The COPD Foundation's COPD360Net Initiative Approach to Patient-Centric Drug Development: A Case Study in Using Patient Surveys to Inform New Treatments for Viral Respiratory Infections.

Patient-centric drug development is crucial to creating treatments that address unmet patient needs but is often ignored. The COPD Foundation's COPD360Net® includes a multistakeholder approach for operationalizing patient-centric development of treatments where patients, caregivers, scientists, and clinicians review opportunities based on scientific merit, potential to address an unmet need, and feasibility of adoption. COPD360Net deploys large-scale online community surveys to review profiles of potential therapies based on those criteria. This approach was implemented to inform the development of an intranasal spray to prevent viral respiratory infections (VRIs), a major cause of exacerbations in people with chronic lung diseases. Insights included: Of the 376 respondents with COPD surveyed, frequent exacerbators reported strong interest in a new type of antiviral nasal spray to prevent VRI.Patient survey and advisory committee insights demonstrated that a pan antiviral nasal spray has potential high value to both clinicians and patients and informed the COPD360Net decision to partner on its development.Including patient perspectives from the outset can be conducted efficiently by mobilizing an engaged online patient community.

Outcomes Important to Patients Diagnosed with Both COPD and Sleep Apnea: Findings from the O2VERLAP Study Focus Groups.

INTRODUCTION: Few studies have asked chronic obstructive pulmonary disease (COPD) -obstructive sleep apnea (OSA) overlap syndrome patients what outcomes of continuous positive airway pressure (CPAP) therapy are important to them, while also considering their self-reported CPAP adherence barriers and facilitators. This study conducted a series of focus groups to learn about those issues with the goal of applying these findings to the design of a larger Patient-Centered Outcomes Research Institute-funded scientific study, the O2VERLAP Study. METHODS: People previously diagnosed with both COPD and OSA, who were current or recent past users of the standard therapy for each condition (i.e., supplemental oxygen and CPAP, respectively), were included. Three qualitative focus groups were run using different communication modalities: (1) teleconference (audio only), (2) in-person, and (3) web-based. RESULTS: The focus groups included a total of 17 participants. The telephone and online focus groups resulted in more relevant and content-rich transcripts, while the in-person focus groups had far fewer excerpts able to be coded. Participants were most concerned about mask fit and comfort. Other key factors affecting CPAP use included nasal dryness and issues concerning insurance. The most important outcome to patients was daytime functioning. DISCUSSION: This work found that telephone and web-based qualitative focus groups resulted in greater topical discussions than in-person focus groups, likely due to the customary socializing that occurs in-person. The study identified: (1) CPAP use barriers and facilitators that helped guide intervention development for the larger study and, (2) daytime functioning as the most important outcome for patients.

Effect of Two Interventional Strategies on Improving Continuous Positive Airway Pressure Adherence in Existing COPD and Obstructive Sleep Apnea Patients: The O2VERLAP Study.

Background: Obstructive sleep apnea (OSA) is a sleep disorder prevalent in >10% of individuals diagnosed with chronic obstructive pulmonary disease (COPD). Continuous positive airway pressure (CPAP) is the first-line therapy for OSA, but many do not use it enough during sleep to effectively manage OSA. The O2VERLAP study compared proactive care (PC)-structured web-based peer-coaching education and support intervention versus reactive care (RC)-education and support based on limited scheduled interactions and patient-initiated contacts. Methods: Participants were primarily recruited from patient communities (COPD, OSA, and the National Patient-Centered Outcomes Research Network [PCORnet]) through electronic methods. Inclusion criteria: ≥40 years old, diagnosis of both COPD and OSA, and currently using CPAP. Participants were then randomly assigned to either the PC or RC group, with outcomes assessed at baseline and 6 and 12 weeks. The primary study outcome was CPAP adherence (hours of use/night) and secondary outcomes were daytime functioning, sleep quality, and daytime sleepiness. Changes in outcomes over time were examined using random effects models. Results: The study enrolled 332 participants of which 294 were randomized. While groups differed significantly in CPAP adherence at baseline (PC: 6.1±3.1, RC: 7.3±2.4 hours/night; P<0.001), there were no significant differences in change of primary and secondary outcomes at either 6 or 12 weeks. Conclusion: In this group of patients with both COPD and OSA on CPAP therapy, no difference was found between the provision of PC and RC. The study did find unexpectedly high baseline CPAP adherence levels, which suggests that any improvement from the intervention would have been very small and difficult to detect.

Developing a patient-driven chronic obstructive pulmonary disease (COPD) research agenda in the U.S.

