Caitlin Thomas

Purpose: Two complement inhibitors are currently used for treating geographic atrophy (GA) secondary to age-related macular degeneration (AMD) and other treatments are in development. We surveyed people with GA to better understand how efficacy, risks, and intravitreal injection frequency impact their treatment preferences. Methods: We used a mixed-methods approach (including qualitative research, pretesting interviews, and clinical expert and patient input) to design an online preference elicitation survey accessible to people with visual impairment. Preferences of US-based adults with GA were elicited with multi-dimensional thresholding. Participants ranked the included treatment characteristics and completed four sets of trade-off exercises (with two to three questions per exercise) containing two characteristics per question. The relative importance of treatment characteristics and participants’ willingness to trade-off between characteristics were evaluated. Results: Participants (N=77; mean age 69.3 years; 32.5% GA treatment-experienced) often preferred treatment over no treatment, ranging from 57% preferring treatment for the "worst” assessed treatment to 99% for the "best” assessed. Participants’ decisions were most influenced by extending the time until they were unable to read (relative importance: 32%) or time to see half as far (27%), followed by injection frequency (16%) and reducing risks of developing wet AMD (14%) and eye inflammation (12%). Most participants (86%) preferred a one-time intravitreal injection (vs every other month or every month). Within an 18-month time treatment timeframe, on average, participants were willing to accept higher risks for increased efficacy (eg a 35% higher risk of eye inflammation or 29% higher risk of wet AMD for a treatment that increases efficacy from 1 to 4 years longer to read and to see half as far). All else equal, participants were willing to accept higher risks for a one-time injection over monthly injections (eg 14% higher risk of eye inflammation or 12% higher risk of wet AMD). Conclusions: Most participants preferred the "worst” treatment profile over no treatment, highlighting a potential unmet need for GA treatment. Treatment choices were most influenced by efficacy, particularly prolonging the time until they are unable to read. Participants were willing to tolerate significantly higher risks for their preferred injection frequency. Shared treatment decision-making is encouraged to ensure alignment with patients’ individual preferences.

Purpose: Two complement inhibitors are US-approved for geographic atrophy (GA) and other treatments are in development. We present the first quantitative assessment of retina specialists’ (RS) preferences for GA treatment. We explored how GA treatment characteristics affect RS’ preferences, the trade-offs RS are willing to make between them, and whether this varies across patient profiles. Methods: US-based RS with ≥ 7 years of clinical experience and ≥ 50 patients with GA in the previous year selected their best and second-best treatment recommendations for specific patient profiles in 12 tasks showing two hypothetical treatment options and no treatment (best-best discrete choice experiment, BB-DCE). Treatment characteristics comprised lesion growth rate reduction, three treatment risks (mild-to-moderate uveitis, progression to wet age-related macular degeneration [AMD], and retinal vasculitis), and intravitreal injection frequency (every month, every other month, and one-time [gene therapy]). Patient profiles were defined by GA lesion location and visual acuity. Preferences were evaluated using a mixed-logit model. Relative importance of each characteristic and trade-offs were analyzed. Results: Of 166 RS, 83.1% had experience treating GA with complement inhibitors. Improving lesion growth rate reduction from 10% to 50% had the greatest impact (almost half of decision-making). Among risks, reducing the chance of progressing to wet AMD from 15% to 1% had the greatest impact (~20% of decision-making). All else equal, RS preferred a one-time over monthly injection(s). Risk tolerance increased with higher lesion growth rate reduction and, to a lesser extent, with less frequent injections; risk tolerance was highest when considering patients with foveal-threatening GA lesions. Conclusion: Across patient profiles, RS prioritized lesion growth rate reduction over risks; intravitreal injection frequency was the least important. However, RS were more risk-tolerant with patients with foveal-threatening lesions. RS would tolerate slightly higher risks for a one-time over monthly injection(s) if lesion growth rate reduction was comparable.

