The roles and potential value of patient preference (PP) data in health technology assessment (HTA) remain to be fully realized despite an expanding literature and various efforts to establish their utility. This article reports lessons learned through a series of collaborative workshops with HTA representatives, organized by the Health Technology Assessment International’s Patient Preferences Project Subcommittee.

Five online workshops were conducted between June 2022 and June 2023, seeking to facilitate collaborative learning and reflection on ways that PP data can be integrated into HTA. Participants included nine HTA representatives from the United States, Canada, Australia, England, and the Netherlands. Workshops were recorded, transcribed, and thematically analyzed.

Despite appreciating the value of PP data, participants were ambivalent about their use in HTA. Some felt that they were already getting the information they needed from the cost-effectiveness analysis or existing patient involvement processes. Others thought that PP data would be very helpful at the initial and final stage of the decision-making process and, particularly, in the following cases: (a) when technology has important non-health benefits; (b) when the clinical and/or cost-effectiveness evidence is marginal; and (c) when treatment is indicated for a large and heterogeneous population. Issues related to the validity and reliability of PP studies were frequently raised, with preference heterogeneity at the core of these concerns.

Collaborating with HTA representatives in the “co-creation” of PP research can help address their concerns and facilitate mutual learning about how PP data can be used in HTA.

Antipsychotic-induced weight gain (AIWG) is a substantial contributor to high obesity rates in psychiatry. Limited management guidance exists to inform clinical practice, and individuals with experience of managing AIWG have had no or minimal input into its development. A lack of empirical research outlining patient values and preferences for management also exists. Recommendations addressing weight management in psychiatry may be distinctly susceptible to ideology and sociocultural values regarding intervention appropriateness and expectations of self-management, reinforcing the need for co-produced management guidance. This study is the first to ask: how do individuals conceptualise preferred AIWG management and how can this be realised in practice?

1. Explore the management experiences of individuals with unwanted AIWG. 2. Elicit their values and preferences regarding preferred management.

Qualitative descriptive methodology informed study design. A total of 17 participants took part in semi-structured interviews. Data analysis was undertaken using reflexive thematic analysis.

Participants reported that clinicians largely overestimated AIWG manageability using dietary and lifestyle changes. They also reported difficulties accessing alternative management interventions, including a change in antipsychotic and/or pharmacological adjuncts. Participants reported current management guidance is oversimplified, lacks the specificity and scope required, and endorses a ‘one-size-fits-all’ management approach to an extensively heterogenous side-effect. Participants expressed a preference for collaborative AIWG management and guidance that prioritises early intervention using the range of evidence-based management interventions, tailored according to AIWG risk, participant ability and participant preference.

Integration of this research into guideline development will help ensure recommendations are relevant and applicable, and that individual preferences are represented.

Wearable digital health technologies (DHTs) have the potential to improve chronic kidney disease (CKD) management through patient engagement. This study aimed to investigate and elicit preferences of individuals with CKD toward wearable DHTs designed to support self-management of their condition.

Using the results of our review of the published literature and after conducting qualitative patient interviews, five-choice attributes were identified and included in a discrete-choice experiment. The design consisted of 10-choice tasks, each comprising two hypothetical technologies and one opt-out scenario. We collected data from 113 adult patients with CKD stages 3–5 not on dialysis and analyzed their responses via a latent class model to explore preference heterogeneity.

Two patient segments were identified. In all preference segments, the most important attributes were the device appearance, format, and type of information provided. Patients within the largest preference class (70 percent) favored information provided in any format except the audio, while individuals in the other class preferred information in text format. In terms of the style of engagement with the device, both classes wanted a device that provides options rather than telling them what to do.

Our analysis indicates that user preferences differ between patient subgroups, supporting the case for offering a different design of the device for different patients’ strata, thus moving away from a one-size-fits-all service provision. Furthermore, we showed how to leverage the information from user preferences early in the R&D process to inform and support the provision of nuanced person-centered wearable DHTs.

Enhancing cancer patients’ sense of control can positively impact psychological well-being. We developed and assessed the psychometric properties of Valued Outcomes in the Cancer Experience (VOICE)TM, a measure of patients’ perceived control over key personal priorities within their cancer experience.

VOICE construction and testing were completed in three phases with separate participant samples: (1) item generation and initial item pool testing (N = 459), (2) scale refinement (N = 623), and (3) confirmatory validation (N = 515).

A 21-item measure was developed that captures cancer patients’ sense of control in seven key domains: (1) Purpose and Meaning, (2) Functional Capacity, (3) Longevity, (4) Quality Care, (5) Illness Knowledge, (6) Social Support, and (7) Financial Capability. VOICE demonstrated adequate internal consistency (full-scale α = 0.93; factor α = 0.67–0.89) and adequate to strong convergent and discriminatory validity.

