Purpose:To understand the trade-offs that patients with episodic migraine are willing to make between attributes of self-injectable calcitonin gene-related peptide (CGRP) monoclonal antibodies (mAbs) and oral small-molecule calcitonin gene-related peptide receptor antagonists (gepants). Patients and Methods:This was an online discrete choice experiment survey among adults with episodic migraine (≥4 and <15 migraine headache days and <15 headache days per month over 3 months) in the United States. Hypothetical treatments were characterized by five attributes: chance of ≥50% migraine reduction, impact on daily activities, onset of treatment effect, reduction in number of acute medications, and treatment administration. The attributes were selected based on insights gained from a previously conducted literature review and focus group study and aligned with the primary and secondary outcome measures in the double-blind Phase IV head-to-head clinical trial of a CGRP mAb versus gepant. Results:601 patients (mean age: 44.8 years) completed the survey. Treatment preferences differed significantly between patients. However, for all patients, the most important driver of treatment preferences was the chance of a ≥50% reduction in monthly migraine headache days (relative attribute importance: 38.3%), followed by the impact on daily activities (23.5%), the onset of treatment effect (19.5%), the reduction in need for acute medication (15.4%), and finally the route of administration (3.4%). Patients were willing to consider a one-week delayed onset of treatment effect or one-day increased need for acute medication for a higher chance (by 2.06% and 2.65% respectively) of a ≥50% reduction in monthly migraine headache days. Patients would trade a reduction of migraine's impact on daily activities from "extreme" to "moderate" or "minimal" with a lower chance (17.09%, 12.06% respectively) of halving the number of monthly migraine headache days. Conclusion:A ≥50% reduction in monthly migraine headache days was the most important treatment attribute for which participants were willing to trade against other attributes. The variation in treatment preferences between patients emphasizes the importance to align decision-making with individual patients' preferences.

Ritlecitinib is an oral Janus kinase 3/tyrosine kinase expressed in hepatocellular carcinoma (JAK3/TEC) family kinase inhibitor approved for the treatment of severe alopecia areata (AA). Benefit-risk profiles of two doses of ritlecitinib (50 mg vs 30 mg once daily) were evaluated by integrating patient preferences and clinical efficacy and safety estimates for ritlecitinib. A discrete-choice experiment (DCE) was utilized to elicit preferences for benefit and safety attributes of systemic AA treatments. Benefits included probabilities of ≥80% scalp hair coverage and achieving moderate to normal eyebrows and eyelashes. Potential risks included 3-year probabilities of serious infection, cancer, and blood clots. Preference estimates were used to calculate the maximum acceptable risk (MAR) that patients would accept for expected increases in benefit from choosing a higher ritlecitinib dose over a lower dose. Ritlecitinib benefits were calculated from the ALLEGRO-2b/3 clinical trial. MARs were calculated separately for each risk and jointly for all possible combinations. Adults (n = 201) with physician-confirmed ≥50% scalp hair loss from AA participated. To achieve expected increases in the probabilities of ≥80% scalp hair coverage or moderate to normal eyebrows and eyelashes when choosing 50 mg over 30 mg of ritlecitinib, patients would be willing to accept increases in each 3-year risk up to a mean of 3.88 absolute percentage points (95% confidence interval [CI], 2.86-4.90) for serious infection, 1.63 (95% CI, 1.08-2.18) for cancer, and 5.30 (95% CI, 3.60-7.00) for blood clots. These results, combined with the estimated differences in risks between the two doses, indicate that patients with AA value increases in the probabilities of scalp, eyebrow, and eyelash hair regrowth, with the average patient accepting increases in potential treatment-related risks for the 50-mg dose in exchange for higher efficacy than 30 mg. The DCE approach to measuring risk tolerance, combined with comparisons to expected benefit and risk differences, can be used to optimize AA treatment dose selection.