To evaluate hematologist-oncologists' preferences for frontline treatment of Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) using tyrosine kinase inhibitors (TKIs) + chemotherapy. An online discrete choice experiment was conducted among US-based hematologist-oncologists. Participants viewed profiles of hypothetical TKI + chemotherapy treatments with varied levels of benefit and risks (minimal residual disease-negative complete remission [MRD-negative CR], arterial occlusive events, grade 3-4 hepatotoxicity, grade 3-4 hematotoxicity) and chose their recommended treatment for five patient profiles: "less-complex" baseline; age ≥ 65; ECOG score 3; diabetes; hypertension. Data were analyzed using mixed multinomial logit models. 121 hematologist-oncologists participated. Increasing MRD-negative CR was most important to hematologist-oncologists, driving 65%-87% of decision-making across patient profiles. Relative importance of benefits/risks varied by patient profile. Pooled across patient profiles, hepatotoxicity was the most concerning risk, driving 14% of decision-making. Based on PhALLCON data and elicited preferences, hematologist-oncologists were predicted to select the profile of ponatinib + chemotherapy over imatinib + chemotherapy for all included patient profiles. Predicted probabilities of choosing ponatinib over imatinib ranged from 87%-98% across patient profiles. Hematologist-oncologists prioritized achieving MRD-negative CR when recommending frontline treatments for Ph+ ALL, accepting some risks if offset by meaningful improvement in MRD-negative CR.

Introduction Self-injectable calcitonin gene-related peptide monoclonal antibodies (CGRP mAbs) and oral CGRP antagonists are currently available for migraine prevention. This study elicited the preferences of participants with migraine for self-injectable CGRP mAb autoinjectors and non-CGRP oral medication and determined the relative importance of autoinjector attributes. Methods Adults from the USA, the UK, and Germany with episodic or chronic migraine who had taken migraine preventive treatments within the past 5 years completed a discrete choice experiment (DCE) online. Participants completed 12 experimental choice tasks, choosing their preferred treatment from three options (two hypothetical self-injectable CGRP mAbs autoinjectors, a non-CGRP oral medication), described by seven autoinjector attributes varied by levels. DCE data were analyzed using an error-component logit model to obtain relative attribute importance (RAI) and to estimate predicted choice probabilities (PCP) for autoinjector profiles. Results In total 1067 participants (51.3% with episodic migraine; 52.6% female; median age 40 years) completed the DCE. Common preventive treatments used were anti-epileptics (47.3%), beta blockers (41.4%), and antidepressants (36.7%). Throughout the DCE, autoinjectors were chosen in 86.3% of cases over non-CGRP oral medication. The most important attribute in participants’ treatment choices was injection duration, with a preference for shorter injection duration (RAI 37.0%), followed by auto-retractable needle removal over manual pull-out (RAI 30.8%), longer storage at room temperature (RAI 15.2%), and no pinching over pinching (RAI 12.5%). Participants were less concerned by dose confirmation (RAI 3.4%), injection steps (RAI 0.6%), and dosing schedule (RAI 0.5%). Elicited preferences suggest that an autoinjector profile comparable to galcanezumab (PCP 44.6%) had a higher likelihood (p < 0.001) of being chosen over profiles comparable to erenumab (PCP 28.8%) or fremanezumab three injections quarterly (PCP 26.6%). Conclusion Participants tended to prefer self-injectable CGRP mAb autoinjectors over non-CGRP oral preventive medications for migraine. Preferences among autoinjectors were driven by injection duration, auto-retractability of needle removal, storage requirements, and autoinjector base and pinching requirements.

