Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey

Overbeeke, Eline van; Hauber, A. Brett; Michelsen, Sissel; Peerlinck, Kathelijne; Lambert, Catherine; Hermans, Cédric; Lê, Phu Quoc; Goldman, M.; Simoens, Steven; Huys, Isabelle

doi:10.1111/hae.14401

Cited by 18 publications

(16 citation statements)

References 34 publications

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“…In studies including attribute ranking, 30,31,33 discrete choice experiments, 32 and trade-offs, 34 the top attributes for decisionmaking include annual bleeding rate, factor level expression, the uncertainty of long-term effects, impact on daily life, chance to stop prophylaxis, dose frequency and durability, impact on ability to participate in physical activity, frequency of monitoring, uncertainty regarding short-term or long-term significant safety issues and duration of follow-up on side effects. A key theme in those studies and the current survey is the unpredictability of the duration of therapeutic response and variability in factor expression.…”

Section: Discussionmentioning

confidence: 99%

Gene therapy preferences and informed decision‐making: Results from a National Hemophilia Foundation Community Voices in research survey

et al. 2022

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Introduction:To inform education and treatment discussions, it is important to understand how persons with haemophilia prefer to learn about and discuss new therapies and to identify variables that influence decision-making. Aim:The aim of this study was to evaluate preferences and variables which influence decision-making related to gene therapy and other novel haemophilia therapies.Methods: An online survey was sent to men with severe haemophilia enrolled in the National Hemophilia Foundation Community Voices in Research online platform for patient-powered research.Results: One hundred four men completed the survey including 33% Hispanics, 96 who had had not gene therapy and 71/96 (74%) who were on prophylaxis. Ninetyfive percent were somewhat or very familiar with gene therapy. Men with haemophilia obtain information about new therapies from several sources, most commonly their haemophilia treatment team, patient advocacy groups and self-study. Participants identified safety and efficacy as well as other educational needs to inform decisionmaking. Of those without prior gene therapy, 73% indicated a high likelihood of considering gene therapy. Hispanic ethnicity and government-issued insurance were associated with a higher likelihood of considering gene therapy as a treatment option. Conclusion:Haemophilia Treatment Centers and patient advocacy groups must be able to educate persons with haemophilia about aspects of novel therapies which are important to the individual, especially short-and long-term safety and efficacy. Further research is needed to determine how patient activation and health literacy influence decision-making and how to achieve equitable access and valid informed consent for novel therapies.

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Section: Discussionmentioning

confidence: 99%

Gene therapy preferences and informed decision‐making: Results from a National Hemophilia Foundation Community Voices in research survey

et al. 2022

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“…The review also identified 3 literature reviews [39][40][41] assessing patient preferences in haemophilia, all of which were not full journal articles. There have been two published studies exploring haemophilia patient preferences in relation to gene therapy: one DCE [37] and one utilising a threshold technique [42] . Surveyed audiences included physicians, patients, pharmacists, healthcare professionals and caregivers (either alone or in combination).…”

Section: Resultsmentioning

confidence: 99%

“…As shown in Table 1, a number of gene therapies are being developed for both haemophilia A and B. Two published studies to date have explored haemophilia patient preferences in relation to gene therapy: one DCE [37] and one utilising a threshold technique [42] . Whilst DCEs have been explored extensively in this paper, the threshold technique is a method that determines the maximal change in one attribute respondents are willing to accept to achieve a given change in another attribute [43] .…”

Section: Head-to-head Comparison Of Gene Therapy Studiesmentioning

confidence: 99%

Discrete choice experiments: An overview of experience to date in haemophilia

Spoors

Miners

Cairns

2022

The Journal of Haemophilia Practice

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Background The patient voice is an important consideration in the availability and choice of pharmaceuticals – however, how to capture this complex area and apply it formally within regulation, health technology assessment and reimbursement remains subject to ongoing debate. Patient preference studies such as discrete choice experiments (DCEs) are being utilised more frequently in healthcare and it is anticipated that patient preference data will be incorporated more frequently into regulatory submissions moving forward. Aim The aim of this review is to provide an overview of DCEs conducted within haemophilia to date and to consider the key issues in response to a rapidly evolving therapeutic pathway. Methods A systematic literature search was undertaken via Ovid MEDLINE and EMBASE CLASSIC + EMBASE. Abstracts were uploaded and analysed via Rayyan systematic review software. Results: Of 478 records identified from the database searches, 12 full text journal articles met the inclusion criteria with a date range from 2005–2021. There have been two published studies exploring haemophilia patient preferences in relation to gene therapy: one DCE and one utilising a threshold technique. Surveyed audiences included physicians, patients, pharmacists, healthcare professionals and caregivers. 50% of the included studies (n=6) were exclusively conducted in the US, whilst 3 recruited participants across multiple countries. The sample size varied considerably between studies with the total sample size ranging from 30 participants to 505 participants. For the studies involving patients and their caregivers, the mean patient age range was 8.2–41.4 years. There was diversity in (a) the scale of the qualitative work undertaken to support the DCEs, (b) the undertaking of pilots, and (c) how extensively these elements were reported in the included studies. There is a notable trend towards using an online web-based format, with 3 out of 4 DCEs since 2019 utilising this approach. The number of attributes observed per DCE ranged from 5–12 with a median of 6 attributes from the included studies. The number of levels per attribute was relatively consistent (range 2–5) with 2–3 (n=4) and 2–4 levels (n=4) being utilised most frequently. Conclusion Patient preferences and the methods for capturing these are likely to be subject to ongoing debate as the haemophilia care pathway evolves to offer more therapeutic options with a range of risks and benefits. Whilst techniques such as DCE are effective at quantifying patient preferences, they tell us little about the reasons driving these decisions and the likelihood that they will change in response to temporal or external factors. DCEs could be particularly useful for estimating the uptake of new products and assessing potential budget impact. Accelerated and reformed regulatory processes are likely to increase demand for patient preference studies. There is therefore an increased requirement to ensure that patient advocacy groups (PAGs) are resourced and have the expertise to support these studies alongside other research commitments, and that manufacturers consider collaborative approaches when formally capturing patient preferences. As more therapeutic options become available in haemophilia care, discrete choice experiment may be a useful means of gauging patient preference © Shutterstock

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“…Non-attendance to the information is evidenced in the short amount of time that respondents spent on the education material regardless of the format (2.36–3.84 minutes, while the video took over 9 minutes to view entirely, and reading the text-based information was expected to take 11 minutes). The long duration of the educational material may have led to non-attendance, but previous research with educational materials of comparable length has found that patients engage with the educational material when the treatment context was novel to the respondents [ 31 , 74 ]. The non-attendance in our study could be related to the high levels of disease experience that the sample had.…”

Section: Discussionmentioning

confidence: 99%

The Impact of Video-Based Educational Materials with Voiceovers on Preferences for Glucose Monitoring Technology in Patients with Diabetes: A Randomised Study

Smith

Whichello

Bekker‐Grob

et al. 2023

Patient

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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. Supplementary Information The online version contains supplementary material available at 10.1007/s40271-022-00612-9.

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Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey

Cited by 18 publications

References 34 publications

Gene therapy preferences and informed decision‐making: Results from a National Hemophilia Foundation Community Voices in research survey

Gene therapy preferences and informed decision‐making: Results from a National Hemophilia Foundation Community Voices in research survey

Discrete choice experiments: An overview of experience to date in haemophilia

The Impact of Video-Based Educational Materials with Voiceovers on Preferences for Glucose Monitoring Technology in Patients with Diabetes: A Randomised Study

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