BACKGROUND:To help navigate the complex treatment landscape of ulcerative colitis (UC), we quantified the benefit-risk trade-offs that patients were willing to make when choosing treatment. METHODS:Patients completed an online discrete choice experiment. Eligible patients had a UC diagnosis for ≥6 months, were aged ≥18 years, and resided in France, Germany, Italy, Spain, or the UK. Patients chose between 2 hypothetical treatments set up to ensure trade-offs were made. Clinical trial data, literature review, and patient interviews identified treatment attributes. Relative attribute importance (RAI) scores and maximum acceptable risks were generated. A patient-centric benefit-risk assessment of 200 mg of filgotinib was conducted as an example to show how measured trade-offs can be used. RESULTS:Overall, 631 patients participated; patients had a mean age of 42.2 years and were predominantly male (75.3%). Achieving and maintaining clinical remission was the most important factor for patients (RAI 32.4%); to achieve this, patients were willing to accept slightly higher risks of blood clots, serious infections, and malignancies compared with lower risk treatment profiles. Patients also valued the convenience of oral treatments, avoiding steroids, and the ability to attend school/work. The patient-centric benefit-risk assessment suggested patients are significantly more likely to prefer Janus kinase 1 preferential inhibitor filgotinib over placebo. CONCLUSIONS:Achieving clinical remission was the highest treatment priority for patients. To attain this, patients were willing to accept some slightly higher risk treatment profiles. Patient choices in the benefit-risk assessment suggested patients were significantly more likely to prefer filgotinib over placebo.

OBJECTIVES:Inhibition of JAK-mediated signaling pathways leads to hair regrowth and improved quality of life in alopecia areata (AA) but may be associated with potential risks. This study assessed the benefit-risk profile of ritlecitinib, an oral selective JAK3/TEC family kinase inhibitor (JAK3/TEC), from the perspective of adult patients with AA to inform regulatory decisions. METHODS:We combined patient preference data from a discrete choice experiment involving 201 adults with AA in the United States and Europe, efficacy data for ritlecitinib 50 mg once daily and placebo from the ALLEGRO phase 2b/3 trial (NCT03732807) of ritlecitinib efficacy and safety, and safety data from an integrated safety analysis of 4 studies in AA. The discrete choice experiment included 6 treatment attributes: 3 benefits (chance of achieving ≥80% scalp hair coverage, moderate or normal eyebrows, and moderate or normal eyelashes) and 3 risks (3-year risks of serious infections, blood clots, and cancer). Choice probabilities were calculated using the estimated utility function. Weighted net benefit scores were calculated using multicriteria decision analysis. Stochastic multicriteria acceptability analysis assessed the impact of uncertainty and preference heterogeneity on net benefit conclusions. RESULTS:Ritlecitinib 50 mg had a higher weighted net benefit score than placebo with a predicted choice probability of 70.9% (95% CI 61.2%-77.5%), indicating that, on average, patients would likely evaluate benefits to outweigh risks. The probability of positive weighted net benefit based on the stochastic multicriteria acceptability analysis was 78%. CONCLUSIONS:Ritlecitinib 50 mg once daily demonstrated a positive benefit-risk profile compared with placebo in adult patients with AA.

Four sphingosine-1-phosphate receptor (S1PR) modulators are currently available in the USA for treating relapsing forms of multiple sclerosis (MS). These S1PR modulators have similar efficacy. Clinicians may therefore consider other factors, such as clinical management considerations, when distinguishing among treatments. This study estimated which S1PR modulator clinicians would choose on the basis of a treatment’s clinical management and quantified how individual aspects of clinical management might drive this choice. A multi-criteria decision analysis (MCDA) was conducted on the basis of clinical management preferences elicited in a discrete choice experiment (DCE) and real-world clinical management profiles of the S1PR modulators currently available to treat relapsing forms of MS (fingolimod, ozanimod, ponesimod, siponimod). The DCE was completed by neurologists in the USA experienced in treating MS and included eight clinical management attributes: first-dose observations, genotyping, liver function tests, eye exams, drug–drug interactions, interactions with antidepressants, interactions with foods high in tyramine, and immune system recovery time. Attribute levels were selected on the basis of S1PR modulator product labels. In the MCDA, partial MCDA scores were created for each attribute and summed to produce an overall MCDA score for each S1PR modulator. The DCE was completed by 200 neurologists. The overall MCDA score was highest for ponesimod (4.78 points), followed by siponimod (4.10 points), fingolimod (3.61 points), and ozanimod (2.38 points). Having fewer drug–drug interactions contributed most to the overall scores (up to 1.56 points), followed by having no first-dose observations (0.95 points), the shortest immune system recovery time (0.94 points), and not interacting with foods high in tyramine (0.86 points). When considering clinical management convenience, the average US-based neurologist treating MS is likely to choose ponesimod over siponimod, fingolimod, or ozanimod. The strongest driver of preferences was the number of drug–drug interactions. This information can help inform recommendations for the treatment of MS and facilitate shared decision-making between clinicians and patients.

