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![Development and evaluation process for decision aids for people considering trial participation. Adapted from the Medical Research Council (MRC) guidance on developing and evaluating complex interventions and the Model Development Process for Decision Aids (Coulter et al. 2013 [19], Craig et al. 2008 [25]).](publication/334270463/figure/fig1/AS:777491379658752@1562379798737/Development-and-evaluation-process-for-decision-aids-for-people-considering-trial.png)
Development and evaluation process for decision aids for people considering trial participation. Adapted from the Medical Research Council (MRC) guidance on developing and evaluating complex interventions and the Model Development Process for Decision Aids (Coulter et al. 2013 [19], Craig et al. 2008 [25]).
Source publication
Abstract Ethical requirements of informed consent stipulate that patients approached to participate in a clinical trial be provided with written information that must cover key aspects of the trial. For consent to be deemed “informed”, potential participants should be provided with a range of information about the trials (e.g., the trial aims, the...
Contexts in source publication
Context 1
... Also, little attention has previously been given to evaluation considerations (and none has acknowledged the cross-learning to decision aids from the complex intervention evaluation literature). We have therefore developed a new and expanded framework to help inform the development and evaluation of trial participation decision aids (Fig. 3). This integrates insights from the MRC complex intervention framework (which we have used as the foundation of this augmented framework), theoretical insights from the ODSF, perspectives integrated from the wider decision-aid literature (including the model development process for treatment decision aids presented by Coulter et al. in ...
Context 2
... insights from the ODSF, perspectives integrated from the wider decision-aid literature (including the model development process for treatment decision aids presented by Coulter et al. in 2013 [19]), the complex intervention evaluation literature, and the experiential learning from our empirical testing process. Our proposed model is presented in Fig. ...
Citations
... Technology has advanced significantly during the initial adoption and use of traditional informed consent, and can be used to enhance the methods of obtaining IC through digital tools that capitalize on participant preferences such as learning styles, culture, and language with text-alternative methods (digital coaching, video, animation, voice-over, etc.) (Entwistle et al., 2012, Gesualdo et al., 2021. In addition, decision aid tools have become more common (Stacey et al., 2017, Gillies andCampbell, 2019). These tools provide a myriad of benefits, which include informing about options, clarifying values, supporting preference construction process, and enabling increased active engagement in shared decision-making (Stacey et al., 2017, Swierenga et al., 2013, Sepucha et al., 2010. ...
The objective of the informed consent (IC) process is to inform potential participants about the purpose, procedures, risks, and benefits associated with clinical research and medical procedures. Traditional paper consent processes are generally long and confusing, especially in busy settings for research such as the emergency department (ED). We describe how we used a tablet-based digital IC process to recruit (N=1,002) older adults for an elder mistreatment study in the ED. Methods: The Virtual Multimedia Interactive Informed Consent (VIC) consent tool was previously developed and tested in an AHRQ-funded R21 study and was found to be usable, acceptable, and it enhanced participants’ comprehension and satisfaction when compared to a traditional paper-based IC process (Abujarad et al., 2021a). VIC was developed using a user-centered design (UCD) approach, incorporating digital coaching, multimedia features such as animated videos to explain research procedures, automated text-to-speech audio, and automated teach-back to emphasize key concepts. The VIC digital consent tool was used to recruit patients for an NIA-funded R01 study evaluating the feasibility of the VOICES Elder Mistreatment Intervention, a self-administered digital health intervention to increase identification of elder mistreatment in ED settings. Due to the complexities of elder mistreatment identification, we recognized the need for an IC process that ensures participant privacy, autonomy, and comprehension, with particular focus on the risks and benefits of recognizing and disclosing mistreatment. A total of 1,002 participants ages 60 and older were consented and enrolled during their visit in the ED. Results: A total of 1,204 of eligible participants agreed to participate in the study and started the consent, of whom 1,012 (84%) participants completed the consent process and enrolled in the VOICES study. Of the 192 (16%) participants who were not enrolled in the study: 158 (13%) did not complete the IC process for varying reasons, the most common reason being due to pain, and 34 (3%) completed the IC fully and chose not to participate in VOICES study. Of the consented participants, 99% fully completed the VOICES study and filled all surveys. Consented participants included older adults from 60 to 102 years old with a mean age of 73.5. Most participants were female, white, and high school educated or higher.Discussion: We believe that the use of digital IC process benefitted the participants who were able to complete the IC process on their own and with minimal help from the study coordinators. We received a high study completion rate among consented participants, and we believe that emphasizing key concepts and using multimedia to explain the more complicated research topics helped better educate potential participants to make a true informed decision about their participation in the VOICES study. It is likely that research participants who have a better understanding of the nature of the study are more likely to finish study procedures, increasing study retention. For the patients who did not complete the IC, they associated that to their chief complaint and medical reasons related to the nature of their visit to the ED. More research is needed to compare traditional and digital consent processes to better evaluate the effectiveness of digital consent.
