Patient and physician preferences for ulcerative colitis treatments in the United States

Abstract

Purpose: This study aimed to elicit patient and physician preferences for ulcerative colitis (UC) treatments in the United States (US).

Patients and methods: The following UC treatment attributes included in the discrete-choice experiment (DCE) were identified during qualitative interviews with both patients and physicians: time to symptom improvement, chance of long-term symptom control, risks of serious infection and malignancy, mode and frequency of administration, and need for steroids. The DCE survey instruments were developed and administered to patients and physicians. A random-parameters logit model was used to estimate preference weights and conditional relative importance for these attributes.

Results: A total of 200 patients with moderate to severe UC (status determined using self-reported medication history) and 200 gastroenterologists completed the survey. Patients’ average age was 42 years; most (59%) were female. Patients considered symptom control 2.5 times as important as time to symptom improvement and 5-year risk of malignancy almost as important as long-term symptom control (relative importance, 0.79 vs 0.96 for long-term symptom control); they preferred oral to subcutaneous or intravenous administration (relative importance, 0.47 vs 0.11 and 0.18, respectively). For physicians, symptom control was the most important attribute and was five times as important as the risk of malignancy.

Conclusion: Both patients and physicians considered long-term symptom control the most important attribute relative to others; however, risk of malignancy was of almost-equal importance to patients but not physicians. Differences between patients’ and physicians’ preferences highlight the need for improved communication about the relevant benefits and risks of different UC treatments to improve therapeutic decision-making.

Keywords:

• ulcerative colitis
• discrete-choice experiments
• maximum acceptable risk
• patient preference
• physician preference

Acknowledgments

The authors gratefully acknowledge Kimberly Moon of RTI Health Solution for overall project management for this study, Dr Allen Mangel for his help during the development of the survey instrument, and Kate Lothman of RTI Health Solutions for her help during the development of this manuscript. This study was funded by Pfizer Inc. through a contract with RTI Health Solutions. The abstract of this paper was presented at the 2018 Advances in Inflammatory Bowel Diseases (AIBD) Conference; December 13, 2018. Orlando, FL as a poster presentation. The poster and the poster’s abstract are available online in the archives of Advances in Inflammatory Bowel Diseases (AIBD): https://aibd.scientificposters.com/epsAbstractAIBD.cfm?id=1.

Abbreviation list

5-ASA, 5-aminosalicylate; CI, confidence interval; DCE, discrete-choice experiment; GED, General Education Development; IBD, inflammatory bowel disease; IV, intravenous; RPL, random-parameters logit; SC, subcutaneous ; SD, standard deviation; SSI, Survey Sampling International; UC, ulcerative colitis; UK, United Kingdom; US, United States.

Ethics approval and informed consent

The RTI International Institutional Review Board (IRB) determined that this study meets the criteria for IRB exemption under the Code of Federal Regulations (section 45CFR46.101(b) specifically) because the research involved the use of survey procedures, the information obtained was recorded in such a manner that human subjects could not be identified, directly or through identifiers linked to the subjects, and any disclosure of the human subjects’ responses outside the research would not reasonably place the subjects at risk of criminal or civil liability or be damaging to the subjects’ financial standing, employability, or reputation. In the qualitative phase, each interview participant signed and dated an informed consent form before the interview. All respondents to the DCE survey provided electronic informed consent and received compensation for time spent participating. The study complied with the Declaration of Helsinki.

Data availability

The data used in the analyses are available from the corresponding author on reasonable request.

Author contributions

Amy Marren and Marco DiBonaventura initiated the study. Marco Boeri, Brett Hauber, Kelley Myers, and Claire Ervin designed the study, with input from Amy Marren, Marco DiBonaventura, and Joseph C Cappelleri. Marco Boeri, Brett Hauber, Kelley Myers, and Claire Ervin conducted the statistical analyses. Marco Boeri led development of the manuscript, with input from Kelley Myers, Claire Ervin, Amy Marren, Marco DiBonaventura, Joseph C Cappelleri, Brett Hauber, and David T Rubin. David T Rubin provided clinical input on the study. All authors contributed to data analysis, drafting and revising the article, gave final approval of the version to be published, and agree to be accountable for all aspects of the work.

Disclosure

Claire Ervin, Kelley Myers, Marco Boeri, and Brett Hauber are employees of RTI Health Solutions, which were paid consultants to Pfizer in connection with the development of this manuscript. Amy Marren, Macro DiBonaventura, and Joseph C Cappelleri are employees and shareholders of Pfizer. Marco Boeri and Brett Hauber received project funding from Pfizer during the conduct of the study. Dr David T Rubin reports grants and personal fees from Abbvie, Genentech/Roche, Janssen Pharmaceuticals, Prometheus Laboratories, Shire, and Takeda, personal fees from Abgenomics, Allergan, Inc., Arena Pharmaceuticals, Biomica, Bristol-Myers Squibb, Dizal Pharmaceuticals, Ferring Pharmaceuticals, Inc., Lilly, Merck & Co., Inc., Medtronic, Napo Pharmaceuticals, Pfizer, and Target PharmaSolutions, Inc., outside the submitted work.  The authors report no other conflicts of interest in this work.