Background: Percentage uptake of some meningococcal vaccines is low in the US. Understanding what drives vaccination preferences may help to increase vaccination rates. Objectives: To determine how attributes of meningococcal vaccines and the availability of a pentavalent (MenABCWY) vaccine profile drive adolescents' and young people's (AYP's) willingness to be vaccinated and parents' and legal guardians' (PLG') willingness for their child to be vaccinated (WTV). To also explore how preferences for meningococcal vaccines vary by participant characteristics. Methods: Vaccine preferences were elicited in a discrete choice experiment (DCE) with AYP aged 16-23 years and PLG of adolescents aged 11-17 years. Participants chose between two hypothetical vaccine profiles that differed in level of protection, dosing, and risks of mild-to-moderate and severe side effects, and a no vaccination profile. Main outcome measures were relative attribute importance (RAI) and WTV. RAI measured the maximum contribution of an attribute to vaccination choice relative to other attributes. WTV compared predicted choice probabilities for the three vaccine profiles. Results: 407 AYP and 394 PLG participated (50.9% male, 78.4% White/Caucasian). Irrespective of vaccine attributes, 59.5% always opted into vaccination and 3.6% always opted out of vaccination. The most important attributes were level of protection (RAI: 33.7%) and risk of mild-to-moderate side effects (RAI: 32.3%). Dosing was more important to PLG (RAI: 5.9%) than AYP (RAI: 2.0%; p < .01). Adding a pentavalent vaccine alternative increased WTV by 3.7 percentage points (PP) for PLG, 2.4 PP for AYP, 16.4 PP for vaccine-hesitant participants, 13.4 PP for participants without health insurance, and 9.6 PP for adults. Conclusion: Level of protection and risk of mild-to-moderate side effects were the most important vaccine attributes. Adding a pentavalent vaccine alternative increased WTV particularly among adults, individuals who were vaccine-hesitant, and individuals without health insurance.

BackgroundQualitative research is fundamental for designing discrete choice experiments (DCEs) but is often underreported in the preference literature. We developed a DCE to elicit preferences for vaccination against invasive meningococcal disease (IMD) among adolescents and young people (AYP) and parents and legal guardians (PLG) in the United States. This article reports the targeted literature review and qualitative interviews that informed the DCE design and demonstrates how to apply the recent reporting guidelines for qualitative developmental work in preference studies.MethodsThis study included two parts: a targeted literature review and qualitative interviews. The Medline and Embase databases were searched for quantitative and qualitative studies on IMD and immunization. The results of the targeted literature review informed a qualitative interview guide. Sixty-minute, online, semi-structured interviews with AYP and PLG were used to identify themes related to willingness to be vaccinated against IMD. Participants were recruited through a third-party recruiter's database and commercial online panels. Interviews included vignettes about IMD and vaccinations and three thresholding exercises examining the effect of incidence rate, disability rate, and fatality rate on vaccination preferences. Participant responses related to the themes were counted.ResultsThe targeted literature review identified 31 concepts that were synthesized into six topics for the qualitative interviews. Twenty AYP aged 16-23 years and 20 PLG of adolescents aged 11-17 years were interviewed. Four themes related to willingness to be vaccinated emerged: attitudes towards vaccination, knowledge and information, perception of IMD, and vaccine attributes. Most participants were concerned about IMD (AYP 60%; PLG 85%) and had positive views of vaccination (AYP 80%; PLG 60%). Ninety percent of AYP and 75% of PLG always chose vaccination over no vaccination, independent of IMD incidence rate, disability rate, or fatality rate.ConclusionWillingness to be vaccinated against IMD was affected by vaccine attributes but largely insensitive to IMD incidence and severity. This article provides an example of how to apply the recent reporting guidelines for qualitative developmental work in preference studies, with 21 out of 22 items in the guidelines being considered.