... More recently, DAs have been developed for people considering participating in clinical trials [28]. DAs support the decision process by providing structured guidance on steps of decision-making, information about available options and their associated outcomes, and information that enables patients to consider what value they place on particular outcomes [29]. A novel DA for proxy decision-making about research has been developed in collaboration with lay advisors and stakeholder groups and informed by theoretical frameworks and empirical research [30]. ...
Background
Randomised trials play a vital role in underpinning evidence-based care. However, trials involving adults with impaired capacity to consent raise a number of ethical and methodological challenges, leading to the frequent exclusion of this group from trials. This includes challenges around involving family members as alternative ‘proxy’ decision-makers. Family members are often given little information about their role as a consultee or legal representative. Some family members find making a decision about trial participation difficult and may experience an emotional and decisional burden as a result. Families have reported a need for greater support and guidance when making such decisions, leading to the development of a decision aid (‘Making decisions about research for others’) for family members acting as consultee/legal representative. The decision aid now requires evaluation to determine its effectiveness in supporting families to make more informed decisions.
Methods
This protocol describes a prospective, multi-centre, randomised-controlled Study Within a Trial (SWAT) to evaluate the effectiveness of the decision aid. The SWAT will initially be embedded in approximately five host trials. SWAT participants will be randomised in a 1:1 ratio to either the intervention (decision aid alongside standard information about the host trial provided to consultees/legal representatives) or control (standard information alone). The primary outcome is the quality of proxy consent decision, assessed by the Combined Scale for Proxy Informed Consent Decisions (CONCORD). The SWAT design is informed by previous qualitative research. Initial feasibility will be explored in one host trial, followed by the main SWAT. An embedded process evaluation and economic evaluation will enable the SWAT findings to be contextualised and identify factors likely to affect implementation.
Discussion
This SWAT will generate the first evidence for recruitment interventions for trials involving adults lacking capacity to consent and add to knowledge about the use of decision support interventions in trial participation decisions. The SWAT will be embedded in a range of trials, and the heterogenous nature of the host trials, settings and populations involved will enable the intervention to be evaluated in a wide range of contexts. However, a pragmatic and flexible approach to conducting the SWAT is needed.
Trial registration
The SWAT is registered as SWAT #159 with the Northern Ireland Hub for Trials Methodology Research SWAT repository (registered 09.08.2020). Each host trial will be registered on a clinical trials registry.
... However, this template does not attend to important nuances of how components are worded or presented to ensure acceptability to the target population. Furthermore, the same components may not be appropriate for all decision aids as health decisions and the context in which they are made vary enormously (15)(16)(17). There is a need for a template specific enough to ensure clear consistency in the decision aid components, but flexible enough to allow content to be tailored for the specific genetic predisposition. ...
