Combining patient preferences with expected treatment outcomes to inform decision-making

  • Megan S. Schuler
  • Laura A. Hatfield


Patient-centered care involves patients in decision-making about their medical care. Effective shared decision-making requires relevant evidence on the expected health outcomes of treatment, assessment of patient preferences for possible health outcomes, and a method to synthesize this information. Few tools exist to help clinicians and patients synthesize clinical evidence and personal preferences. We develop a statistical framework that combines multiple data sources on expected treatment outcomes with individual preferences to produce a personalized preference-weighted outcome score (PWOS). The PWOS can be calculated for multiple treatments and compared to determine which will provide the best balance of risks and benefits given a patient’s preferences. We demonstrate our method by evaluating adjuvant chemotherapy regimens for colorectal cancer. We begin by identifying heterogeneity in patient preferences for health outcomes associated with colorectal cancer disease and treatment, finding classes of patients who value efficacy and side effects differently. Then we fit a hierarchical Bayesian model to randomized clinical trials and produce posterior distributions of expected outcomes under three chemotherapy regimens. We use the PWOS to combine distinct sets of patient preferences with these expected outcomes. Our method preserves estimation uncertainty and accounts for correlation among outcomes. In a simulation study of applying PWOS to make decisions at the population level, we find that our methods are most useful when there are large differences in individual preferences and small distinctions in treatment efficacy (i.e., preference-sensitive settings).


Personalized medicine Multiple outcome modeling Preference-sensitive decisions Utility values Latent profile analysis 



The authors wish to thank the Marshall J. Seidman Center for Studies in Health Economics and Health Care Policy at Harvard Medical School for supporting Dr. Schuler’s work. The authors are grateful to Jennie Best and Lou Garrison for sharing their data on patient utilities and to Dan Sargent and the ACCENT Investigators for sharing the colorectal clinical trial data. Finally, the authors thank Dan Sargent, Tom Kelley, Arlene Ash, the associate editor, and two anonymous reviewers for their helpful comments on the manuscript.


This work was funded by the generous support Marshall J. Seidman Center for Studies in Health Economics and Health Care Policy at Harvard Medical School (Dr. Schuler).

Compliance with ethical standards

Conflict of interest

The authors declare that they have no conflict of interest.

Ethical approval

This article does not contain any studies with human participants or animals performed by any of the authors.