Objective Understanding preferences of patients with rheumatoid arthritis (RA) can facilitate tailored patient-centric care. This study elicited trade-offs that patients with RA were willing to make during treatment selection.Methods Patients with RA completed an online discrete choice experiment, consisting of a series of choices between hypothetical treatments. Treatment attributes were selected based on literature review and qualitative patient interviews. Eligible patients were ≥18 years old, diagnosed with RA, receiving systemic disease-modifying antirheumatic drug therapy, and residents of Europe or USA. Male patients were oversampled for subgroup analyses. Data were analysed using a correlated mixed logit model.Results Of 2090 participants, 42% were female; mean age was 45.2 years (range 18–83). Estimated effects were significant for all attributes (p<0.001) but varied between patients. Average relative attribute importance scores revealed different priorities (p<0.001) between males and females. While reducing pain and negative effect on semen parameters was most important to males, females were most concerned by risk of blood clots and serious infections. No single attribute explained treatment preferences by more than 30%. Preferences were also affected by patients’ age: patients aged 18–44 years placed less importance on frequency and mode of treatment administration (p<0.05) than older age groups. Patients were willing to accept higher risk of serious infections and blood clots in exchange for improvements in pain, daily activities or administration convenience. However, acceptable trade-offs varied between patients (p<0.05).Conclusion Treatment preferences of patients with RA were individual-specific, but driven by benefits and risks, with no single attribute dominating the decision-making.

Introduction New glucose-monitoring technologies have different cost–benefit profiles compared with traditional finger-prick tests, resulting in a preference-sensitive situation for patients. This study aimed to assess the relative value adults with diabetes assign to device attributes in two countries. Research design and methods Adults with type 1 or 2 diabetes from the Netherlands (n=226) and Poland (n=261) completed an online discrete choice experiment. Respondents choose between hypothetical glucose monitors described using seven attributes: precision, effort to check, number of finger pricks required, risk of skin irritation, information provided, alarm function and out-of-pocket costs. Panel mixed logit models were used to determine attribute relative importance and to calculate expected uptake rates and willingness to pay (WTP). Results The most important attribute for both countries was monthly out-of-pocket costs. Polish respondents were more likely than Dutch respondents to choose a glucose-monitoring device over a standard finger prick and had higher WTP for a device. Dutch respondents had higher WTP for device improvements in an effort to check and reduce the number of finger pricks a device requires. Conclusion Costs are the primary concern of patients in both countries when choosing a glucose monitor and would likely hamper real-world uptake. The costs-benefit profiles of such devices should be critically reviewed.

