Literature Review
The first step in our approach was a targeted literature review, performed to understand published literature regarding patient needs and expectations related to COPD. The findings of this literature review revealed that most of the patient preference studies (15 of the total 22 retrieved studies in COPD) have traditionally evaluated key attributes around device preferences. Only two studies focused on the disease-related aspects that patients would most likely to alleviate or on an ideal treatment profile for COPD from the patients’ perspective [9, 10]. This analysis offered us an understanding of the current knowledge gap concerning preferences of COPD patients and provided the basis for developing the search strategy for the subsequent social media analysis.
SML
The next step was an SML study—an analysis of online conversations in open-access platforms among patients with COPD. Analysis of patient interactions on social media is a quick approach to obtain rich information about patient unmet needs, disease management, and delivery of care [11]. Importantly, SML allows researchers to “listen” to the open discussions that are already happening online in the patients’ own words, without influencing those conversations and imposing any further research burden on the patient community [12, 13]. Although it is convenient to gather patient inputs via studying social media postings, there are inherent limitations due to the nature of SML, such as self-selection bias (not everyone engages online; greater likelihood of negative aspects being posted; may include a narrow representation of clinical/demographic characteristics) limiting generalizability; no verification of identity; and the passive observational nature not allowing further probing to seek clarification on the posted views [14, 15]. At this time there is a lack of guidance surrounding the key ethical issues such as privacy, anonymity, and informed consent (voluntary participation) that may arise in analysing the content posted on social media [16]. To respect patient privacy during the SML study conduct and reporting, we adhered to the data privacy obligations in accordance with the Health Insurance Portability and Accountability Act (HIPAA) [17]. All the data for SML study were obtained from publicly accessible sources without accessing password-protected information and only aggregated qualitative findings derived based on the anonymized content were reported. The detailed methodology along with results of COPD SML study has been reported elsewhere [6]. As a follow-up to the SML study, it is important to conduct qualitative research studies with patients to confirm and expand on the findings from SML.
OBB
Subsequently we conducted an OBB study to further validate and explore the patient insights revealed from SML in greater detail with a qualitative approach. Twenty COPD patients (UK, n = 10; USA, n = 10) participated in the OBB that ran for over 2 weeks [7]. Being an asynchronous, moderated, closed online community platform (similar to a private chat room) for qualitative patient research, an OBB allows participants to answer predefined questions in a comprehensive manner [12]. Information derived from the literature search and SML study formed the basis for the questions developed for the OBB. An OBB offers the interactive benefits of a face-to-face focus group discussion, but without certain individuals dominating the conversation. It also has the potential to generate deep insights, including emotional and sensitive aspects of living with a disease, which may not surface in a traditional interview settings [12, 18]. A key strength of this exercise is that it provides a platform for candid, honest, and shared patient-to-patient interactions while maintaining anonymity through the study [12, 19]. When the participating patients were contacted for debriefing at the end of the OBB session, the patients expressed agreement with the conclusions derived from the discussions and also expressed that they were very engaged throughout their participation in the study. The comments shared by patients on the final day of the OBB study reflected that patients really valued the opportunity to participate in this form of research and share experiences with ‘like’ patients.
The detailed findings from the qualitative online insight gathering approaches (SML and OBB studies) in COPD have been reported in their respective full publications [6, 7]. In short, the results of these studies indicated that besides dyspnoea and exacerbations, cough and mucus production are very important aspects of the disease for COPD patients. The findings from the SML study showed that cough and mucus production were the symptoms affecting quality of sleep, work, and daily activities irrespective of severity of disease [6]. These findings also revealed that relief from cough, mucus production, and shortness of breath would be the most desirable aspects of disease management from a patient perspective (Table 2) [6, 7].
Table 2 Key findings from the SML and OBB studies [6, 7] The results from the OBB study conducted with patients suggested that the symptoms of cough and excess mucus production are particular issues for the patients early in the day, with the effort of mucus expulsion leaving them tired and exhausted for the day ahead. These cough and mucus symptoms also result in the need for continuous breaks during the daytime making most patients exhausted. The symptoms also manifest during the night (phlegm accumulates when lying down and introduces sense of suffocation, together with the cough causing sleep disturbance) [7]. Another important observation from the OBB study was that patients with COPD experience urinary incontinence associated with cough but tend to be uncomfortable talking openly about such sensitive symptoms (hence this was not a prominent complaint in social media posts); in fact, two-third of the study participants from OBB who suffered from chronic cough acknowledged incontinence to be a significant problem [6, 7]. To build further on the significance and relative importance of these symptoms to patients (cough and mucus secretion and consequences thereof such as sleep disturbance, fatigue, incontinence) compared with the more commonly measured end points (lung capacity or breathlessness; exacerbations/hospitalizations) in COPD, there was a need to evaluate them in a quantitative patient preference study.
Web-Based Patient Preference Study
Patient preferences reflect patients’ choices among different disease state alternatives or particular health interventions (drug or medical device) over other available options, based on trade-offs between the advantages and disadvantages of the preferred choice [20, 21]. To make a choice or stated preference, patients need to weigh the advantages and disadvantages of each disease state or intervention and compare them with the alternatives. The methods to derive patient preferences focus on understanding the relative importance of attributes by patients and provide an alternative to characterize patient needs and desires [21]. We are evaluating the findings from the SML and OBB studies in a quantitative patient preference study. This study is currently ongoing via a discrete-choice experiment (DCE) online survey in patients with COPD, which will further quantify the relative importance of different symptoms from the patients’ perspective.
While designing the preference study we approached a health technology assessment (HTA) agency to obtain input on study design—a first of its kind ‘scientific advice’ from an HTA body. We sought input from NICE (The National Institute for Health and Care Excellence) on the COPD patient preference study design and incorporated many suggestions received through this early scientific advice into the study protocol [22]. In particular, considering the recommendations by NICE, the study protocol was updated for eligibility criteria, quality of life questionnaire instrument selection, and total number of patients to be recruited for selected countries.
The patient preference study will use a DCE design, built on the principle of random utility theory, that assumes that disease states can be decomposed into various attributes (symptoms) and the attractiveness/unattractiveness of a disease state depends on patients’ relative preferences for the attributes (symptoms) expressed by the frequency with which they choose their preferred disease profile [23]. In our DCE survey, patients with COPD will be asked to choose between two disease states with varying levels of the aforementioned attributes, as expressed by their willingness to accept trade-offs among them [24]. This ‘disease state’ preference survey is a fairly novel approach as typically the discrete choice is made between two (hypothetical) treatment options. We believe the choice among disease states is a more appropriate exercise for patients when investigating the relative importance of symptoms to them, as it avoids any confusion between attributes/symptoms being incorrectly interpreted as side effects of a product. The results of this ongoing preference study will provide estimates of the relative importance of different symptoms to patients, which in turn will provide evidence about the importance of incorporating these patient-relevant end points in clinical trials. Furthermore, the results will provide a basis for determining the relative value, from the patients’ perspective, that future treatments would offer that address these same symptoms to a greater or lesser degree.