Background
People with genetic predispositions to cancer are faced with complex health decisions about managing their risk. Decision aids can support informed, values-based decisions, alongside shared decision-making with a clinician. Whilst diagnoses of genetic predispositions to cancer are increasing, there is no scalable decision aid to support these people. This paper presents an accessible, relevant decision aid template which can be adapted for different predispositions to cancer.
Methods
The decision aid template was co-developed with 12 patients affected by cancer and informed by empirical and theoretical literature. In addition, consultations were conducted with a further 19 people with Lynch syndrome; a specific genetic predisposition to cancer. Clinical stakeholders were consulted regularly. Coulter's framework for decision aid development guided the process, and these activities were complemented by the International Patient Decision Aid Standards, and the latest evidence on communicating risk in decision aids. Programme theory was developed to hypothesise how the decision aid would support decision-making and contextual factors which could influence the process. Guiding principles co-developed with the patient panel described how the decision aid could effectively engage people.
Results
The in-depth co-design process led to the identification of five core components of an accessible decision aid template for people with a genetic predisposition to cancer: defining the decision; option grid showing implications of each option; optional further details such as icon arrays to show tailored risk and personal narratives; values clarification activity; and a summary to facilitate discussion with a clinician. Specific guidance was produced describing how to develop each component. The guiding principles identified that the decision aid template needed to promote trust, reduce distress, and be comprehensive, personally relevant and accessible in order to engage people.
Conclusion
Adopting a co-design process helped ensure that the decision aid components were relevant and accessible to the target population. The template could have widespread application through being adapted for different genetic predispositions. The exact content should be co-designed with people from diverse backgrounds with lived experience of the specific predisposition to ensure it is as useful, engaging and relevant as possible.
... The focus on the content and structure of information as decision support and on assessing the level of knowledge gained has been criticised for neglecting the social context of decision processes. A review done by Gillies and colleagues [45] on decision aids found that discussions, compared to information received during the informed consent process, increased understanding, which suggests that it is not just information but the chance to reflect and share thoughts along the way that aid the decision-making process. Among the included studies three used a QPL, which is one way to give patients the opportunity to discuss their thoughts on trial participation. ...
Objectives
Existing research on the perspectives of patients with cancer and health care professionals indicates that patient decision making on cancer clinical trial participation is a complex process and may be poorly understood, possibly compromising their decision to participate. This systematic review investigates interventions that support patients in their decision-making processes regarding whether to participate or not and assesses the qualities of the interventions, measures used and related outcomes.
Methods
Six databases were systematically searched and only studies evaluating interventions that support the decision making of adult patients offered to enter a cancer clinical trial were included. Ten articles met the criteria and were analysed using a narrative synthesis approach.
Results
The research focus of the included studies reflected the multifactorial nature of what constitutes support for patient decision making in terms of entering a cancer clinical trial. However, most interventions were based on the hypothesis that more information leads to support in decision making, and did not take other factors, such as the relationship to the clinical staff or relatives, the patients’ strong hope for therapeutic benefit or other existential needs into account. The interventions were primarily based on a specific tool, executed once, which seems to imply that decisions need only to be supported once and not at several time points throughout the decision process, and did not assess the importance of a patient’s family- or social relations. Moreover, few interventions focused on the patients’ counselling experience or assessed patient preferences in relation to decision making.
Conclusions
The findings demonstrate a lack of research on interventions to support patients’ decision making that takes other factors, apart from improving knowledge of trials, into account. Limited evidence exists on the effectiveness of decision support interventions to improve the experience of support in adult patients with cancer. Interventions that take patient preferences in relation to decision making and the social context of decision processes into account need to be developed and assessed.
... One intervention recently developed to support proxies in making decisions about non-emergency research participation is a decision aid (DA) intended to help families to make more informed and supported decisions when acting as a proxy [16]. Using decision science principles, DAs can help when making complex and preferencesensitive decisions, including decisions about participating in clinical trials [17,18]. DAs differ from traditional information materials in that they do not focus solely on improving the delivery of information [18], but instead are intended to facilitate decision-making and lead to decisions which are more informed and consistent with the person's values [19]. ...