Background: Patients with follicular lymphoma (FL) often relapse or become refractory to treatment (R/R). While the R/R FL treatment landscape evolves, little is known about the priorities of patients and physicians. This discrete‐choice experiment (DCE) study assessed patients' and physicians' treatment preferences, and the trade‐offs they would be willing to make between efficacy, tolerability, and administration.Methods: An online survey was conducted in US‐based patients (≥18 years) with R/R FL and FL‐treating physicians. The DCE was informed by a targeted literature review, clinical data, expert oncologist input, and pilot interviews. Participants completed eight experimental choice tasks where they chose between two hypothetical treatment profiles defined by six attributes: progression‐free survival (PFS), administration/monitoring, risks of laboratory abnormalities requiring intervention, severe infections, diarrhea, and cytokine release syndrome (CRS). Relative attribute importance (RAI) and willingness to trade‐off between PFS and other attributes were estimated.Results: Two‐hundred patients (mean age 63.5 years; median three prior lines of therapy) and 151 FL‐treating physicians participated. Increasing PFS was most important for both groups, although it was relatively less important to patients than physicians (RAI 35.2% vs. 45.7%). Administration/monitoring was three times more important to patients than physicians (RAI 28.8% vs. 9.5%); patients preferred oral treatment and would be willing to tolerate a significant reduction in PFS for oral administration over weekly intravenous infusions. Avoiding CRS was less important to patients than to physicians (RAI 7.7% vs. 15.8%). Both groups would accept shorter PFS for reduced risks of side effects (especially of laboratory abnormalities for patients and of CRS for physicians).Conclusion: Although PFS was the most important attribute to patients and physicians, both would tolerate lower PFS for reduced side effects. Patients would also accept a substantial reduction in PFS for oral administration. Differences between the preferences/priorities of patients and physicians highlight the importance of shared decision‐making.

Introduction: Newer treatment options for relapsed/refractory multiple myeloma (RRMM) with efficacy and safety profiles that differ from traditional therapies have facilitated personalized management strategies to optimize patient outcomes. In the context of such personalized management, understanding how treatment characteristics influence patients’ preferences is essential. This study assessed patients’ preferences for RRMM treatment attributes and determined trade-offs between potential benefits, administration procedures, and adverse effects.Methods: Patients’ preferences were evaluated using a discrete choice experiment (DCE). Patients with RRMM who reported failing two lines of anti-myeloma treatment (immunomodulatory agent and a proteasome inhibitor [PI]) or ≥ 3 lines (including ≥1 PI, immunomodulatory agent, or anti-CD38 monoclonal antibody), were recruited across the US, UK, Italy, Germany, France, and Spain. DCE attributes and levels were identified using a targeted literature review, a review of clinical data for relevant RRMM treatments, qualitative patient interviews, and input from clinical and myeloma patient experts. The DCE was administered within an online survey from February–June 2022. Preference data were analyzed using an error-component logit model and willingness to make trade-offs for potential benefits, and relative attribute importance scores were calculated.Results: Overall, 296 patients from the US (n = 100), UK (n = 49), Italy (n = 45), Germany (n = 43), France (n = 39), and Spain (n = 20) participated in the DCE. Mean (standard deviation) age was 63.8 (8.0) years, 84% had a caregiver, and patients had a median of 3 (range: 2–8) prior lines of therapy. Efficacy attributes most influenced patients’ preferences, with increasing overall response rate (25–85%) and overall survival (6 months to 2 years) contributing to ~50% of treatment decision-making. Administration procedures were also considered important to patients. Avoiding individual side effects was considered relatively less important, with patients willing to tolerate increases in side effects for gains in efficacy. Patient characteristics such as rate of disease progression, sociodemographics, or clinical characteristics also influenced treatment preferences.Conclusion: Patients with RRMM were willing to tolerate increased risk of side effects for higher efficacy. Preferences and risk tolerance varied between patients, with preference patterns differing by certain patient characteristics. This highlights the importance of shared decision-making for optimal treatment selection and patient outcomes.

