One of the best ways to ensure that a sponsor’s clinical and commercial programs reflect how patients experience a disease or a treatment is to interview patients directly over time. This practice – longitudinal qualitative research – is an emerging science that can guide development efforts as well as inform communication strategies for treatments in any therapy area. Here, Sally Lanar and Alexia Marrel of ICON plc, outline what longitudinal qualitative research is, when and how it can be used and the key factors for success when using this type of research. A Maturing Field of Research First and foremost, what is longitudinal qualitative research? Put simply, this research is conducted through interviews with patients at various time points during their study participation. The interviews should be conducted by qualified interviewers who have been trained in how to establish rapport with a patient, how to remain objective while eliciting feedback, and how to follow an interview guide and adapt it as needed to the patient. This research is important in clinical development for two reasons. Firstly, the life sciences industry is committed to taking a patient-centric view of clinical development. Secondly, qualitative research methodologies have matured. Interviewing patients in clinical trials is a valid approach to understanding the patient perspective of a disease or treatment, providing context for explaining and interpreting quantitative clinical data, and informing future outcome assessment strategies and trials.1 The practice is in line with the US Food and Drug Administration’s recent guidance that recommends interviewing patients enrolled in clinical trials at screening and at an exit in order to assess change over time.2 Qualitative longitudinal research does just that – it uncovers how patients experience healthcare over time, providing the context and details needed to understand and assess meaningful change in their condition. This includes capturing the critical moments’ patients go through that cannot be captured by quantitative patient-reported outcomes (PROs).3

The research can be applied to:

• Documenting the impact of a potential therapy from a patient perspective

• Studying complex phenomena in rare diseases, for example, the onset and duration of symptoms when this is not described in the literature

• Evaluating patient/physician relationships over time

The approach has the advantage of giving a much fuller picture of the patient experience than a single interview conducted at one point in time. Holding multiple interviews also avoids the recall bias that can be present in exit interviews. The depth and frequency of the conversations yield granular data that can offer an extremely vivid picture from a relatively small sample of patients.

When to Use

The qualitative data gathered via patient interviews can be valuable in any therapeutic area but are particularly useful in studies of rare diseases, chronic diseases, and surgical interventions. Possible objectives of such research include:

• The natural history of the disease is poorly known or not well described in the literature
• Insights will be sought on clinical parameters not measured in a trial (such as the onset of action or duration of therapy)

• A small patient sample and lack of valid, specific Clinical Outcomes Assessment measures will limit benefit/risk assessment and value communication

• Regulators are likely to challenge the clinical meaningfulness of the magnitude of change or difference

• It will be important to develop strategies to promote adherence to the therapy

Designing Qualitative Studies The benefits are compelling, so how best to go about designing such studies? These must be designed carefully to produce clear and replicable results, the data must be analysed according to scientifically valid methods, and the findings must be communicated in a way that gives voice to patient views. The research question and a clear vision of how the data will be collected and analysed should guide the study design. Once the purpose is clearly defined, the next step is to develop the interview guide which calls for the experience of a qualitative research scientist to ensure that the questions will elicit meaningful answers. The interview guide should be the same for all patients for the first interview, as it will set the foundation for further assessments and serve as a baseline in following the patient journey. Then, for subsequent interviews, the interview guide can either continue to be the same for all patients, or it can be customised for each patient based on the results of the baseline interviews. In either case, there should be sufficient flexibility in the protocol design to allow for openness in the interview – as directed by a trained and skilled interviewer. The frequency and timing of interviews could either align with the time points for collecting patient-reported outcomes (PRO) in the trial or synchronise with the timing of expected effects of the therapy. Longitudinal qualitative research can be performed as a standalone study, or it can occur within a quantitative study in conjunction with the collection of clinical data, for example within a clinical trial or an observational (natural history) study. In the latter case, the results can be triangulated with the clinical data.3