... Using decision science principles, DAs can help when making complex and preferencesensitive decisions, including decisions about participating in clinical trials [17,18]. DAs differ from traditional information materials in that they do not focus solely on improving the delivery of information [18], but instead are intended to facilitate decision-making and lead to decisions which are more informed and consistent with the person's values [19]. We developed a DA for proxy decisions about trial participation in response to family members identifying a need for better information and support when making these challenging decisions [16]. ...
Background
Recruitment of adults lacking the capacity to consent to trials requires the involvement of an alternative ‘proxy’ decision-maker, usually a family member. This can be challenging for family members, with some experiencing emotional and decisional burdens. Interventions to support proxy consent decisions in non-emergency settings are being developed. However, the ability to evaluate interventions is limited due to a lack of measures that capture outcomes of known importance, as identified through a core outcome set (COS).
Methods
Using established measure development principles, a four-stage process was used to develop and refine items for a new measure of proxy decision quality: (1) findings from a recent scoping review and consensus study were reviewed to identify items for inclusion in the scale and any existing outcome measures, (2) assessment of content coverage by existing measures and identification of insufficiency, (3) construction of a novel scale, and (4) cognitive testing to explore comprehension of the scale and test its content adequacy through interviews with family members of people with impaired capacity.
Results
A range of outcome measures associated with healthcare decision-making and informed consent decisions, such as the Decisional Conflict Scale, were identified in the scoping review. These measures were mapped against the key constructs identified in the COS to assess content coverage. Insufficient coverage of areas such as proxy-specific satisfaction and knowledge sufficiency by existing instruments indicated that a novel measure was needed. An initial version of a combined measure (the CONCORD scale) was drafted and tested during cognitive interviews with eleven family members. The interviews established comprehension, acceptability, feasibility, and content adequacy of the scale. Participants suggested re-phrasing and re-ordering some questions, leading to the creation of a revised version.
Conclusions
The CONCORD scale provides a brief measure to evaluate the quality of decisions made on behalf of an adult who lacks the capacity to consent in non-emergency settings, enabling the evaluation of interventions to improve proxy decision quality. Initial evaluation indicates it has content adequacy and is feasible to use. Further statistical validation work is being undertaken.
... More recently, DAs have been developed for people considering participating in clinical trials [24]. DAs support the decision process by providing structured guidance on steps of decision making, information about available options and their associated outcomes, and information that enables patients to consider what value they place on particular outcomes [25]. A novel DA for proxy decision-making about research has been developed in collaboration with lay advisors and stakeholder groups and informed by theoretical frameworks and empirical research [26]. ...
Background
Randomised trials play a vital role in underpinning evidence-based care. However, trials involving adults with impaired capacity to consent raise a number of ethical and methodological challenges, leading to the frequent exclusion of this group from trials. This includes challenges around involving family members as alternative ‘proxy’ decision-makers. Family members are often given little information about their role as a consultee or legal representative. Some family members find making a decision about trial participation difficult and may experience an emotional and decisional burden as a result. Families have reported a need for greater support and guidance when making such decisions, leading to the development of a decision aid (‘Making decisions about research for others’) for family members acting as consultee/legal representative. The decision aid now requires evaluation to determine its effectiveness in supporting families to make more informed decisions.
Methods
This protocol describes a prospective, multi-centre, randomised-controlled Study Within a Trial (SWAT) to evaluate the effectiveness of the decision aid. The SWAT will initially be embedded in approximately five host trials. Participants will be randomised in a 1:1 ratio to either the intervention (decision aid alongside standard information about the host trial provided to consultees/legal representatives) or control (standard information alone). The primary outcome is the quality of proxy consent decision, assessed by the Combined Scale for Proxy Informed Consent Decisions (CONCORD). The SWAT design is informed by previous qualitative research. Initial feasibility will be explored in one host trial, followed by the main SWAT. An embedded process evaluation and economic evaluation will enable the SWAT findings to be contextualised and identify factors likely to affect implementation.