Background The rheumatoid arthritis (RA) treatment landscape is diverse, with multiple therapies available that differ in several attributes such as mode of administration and benefit-risk profile. Patients and prescribers face challenging trade-offs during treatment selection to accommodate patients' circumstances in order to ensure comprehensive disease management. EULAR recommendations for RA management emphasize the need to recognize patient preferences in shared decision-making (SDM). Therefore, it is essential to understand how preferences differ in the RA patient population. Objectives This study elicited trade-offs that RA patients were willing to make during treatment selection while accounting for preference heterogeneity. Methods An online discrete choice experiment (DCE) was conducted from September to October 2021 in which RA patients were required to elicit their preferences for attributes of treatments for RA (Figure 1) and make trade-offs between them. Attributes were selected and defined based on literature review and qualitative patient interviews, and were then tested in a quantitative pilot. Main data collection consisted of an online survey in which participants were asked to repeatedly choose between hypothetical treatments. Eligible patients were ≥18 years old, diagnosed with RA, currently received systemic disease-modifying anti-rheumatic drug therapy for RA, and were residents of France, Germany, Italy, Spain, United Kingdom, or United States. Male patients were oversampled to support subgroup analysis of preferences for effects on sperm parameters. Data were analyzed using a correlated mixed logit model and differences in preferences between sex and age were explored. Relative attribute importance (RAI) scores and maximum acceptable risk (MAR) measures were derived from the estimates. Results A total of 2,090 patients participated; 42% were female with predefined oversampling of male patients, with a mean age of 45.2 years (range 18–83). Estimated effects were significant for all attributes (p<0.001), implying that they all influenced treatment choices and suggesting preferences differed between participants. Average RAI scores revealed different priorities (p<0.001) between males and females (Figure 1). While reducing pain and negative effect on semen parameters was most important to male patients, female patients were most concerned by risk of blood clots and serious infections. The remaining attributes were of lower importance but were still relevant. However, no single attribute explained treatment preferences by more than 30%. Preferences were also affected by patients' age: patients aged 18-44 years placed less importance on frequency and mode of treatment administration (p<0.05) than older age groups. Patients were willing to make benefit-risk trade-offs; they accepted extra risks of blood clots (male: 1.8%; female: 0.8%), serious infections (male: 2.5%; female: 1.0%), or negative effects on sperm (male: 7.4%) for an oral pill every day instead of injection once a week. They also accepted extra risks of blood clots (male: 2.3%; female: 1.2%), serious infections (male: 3.2%; female: 1.6%), or negative effects on sperm (male: 10.4%) for reducing amount of pain from 30% to 10%. Similar observations were made for improved performance of daily activities. However, acceptable trade-offs varied between patients (p<0.05). Conclusion Preferences of RA patients were driven by benefits and risks of RA treatments, with no single attribute dominating the decision making. Patients were willing to accept higher risk of serious infections and blood clots in exchange for improvements in pain, daily activities, or administration convenience. These findings emphasize the importance of considering the entire treatment profile, including benefits, risks, and administration to support SDM between providers and patients.Preference drivers: males – pain, blood clots; females – blood clots, infections, pain. Sperm risk data are based on male responses only. Acknowledgements This study was funded by Galapagos NV (Mechelen, Belgium). Publication coordination was provided by Fabien Debailleul, PhD, of Galapagos NV. Medical writing support was provided by Brooke Middlebrook (Evidera) and publications management was provided by Aspire Scientific Ltd (Bollington, UK), funded by Galapagos NV. Disclosure of Interests Rieke Alten Consultant of: AbbVie, Amgen, Biogen, BMS, Celltrion, Gilead, Janssen, Lilly, Medac, MSD, Mylan, Novartis, Pfizer, Roche, Sandoz, Sanofi-Genzyme, UCB, Viatris, Juan Carlos Nieto González Speakers bureau: AbbVie, Amgen, Biogen, BMS, Celgene, FAES Farma, Gebro, Janssen, Lilly, MSD, Nordic Pharma, Novartis, Pfizer, Roche, Sandoz, Sanofi, UCB Pharma, Consultant of: AbbVie, Amgen, GSK, Galapagos, Janssen, Lilly, MSD, Peggy Jacques: None declared, Carlomaurizio Montecucco Speakers bureau: AbbVie, BMS, Boehringer, Eli Lilly, Galapagos, Pfizer, Roche, Sanofi, Consultant of: AbbVie, BMS, Gilead, Robert Moots Speakers bureau: Amgen, Galapagos, Consultant of: Ferring, Grant/research support from: Novartis, Helga Radner Speakers bureau: Gilead Sciences, Janssen, MSD, Pfizer Corporation Austria, Sebastian Heidenreich Employee of: Evidera Inc, which is part of Thermo Fisher Scientific's Clinical Research Group. Evidera received payment for conducting the work outlined in this work., Chiara Whichello Employee of: Evidera Inc, which is part of Thermo Fisher Scientific's Clinical Research Group. Evidera received payment for conducting the work outlined in this work., Nicolas Krucien Employee of: Evidera Inc, which is part of Thermo Fisher Scientific's Clinical Research Group. Evidera received payment for conducting the work outlined in this work., Monia Zignani Shareholder of: Galapagos, Employee of: Galapagos, Harald Vonkeman Speakers bureau: AbbVie, Boehringer Ingelheim, Galapagos, Janssen, Novartis, Pfizer, UCB, Grant/research support from: AbbVie, Boehringer Ingelheim, Galapagos, Janssen, Novartis, Pfizer, UCB, Katrien Van Beneden Shareholder of: Galapagos, Employee of: Galapagos.Figure 1RAI overall and by sex

