Patient listening on social media for patient-focused drug development: a synthesis of considerations from patients, industry and regulators

Patients, life science industry and regulatory authorities are united in their goal to reduce the disease burden of patients by closing remaining unmet needs. Patients have, however, not always been systematically and consistently involved in the drug development process. Recognizing this gap, regulatory bodies worldwide have initiated patient-focused drug development (PFDD) initiatives to foster a more systematic involvement of patients in the drug development process and to ensure that outcomes measured in clinical trials are truly relevant to patients and represent significant improvements to their quality of life. As a source of real-world evidence (RWE), social media has been consistently shown to capture the first-hand, spontaneous and unfiltered disease and treatment experience of patients and is acknowledged as a valid method for generating patient experience data by the Food and Drug Administration (FDA). While social media listening (SML) methods are increasingly applied to many diseases and use cases, a significant piece of uncertainty remains on how evidence derived from social media can be used in the drug development process and how it can impact regulatory decision making, including legal and ethical aspects. In this policy paper, we review the perspectives of three key stakeholder groups on the role of SML in drug development, namely patients, life science companies and regulators. We also carry out a systematic review of current practices and use cases for SML and, in particular, highlight benefits and drawbacks for the use of SML as a way to identify unmet needs of patients. While we find that the stakeholders are strongly aligned regarding the potential of social media for PFDD, we identify key areas in which regulatory guidance is needed to reduce uncertainty regarding the impact of SML as a source of patient experience data that has impact on regulatory decision making

COPD depicted - patients drawing their lungs

Ad A Kaptein,1 Jitske Tiemensma,2 Elizabeth Broadbent,3 Guus M Asijee,4,5 Maarten Voorhaar4,5 1Medical Psychology, Leiden University Medical Centre (LUMC), Leiden, the Netherlands; 2Psychological Sciences, University of California, Merced, CA, USA; 3Psychology Department, Auckland University Medical School, Auckland, New Zealand; 4CAPHRI School for Public Health and Primary Care, Maastricht University, Maastricht, the Netherlands; 5Boehringer Ingelheim Pharma GmbH & Co. KG, Ingelheim am Rhein, Germany Background: Given the increasing importance of patient-reported outcomes (PRO) in quality medical care, we examined the value and feasibility of an innovative method for assessing patients’ illness perceptions, represented in drawings made by patients with COPD of their lungs. Aim: The aim of our study was: to study patients’ representation of COPD as reflected in their drawings of their lungs; and to examine scores on a validated measure that assesses illness perceptions (ie, Brief Illness Perception Questionnaire [B-IPQ]). Patients and methods: One hundred outpatients with COPD, mean age 70 years, selected from a pharmacy database, participated and 98 filled out the B-IPQ. Eighty-seven patients completed the drawing task. Results: The illness perceptions as reflected in the responses to the B-IPQ scales represented a quite optimistic view of COPD and its consequences. The drawings of the lungs reflected a considerable discordance between patients’ representations and medically accepted representations of lungs of a person with COPD. Conclusion: Assessing illness perceptions in clinical care and research about COPD offers opportunities to identify goals for patient education and self-management. Inviting patients to draw their illness is an innovative and promising approach to assessing PRO. Keywords: COPD, drawings, illness perceptions, PRO, quality of life, self-managemen

Illness perceptions and coping determine quality of life in COPD patients

Jitske Tiemensma,1 Erin Gaab,2 Maarten Voorhaar,3,4 Guus Asijee,3,5 Adrian A Kaptein6 1Psychological Sciences, 2Health Sciences Research Institute, University of California, Merced, CA, USA; 3Department of Family Medicine, Maastricht University, Care and Public Health Research Institute School for Public Health and Primary Care, Maastricht, the Netherlands; 4Boehringer Ingelheim, Alkmaar, the Netherlands; 5Boehringer Ingelheim, Amsterdam, the Netherlands; 6Department of Medical Psychology, Leiden University Medical Centre, Leiden, the Netherlands Background: A key goal of chronic obstructive pulmonary disease (COPD) care is to improve patients’ quality of life (QoL). For outcomes such as QoL, illness perceptions and coping are important determinants.Aim: The primary aim was to assess the associations between illness perceptions, coping and QoL in COPD patients. A secondary aim was to compare illness perceptions and coping of patients with reference values derived from the literature.Patients and methods: A total of 100 patients were included in the study. Patients were asked to complete the Brief Illness Perception Questionnaire (B-IPQ), the Utrecht Proactive Coping Competence scale (UPCC), and a QoL item. Correlations and linear regression models were used to analyze the data. Student’s t-tests were used to compare patients with COPD with reference values derived from the literature.Results: Patients with better understanding of COPD utilized more proactive coping strategies (P=0.04). A more intense emotional response to COPD was related to less proactive coping (P=0.02). Patients who reported using more proactive coping techniques also reported to have a better QoL (P<0.01). Illness perceptions were also related to QoL: more positive illness perceptions were related to a better QoL (all P<0.05). Patients with COPD reported more negative illness perceptions than people with a common cold or patients with asthma (all P<0.01), but reported similar perceptions compared with patients with diabetes.Conclusion: Patients with COPD reported a moderate QoL, but appeared to be proficient in proactive coping. Illness perceptions, coping, and QoL were all associated with each other. Patients reported more strongly affected illness perceptions compared to people with a cold and patients with asthma. We postulate that a self-management intervention targeting patients’ illness perceptions leads to improved QoL. Keywords: COPD, illness perceptions, coping, QoL, common sense mode

