Patient preferences for inflammatory bowel disease treatments: protocol development of a global preference survey using a discrete choice experiment.

Background: As the therapeutic landscape for inflammatory bowel disease (IBD) continues to expand, a need exists to understand how patients perceive and value different attributes associated with their disease as well as with current and emerging treatments. These insights can inform the development and regulation of effective interventions for IBD, benefiting various stakeholders including healthcare professionals, drug developers, regulators, Health Technology Assessment bodies, payers, and ultimately patients suffering from IBD. In response to this, the present patient preference study was developed with the aim to (1) determine the relative preference weights for IBD treatment and disease related attributes, and (2) explain how preferences may differ across patients with different characteristics (preference heterogeneity). Methods: The patient preference study (PPS) was developed through an 8-step process, with each step being informed by an advisory board. This process included: (1) stated preference method selection, (2) attribute and level development (including a scoping literature review, focus group discussions, and advisory board meetings), (3) choice task construction, (4) sample size estimation, (5) survey implementation, (6) piloting, (7) translation, and (8) pre-testing. The resulting discrete choice experiment (DCE) survey comprises 14 attributes with between two and five varying levels. Participants will answer 15 DCE questions with a partial profile design, where each of the choice questions encompasses two hypothetical treatment profiles showing four attributes. Additionally, questions about patients' socio-demographic and clinical characteristics, as well as contextual factors are implemented. The survey is available in 15 different languages and aims to minimally recruit 700 patients globally. Discussion: This protocol gives valuable insights toward preference researchers and decision-makers on how PPS design can be transparently reported, demonstrating solutions to remaining gaps in preference research. Results of the PPS will provide evidence regarding the disease and treatment related characteristics that are most important for IBD patients, and how these may differ across patients with different characteristics. These findings will yield valuable insights applicable to preference research, drug development, regulatory approval, and reimbursement processes, enabling decision making across the medicinal product life cycle that is aligned with the true needs of IBD patients.

Indirect Costs of Inflammatory Bowel Diseases: A Comparison of Patient-Reported Outcomes Across 12 European Countries.

BACKGROUND: National studies report a high variability of indirect costs of inflammatory bowel disease (IBD). In this study, selected aspects of the societal burden of IBDs were compared between 12 European countries. METHODS: A questionnaire-based study among adult patients with IBD was performed. Data on patient characteristics, productivity loss, and informal care were collected. The costs of productivity loss were assessed from the social perspective. The cost of absenteeism and presenteeism was valuated using the gross domestic product per worker. Informal care was measured by time inputs of relatives and friends to assist patients. Productivity loss among informal caregivers outside their paid work was valuated with the average wage. The results were adjusted for confounders and multiplicity. RESULTS: Responses from 3687 patients (67% employed) were analyzed. Regular activity (outside paid work) impairment did not differ between countries, but a significant difference in informal care and productivity loss was observed. There were no differences in indirect costs between the types of IBD across the countries. The mean annual cost of absenteeism, presenteeism, and informal care varied from €1253 (Bulgaria) to €7915 (Spain), from €2149 (Bulgaria) to €14 524 (Belgium), and from €1729 (Poland) to €12 063 (Italy), respectively. Compared with patients with active disease, those with IBD in remission showed a lower indirect cost by 54% (presenteeism, P < .001) or 75% (absenteeism, informal care, P < .001). CONCLUSIONS: The study showed a high relevance of the indirect cost of IBD in the context of economic evaluation, as well as a between-country variability of work-related impairment or informal care.

The study showed a high relevance of the indirect cost of inflammatory bowel disease in the context of economic evaluation, as well as a between-country variability of work-related impairment or informal care.

Out-of-pocket expenses of patients with inflammatory bowel disease: a comparison of patient-reported outcomes across 12 European countries.