Discussion
This SWAT will generate the first evidence for recruitment interventions for trials involving adults lacking capacity to consent and add to knowledge about the use of decision support interventions in trial participation decisions. The SWAT will be embedded in a range of trials, and the heterogenous nature of the host trials, settings and populations involved will enable the intervention to be evaluated in a wide range of contexts. However, a pragmatic and flexible approach to conducting the SWAT is needed.
Trial Registration
The SWAT is registered as SWAT #159 with the Northern Ireland Hub for Trials Methodology Research SWAT repository (registered 09.08.2020). Each host trial will be registered on a clinical trials registry.
... Good healthcare decisions require patients to consider both factual and probabilistic information, along with their personal values and preferences, and then choose the option that is most concordant with those values and preferences [16,30]. Research participation decisions are also known to be preference-sensitive decisions, where good decisions are considered to have a match between the decision (participation or non-participation) and the features that matter most to the informed patient [31]. Whilst the terms values and preferences were not defined in the literature synthesised here, and indeed were often used interchangeably, values refers to high level constructs or attitudes such as truth-seeking or risk aversion, whereas preferences involves ranking the various attributes of options, such as drug side-effects which a patient prefers to avoid and hence will avoid that associated option [24]. ...
... The importance of this congruence (also termed concordance) has its basis in autonomy which is considered to have two important elements-agency (the ability to make choices) [41], and authenticity (the ability to live a life of one's own choosing) [42]. 'Good' decisions can be viewed as those where there is a close match between the chosen decision and the features that matter most to the informed patient [31]. The proxy aims to make a values-congruent decision during the deliberation stage of the decision-making process, and it is their uncertainty about whether participation in research is congruent with the person's values that can lead to the decisional conflict they may experience. ...
... Whilst the sequential process reported in the study is reflective of the findings from this synthesis, the study was intended to be a descriptive characterisation of what proxy consent in critical care is, rather than identifying what constitutes quality decision-making as outlined in this paper. Similarly, studies that have sought to explicate the concepts that capture the quality of informed consent given for oneself have primarily focused on information provision and identifying knowledge-deficits [70], rather than considering other aspects of decision quality included here such as decisional conflict or regret [31]. ...
People who are unable to make decisions about participating in research rely on proxies to make a decision based on their wishes and preferences. However, patients rarely discuss their preferences about research and proxies find it challenging to determine what their wishes would be. While the process of informed consent has traditionally been the focus of research to improve consent decisions, the more conceptually complex area of what constitutes ‘good’ proxy decision-making for research has remained unexplored. Interventions are needed to improve and support proxy decision-making for research but are hampered by a lack of understanding about what constitutes decision quality in this context. A global increase in conditions associated with cognitive impairment such as dementia has led to an urgent need for more research into these conditions. The COVID-19 pandemic and subsequent necessity to conduct research with large numbers of critically ill patients has made this need even more pressing. Much of the empirical research centres on the desire to improve decision accuracy, despite growing evidence that authenticity is more reflective of the aim of proxy decisions and concerns about the methodological flaws in authenticity-focused studies. Such studies also fail to take account of the impact of decision-making on proxies, or the considerable body of research on improving the quality of healthcare decisions. This paper reports a concept synthesis of the literature that was conducted to develop the first conceptualisation of ‘good’ proxy decisions about research participation. Elements of decision quality were identified across three stages of decision-making: proxy preparedness for decision-making which includes knowledge and understanding, and values clarification and preference elicitation; the role of uncertainty, decisional conflict, satisfaction and regret in the decision-making process; and preference linked outcomes and their effect. This conceptualisation provides an essential first step towards the future development of interventions to enhance the quality of proxy decision-making and ensure proxy decisions represent patients’ values and preferences.
