Overview
In a collective effort to move towards a more patient-centric approach to medicinal product development,1–3 drug companies, regulators and assessment bodies are increasingly seeking to incorporate patients’ preferences regarding therapeutic options and attributes as a formal element in their decision-making regarding the benefit-risk profile of a product. Among the key drivers of this change is the notion that patients are not only the ones who directly benefit from a therapy but also the ones who bear its burden in that they are exposed to the treatment’s side effects.4 Patient preference studies capture patients’ views on which treatment and disease-related characteristics are important to them, the trade-offs they are willing to make between positive and negative aspects of different therapies and treatment attributes, and the impact of individual patient characteristics on the formation of such preferences.5,6 The present commentary discusses opportunities for conducting patient preferences studies using web-based modes of data collection in the context of the Patient Preference in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) project. As part of the Innovative Medicines Initiative (Horizon 2020), this 5-year research project aims to develop evidence-based recommendations to guide industry, regulatory authorities, health technology assessment (HTA) bodies, and other researchers on when and how patient preference studies could be used to inform decision-making throughout the medical product lifecycle (for an overview, see https://www.imi-prefer.eu/ or https://www.imi.europa.eu/projects-results/project-factsheets/prefer). The project features three patient preference core case studies within three disease areas: non-small-cells lung cancer (NSCLC), rheumatology, and neuromuscular disorders.
The application of web-based surveys administered via use of a remote modality is becoming increasingly popular due to the fact that it offers numerous advantages over traditional paper-based or computer-assisted surveys.7 During the COVID-19 pandemic, clinical studies were forced to rely more on remotely administered online surveys to gather data than initially planned. However, it is unclear whether complex preference elicitation tasks administered online in highly vulnerable patient populations are also feasible.
In order to discuss the feasibility of web-based preference surveys in vulnerable populations, we refer to the non-small cell lung cancer (NSCLC) quantitative study being conducted as a part of PREFER. This case study involved a large cohort of patients with NSCLC at stages from I to IV recruited from three cancer treatment centers across Italy and Belgium.8 The clinical aim of this case study was to assess how patients trade-off benefits and risks related to treatment alternatives, by using both discrete choice experiment (DCE) and swing weighting (SW) methods. DCE asks respondents to make choices among sets of profiles in a series of choice questions, with different levels of benefits and risks associated with the choices. A typical DCE question is “Which of the (two) treatments do you prefer?”. Conversely, SW asks patients to rank and rate different treatment attributes, and the preferences that inform this decision are assumed to be directly captured with the elicitation task.
Lung cancer patients represent a vulnerable population in that they tend to be older (65 years of age or older at the time of initial diagnosis) and report high levels of physical and psychological morbidities associated with their disease.9 In this paper, we describe opportunities and challenges associated with conducting web-based preference studies derived specifically from the PREFER Lung Cancer case study.
Web-Based Data Collection in Patient Preference Studies: General Methodological Considerations
While preference elicitation tasks have traditionally been administered in paper- or computer-based formats requiring the researcher’s presence (for review see10), the last 10 years have seen a rapid increase in the use of surveys and preference studies that are self-administered by participants online.7
Online data collection is a method that allows researchers to collect data from large and diverse populations in a quick and cost-effective manner.11 Several studies have investigated the validity of web-based surveys by comparing the results of online studies with studies conducted via traditional modalities (eg, mail, in-person interviews). Results indicated that the validity and reliability of data obtained online are comparable to those obtained by classical methods.12–14 However, there are several limitations that should be kept in mind when choosing a web-based survey approach for assessing patient preferences. One issue is the potential lack of inclusiveness and representativeness of this research method, whereby populations who do not have access to the internet and digital computing resources (eg, elderly or people with a low socio-economic status) may be underrepresented in the results.15 Some patient populations may live in rural contexts (with poor internet connectivity), maybe less educated, with lower literacy more in general. In those instances, an online survey would be an obstacle to their participation, whereas the presence of an experimenter helping them in understanding tasks and choosing responses to the survey plays a very important role. Another limitation is digital literacy and the age of the participants, whereby low digital skills and older age may limit participation in a self-administered web-based survey.16,17 Web surveys must therefore be designed in such a way that they are simple to complete by patients with lower digital literacy, with instructions that are clearly stated so as to obviate the need for clarifications by the experimenter. An additional concern regards ethical matters and, particularly, challenges regarding participants’ consent, privacy limitations, anonymity and confidentiality posed by websites.
