Treatment preferences for preventive interventions for rheumatoid arthritis: protocol of a mixed methods case study for the Innovative Medicines Initiative PREFER project

Falahee, Marie; Simons, Gwenda; DiSantostefano, Rachael L.; Valor‐Méndez, Larissa; Radawski, Christine; Englbrecht, Matthias; Bywall, Karin Schölin; Tcherny-Lessenot, Stéphanie; Kihlbom, Ulrik; Hauber, Brett; Veldwijk, Jorien; Raza, Karim

doi:10.1136/bmjopen-2020-045851

Cited by 9 publications

(12 citation statements)

References 52 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…This study is part of a case study for the Innovative Medicines Initiative project ‘PREFER’ (Patient Preferences in Benefit-Risk Assessments during the Medical Product Lifecycle), which aimed to develop evidence-based recommendations on how and when preference studies can inform decision-making during drug development. 14 The case study protocol 27 and other results from the case study 21 have been published previously.…”

Section: Methodsmentioning

confidence: 99%

“…The selection of treatment attributes included in the PTT was informed by a literature review, 17 a qualitative study and an attribute ranking survey. 27 The final attributes ( table 1 ) were agreed upon by an international team of clinical researchers, consultant rheumatologists, preference elicitation experts and patient partners. Attribute levels were estimated with input from clinical experts, including researchers leading the development and/or clinical trials of preventive treatments for RA.…”

Section: Methodsmentioning

confidence: 99%

See 1 more Smart Citation

Acceptable risks of treatments to prevent rheumatoid arthritis among first-degree relatives: demographic and psychological predictors of risk tolerance

et al. 2022

Self Cite

View full text Add to dashboard Cite

ObjectivesTo quantify tolerance to risks of preventive treatments among first-degree relatives (FDRs) of patients with rheumatoid arthritis (RA).MethodsPreventive treatments for RA are under investigation. In a preference survey, adult FDRs assumed a 60% chance of developing RA within 2 years and made choices between no treatment and hypothetical preventive treatment options with a fixed level of benefit (reduction in chance of developing RA from 60% to 20%) and varying levels of risks. Using a probabilistic threshold technique, each risk was increased or decreased until participants switched their choice. Perceived risk of RA, health literacy, numeracy, Brief Illness Perception Questionnaire and Beliefs about Medicines Questionnaire-General were also assessed. Maximum acceptable risk (MAR) was summarised using descriptive statistics. Associations between MARs and participants’ characteristics were assessed using interval regression with effects coding.Results289 FDRs (80 male) responded. The mean MAR for a 40% reduction in chance of developing RA was 29.08% risk of mild side effects, 9.09% risk of serious infection and 0.85% risk of a serious side effect. Participants aged over 60 years were less tolerant of serious infection risk (mean MAR ±2.06%) than younger participants. Risk of mild side effects was less acceptable to participants who perceived higher likelihood of developing RA (mean MAR ±3.34%) and more acceptable to those believing that if they developed RA it would last for a long time (mean MAR ±4.44%).ConclusionsAge, perceived chance of developing RA and perceived duration of RA were associated with tolerance to some risks of preventive RA therapy.

show abstract

Section: Methodsmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Acceptable risks of treatments to prevent rheumatoid arthritis among first-degree relatives: demographic and psychological predictors of risk tolerance

et al. 2022

Self Cite

View full text Add to dashboard Cite

show abstract

“…Further investigation is needed to assess preferences in different countries with different types of healthcare provision and also in low and middle income countries. Existing choice-based studies have not yet identified participant characteristics (e.g., gender; health literacy; and numeracy) associated with preference heterogeneity (52), though this is currently under investigation (58).…”

Section: Discussionmentioning

confidence: 99%

“…Finally, the protocol of a stated choice survey employing both a DCE and a probabilistic threshold technique to assess preferences for preventive treatments for RA has been published (58). That study recruits large samples of the general population via survey panels in the UK, Germany and Romania, and also recruits FDRs of confirmed RA patients.…”

Section: Quantitative Investigationsmentioning

confidence: 99%

Perspectives of at-Risk Individuals on Preventive Intervention for Rheumatoid Arthritis: A Mini Review

Falahee

Raza

2022

Front. Immunol.