... For real-world implementation of patient decision aids and shared decision making at scale, some developers may use templates, standardized processes, or common resources for producing new decision aids. 13,14 Such templates may be initially developed and tested thoroughly in a similar way as the maximum process presented above. Then, when the template is used as a starting point for developing new patient decision aids for a specific clinical situation, it may be only necessary to use a minimal process or similarly abridged process. ...
Background
The 2013 update of the evidence informing the quality dimensions behind the International Patient Decision Aid Standards (IPDAS) offered a model process for developers of patient decision aids.
Objective
To summarize and update the evidence used to inform the systematic development of patient decision aids from the IPDAS Collaboration.
Methods
To provide further details about design and development methods, we summarized findings from a subgroup ( n = 283 patient decision aid projects) in a recent systematic review of user involvement by Vaisson et al. Using a new measure of user-centeredness (UCD-11), we then rated the degree of user-centeredness reported in 66 articles describing patient decision aid development and citing the 2013 IPDAS update on systematic development. We contacted the 66 articles’ authors to request their self-reports of UCD-11 items.
Results
The 283 development processes varied substantially from minimal iteration cycles to more complex processes, with multiple iterations, needs assessments, and extensive involvement of end users. We summarized minimal, medium, and maximal processes from the data. Authors of 54 of 66 articles (82%) provided self-reported UCD-11 ratings. Self-reported scores were significantly higher than reviewer ratings (reviewers: mean [SD] = 6.45 [3.10]; authors: mean [SD] = 9.62 [1.16], P < 0.001).
Conclusions
Decision aid developers have embraced principles of user-centered design in the development of patient decision aids while also underreporting aspects of user involvement in publications about their tools. Templates may reduce the need for extensive development, and new approaches for rapid development of aids have been proposed when a more detailed approach is not feasible. We provide empirically derived benchmark processes and a reporting checklist to support developers in more fully describing their development processes. [Box: see text]
... First, participation in clinical research is often a difficult decision that could benefit from shared decision-making (SDM), a framework originally designed to help people work systematically through difficult treatment decisions, often with decision-making tools called patient decision aids that seek to achieve the clear goals of improving knowledge of key aspects of the decision, understanding outcome probabilities, and ensuring a match between outcome preferences and the choice made [15][16][17]. More recently, SDM principles are being applied to choices related to clinical trial involvement [18][19][20][21][22], such as consent [18][19][20][21] and participation decisions [22][23][24]. This framework provides a fundamentally different model about what informed consent and recruitment could look like, emphasizing shared, deliberative decision-making between patient and provider, and improved quality of decision-maker experience by ensuring understanding of the probability of relevant outcomes and a match between outcomes most valued by the decision maker and the decision made. ...
... First, participation in clinical research is often a difficult decision that could benefit from shared decision-making (SDM), a framework originally designed to help people work systematically through difficult treatment decisions, often with decision-making tools called patient decision aids that seek to achieve the clear goals of improving knowledge of key aspects of the decision, understanding outcome probabilities, and ensuring a match between outcome preferences and the choice made [15][16][17]. More recently, SDM principles are being applied to choices related to clinical trial involvement [18][19][20][21][22], such as consent [18][19][20][21] and participation decisions [22][23][24]. This framework provides a fundamentally different model about what informed consent and recruitment could look like, emphasizing shared, deliberative decision-making between patient and provider, and improved quality of decision-maker experience by ensuring understanding of the probability of relevant outcomes and a match between outcomes most valued by the decision maker and the decision made. ...
Background
Clinical trial recruitment is a continuing challenge for medical researchers. Previous efforts to improve study recruitment have rarely been informed by theories of human decision making and behavior change. We investigate the trial recruitment strategies reported by study recruiters, guided by two influential theoretical frameworks: shared decision-making (SDM) and the Theoretical Domains Framework (TDF) in order to explore the utility of these frameworks in trial recruitment.
