Patient-Patient Centered Outcomes Research

Public Preferences and Willingness to Pay for a Multidisciplinary Colorectal and Pelvic Reconstruction Service
Pan T, Trajanoversuska M, Meng Y, Best S, King SK and Goranitis I
Children with congenital colorectal conditions require care from multiple health professionals. However, evidence on the value of a multidisciplinary care model is lacking. This study aimed to elicit public preferences and willingness to pay for a multidisciplinary care model for children with congenital colorectal conditions.
ePROMs are Best Used Alongside 'A High-Quality Conversation' as Part of a Therapeutic Relationship. A Qualitative Study of Children with Life-Altering Skin Conditions
Tyack Z, Simons M, Hartshorn E, Kimble RM and Killey J
The benefits and barriers of using patient-reported outcome measures (PROMs) in routine clinical practice have been examined extensively, but there has been limited in-depth exploration of the experience of PROMs by stakeholders and consumers. This study sought to understand the experiences of children with life-altering skin conditions, their caregivers and treating health professionals regarding the routine use of electronic PROMs.
How Important is Healthcare-Contact Time to Systemic Treatment Decision-Making in Advanced Gastrointestinal Cancers: Developing Attributes to Include in a Discrete Choice Experiment
Stevens SX, El-Katateny E, Addo IY, Street D, Booth C, Shaw J, Vardy JL and De Abreu Lourenco R
People receiving treatment for advanced cancer invest substantial portions of their survival time receiving healthcare, labelled the 'time toxicity' of treatment. Although qualitative research has examined the impact of time burden on patients and their caregivers, its influence on treatment decision-making is unclear.
Factors influencing Patient Preferences for BRCA Testing and Adjuvant Therapy in HER2-Negative Early Breast Cancer in the United States: Best-Worst Scaling and Discrete Choice Experiment
Mishkin K, Li Q, Earla JR, Mejia JA, Hirshfield KM, Krupsky K, Lankin J, Beusterien K, Mulvihill E, Honomichl R, Gordon A and Xu X
Poly(ADP-ribose) polymerase inhibitors (PARPi) have survival benefits for patients with high-risk (High-risk disease is defined per the phase III OlympiA trial as follows: for triple-negative breast cancer, residual disease after neoadjuvant chemotherapy or node-positive or ≥ 2 cm tumors after adjuvant chemotherapy; for hormone receptor-positive disease, four or more positive nodes after adjuvant chemotherapy or a CPS + EG score ≥ 3 after incomplete response to neoadjuvant chemotherapy. The CPS + EG score accounts for clinical/pathologic stage, ER status, and grade (Giaquinto et al. in CA Cancer J Clin 72:524-541, 2022)), human epidermal growth factor receptor 2 (HER2)-negative early breast cancer (eBC) with germline BReast CAncer gene mutations (gBRCAm). However, many patients are unaware of their gBRCA status; this can impact eligibility for targeted treatment. We sought to evaluate patient preferences for BRCA testing and treatment decision-making as they relate to HER2-negative eBC.
Reducing Primary Healthcare Bypass Behaviour: A Discrete Choice Experiment Study Exploring the Preferences for Primary Eye Care Services in Rural Xinjiang
Dong X, Zhao J, Zhao Z, Su C and Ma X
Bypassing primary healthcare (PHC) in favor of higher-level providers is a persistent issue in many low- and middle-income settings, particularly in rural China, where primary eye care (PEC) remains underutilized. Understanding patient preferences and determinants of care-seeking behavior is crucial for promoting patient-centered outcomes and strengthening PHC utilization.
Patient Preferences in Anticoagulation Treatment: A Review of Discrete Choice Experiments
Brugman IM, van Eikenhorst L, Schlinkert C and Wagner C
Patient engagement is expected to improve the safety and efficacy of anticoagulant medication. By identifying the factors and patient characteristics influencing patients' decisions regarding anticoagulant medication, we aim to support healthcare professionals regarding patient engagement. This review of discrete choice experiments (DCEs) explores the considerations and preferences of patients with various underlying conditions when choosing their anticoagulant medication. Two international databases were searched in March 2024: PubMed and Web of Science Core Collection. The search was updated in July 2025. Eligible studies included original DCE studies that explored the considerations and preferences of patients and covered long-term anticoagulant use beyond the hospital setting (direct oral anticoagulants, vitamin K antagonists, antiplatelet agents, and low molecular weight heparins). The initial search identified a total of 174 records, after which two authors independently assessed the articles for both the title and abstract and the full-text inclusion rounds. After the update, this resulted in the inclusion of 13 articles. The PRISMA 2020 statement was followed and the quality of the included studies was assessed. The results show that overall patients prioritize safety and effectiveness of anticoagulants over convenience factors. If only convenience factors were taken into account, frequency of administration emerges as the most important. Subgroup analysis showed that patient preferences vary based on geographic, demographic, and socioeconomic factors, health status, as well as previous experiences with anticoagulation medication or related health issues. By acknowledging the impact of these diverse factors on patient preferences, healthcare professionals can better support safe and effective anticoagulant care tailored to the needs of individual patients.