BACKGROUND: To document a generalizable process for developing a patient-prioritized chronic obstructive pulmonary disease (COPD) research agenda and to provide an overview of domains that were developed in response to people living with COPD and caregivers' suggestions for research. METHODS: Adults with COPD and caregivers who are members of the COPD Patient-Powered Research Network (PPRN) provided suggestions for COPD-related research through a self-administered, online survey. These responses were analyzed with a content analysis approach: domains for categorizing all survey responses were created, then all responses were categorized independently by a group of researchers, then these categorizations were adjudicated, and finally a density map was created that represented the number of responses in each of the domains. RESULTS: At the time of analysis, 6157 adults had fully completed the baseline survey. Survey responses were categorized across seven domains as follows: 22.5% of all responses fell into the domain family/social/community research, 20.8% of all responses fell into the domain well-being, 15% of all responses fell into the domain curative research, 14.6% of all responses fell into the domain biomedical therapies, 10.5% of all responses fell into the domain policy concerns, 6% of all responses fell into the domain holistic therapies and 10.7% of all responses fell into the domain ambiguous comments that could not be translated into concrete research topics. CONCLUSION: Using qualitative open-ended survey responses from the COPD PPRN registrants, we were able to identify six key domains of research about COPD that are considered most important by patients. These domains differ in content from prior scientist-led efforts to develop priorities for COPD research, demonstrating the ongoing importance of involving patients and their caregivers in determining research priorities. The results suggest the field can more closely align research efforts to patient priorities by considering the identified domains.

The COPD Patient-Powered Research Network (PPRN) asked adults with COPD and their caregivers to suggest areas for future research on COPD. Researchers analyzed 7336 survey responses and created six research areas to reflect their suggestions: (1) family/social/community research, (2) well-being, (3) research on a cure for COPD, (4) new drugs and treatments, (5) policy concerns, and (6) natural therapies. Several research topics patients and their caregivers identified as important have received little attention from researchers in the past and could be valuable topics for researchers to explore.

Self-reported COPD Medication Use and Adherence in the COPD Foundation Patient- Powered Registry Network.

PURPOSE: Pharmacotherapy is one cornerstone of Chronic Obstructive Pulmonary Disease (COPD) management. Published U.S. data seldom includes patient-reported COPD medication use and adherence. We add this patient perspective to the commonly reported administrative prescribing and fill data. METHODS: This survey study used inhaler and nebulizer pictures and lists of oral COPD medications to query members of the COPD Foundation Patient-Powered Research Network, a national self-reported online registry. Medications used, adherence, inhaler education, cost concerns, previous exacerbations, and COPD Assessment Test scores were assessed and summarized using simple descriptive statistics and hazard ratios controlling for age, gender, and disease burden. RESULTS: Respondents mean age was 68 years, 60% were women, >69% with the COPD Assessment Test (CAT) scores >15, and >50% reported 2 or more exacerbations in the past 12 months. Overall, >98% used one or more inhaled COPD medications, 7.6% rescue inhaler only, 17.8% used long-acting bronchodilator only therapy (11.1% dual), and 72.8% using corticosteroid therapies, including 53% triple therapy. Nebulizers were used by 59.4% and 34.8% use oral COPD medications. Reported adherence rates were high (80.1%), but 41% reported trouble paying for medications, with 20.1% reported missing medications due to cost. CONCLUSIONS: In this population, COPD had a high burden with >50% of respondents using triple therapy, and one in eight maintenance oral corticosteroids. Self-reported adherence was high, but with significant cost concerns reported resulting in missed medications.

Pilot Study of a Patient Experience with an ELLIPTA Inhaler Electronic Medication Monitor and Associated Integrated System: A Prospective Observational Study Using the COPD Patient-Powered Research Network.

BACKGROUND: Electronic medication monitors (EMMs) are associated with decreased rescue inhaler use, symptom burden, and increased medication adherence in asthma. However, the use of EMMs in people with chronic obstructive pulmonary disease (COPD) using the ELLIPTA dry powder inhaler has not been studied. METHODS: This was an open-label, single-arm, prospective observational study of EMMs and associated application (app) use over 12 weeks and up to 24 weeks (April-October 2019) in people with self-reported COPD aged ≥40 years enrolled in the COPD Patient-Powered Research Network, using an ELLIPTA inhaler. The primary outcome was daily active use of the app over 12 weeks. Treatment adherence, rescue inhaler use, and participant satisfaction were assessed over the same period. RESULTS: Among the 122 participants, mean (standard deviation [SD]) proportion of days participants opened the app was 59.5% (31.4), 51.1% (33.5) and 41.3% (34.2) for Days 1-30, 31-60 and 61-90, respectively. Mean (SD) adherence to maintenance medication remained stable: 80.2% (22.7) and 79.9% (26.7) for Days 1-30 and 61-90, respectively. In participants using a rescue inhaler and EMM, mean (SD) rescue-free days increased from 18.5 (10.0; Days 1-30, n=51) to 21.4 (9.6; Days 61-90, n=48). Participants reported high levels of confidence in using the EMM, valued app reminders highly and reported high system satisfaction (mean [SD] scale: 1=low, 5=high; 4.6 [1.1], 4.3 [1.1] and 4.1 [1.1], respectively). CONCLUSIONS: Use of an ELLIPTA EMM with frequent app engagement, high participant satisfaction and decreased rescue medication use may aid COPD management.