Methods
We interviewed all nine active study recruiters from a multi-site, open-label pilot trial assessing the feasibility of a large-scale randomized trial. Recruiters were primarily nurses or master's-level research assistants with a range of 3 to 30 years of experience. The semi-structured interviews included questions about the typical recruitment encounter, questions concerning the main components of SDM (e.g. verifying understanding, directive vs. non-directive style), and questions investigating the barriers to and drivers of their recruitment activities, based on the TDF. We used directed content analysis to code quotations into TDF domains, followed by inductive thematic analysis to code quotations into sub-themes within domains and overarching themes across TDF domains. Responses to questions related to SDM were aggregated according to level of endorsement and informed the thematic analysis.
Results
The analysis helped to identify 28 sub-themes across 11 domains. The sub-themes were organized into six overarching themes: coordinating between people, providing guidance to recruiters about challenges, providing resources to recruiters, optimizing study flow, guiding the recruitment decision, and emphasizing the benefits to participation. The SDM analysis revealed recruiters were able to view recruitment interactions as successful even when enrollment did not proceed, and most recruiters took a non-directive (i.e. providing patients with balanced information on available options) or mixed approach over a directive approach (i.e. focus on enrolling patient in study). Most of the core SDM constructs were frequently endorsed.
Conclusions
Identified sub-themes can be linked to TDF domains for which effective behavior change interventions are known, yielding interventions that can be evaluated as to whether they improve recruitment. Despite having no formal training in shared decision-making, study recruiters reported practices consistent with many elements of SDM. The development of SDM training materials specific to trial recruitment could improve the informed decision-making process for patients.
... These findings resonate with Houghton et al's conceptual model which indicates that prospective randomised controlled trial participants will consider what they stand to gain or lose, altruistic motives, social influences and the burden of participation. 10 However, unique to the early phase cancer trial context is that patients often express therapeutic misconception (believing the intent of the trial is to benefit them) 31 50 and unrealistic optimism (believing they will experience better outcomes than others), 18 19 posing ethical challenges for trialists. 51 Consistent with existing guidance on developing decision aids for clinical trials, 48 it will, thus, be important to present CAR-T cell therapy safety and efficacy findings with an emphasis on how safety and efficacy data compare to usual care and other available options. Though the threat of adverse events remains, the likelihood of experiencing adverse side effects may be comparable to treatments they have already endured. ...
Objectives
Bench to bedside translation of groundbreaking treatments like chimeric antigen receptor T (CAR-T) cell therapy depends on patient participation in early phase trials. Unfortunately, many novel therapies fail to be adequately evaluated due to low recruitment rates, which slows patient access to emerging treatments. Using the Theoretical Domains Framework (TDF), we sought to identify potential patient barriers and enablers to participating in an early phase CAR-T cell therapy trial.
Design
We used qualitative semistructured interviews to identify potential barriers and enablers to patients’ hypothetical participation in an early phase CAR-T cell therapy trial. We used the TDF and directed content analysis to identify relevant domains based on frequency, relevance and the presence of conflicting beliefs.
Participants
Canadian adult patients diagnosed with haematological malignancies.
Results
In total, we interviewed 13 participants (8 women, 5 men). Participants ranged in age from 18 to 73 (median=56) and had been living with haematological cancer from a few months to several years. We found participants were unfamiliar with CAR-T cell therapy but wished to know more about treatment safety, efficacy and trial logistics (domains: knowledge, beliefs about consequences). They were motivated by altruistic considerations, though many prioritised personal health benefits despite recognising the goals (ie, establishing safety) of early phase clinical trials (domains: goals, intentions). Every participant valued receiving medical advice from their haematologists and oncologists, though some preferred impartial medical experts to inform their decision making (domain: social influences). Finally, participants indicated that improving access to financial and social supports would improve their trial participation experience (domain: environmental context and resources).
Conclusion
Using the TDF allowed us to identify factors that might undermine participation to a CAR-T cell therapy trial and to optimise recruitment processes by considering patient perspectives to taking part in early phase trials.
Trial regestration: NCT03765177 ; Pre-results.