PRODUCTS with janus kinase (JAK) inhibition have been shown to promote hair regrowth in patients with alopecia areata (AA). To guide drug-approval and treatment decisions, it is important to understand patients' willingness to accept the potential risks of JAK inhibition in exchange for potential benefits. We quantified the treatment preferences of adult (≥18 years) and adolescent patients (12-17 years) with AA in the US and Europe to determine the trade-offs they are willing to make between benefits and risks. Preferences for oral AA treatment attributes were elicited using a discrete choice experiment consisting of 12 tasks in which patients chose between two hypothetical treatment alternatives and no treatment. Benefits included the probability of 80%-100% scalp hair regrowth (Severity of Alopecia Tool score ≤ 20) and achieving moderate-to-normal eyebrow and eyelash hair. Treatment-related risks included 3-year probabilities of serious infection, cancer, and blood clots. Preference estimates were used to calculate the maximum level of each risk that patients were willing to accept for increases in treatment benefits. The most important attribute to both adults (n = 201) and adolescents (n = 120) was a 50% probability of achieving hair regrowth on most or all the scalp; however, adolescents placed greater relative importance on this attribute than did adults. Adults were averse to the risks of serious infection, cancer, and blood clots, whereas adolescents were averse to the risk of cancer. For a 20% increase in the probability of 80%-100% scalp hair regrowth, adults were willing to accept a mean (95% confidence interval) 3-year risk of serious infection, cancer, and blood clots of 7.4% (5.5-9.3), 2.5% (1.9-3.1), and 9.3% (6.4-12.2). Adolescents were willing to accept a 3-year risk of cancer of 3.3% (2.4-4.2). Patients with AA in the US and Europe are willing to accept substantial risks to obtain an effective treatment.

To explore factors driving young peoples' and parents' views on invasive meningococcal disease (IMD) and their willingness to vaccinate (WTV).

IntroductionLimited evidence exists for how patient preference elicitation methods compare directly. This study compares a discrete choice experiment (DCE) and swing-weighting (SW) by eliciting preferences for glucose-monitoring devices in a population of diabetes patients. MethodsA sample of Dutch adults with type 1 or 2 diabetes (n = 459) completed an online survey assessing their preferences for glucose-monitoring devices, consisting of both a DCE and a SW exercise. Half the sample completed the DCE first; the other half completed the SW first. For the DCE, the relative importance of the attributes of the devices was determined using a mixed-logit model. For the SW, the relative importance of the attributes was based on ranks and points allocated to the 'swing' from the worst to the best level of the attribute. The preference outcomes and self-reported response burden were directly compared between the two methods. ResultsParticipants reported they perceived the DCE to be easier to understand and answer compared to the SW. Both methods revealed that cost and precision of the device were the most important attributes. However, the DCE had a 14.9-fold difference between the most and least important attribute, while the SW had a 1.4-fold difference. The weights derived from the SW were almost evenly distributed between all attributes. ConclusionsThe DCE was better received by participants, and generated larger weight differences between each attribute level, making it the more informative method in our case study. This method comparison provides further evidence of the degree of method suitability and trustworthiness.