Given the numerous advantages of online surveys and the surge in the use of technology prompted by the recent COVID-19 pandemic, remote/online interviews and web-based surveys (“e-surveys”) are likely to become increasingly popular modes of administration. The present article contributes to the field by exploring this format for organizing and conducting patient preference studies in a frail and elderly population, namely lung cancer patients, involving virtual interactive content that can be administered online. This commentary focuses on two quantitative preference elicitation tasks used in the PREFER project (ie, DCE and SW), and highlights the strengths and limitations of web-based surveys administered remotely in a diseased population.
A Web-Based Implementation of Preference Surveys in an Elderly and Diseased Population
Methods Overview
Due to the SARS-CoV-2 pandemic, the original protocol of the PREFER lung cancer quantitative case study8 had to be modified. Originally, the protocol specified that eligible NSCLC participants could be contacted either by phone or approached in the waiting room either before or after a medical visit, at which point the researcher would introduce the study. In the original protocol it was anticipated that surveys were to be run on tablets during a clinic visit, and should last about 1.5 hours. In response to COVID-19-related restrictions regarding in-person contact, the protocol was adapted to an online mode of recruitment and to a web-based self-completed modality of survey administration. Recruitment had to be carried out primarily via telephone so as to limit physical contact between patients and researchers. Once patients had indicated interest in participating, they would receive an invitation letter via e-mail, as well as a participant information sheet, consent form, and contact information of the principal investigators. After returning the forms to the researcher, participants would then receive a personalized link to fill in the survey online. Upon entering the survey, patients were again probed to give their consent by selecting “Yes, I want to participate” in the corresponding informed consent online, and then asked to complete all the survey sections (without the presence of the researcher as planned in the original protocol). The project received ethical approval by the Ethische Commissie Onderzoek UZ/KU Leuven (reference S64022) in Belgium, and by the “Ethical Committee of the European Institute of Oncology IRCCS (reference R1142/20-IEO 1206) in Italy.
The platform chosen to host the survey was Sawtooth software,18 which handles both traditional survey questions and choice analysis. Within the survey, training material was provided in the form of interactive tutorials developed specifically for the case study that contained graphics, pictograms, icon arrays, voice-over, and click-on functions (for a recent description see19) (Figures 1A and B). This e-learning tool (referred to as an educational tool hereafter) introduced participants to general health-related terminology, as well as more specific disease-related content (eg, existing therapies for lung cancer), explained the basic aims of the choice tasks, and instructed participants as to how to perform the task and state their preferences. The tool introduced contents in a direct fashion using plain language and provided practical examples to complement the verbal instructions. In this way, the tool was intended to assist patients by increasing their functional health literacy (ie, the individual’s capacity to obtain, process, and understand basic health information and make appropriate health decisions critical to perform the preference elicitation tasks in our case), while explaining the task in simplified terms.
 | Figure 1 Illustration of PREFER survey. (A) Educational tool introducing discrete choice experiment (DCE); (B) educational tool introducing swing weighting (SW). |
A first version of the survey including the educational tool was tested in a pilot study with a small group of patients (N = 5) who provided feedback regarding how understandable the material was and whether the survey length and burden to the patient was acceptable. The tool content and format were then revised according to this feedback. During the pilot testing, the researcher shared the screen with the participant and used the think aloud technique20 to elicit thoughts or insights.
Opportunities and Challenges of Web-Based Survey for Preference Studies
Opportunities
The implementation of a web-based and remotely administered survey for the elicitation of patient preferences in PREFER in vulnerable populations had several advantages, which should be considered when preparing to conduct future studies of this type. First, as the result of using an online survey, we were able to enroll a larger portion of eligible patients, including those who reside in distant geographical areas and those who are physically and psychologically too frail to travel for research purposes, which constitute a large portion of our lung cancer (LC) patient population. Out of all LC patients enrolled in our study, roughly 39% lived outside the regions where the clinical centers were located – making it unlikely that they would have accepted to take part in a face-to-face study. Additionally, web-based questionnaires reduced the financial costs and time associated with using paper-based, computer-assisted approaches to survey administration,21–23 thereby accelerating data collection. Indeed, we were able to recruit a sample of 307 patients over a 24-week period, a sample size that would have been very difficult to achieve if using a face-to-face design. Moreover, this modality reduced the burden on the patients – who did not need to spend additional time in the hospital beyond the clinical and therapeutic commitments, as well as on the…