Forbes, A., Joly, F., Heinze, H. et al. (2012). Development and validation of the disease-specific Short Bowel Syndrome-Quality of Life (SBS-QoL™) scale. Clinical Nutrition (in press).
Jeppesen, P. B., Gilroy, R., Pertkiewicz, M., Allard, J. P., Messing, B., & O’Keefe, S. J. (2011). Randomised placebo-controlled trial of teduglutide in reducing parenteral nutrition and/or intravenous fluid requirements in patients with short bowel syndrome. Gut, 60(7), 902–914.
Brazier, J., Czoski-Murray, C., Roberts, J., Brown, M., Symonds, T., & Kelleher, C. (2008). Estimation of a preference-based index from a condition-specific measure: The King’s Health Questionnaire. Medical Decision Making, 28, 113–126.
Kind, P., & Macran, S. (2005). Eliciting social preference weights for functional assessment of cancer therapy-lung health states. Pharmacoeconomics, 23, 1143–1153.
Brazier, J., Roberts, J., & Deverill, M. (2002). The estimation of a preference-based measure of health from the SF-36. Journal of health economics, 21, 271–292.
Kind, P. (1996). The EuroQoL instrument: An index of health-related quality of life. Quality of life and pharmacoeconomics in clinical trials, 2, 191–201.
Rowen, D., Brazier, J. E., Young, T. A., Gaugrist, S., Craig, B. M., King, M. T., et al. (2010). Deriving a preference-based measure for cancer using the EORTC QLQ-C30. Value in Health, 14, 721–731.
Yang, Y., Brazier, J. E., Tsuchiya, A., & Young, T. A. (2011). Estimating a preference-based index for a 5-dimensional health state classification for asthma derived from the asthma quality of life questionnaire. Medical Decision Making, 31, 281–291.
Berghoefer, P., Mittelstadt, S., & Vogt-Humberg, I. (2010). Health-related quality of life (HRQoL) in patients with SBS. Internal Report by Peirrel Research Germany GmbH (former IFE Germany GmbH) for Nycomed GmbH.
Boyd, N. F., Sutherland, H. J., Heasman, K. Z., Tritchler, D. L., & Cummings, B. J. (1990). Whose utilities for decision analysis? Medical Decision Making, 10, 58–67.
Shiroiwa, T., Fukuda, T., & Tsutani, K. (2009). Health utility scores of colorectal cancer based on societal preference in Japan. Quality of Life Research, 18, 1095–1103.
Ubel, P. A., Loewenstein, G., & Jepson, C. (2003). Whose quality of life? A commentary exploring discrepancies between health state evaluations of patients and the general public. Quality of Life Research, 12, 599–607.
Devlin, N. J., Buckingham, K., Shah, K., Tsuchiya, A., Tilling, C., Wilkinson, G. et al. (2011). A comparison of alternative variants of the lead and lag time TTO. Health Economics, 22(5), 517–532.
Devlin, N. J., Tsuchiya, A., Buckingham, K., & Tilling, C. (2011). A uniform time trade off method for states better and worse than dead: Feasibility study of the ‘lead time’ approach. Health Economics, 20, 248–361.
Kind, P., Dolan, P., Gudex, C., & Williams, A. (1998). Variations in population health status: Results from a United Kingdom national questionnaire survey. BMJ, 316, 736–741.
Acknowledgments
This study was funded by Nycomed—a Takeda company. The authors would like to thank Professor Alastair Forbes, Professor Palle Bekker Jeppesen and Dr. Marcus Harbord for their expert review and input during item selection for development of the health states.
Author information
Authors and Affiliations
Corresponding author
Appendix
Appendix
Rights and permissions
About this article
Cite this article
Lloyd, A., Kerr, C., Breheny, K. et al. Economic evaluation in short bowel syndrome (SBS): an algorithm to estimate utility scores for a patient-reported SBS-specific quality of life scale (SBS-QoL™). Qual Life Res 23, 449–458 (2014). https://doi.org/10.1007/s11136-013-0516-4
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1007/s11136-013-0516-4