A Mixed-Methods Study to Better Measure Patient-Reported Pain and Fatigue in Soft Tissue Sarcoma

Abstract Introduction Pain and fatigue are commonly reported by patients with soft tissue sarcoma (STS) as distressing symptoms, yet no patient-reported outcome (PRO) measures have been validated or developed specifically for STS. This study aimed to develop novel PRO scales using existing item banks to measure pain and fatigue in STS. Methods A three-stage mixed-methods approach was used. Stage 1: a literature review examined the development and validation of the European Organization for Research and Treatment of Cancer (EORTC) library, Patient-Reported Outcomes Measurement Information System (PROMIS) pain/fatigue item banks, Functional Assessment of Cancer Therapy-General, and FACIT-Fatigue. Conceptual models were developed for pain and fatigue. Stage 2: semi-structured interviews were conducted with clinical experts (n = 3) and STS patients (n = 28) to ensure conceptual coverage and cognitively debrief the selected PRO items. Stage 3: exploratory Rasch measurement theory (RMT) analyses were performed to examine the measurement properties of the proposed scales. Results Stage 1: The conceptual model for fatigue was organized into two overarching domains: fatigability and fatigue, further split into two subdomains: symptoms and impact. The conceptual model for pain had one overarching domain split into two subdomains: descriptors and impact. Pain (n = 56) and fatigue (n = 40) items were selected from the EORTC item library. Stage 2: qualitative findings ensured conceptual coverage, provided insight into the relevance and comprehension of the items, and informed subsequent item reduction. Stage 3: The total item number was reduced to 43 (pain n = 18, fatigue n = 25). Exploratory RMT analyses supported the final scales’ psychometric properties. Conclusions This mixed-methods research generated important information on the experience of pain and fatigue in specific subtypes of STS. Five novel PRO scales have been developed through careful item selection in consultation with experts and supported by qualitative and quantitative evidence. These scales may be of value to future clinical trials for STS

Validation of the Dutch version of the primary care resources and support for self-management tool:A tool to assess the quality of self-management support

INTRODUCTION: Enhancing the self-management activities of patients improves the quality of care and is an integrated element of current healthcare provision. However, self-management support (SMS) is not yet common in healthcare. The Primary Care Resources and Support for Self-Management (PCRS) is a tool for healthcare professionals to assess the quality of SMS. In this study, we assessed the validity and reliability of the Dutch version of the PCRS. METHOD: The validation of the PCRS was performed in Dutch healthcare centres. Correlations between the PCRS scores and the Assessment of Chronic Illness Care (ACIC) and Clinician Support for Patient Activation Measure (CS-PAM) scores were calculated to assess the convergent and discriminant validity. A confirmatory factor analysis (CFA) was performed to test the factor structure. Lastly, the internal consistency and face validity were assessed. RESULTS: The convergent and discriminant validity were good, with respective correlations of 0.730 (p 0.050) between the PCRS and the ACIC SMS subscale and the PCRS and the CS-PAM. Although 49% of the variance of the PCRS was explained by one factor, the CFA could not confirm a fit between a one-factor model and the data. The reliability was excellent (Cronbach's α = 0.921). CONCLUSION: The PCRS showed good validity and excellent internal consistency. However, the evidence for its validity was inconclusive. We therefore suggest rephrasing specific items

Patient listening on social media for patient-focused drug development: a synthesis of considerations from patients, industry and regulators

Cimiano P, Collins B, De Vuono MC, et al. Patient listening on social media for patient-focused drug development: a synthesis of considerations from patients, industry and regulators. Frontiers in Medicine. 2024;11: 1274688.Patients, life science industry and regulatory authorities are united in their goal to reduce the disease burden of patients by closing remaining unmet needs. Patients have, however, not always been systematically and consistently involved in the drug development process. Recognizing this gap, regulatory bodies worldwide have initiated patient-focused drug development (PFDD) initiatives to foster a more systematic involvement of patients in the drug development process and to ensure that outcomes measured in clinical trials are truly relevant to patients and represent significant improvements to their quality of life. As a source of real-world evidence (RWE), social media has been consistently shown to capture the first-hand, spontaneous and unfiltered disease and treatment experience of patients and is acknowledged as a valid method for generating patient experience data by the Food and Drug Administration (FDA). While social media listening (SML) methods are increasingly applied to many diseases and use cases, a significant piece of uncertainty remains on how evidence derived from social media can be used in the drug development process and how it can impact regulatory decision making, including legal and ethical aspects. In this policy paper, we review the perspectives of three key stakeholder groups on the role of SML in drug development, namely patients, life science companies and regulators. We also carry out a systematic review of current practices and use cases for SML and, in particular, highlight benefits and drawbacks for the use of SML as a way to identify unmet needs of patients. While we find that the stakeholders are strongly aligned regarding the potential of social media for PFDD, we identify key areas in which regulatory guidance is needed to reduce uncertainty regarding the impact of SML as a source of patient experience data that has impact on regulatory decision making