Feasibility of Routine Quality-of-Life Measurement in Residential Aged Care: Results from a Pilot Study in Australia
Engel L, Devlin N, Dow B, Gilbert A, Mulhern B, Peasgood T, Viney R and Batchelor F
This study presents findings from a pilot study that aimed to examine the feasibility of routine measurement of quality of life in residential aged care, including the examination of barriers to and facilitators of collecting and using that data to improve quality of care.
A Systematic Review of Patient-Reported Measures for Individuals Who Underwent Genetic Testing for Heritable Cancer
Raspin K, Nowakowski D, Dickinson JL and Roydhouse J
Advances in genomic technologies have driven a substantial shift in cancer care, including early screening and targeted interventions for high-risk individuals who have not received a cancer diagnosis. Understanding patients' experience of care and their associated outcomes is essential to effectively delivering precision medicine. These outcomes are usually evaluated through patient-reported measures (PRMs), rather than administrative data.
Patient-Reported Outcomes in Early-Phase Oncology Clinical Trials: A Stepping Stone to a Patient-Centered Drug Development
Fiteni F, Meilhoc A, Blin O and Haenel E
In phase I clinical trials, the recommended phase II dose (RP2D) is usually set at or near the maximum tolerated dose (MTD), which is determined based on the observation of dose-limiting toxicities (DLTs). Clinicians typically evaluate toxicities using the National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE), with grade three or higher toxicities classified as DLTs. However, it has been repeatedly demonstrated that physicians tend to underestimate patient's symptoms. Therefore, patient-reported outcomes (PROs), especially the NCI PRO-CTCAE questionnaire, can complement clinician assessments by providing direct patient input on adverse events. This integration could lead to a more accurate definition of DLT and better informed RP2D decisions. Moreover, PROs could optimize sample size strategies in later-stage trials and enable comparison of health-related quality of life (HRQoL) data with synthetic control arms to confirm the benefit of a drug, especially in rare oncogene-driven subsets. Whilst stakeholders and regulatory authorities acknowledge the value of integrating PROs early in drug development, they emphasize the lack of methodological guidelines to support broader adoption. The integration of PROs represents an opportunity to improve the patient-centeredness of phase I trials, ultimately strengthening the drug development process.
Acknowledgement to Referees
Correction: Preferences for Neurodevelopmental Follow-Up Care for Children: A Discrete Choice Experiment
Sharma P, Kularatna S, Abell B, McPhail SM and Senanayake S
Patient Insights on Acne Hyperpigmentation, Trifarotene, and Skincare: 6-Month, LEAP Phase 4 Qualitative Exit Interview Findings
Dias-Barbosa C, Taylor N, Chan K, York JP, Puelles J and Chavda R
Acne-induced hyperpigmentation (AIH) has a considerable impact on patients' quality of life (QoL). In the phase 4 LEAP trial, patients treated with the topical retinoid trifarotene showed faster improvement in AIH compared to patients treated with its vehicle cream (VC) at week (W) 12.
The Patient Voice in Atopic Eczema Guidelines: How Do We Make it Standard Practice?
Arents B, Capozza K, Dlova NC, Funk M, Koh MJA and Ogola R
Patient and caregiver involvement in developing clinical guidelines is widely recommended. However, among current atopic eczema guidelines, only a minority incorporates the patient perspective. Much of the general guidance on patient involvement can be applied to atopic eczema. However, there are specific factors to consider in order to embed sustainable patient involvement in the atopic eczema guideline development process. These include the selection of patients and caregivers to represent the heterogeneity of the disease and of the patient experience. The psychosocial impact of the disease suggests support requirements that enable patients to be equal partners with other stakeholders. The priorities and feedback of patients  with atopic eczema can differ from those of healthcare professionals and are central to shaping the research and implementation that form part of a guideline cycle. Barriers to patient involvement in terms of resources or healthcare policy need to be addressed so that patients and caregivers can be active participants in developing guidelines that are culturally, racially, socially and geographically appropriate. We suggest that steps can be taken to facilitate patient involvement in patients and caregivers living with atopic eczema and offer a framework to support patient involvement as standard practice in guideline development.