  1. Andre, T., Boni, C., Mounedji-Boudiaf, L., Navarro, M., Tabernero, J., Hickish, T., Topham, C., Zaninelli, M., Clingan, P., Bridgewater, J., Tabah-Fisch, I., de Gramont, A.: Oxaliplatin, fluorouracil, and leucovorin as adjuvant treatment for colon cancer. N. Engl. J. Med. 350(23), 2343–2351 (2004)CrossRefPubMedGoogle Scholar
  2. Arnold, D., Girling, A., Stevens, A., Lilford, R.: Comparison of direct and indirect methods of estimating health state utilities for resource allocation: review and empirical analysis. BMJ 339, b2688 (2009)CrossRefPubMedPubMedCentralGoogle Scholar
  3. Balogh, E., Ganz, P., Murphy, S., Nass, S., Ferrell, B., Stovall, E.: Patient-centered cancer treatment planning: improving the quality of oncology care. Summary of an Institute of Medicine workshop. Oncologist 16(12), 1800–1805 (2011)CrossRefPubMedPubMedCentralGoogle Scholar
  4. Best, J.H., Garrison, L.P., Hollingworth, W., Ramsey, S.D., Veenstra, D.L.: Preference values associated with stage III colon cancer and adjuvant chemotherapy. Qual. Life Res. 19(3), 391–400 (2010)CrossRefPubMedGoogle Scholar
  5. Cheung, W., Shi, Q., O’Connell, M., Cassidy, J., Blanke, C., Kerr, D., Meyers, J., Cutsem, E.V., Alberts, S., Yothers, G., Sargent, D.: The predictive and prognostic value of sex in early-stage colon cancer: a pooled analysis of 33,345 patients from the ACCENT database. Clin. Colorectal Cancer 12(3), 179–187 (2013)CrossRefPubMedPubMedCentralGoogle Scholar
  6. Clancy, C., Collins, F.S.: Patient-centered outcomes research institute: the intersection of science and health care. Sci. Transl. Med. 2(37), 37cm18 (2010)CrossRefPubMedGoogle Scholar
  7. Cox, C.E., White, D.B., Abernethy, A.P.: A universal decision support system: addressing the decision-making needs of patients, families, and clinicians in the setting of critical illness. Am. J. Respir. Crit. Care Med. 190(4), 366–373 (2014)CrossRefPubMedPubMedCentralGoogle Scholar
  8. de Bekker-Grob, E., Rose, J., Donkers, B., Essink-Bot, M., Bangma, C., Steyerberg, E.: Men’s preferences for prostate cancer screening: a discrete choice exper- iment. Br. J. Cancer 108(3), 533–541 (2013)CrossRefPubMedPubMedCentralGoogle Scholar
  9. Djalalov, S., Rabeneck, L., Tomlinson, G., Bremner, K.E., Hilsden, R., Hoch, J.S.: A review and meta-analysis of colorectal cancer utilities. Med. Decis. Making 34(6), 809–818 (2014)CrossRefPubMedGoogle Scholar
  10. Elkin, E., Kim, S., Casper, E., Kissane, D., Schrag, D.: Desire for information and involvement in treatment decisions: elderly cancer patients’ preferences and their physicians’ perceptions. J. Clin. Oncol. 25(33), 5275–5280 (2007)CrossRefPubMedGoogle Scholar
  11. Engstrom, P.F., Arnoletti, J.P., Benson, A.B., Chen, Y.J., Choti, M.A., Cooper, H.S., Covey, A., Dilawari, R.A., Early, D.S., Enzinger, P.C., Fakih, M.G., Fleshman, J., Fuchs, C., Grem, J.L., Kiel, K., Knol, J.A., Leong, L.A., Lin, E., Mulcahy, M.F., Rao, S., Ryan, D.P., Saltz, L., Shibata, D., Skibber, J.M., Sofocleous, C., Thomas, J., Venook, A.P., Willett, C.: NCCN clinical practice guidelines in oncology: colon cancer. J. Natl. Compr. Cancer Netw. 7(8), 778 (2009)Google Scholar
  12. Gaston, C., Mitchell, G.: Information giving and decision-making in patients with advanced cancer: a systematic review. Soc. Sci. Med. 61(10), 2252–2264 (2005)CrossRefPubMedGoogle Scholar
  13. Gelman, A.: Prior distributions for variance parameters in hierarchical models. Bayesian Anal. 1(3), 515–533 (2006)CrossRefGoogle Scholar
  14. Gomes, M., Hatfield, L.A., Normand, S.L.T.: Handling missing data in meta-analysis of individual participant data with correlated mixed outcomes. Stat. Med. 35(21), 3676–3689 (2016)CrossRefPubMedPubMedCentralGoogle Scholar
  15. Haller, D.G., Tabernero, J., Maroun, J., de Braud, F., Price, T., Van Cutsem, E., Hill, M., Gilberg, F., Rittweger, K., Schmoll, H.J.: Capecitabine plus oxaliplatin compared with fluorouracil and folinic acid as adjuvant therapy for stage III colon cancer. J. Clin. Oncol. 29(11), 1465–1471 (2011)CrossRefPubMedGoogle Scholar