Background: Research about the decision to participate in a clinical study has tended to be limited to single indications and has focused on narrow sets of study and participant characteristics. This study applied stated preference methods to understand the clinical trial design attributes that most influence willingness to participate and how this varied with participant characteristics. Methods: Adults residing in the US, China, or Poland with a self-reported diagnosis of cancer, heart disease, migraine, rheumatoid arthritis, or multiple sclerosis completed an online survey. Participants were asked whether they would participate in clinical studies defined by seventeen attributes within five categories (payment/support, administration/procedures, treatment-related, study location/time commitment, and data collection/feedback). Participants saw six different hypothetical clinical study profiles. Depending on their participation decision to an initial clinical study profile, the subsequent five questions had one design attribute (randomly selected per question) consecutively improved or deteriorated to elicit preferences. A logistic regression was used to determine which participant characteristics influenced participation decisions. A latent class logit model was used to identify how the influence of study design features varied between participants and whether groups of participants with similar preferences could be identified. Results: The survey was completed by 487 participants (32% China, 35% Poland, 33% US; 8%–19% per indication). Willingness to participate was found to be a function of participant age, certain elements of quality of life, and previous treatment experience, in particular number of lines of treatment received and experience of adverse events. Willingness to participate was influenced by study design features such as payment, study duration, and time commitment – both the overall time and whether the time was at home or away from home, with the latter being particularly relevant to participants experiencing fatigue due to their disease. Conclusions: This study quantifies how study designs influence willingness to participate and how this varies with participant types. These findings suggest that it is how an indication influences quality of life and treatment experience, rather than the indication alone, that impacts participation rates, opening the way for insights that are transferrable across indications, which may be particularly useful when considering rare diseases.

Introduction Information about patient preferences for the treatment of anaemia associated with chronic kidney disease (CKD) is scarce. Hence, our aim was to examine how patients with non-dialysis-dependent CKD valued attributes of alternative hypothetical anaemia treatments. Methods A discrete choice experiment (DCE) was conducted in adult patients who reported a clinical diagnosis of CKD-related anaemia. Treatment attributes included mode and frequency of administration, need for iron supplementation, risk of gastrointestinal side effects, risk of major cardiovascular events and impact on energy levels (as defined by the vitality section of the SF-6D health index). Logit models were used to analyse patients’ preferences. Results The DCE was completed by 200 patients in four countries. Patients preferred an oral mode of administration. Patients were willing to tolerate a 5.1% (95% CI 2.0–8.3%) increase in the risk of a major cardiovascular event and an 11.7% (95% CI 5.0–18.5%) increase in the risk of gastrointestinal side effects to switch from an at-home subcutaneous injection administered once every 2 weeks to an at-home oral pill administered three times a week. Patients were willing to tolerate a 20.3% (95% CI 15.0–25.6%) increase in the risk of gastrointestinal side effects and an 8.9% (95% CI 6.1–11.7%) increase in the risk of a major cardiovascular event to transition from ‘Sometimes having a lot of energy’ to ‘Always having a lot of energy’. Conclusions Patients with non-dialysis-dependent CKD-related anaemia demonstrated clear treatment preferences and were willing to accept increased gastrointestinal or cardiovascular risks in exchange for more energy or an oral treatment.

Objectives We aimed to quantify patient preferences for efficacy, safety and convenience features of atopic dermatitis (AD) treatments. Design and setting Online discrete choice experiment survey. Participants Adults in the UK, France and Spain who had used AD treatments during the past 2 years. Primary and secondary outcome measures Preferences for attributes were analysed using a multinomial logit model. Willingness to make trade-offs was expressed as the maximum acceptable decrease (MAD) in the probability of achieving clear/almost clear skin at week 16. Results The survey was completed by 404 patients (44.1±12.0 years; 65% women; 64% moderate/severe eczema). Most patients (68%) had no prior experience of using self-injectable treatments for AD or any other illness. Participants most valued increasing the chance of achieving a meaningful reduction in itch at week 16 from 20% to 50%, followed by reducing the risks of serious infections from 6% to 0% and of eye inflammation from 20% to 0%. Participants were willing to accept a decrease in the possibility of achieving clear/almost clear skin to obtain a treatment that can be paused (MAD=24.1%), requires occasional check-ups (MAD=16.1%) or no check-ups (MAD=20.9%) over frequent check-ups, is administered as a one time per day or two times per day oral pill versus a subcutaneous injection every 2 weeks (MAD=16.6%), has a 2-day over 2-week onset of action (MAD=11.3%), and can be used for flare management (MAD=5.8%). Conclusions Although patients with AD most valued treatment benefits and risks, they were willing to tolerate reduced efficacy to obtain a rapid onset, oral administration, less frequent monitoring and a treatment that can be paused. Understanding patients’ preferences for AD therapies, including new targeted therapies, can aid shared decision-making between clinicians and patients and support health technology assessments.