Women's Preferences in Early Pregnancy Loss Management: A Focus on Psychological Considerations Using the Analytic Hierarchy Process
Peters MEC, Icks A and Dintsios CM
Early pregnancy loss (EPL) is associated with significant emotional burden. While multiple management options exist, psychological aspects are often underrepresented in decision-making support. The objective of this study is to identify criteria that are important to women when choosing between expectant, medical, and surgical management of EPL, with particular attention to psychological aspects.
Who Prefers Death to Life in Composite Time Trade-off Interviews, and Why? A Mixed-Methods Study among Asians in Singapore
Cheng LJ, Vasan Thakumar A, Chen LA, Tan RL, Liao M and Luo N
The EuroQol Valuation Technology (EQ-VT) uses composite time trade-off (cTTO) with a 10-year lead-time to value health states worse-than-dead (WTD) ranging between - 1 and 0 (dead). While WTD responses are common in EQ-5D-5L studies, their drivers remain understudied in Asia. This mixed-methods study explored socio-demographic factors and reasoning behind WTD preferences in Singapore.
Eliciting Patient-Centric Value Parameters: A National Best-Worst Scaling Profile Case Survey for Second-Line Antidiabetic Drugs in China
Lian D, Liu J, Wei Y, Yang Y, Ren Y, Bao S, Liu L, Liu S and Chen Y
China faces the world's largest diabetes burden, with a 12.4% prevalence. A wide range of second-line therapies is available for patients with type 2 diabetes mellitus (T2DM), each with distinct characteristics. Although current guidelines advocate for a patient-centered approach in selecting second-line treatments, empirical evidence on Chinese patient's preferences for these therapies remains limited. We therefore elicited population‑level preferences using a Best-Worst Scaling profile‑case (BWS‑2) survey to inform decision making.
Correction: Patients' Needs and Preferences for Cardiac Pacemaker Implantation: A Qualitative Study on Disease and Medical Device Characteristics to Inform a Quantitative Preference Study
Vanneste A, Sinnaeve P, Huys I, Adriaenssens T and Garweg C
Public Preferences for Emerging Cancer Screening Modalities: A Systematic Review of Discrete Choice Experiments
Har CE, Gao Q, Chen W and Wang Y
Early detection of cancer reduces mortality and morbidity, but conventional screening methods often face challenges such as invasiveness, limited accessibility and high resource demands. Emerging cancer screening technologies could overcome these barriers, yet their adoption depends heavily on public acceptance. This systematic review synthesises evidence from discrete choice experiment (DCE) studies examining population preferences for emerging cancer screening technologies.
Eliciting Patient Preferences for the Hospital at Home Programme in Singapore; A Discrete Choice Experiment
Kularatna S, Leng LL, Jen MTW, Hoe TW, Boon TW, Feng LY, Graves N and Senanayake S
Hospital overcrowding in Singapore has reached a critical level, primarily owing to an ageing population and an increased demand for medical services. Alternative care models such as hospital-at-home (H@H) programmes have been introduced to address this issue, utilising telehealth and home-based care. We conducted a choice experiment to determine the most preferred characteristics of a hospital-at-home programme in Singapore.
PAIRED: Quantitative and Qualitative PAtIent-Reported Experiences and perceiveD Benefit of Treatment with Dolutegravir/Lamivudine in the United States
Slim J, Brogan AP, Harper G, Mycock K, McMillan A, Merrill D and Verdier G
Understanding the treatment experiences and satisfaction of people with HIV using two-drug regimens, including dolutegravir/lamivudine (DTG/3TC), is crucial to assess ongoing needs and meet Ending the HIV Epidemic goals.
Strategies to Translate PROMs to Linguistically Diverse and Lesser-Spoken Languages: Lessons from Wales
Palmer RI, Waddington H and Withers K
Patient-reported outcome measures (PROMs) are widely implemented in Wales to achieve Value-Based Healthcare (VBHC). All patient-facing information in Wales must be available bilingually; therefore, Welsh-language PROM translation is necessary. Conceptually equivalent and patient-friendly translations of PROMs are essential to ensure that the data they collect are equivalent to that collected via their corresponding original language versions. As a result, the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) have developed best practice principles for PROM translations. This article reflects on a body of work undertaken over 8 years between 2016 and 2024 to translate a series of PROMs from English to Welsh by attempting to follow these principles. While effective in most areas, they do not provide guidance on reaching an appropriate register in languages with more than one register, dealing with multiple dialects, or how to recruit enough participants for cognitive debriefing when there are limited numbers of native speakers. Lessons have been learned while trying to manage these challenges in Wales, and recommendations for future practice are provided.