BACKGROUND: There is a high variability of out-of-packet patient costs of inflammatory bowel diseases (IBDs), but the issue is not widely recognised. Therefore, we compared patient costs of IBDs between 12 European countries. METHODS: A questionnaire-based study was conducted among adult patients with IBD. Data on patient characteristics and out-of-pocket expenses were anonymously collected. Ordered logit regression models were used to analyse the responses provided by patients. The results were adjusted for confounders and multiplicity. RESULTS: The questionnaires obtained from 3687 patients were analysed. Patients with comorbidities and active disease indicated higher out-of-pocket expenses than those without comorbidities and with disease in remission, respectively. Compared with other IBD, patients with ulcerative colitis indicated higher expenses on medications prescribed or recommended by physicians [odds ratio (OR) 1.99, 95% CI 1.48-2.67]. Expenses on dietary supplements, special diet or equipment, ostomy pouches, and transportation to a medical facility differed slightly between patients at different ages and were lower among men than among women (OR 0.71, 95% CI 0.54-0.93). The expenses differed significantly between countries. An adjusted mean patient cost per month varied from €77 (patient with Crohn disease in remission from Denmark) to €376 (patient with active ulcerative colitis from Romania). Compared with active disease, patients with IBD in remission had a lower out-of-pocket cost by 29-62% (€10-€22 monthly; p < 0.001). CONCLUSIONS: The study revealed a high relevance of the out-of-pocket cost of IBD in the context of economic evaluation and a high variability of the cost between countries.

What are the Unmet Needs and Most Relevant Treatment Outcomes According to Patients with Inflammatory Bowel Disease? A Qualitative Patient Preference Study.

BACKGROUND AND AIMS: As more therapeutic options with their own characteristics become available for inflammatory bowel disease [IBD], drug development and individual treatment decision-making needs to be tailored towards patients' preferences and needs. This study aimed to understand patient preferences among IBD patients, and their most important treatment outcomes and unmet needs. METHODS: This qualitative study consisted of [1] a scoping literature review, [2] two focus group discussions [FGDs] with IBD patients [n = 11] using the nominal group technique, and [3] two expert panel discussions. RESULTS: IBD patients discussed a multitude of unmet needs regarding their symptoms, side-effects, and psychological and social issues for which they would welcome improved outcomes. In particular, IBD patients elaborated on the uncertainties and fears they experienced regarding the possible need for surgery or an ostomy, the effectiveness and onset of action of their medication, and the medication's long-term effects. Furthermore, participants extensively discussed the mental impact of IBD and their need for more psychological guidance, support, and improved information and communication with healthcare workers regarding their disease and emotional wellbeing. The following five characteristics were identified during the attribute grading as most important: prevent surgery, long-term clinical remission, improved quality of life [QoL], occurrence of urgency and improved labour rate. CONCLUSIONS: This study suggests that IBD drug development and treatment decision-making are needed to improve IBD symptoms and adverse events that significantly impact IBD patients' QoL. Furthermore, this study underlines patients' need for a shared decision-making process in which their desired treatment outcomes and uncertainties are explicitly discussed and considered.

Multi-national observational study to assess quality of life and treatment preferences in patients with Crohn's perianal fistulas.

BACKGROUND: Patients with Crohn's disease (CD) are at risk of developing complications such as perianal fistulas. Patients with Crohn's perianal fistulas (CPF) are affected by fecal incontinence (FI), bleeding, pain, swelling, and purulent perianal discharge, and generally face a higher treatment burden than patients with CD without CPF. AIM: To gain insights into the burden of illness/quality of life in patients with CPF and their treatment preferences and satisfaction. METHODS: This cross-sectional observational study was conducted in patients with CD aged 21-90 years via a web-enabled questionnaire in seven countries (April-August 2021). Patients were recruited into three cohorts: Cohort 1 included patients without perianal fistulas; cohort 2 included patients with perianal fistulas without fistula-related surgery; and cohort 3 included patients with perianal fistulas and fistula-related surgery. Validated patient-reported outcome measures were used to assess quality of life. Drivers of treatment preferences were measured using a discrete choice experiment (DCE). RESULTS: In total, 929 patients were recruited (cohort 1, n = 620; cohort 2, n = 174; cohort 3, n = 135). Short Inflammatory Bowel Disease Questionnaire scores were worse for patients with CPF (cohorts 2 and 3) than for those with CD without CPF (cohort 1): Mean score 3.8 and 3.7 vs 4.1, respectively, (P < 0.001). Similarly, mean Revised FI and FI Quality of Life scores were worse for patients with CPF than for those with CD without CPF. Quality of Life with Anal Fistula scores were similar in patients with CPF with or without CPF-related surgery (cohorts 2 and 3): Mean score 41 and 42, respectively. In the DCE, postoperative discomfort and fistula healing rate were the most important treatment attributes influencing treatment choice: Mean relative importance 35.7 and 24.7, respectively. CONCLUSION: The burden of illness in CD is significantly higher for patients with CPF and patients rate lower postoperative discomfort and higher healing rates as the most desirable treatment attributes.