Objective: Patients have unique insights and are (in-)directly affected by each decision taken throughout the life cycle of medicinal products. Patient preference studies (PPS) assess what matters most to patients, how much, and what trade-offs patients are willing to make. IMI PREFER was a six-year European public-private partnership under the Innovative Medicines Initiative that developed recommendations on how to assess and use PPS in medical product decision-making, including in the regulatory evaluation of medicinal products. This paper aims to summarize findings and recommendations from IMI PREFER regarding i) PPS applications in regulatory evaluation, ii) when and how to consult with regulators on PPS, iii) how to reflect PPS in regulatory communication and iv) barriers and open questions for PPS in regulatory decision-making.Methods: PREFER performed six literature reviews, 143 interviews and eight focus group discussions with regulators, patient representatives, industry representatives, Health Technology Assessment bodies, payers, academics, and clincians between October 2016 and May 2022.Results: i) With respect to PPS applications, prior to the conduct of clinical trials of medicinal products, PPS could inform regulators' understanding of patients' unmet needs and relevant endpoints during horizon scanning activities and scientific advice. During the evaluation of a marketing authorization application, PPS could inform: a) the assessment of whether a product meets an unmet need, b) whether patient-relevant clinical trial endpoints and outcomes were studied, c) the understanding of patient-relevant effect sizes and acceptable trade-offs, and d) the identification of key (un-)favorable effects and uncertainties. ii) With respect to consulting with regulators on PPS, PPS researchers should ideally have early discussions with regulators (e.g., during scientific advice) on the PPS design and research questions. iii) Regarding external PPS communication, PPS could be reflected in the assessment report and product information (e.g., the European Public Assessment Report and the Summary of Product Characteristics). iv) Barriers relevant to the use of PPS in regulatory evaluation include a lack of PPS use cases and demonstrated impact on regulatory decision-making, and need for (financial) incentives, guidance and quality criteria for implementing PPS results in regulatory decision-making. Open questions concerning regulatory PPS use include: a) should a product independent broad approach to the design of PPS be taken and/or a product-specific one, b) who should optimally be financing, designing, conducting, and coordinating PPS, c) when (within and/or outside clinical trials) to perform PPS, and d) how can PPS use best be operationalized in regulatory decisions.Conclusion: PPS have high potential to inform regulators on key unmet needs, endpoints, benefits, and risks that matter most to patients and their acceptable trade-offs. Regulatory guidelines, templates and checklists, together with incentives are needed to foster structural and transparent PPS submission and evaluation in regulatory decision-making. More PPS case studies should be conducted and submitted for regulatory assessment to enable regulatory discussion and increase regulators' experience with PPS implementation and communication in regulatory evaluations.

INTRODUCTION:Ensuring patients have enough information about healthcare choices prior to completing a preference study is necessary to support the validity of the findings. Patients are commonly informed using text-based information with supporting graphics. Video-based information may be more engaging for the general patient population. This study aimed to assess (1) the impact that educating patients using video-based educational materials with a voiceover has on patient preferences compared to traditional text, and (2) whether this impact is consistent between two countries.MATERIALS AND METHODS:A video-based educational tool was developed to inform patients prior to completing a discrete choice experiment assessing preferences for glucose monitors. Patients with diabetes from the Netherlands and Poland were recruited through an online research panel. Respondents were randomised to receive information in either a text or a video with animations and a voiceover. Data were analysed using a mixed-logit model.RESULTS:N = 981 completed surveys were analysed from the Netherlands (n = 459) and Poland (n = 522). Differences were found between the countries, but no interpretable pattern of differences was found between the two types of educational materials. Patients spent less time in the educational material than would be necessary to fully review all of the content.CONCLUSIONS:Simply providing educational material in a video with animations and voiceovers does not necessarily lead to better engagement from respondents or different preference outcomes in a sample of diabetes patients when compared to text. Increasing engagement with educational materials should be a topic of future research for those conducting patient preference research as no amount of educational material will be helpful if respondents do not access it.

To evaluate benefit-risk profiles of two doses of ritlecitinib (50 mg vs. 30 mg daily) integrating patient preferences and clinical efficacy estimates for ritlecitinib, an investigational treatment for alopecia areata (AA).

When assessing patient preferences, it is necessary to ensure that patients are informed prior to completing a preference study as this supports the validity of the findings. Most often researchers inform patients through the use of text-based information which may be supported with simple graphics. However, video-based information may be more accessible to the general patient population where health literacy may be an issue. This study aimed to assess 1) the impact that educating patients using video-based educational materials with a voiceover has on patient preferences compared to traditional text, and 2) whether this impact is consistent between two countries. A video-based educational tool was developed using a structured approach in order to inform patients prior to completing a discrete choice experiment assessing their preferences for glucose monitors. Patients from two countries (the Netherlands and Poland) were recruited through an online research panel. Respondents were randomized to receive information using either text with simple graphics or a video with animations and a voiceover. Data was analyzed using a mixed-logit model. N=981 completed surveys were analyzed from the Netherlands (n=459) and Poland (n=522). Differences in preferences were found between the countries, but no pattern of differences were found between the two types of educational materials. Patients spent less time in the educational material than would be necessary to fully view the information. Simply providing educational material in a video with animations and voiceovers does not necessarily lead to better informed respondents or different preference outcomes in the general diabetes population when compared to the traditional text format. Future research should look at ways to increase engagement with educational materials where a need for information has been identified.