VOICE measures cancer patients’ perceived control across a diverse range of personal priorities, creating a platform for elevating patient perspectives and identifying pathways to enhance patient well-being. VOICE is positioned to guide understanding of the patient experience and aid the development and evaluation of supportive care interventions to enhance well-being.

This research examined the perspective of the Huntington’s disease (HD) community regarding the use of predictive biomarkers as endpoints for regulatory approval of therapeutics to prevent or delay the onset of clinical HD in asymptomatic mutation carriers.

An online, choice-based conjoint survey was shared with HD community members including untested at-risk individuals, presymptomatic mutation carriers, and symptomatic individuals. Across 15 scenarios, participants chose among two proposed therapies with differing degrees of biomarker improvement and side effects or a third option of no treatment.

Two hundred and thirty-eight responses were received. Attributes reflecting biomarker efficacy (e.g., prevention of brain atrophy on magnetic resonance imaging, reduced mutant huntingtin, or reduced inflammation biomarkers) had 3- to 7-fold greater importance than attributes representing side effects (e.g., increased risk of heart disease, cancer, and stroke over 20 years) and were more influential in directing choice of treatments. Reduction in mutant huntingtin protein was the most valued attribute overall. Multinomial logit model simulations based on survey responses demonstrated high interest among respondents (87–99% of the population) for drugs that might prevent or delay HD solely based upon biomarker evidence, even at the risk of serious side effects.

These results indicate a strong desire among members of the HD community for preventive therapeutics and a willingness to accept significant side effects, even before the drug has been shown to definitively delay disease onset if the drug improves biomarker evidence of HD progression. Preferences of the HD community should inform regulatory policies for approving preventive therapies.

Consideration of ethical, legal, and social issues plus patient values (ELSI+) in health technology assessment (HTA) is challenging because of a lack of conceptual clarity and the multi-disciplinary nature of ELSI+. We used concept mapping to identify key concepts and inter-relationships in the ELSI+ domain and provide a conceptual framework for consideration of ELSI+ in HTA.

We conducted a scoping review (Medline and EMBASE, 2000–2016) to identify ELSI+ issues in the HTA literature. Items from the scoping review and an expert brainstorming session were consolidated into eighty ELSI+-related statements, which were entered into Concept Systems® Global MAX™ software. Participants (N = 38; 36 percent worked as researchers, 21 percent as academics; 42 percent self-identified as HTA experts) sorted the statements into thematic groups, and rated them on importance in making decisions about adopting technologies in Canada, from 1 (not at all important) to 5 (extremely important). We used Concept Systems® Global MAX™ software to create and analyze concept maps with four to sixteen clusters.

Our final ELSI+ map consisted of five clusters, with each cluster representing a different concept and the statements within each cluster representing the same concept. Based on the concepts, we named these clusters: patient preferences/experiences, patient quality of life/function, patient burden/harm, fairness, and organizational. The highest mean importance ratings were for the statements in the patient burden/harm (3.82) and organizational (3.92) clusters.

This study suggests an alternative approach to ELSI+, based on conceptual coherence rather than academic disciplines. This will provide a foundation for incorporating ELSI+ into HTA.

Neuroendocrine tumors (NETs) are rare, slow-growing malignant tumors. So far, there are no data on patient preferences regarding its therapy. This empirical study aimed to elicit patient preferences in the drug treatment of NET.

Based on qualitative patient interviews and an analytic hierarchy process, six patient-relevant attributes were analyzed and weighted using a discrete-choice experiment. Patients were recruited with the help of a NET support group. An experimental 3*3 + 6*3 –MNL design was created using NGene. The design consisted of eighty-four choices, divided into seven blocks. Participants were randomly assigned to these blocks. The analysis included random parameter logit and latent class models.

A total of 275 participants (51.6 percent female; mean age, 58.4 years) were included. The preference analysis within the random parameter logit model, taking into account the 95 percent confidence interval, showed predominance for the attribute “overall survival.” The attributes “response to treatment” and “stabilization of tumor growth” followed. The side effects “nausea/vomiting” and “diarrhea” were considered of relatively equal importance. Latent class analysis of possible subgroup differences revealed three preference patterns.

Preferences can influence therapeutic decisions. Preference analyses indicated that “overall survival” had the strongest influence, with participants clearly weighing outcome attributes higher than side effect attributes. In conclusion, mono-criterial decisions would not fully reflect patient perspectives.