Self Cite

View full text Add to dashboard Cite

There has been intense research focus on the biological mechanisms underlying the transition from health to disease for rheumatoid arthritis (RA) over recent years, and it is now well established that a state of autoimmunity precedes the development of symptoms for a large proportion of patients. This has led to an increased interest in the identification of at-risk groups and the potential for preventive intervention. The ability of several immunomodulatory agents to delay or prevent RA is under investigation and novel cellular therapies are in development. Preventive approaches are also being assessed in other chronic autoimmune diseases. For example, an anti-CD3 antibody has recently been shown to delay progression to type 1 diabetes in non-diabetic relatives of patients identified as being at high risk. The identification and treatment of individuals as being at risk of a disease where there is a degree of uncertainty around the potential for benefit is socially and ethically challenging. Recently reported difficulties in recruitment to RA prevention trials have underlined the importance of understanding the perspectives of at-risk individuals to identify barriers and facilitators that need to be addressed in order for preventive strategies to be acceptable. Understanding of their preferences for benefits and risks of preventive interventions can inform efficient intervention prioritization, prevention trial design and the development of informational resources for those at risk. In this review we summarize current knowledge of preferences for RA prevention and make recommendations for further research needed to ensure efficient development of preventive therapies and clinical implementation.

show abstract

“…The Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) project aims to assess when and how patient preferences on benefits and risks should be incorporated into treatment decisions (46). Through PREFER, a multitude of preference studies have been conducted across chronic conditions, with consideration to the inclusion of patient preferences in the process of clinical trials and drug development, from pre-approval of treatments to post-authorization (46)(47)(48). Both the FDA and PREFER have highlighted the promise and potential value of including patient preferences when designing clinical trials.…”

Section: Discussionmentioning

confidence: 99%

The Application of Preference Elicitation Methods in Clinical Trial Design to Quantify Trade-Offs: A Scoping Review

Thomas

Marshall

Choudhary

et al. 2021

Patient

View full text Add to dashboard Cite

Background and Objective: Patients can express preferences for different treatment options in a healthcare context, which can be measured with quantitative preference elicitation methods.Our objective was to conduct a scoping review to determine how preference elicitation methods have been used to design clinical trials. Methods:We conducted a scoping review to identify primary research studies involving any health condition, that used quantitative preference elicitation methods, including direct utilitybased approaches, and stated preference studies, to value heath trade-offs in the context of clinical trial design. Studies were identified by screening existing systematic and scoping reviews, and a primary literature search in MEDLINE from 2010-present. We extracted study characteristics and the application of preference elicitation methods to clinical trial design according to the SPIRIT checklist from primary studies and summarized the findings descriptively. Results:We identified 18 eligible studies. The included studies applied patient preferences to 5 areas of clinical trial design: intervention selection (n=1), designing N-of-1 trials (n=1), outcome selection and weighting composite and ordinal outcomes (n=12), sample size calculations (n=2), and recruitment (n=2). Using preference elicitation methods led to different decisions being made, such as using preference-weighted composite outcomes instead of equally weighted composite outcomes.Scoping review on preferences and trials 5 Conclusion: Preference elicitation methods are infrequently used to design clinical trials but may lead to changes throughout the trial which could impact the evidence generated. Future work should be done to consider measurement challenges and explore stakeholder perceptions.

show abstract

Treatment preferences for preventive interventions for rheumatoid arthritis: protocol of a mixed methods case study for the Innovative Medicines Initiative PREFER project

Cited by 9 publications

References 52 publications

Acceptable risks of treatments to prevent rheumatoid arthritis among first-degree relatives: demographic and psychological predictors of risk tolerance

Acceptable risks of treatments to prevent rheumatoid arthritis among first-degree relatives: demographic and psychological predictors of risk tolerance

Perspectives of at-Risk Individuals on Preventive Intervention for Rheumatoid Arthritis: A Mini Review

The Application of Preference Elicitation Methods in Clinical Trial Design to Quantify Trade-Offs: A Scoping Review

Contact Info

Product

Resources

About