  16. IMPACT Investigators: Efficacy of adjuvant fluorouracil and folinic acid in colon cancer. Lancet 345(8955), 939–944 (1995)CrossRefGoogle Scholar
  17. Institute of Medicine: Crossing the quality chasm: a new health system for the 21st century. National Academy Press, Washington (2001)Google Scholar
  18. Kaltoft, M., Cunich, M., Salkeld, G., Dowie, J.: Assessing decision quality in patient-centered care requires a preference-sensitive measure. J. Health Serv. Res. Policy 19(2), 110–117 (2014)CrossRefPubMedPubMedCentralGoogle Scholar
  19. Keirns, C.C., Goold, S.D.: Patient-centered care and preference-sensitive decision making. J. Am. Med. Assoc. 302(16), 1805–1806 (2009)CrossRefGoogle Scholar
  20. Krouss, M., Croft, L., Morgan, D.J.: Physician understanding and ability to communicate harms and benefits of common medical treatments. JAMA Intern. Med. 176, 1565–1567 (2016)CrossRefPubMedGoogle Scholar
  21. Kuebler, J.P., Wieand, H.S., O’Connell, M.J., Smith, R.E., Colangelo, L.H., Yothers, G., Petrelli, N.J., Findlay, M.P., Seay, T.E., Atkins, J.N., Zapas, J.L., Goodwin, J.W., Fehrenbacher, L., Ramanathan, R.K., Conley, B.A., Flynn, P.J., Soori, G., Colman, L.K., Levine, E.A., Lanier, K.S., Wolmark, N.: Oxaliplatin combined with weekly bolus fluorouracil and leucovorin as surgical adjuvant chemotherapy for stage II and III colon cancer: results from NSABP C-07. J. Clin. Oncol. 25(16), 2198–2204 (2007)CrossRefPubMedGoogle Scholar
  22. Masya, L., Young, J., Solomon, M., Harrison, J., Dennis, R., Salkeld, G.: Preferences for outcomes of treatment for rectal cancer: patient and clinician utilities and their application in an interactive computer-based decision aid. Dis. Colon Rectum 52(12), 1994–2002 (2009)CrossRefPubMedGoogle Scholar
  23. McCleary, N., Meyerhardt, J., Green, E., Yothers, G., de Gramont, A., Cutsem, E.V., O’Connell, M., Twelves, C., Saltz, L., Haller, D., Sargent, D.: Impact of age on the efficacy of newer adjuvant therapies in patients with stage II/III colon cancer: findings from the ACCENT database. J. Clin. Oncol. 31(20), 2600–2606 (2013)CrossRefPubMedPubMedCentralGoogle Scholar
  24. Meghani, S., Lee, C., Hanlon, A., Bruner, D.: Latent class cluster analysis to understand heterogeneity in prostate cancer treatment utilities. BMC Med. Inform. Decis. Mak. 9, 47 (2009)CrossRefPubMedPubMedCentralGoogle Scholar
  25. Moertel, C.G., Fleming, T.R., Macdonald, J.S., Haller, D.G., Laurie, J.A., Goodman, P.J., Ungerleider, J.S., Emerson, W.A., Tormey, D.C., Glick, J.H., et al.: Levamisole and fluorouracil for adjuvant therapy of resected colon carcinoma. N. Engl. J. Med. 322(6), 352–358 (1990)CrossRefPubMedGoogle Scholar
  26. Muhlbacher, A., Bethge, S.: Patients’ preferences: a discrete-choice experiment for treatment of non-small-cell lung cancer. Eur. J. Health Econ. 16(6), 657–670 (2015)CrossRefPubMedGoogle Scholar
  27. Ness, R., Holmes, A., Klein, R., Dittus, R.: Utility valuations for outcome states of colorectal cancer. Am. J. Gastroenterol. 94(6), 1650–1657 (1999)CrossRefPubMedGoogle Scholar
  28. O’Connor, A., Legare, F., Stacey, D.: Risk communication in practice: the contribution of decision aids. Br. Med. J. 327(7417), 736–740 (2003)CrossRefGoogle Scholar
  29. O’Connor, E., Greenblatt, D., LoConte, N., Gangnon, R., Liou, J., Heise, C., Smith, M.: Adjuvant chemotherapy for stage II colon cancer with poor prognostic features. J. Clin. Oncol. 29(25), 3381–3388 (2011)CrossRefPubMedPubMedCentralGoogle Scholar
  30. Plummer M.: JAGS: a program for analysis of Bayesian graphical models using Gibbs sampling. In: Proceedings of the 3rd International Workshop of Distributed Statistical Computing (DSC 2003), Vienna, Austria, vol ISSN 1609- 395X (2003)Google Scholar