Health-related quality of life (HRQoL) data generated by generic, preference-based instruments (i.e., EQ-5D) are highly demanded in health policy decision making, because they allow for direct comparisons of HRQoL outcomes between disease areas. We aimed to quantify HRQoL outcomes in breast cancer (BC), rheumatoid arthritis (RA), multiple sclerosis (MS), rare cancers (RC), and rare disease (RD) patients and understand the patterns that differentiate HRQoL outcomes between these disease areas, and more specifically between rare and more common disease population groups.

An international, Web survey of patients measured HRQoL (EQ-5D-5L), self-perceived health (EQ-5D-5L Visual Analogue Scale), and additional QoL dimensions, such as patient disability level.

We received 675 completed responses. Average utility loss was 53.5 percent, 32.5 percent, and 33.3 percent for RD, RA, and MS patients, respectively, in contrast to 18.6 percent for BC and RC patients. Statistically significant differences (p < .05) were observed between disease groups in all EQ-5D-5L domain outcomes, apart from that of “Anxiety/Depression.” Severe and/or extreme problems were reported in performing usual activities for RD and RC (34 percent and 13 percent of overall problems reported respectively), mobility for MS (18 percent), pain/discomfort for RA (13 percent), and anxiety/depression for BC (7 percent) patients.

We demonstrated significant differences in the dimensions that drive HRQoL outcomes between rare and more common diseases and showcased that the same EQ-5D utility may reflect very different severities depending on the patient population under investigation. Future research should examine whether outcomes in other, critical HRQoL domains not included in generic measures also highlight significant differences across disease areas.

Our aim was to gain insight into the perspectives of patients, close relatives, nurses, and physicians on medication management for patients with a life expectancy of less than 3 months.

We conducted an empirical multicenter study with a qualitative approach, including in-depth interviews with patients, relatives, nurses, specialists, and general practitioners (GPs). We used the constant comparative method and ATLAS.ti (v. 7.1) software for our analysis.

Saturation occurred after 18 patient cases (76 interviews). Some 5 themes covering 18 categories were identified: (1) priorities in end-of-life care, such as symptom management and maintaining hope; (2) appropriate medication use, with attention to unnecessary medication and deprescription barriers; (3) roles in decision making, including physicians in the lead, relatives' advocacy, and pharmacists as suppliers; (4) organization and communication (e.g., transparency of tasks and end-of-life conversations); and (5) prerequisites about professional competence, accessibility and quality of medical records, and financial awareness. Patients, relatives, nurses, specialists, and GPs varied in their opinions about these themes.

This study adds to our in-depth understanding of the complex practice of end-of-life medication management. It provides knowledge about the diversity of the perspectives of patients, close relatives, nurses, and physicians regarding beliefs, attitudes, knowledge, skills, behavior, work setting, the health system, and cultural factors related to the matter. Our results might help to draw an interdisciplinary end-of-life medication management guide aimed at stimulating a multidisciplinary and patient-centered pharmacotherapeutic care approach.

Communication based on patient preferences can alleviate their psychological distress and is an important part of patient-centered care for physicians who have the task of conveying bad news to cancer patients. The present study aimed to explore the demographic, medical, and psychological factors associated with patient preferences with regard to communication of bad news.

Outpatients with a variety of cancers were consecutively invited to participate in our study after their follow-up medical visit. A questionnaire assessed their preferences regarding the communication of bad news, covering four factors—(1) how bad news is delivered, (2) reassurance and emotional support, (3) additional information, and (4) setting—as well as on demographic, medical, and psychosocial factors.

A total of 529 outpatients with a variety of cancers completed the questionnaire. Multiple regression analyses indicated that patients who were younger, female, had greater faith in their physician, and were more highly educated placed more importance on “how bad news is delivered” than patients who were older, male, had less faith in their physician, and a lower level of education. Female patients and patients without an occupation placed more importance on “reassurance and emotional support.” Younger, female, and more highly educated patients placed more importance on “additional information.” Younger, female, and more highly educated patients, along with patients who weren't undergoing active treatment placed more importance on “setting.”

Patient preferences with regard to communication of bad news are associated with factors related to patient background. Physicians should consider these characteristics when delivering bad news and use an appropriate communication style tailored to each patient.

Antidepressants are frequently prescribed but results regarding their efficacy have been equivocal for different spectra of the severity continuum and their side-effects are often burdensome. Non-adherence is a likely consequence. The objective was therefore to examine patients’ trade-offs between the efficacy, side-effects and other drawbacks of antidepressants and whether these trade-offs predicted non-adherence.

Trade-offs from 225 antidepressant users, recruited through community pharmacies, were assessed with an Adaptive Conjoint Analysis (ACA) choice task that was customized to each individual patient. From the estimated utilities, relative importance scores of treatment properties were calculated. Non-adherence was measured through self-report and pharmacy refill data.