Electronic Health Records and Pulmonary Function Data: Developing an Interoperability Roadmap. An Official American Thoracic Society Workshop Report.

A workshop "Electronic Health Records and Pulmonary Function Data: Developing an Interoperability Roadmap" was held at the American Thoracic Society 2019 International Conference. "Interoperability" is defined as is the ability of different information-technology systems and software applications to directly communicate, exchange data, and use the information that has been exchanged. At present, pulmonary function test (PFT) equipment is not required to be interoperable with other clinical data systems, including electronic health records (EHRs). For this workshop, we assembled a diverse group of experts and stakeholders, including representatives from patient-advocacy groups, adult and pediatric general and pulmonary medicine, informatics, government and healthcare organizations, pulmonary function laboratories, and EHR and PFT equipment and software companies. The participants were tasked with two overarching Aobjectives: 1) identifying the key obstacles to achieving interoperability of PFT systems and the EHR and 2) recommending solutions to the identified obstacles. Successful interoperability of PFT data with the EHR impacts the full scope of individual patient health and clinical care, population health, and research. The existing EHR-PFT device platforms lack sufficient data standardization to promote interoperability. Cost is a major obstacle to PFT-EHR interoperability, and incentives are insufficient to justify the needed investment. The current vendor-EHR system lacks sufficient flexibility, thereby impeding interoperability. To advance the goal of achieving interoperability, next steps include identifying and standardizing priority PFT data elements. To increase the motivation of stakeholders to invest in this effort, it is necessary to demonstrate the benefits of PFT interoperability across patient care and population health.

The Burden of Cough and Phlegm in People With COPD: A COPD Patient-Powered Research Network Study.

RATIONALE: Cough and phlegm are common symptoms of chronic obstructive pulmonary disease (COPD) and may significantly affect quality of life. This study assessed the burden of cough and phlegm on clinical outcomes and quality of life among people with a self-reported physician diagnosis of COPD. METHODS: Patient-reported data from the COPD Foundation's Patient-Powered Research Network (COPD PPRN) were utilized. Cough and phlegm severity and frequency were assessed by responses to questions on the COPD Assessment Test (CAT) and categorized into none/low, moderate and severe. Quality of life domains were evaluated using the Patient-Reported Outcome Measurement Information System (PROMIS-29). Associations between cough and phlegm levels and PROMIS-29 domains were examined using multivariate analysis of variance (MANOVA). RESULTS: The 5286 participants were average age 64.4 years (SD=11.4), 87.9% white, 60.4% female, 51.2% married, and 42.2% with caregivers. Approximately three-fourths of the participants had moderate or severe cough or phlegm levels. Respondents with moderate and high cough or phlegm had significantly worse dyspnea (p<0.0001), more exacerbations in the previous one year (p<0.0001), worse physical and social functioning, and more symptoms of anxiety and depression on PROMIS-29 compared to those with no/low cough and phlegm. CONCLUSIONS: In this group of people with COPD, higher levels of cough and phlegm are associated with worse clinical and quality of life outcomes.

Patient-Reported Consequences of Community-Acquired Pneumonia in Patients with Chronic Obstructive Pulmonary Disease.

Community acquired pneumonia (CAP) carries high morbidity, mortality, and economic burden, which is even higher in adults diagnosed with chronic obstructive pulmonary disease (COPD). While several studies have assessed the clinical burden and mortality risk of CAP and COPD, very few studies focus on CAP burden from a COPD patient perspective. Individuals recently diagnosed with CAP and with pre-existing COPD were recruited through the COPD Foundation. The CAP Burden of Illness Questionnaire (CAP-BIQ), a content validated questionnaire assessing CAP symptomatology, duration of symptoms and CAP impact on work, activities and family, was administered at baseline and at 30-days follow-up. Of the 490 participants recruited, 481 had data sufficient for analysis. The prevalence of respiratory-related symptoms was very high (>90%) at the time of diagnosis with other generalized symptoms such as fatigue, trouble sleeping, headaches and confusion present in more than 60% of participants. Mean duration of symptoms varied from approximately 2 weeks for headaches and fever to more than a month for fatigue, wheezing, dyspnea, and cough. Employed participants missed an average of 21 days of work and those not employed missed 36 days of usual activities. Over 84% required help from family, friends or care givers. CAP is a serious and burdensome condition for people with COPD, a condition that can impair activities for weeks, frequently requires care from family or friends, and includes lingering symptoms. The patient-reported impact of CAP reported in this study underscores the need for prevention strategies in this population.