  31. Plummer M.: rjags: Bayesian graphical models using MCMC., r package version 4-5 (2016)
  32. Politi, M.C., Studts, J.L., Hayslip, J.W.: Shared decision making in oncology practice: What do oncologists need to know? Oncologist 17(1), 91–100 (2012)CrossRefPubMedPubMedCentralGoogle Scholar
  33. Renfro, L., Grothey, A., Xue, Y., Saltz, L., Andre, T., Twelves, C., Labianca, R., Allegra, C., Alberts, S., Loprinzi, C., Yothers, G., Sargent, D.: ACCENT-based web calculators to predict recurrence and overall survival in stage III colon cancer. J. Natl. Cancer Inst. 106(12), dju333 (2014)CrossRefPubMedPubMedCentralGoogle Scholar
  34. Rosato, R., Testa, S., Oggero, A., Molinengo, G., Bertolotto, A.: Quality of life and patient preferences: identification of subgroups of multiple sclerosis patients. Qual. Life Res. 24(9), 2173–2182 (2015)CrossRefPubMedGoogle Scholar
  35. Sanoff, H., Carpenter, W., Sturmer, T., Goldberg, R., Martin, C., Fine, J., McCleary, N., Meyerhardt, J., Niland, J., Kahn, K., Schymura, M., Schrag, D.: Effect of adjuvant chemotherapy on survival of patients with stage III colon cancer diagnosed after age 75 years. J. Clin. Oncol. 30(21), 2624–2634 (2012)CrossRefPubMedPubMedCentralGoogle Scholar
  36. Sassi, F.: Calculating QALYs, comparing QALY and DALY calculations. Health Policy Plan. 21(5), 402–408 (2006)CrossRefPubMedGoogle Scholar
  37. Schnipper, L., Davidson, N., Wollins, D., Tyne, C., Blayney, D., Blum, D., Dicker, A., Ganz, P., Hoverman, J., Langdon, R., Lyman, G., Meropol, N., Mulvey, T., Newcomer, L., Peppercorn, J., Polite, B., Raghavan, D., Rossi, G., Saltz, L., Schrag, D., Smith, T., Yu, P., Hudis, C., Schilsky, R.: American Society of Clinical Oncology statement: a conceptual framework to assess the value of cancer treatment options. J. Clin. Oncol. 33(23), 2563–2577 (2015)CrossRefPubMedPubMedCentralGoogle Scholar
  38. Shabaruddin, F., Chen, L.C., Elliott, R., Payne, K.: A systematic review of utility values for chemotherapy-related adverse events. Pharmacoeconomics 31(4), 277–288 (2013)CrossRefPubMedGoogle Scholar
  39. Shiroiwa, T., Fukuda, T., Tsutani, K.: Health utility scores of colorectal cancer based on societal preference in Japan. Qual. Life Res. 18(8), 1095–1103 (2009)CrossRefPubMedGoogle Scholar
  40. Stein, D., Joulain, F., Naoshy, S., Iqbal, U., Muszbek, N., Payne, K., Ferry, D., Goey, S.: Assessing health-state utility values in patients with metastatic colorectal cancer: a utility study in the United Kingdom and the Netherlands. Int. J. Colorectal Dis. 29(10), 1203–1210 (2014)CrossRefPubMedGoogle Scholar
  41. Thacher, J., Morey, E., Craighead, W.: Using patient characteristics and attitudinal data to identify depression treatment preference groups: a latent-class model. Depress. Anxiety 21(2), 47–54 (2005)CrossRefPubMedGoogle Scholar
  42. Ubel, P., Angott, A., Zikmund-Fisher, B.: Physicians recommend different treatments for patients than they would choose for themselves. Arch. Intern. Med. 171(7), 630–634 (2011)CrossRefPubMedGoogle Scholar
  43. US Food and Drug Administration.: Enhancing benefit-risk assessment in regulatory decision-making. (2016)
  44. Whitney, S.N., Holmes-Rovner, M., Brody, H., Schneider, C., McCullough, L.B., Volk, R.J., McGuire, A.L.: Beyond shared decision making: an expanded typology of medical decisions. Med. Decis. Mak. 28(5), 699 (2008)CrossRefGoogle Scholar
  45. Yothers, G., Sargent, D., Wolmark, N., Goldberg, R., O’Connell, M., Benedetti, J., Saltz, L., Dignam, J., Blackstock, A.: Outcomes among black patients with stage II and III colon cancer receiving chemotherapy: an analysis of ACCENT adjuvant trials. J. Natl Cancer Inst. 103(20), 1498–1506 (2011)CrossRefPubMedPubMedCentralGoogle Scholar

Copyright information

© Springer Science+Business Media New York 2016

Authors and Affiliations

  1. 1.Department of Health Care PolicyHarvard Medical SchoolBostonUSA

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