Relapse prevention and symptom relief were on average equally important. Side-effects were as important and the side-effect stomach and intestine complaints was on average even slightly more important than relapse prevention and symptom relief. Additional treatment with psychotherapy was preferred by 61% of the patients. A benefit/drawback ratio revealed that 18% of the patients did not consider the efficacy to outweigh the drawbacks. A higher benefit/drawback ratio was associated with a decreased odds of intentional non-adherence [odds ratio (OR) 0.2, 95% confidence interval (CI) 0.07–0.7, Wald = 6.7, p = 0.01).

For nearly one in five patients, the efficacy of antidepressants does not outweigh their drawbacks. Knowing patients’ trade-offs is likely to aid both physicians and patients to identify important treatment preferences, to improve adherence and to make more deliberate decisions on whether or not to continue treatment.

To explore patients' understanding of decision making in the treatment of advanced cancer and to determine the factors they believe important to these processes in their care.

Surveys were distributed to consecutive outpatients with advanced malignancy attending a comprehensive cancer treatment center.

Patients believed that the medical condition (94%), their doctors' experience (81%), and the medical literature (73%) are the most important factors for decisions made in their care. They also value their relationship with the doctor (63%) and their own (the patients') values (63%), and just over a third considered their family's values and the doctors' personality important. Most did not believe the doctors' values should influence decisions made. They were mindful of the uncertainty involved in decisions in the setting of advanced cancer.

Overall, patients were satisfied with the decision-making processes and they understood and highly regarded the incorporation of factors, other than their medical condition, in their care.

To compare induction, pre- and post-discharge recovery characteristics and patient preferences between four anaesthetic regimens in adult day-surgery.

Randomized controlled trial. In all, 1158 adults assigned to: propofol induction and maintenance, propofol induction with isoflurane/N2O, or sevoflurane/N2O maintenance, or sevoflurane/N2O alone. We prospectively recorded induction and pre-discharge recovery characteristics, collected 7-day post-discharge recovery characteristics using patient diaries and patient preferences by telephone follow-up.

Recruitment rate was 73% – of the 425 refusals, 226 were not willing to risk a volatile induction. During induction, excitatory movements and breath holding were more common with sevoflurane only (P < 0.01). Injection pain and hiccup were more common with propofol induction (P < 0.01). In the recovery room and the postoperative ward, both nausea and vomiting were more common with sevoflurane only (P < 0.01). This difference disappeared within 48 h. There was no difference between groups in the mental state on awakening, recovery time, time to discharge or overnight admissions; then was also no difference in pain between the four groups for each of the seven postoperative days (P < 0.01), nor any differences in concentration or forgetfulness. Patients took 6.5 days (95% CI: 6.0–7.0, n = 693) to resume normal activities. Patients who received sevoflurane only were more likely to recall an unpleasant induction and least likely to want the same induction method again (P < 0.01).

Differences in outcome between the four regimens are transient; sevoflurane is not an ideal sole agent for adult day case anaesthesia and, in this setting, patients base their preferences for future anaesthetics on the method of induction.

Despite growing recognition of the importance of communication with children with life-limiting illnesses and their families, there has been limited research that includes the child's perspective. The purpose of the current study was to identify the aspects of physician communication that children with life-limiting illnesses and their parents perceived to be facilitative or obstructive in pediatric palliative care.

This qualitative study reports on the first 20 parent and child pairs of pediatric oncology and cardiology patients (mean age 14.25 years, range 9-21 years) with a poor prognosis (physician reported likely <20% chance of survival beyond 3 years) from two children's hospitals and one pediatric hospice in Los Angeles, California. Perspectives on physician communication were elicited from children's and parents' individual narratives, recorded, coded, and analyzed using qualitative grounded theory methodology.

Both children and parents identified five domains of physician communication deemed to be highly salient and influential in quality of care. These included relationship building, demonstration of effort and competence, information exchange, availability, and appropriate level of child and parent involvement. Parents identified coordination of care as another important communication domain. The characteristics of physicians that were deemed most harmful to satisfying communication included having a disrespectful or arrogant attitude, not establishing a relationship with the family, breaking bad news in an insensitive manner, withholding information from parents and losing their trust, and changing a treatment course without preparing the patient and family.

The six positive communication domains are areas for clinicians to recognize and monitor in communicating with children and families in the pediatric palliative care setting. Knowledge of the qualities of communication that are satisfying to and valued by children and their parents have the potential to lead to more effective communication around the difficult decisions faced by physicians, parents, and children with life-threatening conditions.