ARCH CONFERENCE Integrating Patients into Meaningful Real-World Research Susan J. Bartlett1, Teresa Barnes2, and R. Andrew McIvor3 1
Department of Medicine, McGill University, Respiratory Epidemiology and Clinical Research Unit, and Division of Clinical Epidemiology, Royal Victoria Hospital, Montreal, Quebec, Canada; 2American Thoracic Society Public Advisory Round Table and Vice President of the Coalition for Pulmonary Fibrosis, Culver City, California; and 3Department of Medicine, Firestone Institute for Respiratory Health, McMaster University, Hamilton, Ontario, Canada
Abstract Research in respiratory, sleep, and critical care medicine has historically been the domain of scientists and clinicians attempting to understand pathophysiological mechanisms and consequences of disease in an effort to develop effective treatments. This traditional approach of placing scientific rigor before the patient’s reality is changing. There is growing recognition of the importance of integrating patient perspectives (e.g., preferences, expectations, and expanded definitions of what constitutes “successful” outcomes) into clinical research to achieve meaningful results for a broader group of stakeholders. This evolution is reflected in the growth of patientcentered organizations and patient advocacy groups that seek to meaningfully integrate patients into the process of prioritizing research needs and creating alliances wherein patients and
researchers can partner together to accomplish research goals. In tandem, a growing number of real-world trials (i.e., those with broader, more representative patient populations and routine care pathways) now complement findings from traditional randomized controlled trials and offer new opportunities to design studies that better reflect patients’ healthcare preferences and experiences. Patients’ perspectives are key determinants of treatment adherence and outcomes, as well as the feasibility and likely value of implementing care pathways. The advent of smartphone and push technologies offer new opportunities for the collection of more patient-centered and ecologically valid patient data, thereby adding new dimensions to meaningfully integrate patients into real-world research. Keywords: patient-centered research; patient-centered outcomes; patient engagement
(Received in original form September 26, 2013; accepted in final form December 5, 2013 ) Supported by the Respiratory Effectiveness Group, which paid the publication costs. Author Contributions: All authors contributed to conception, critical revision, and final approval of the manuscript. S.J.B. integrated sections drafted by each author to develop the first draft. Correspondence and requests for reprints should be addressed to Susan J. Bartlett, Ph.D., Associate Professor of Medicine, McGill University/Royal Victoria Hospital, 687 av des Pins Ouest, Ross 4.31, Montreal, PQ, H3A 1A1 Canada. E-mail: [email protected] Ann Am Thorac Soc Vol 11, Supplement 2, pp S112–S117, Feb 2014 Copyright © 2014 by the American Thoracic Society DOI: 10.1513/AnnalsATS.201309-327RM Internet address: www.atsjournals.org
Chronic pulmonary conditions, critical illness, and sleep disorders impact multiple aspects of an individual’s physical, emotional, and mental health. Beyond reducing symptoms, clinical interventions are increasingly judged not only on their ability to alleviate symptoms but also on their ability to reduce impairments to each of these health domains. This shift toward incorporating a more patient-centered view of health and wellbeing began many years ago with the development of specific health-related quality of life (HRQL) tools. Although the growing trend of moving beyond classically designed randomized controlled trials (RCTs) to developing studies S112
that better capture “real life” experiences and outcomes valued by patients is a necessary step, there is also a need for proactive patient engagement to ensure the patient perspective is integrated early in the conception of a study to ensure that outcomes are meaningful and relevant to patients and other healthcare stakeholders.
The Need for Patient-centered and “Real World” Research Research in pulmonary medicine, critical illness, and sleep has historically been driven by questions identified by scientists and
clinicians who focused largely on understanding pathophysiological mechanisms and consequences of disease in an effort to develop effective therapeutics. This failure to engage patients in the design of traditional research may have, inadvertently, placed scientific rigor before the patient’s reality. Indeed, the research contributions of individuals living with chronic diseases has been largely relegated to whether or not they wish to participate as “subjects” in clinical trials. There has been little opportunity for patients to help identify important research questions, let alone participate in the design, analysis, interpretation, or dissemination of study
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ARCH CONFERENCE results. Furthermore, when the evidence base used to develop treatment guidelines relies on findings from RCTs that enroll highly selected participants, the result is often interventions and care pathways that are challenging or, at times, unrealistic to implement. In an era of healthcare reform, patients are increasingly acknowledged as having unique and important perspectives about treatment goals, preferences, and healthcare experiences—all of which are key determinants of adherence and real-life outcomes. Observational studies and pragmatic trials represent an alternative approach to gathering information across a broader group of patients. Health decision makers increasingly seek “real world” data from observational trials to complement those generated by RCTs (1). (We acknowledge the term “real world” is disputed by some. Just as the strong internal validity of RCTs also reduces generalizability of results, observational studies, too, have important limitations, including selection bias and confounding, which often remain unmeasured but have the potential to significantly influence outcomes observed. All studies, too, are vulnerable to the Hawthorne effect.) Mixed method approaches now add qualitative elements (e.g., individual interviews, focus groups) to traditional RCTs to gain insight into participants’ expectations, interpretation of symptoms, treatment, healthcare experiences, and other factors that influence persistence with therapeutic regimens. However, to create research that reflects the lived experiences of patients and offers results that are meaningful results to patients and other healthcare stakeholders, it is necessary not only to incorporate the patient voice but also to actively engage patients as partners and key stakeholders in the research process.
Understanding What Matters Most to Patients To have a true appreciation for the perspective of medical consumers (patients, families, caregivers), it is important to recognize that they are not only stakeholders in healthcare and the healthcare system but that they are the ultimate stakeholder—and, arguably, the ones with the most at stake. Decisions that patients make about whether to initiate and adhere to prescribed
treatments reflect a complex and evolving process that often begins well before scripts are provided at clinic visits or participants enroll in clinical trials. For many, decisions about the necessity and potential benefits of treatment emerge around the time of diagnosis and continue to evolve in relation to ongoing treatment experiences. Historically, patients have been expected to “comply” with medical advice, even if their understanding of their disease has been limited and no attempt has been made to engage them in decision making about their treatment. There has been longstanding concern that it may be unrealistic and potentially burdensome to expect patients to understand the myriad of considerations of complex diseases and multimorbidity to participate in medical decision making. Rather than asking patients directly about treatment preferences and needs, the medical community has traditionally examined indirect evidence to gain insight into patient perspectives and preferences. For example, factors such as recruitment rates and study withdrawals in clinical trials have been interpreted as evidence of the acceptability, tolerability, and relative value and burden of treatments. Treatment preferences among patients are often surmised by examining adherence, for example, to oral versus inhaled therapies as well as to specific types of inhalation devices. Up to half of all medicines prescribed for chronic conditions are not taken as prescribed (2). Often suboptimal adherence, or even treatment refusal, is viewed as evidence of patient aversion to treatmentassociated risks. However, patients are generally willing to accept risk if they also perceive the trade-off is the opportunity for benefits they value. There is an unwritten understanding among patients, especially those suffering from rare diseases or lifethreatening conditions with few or no lifesaving options, that “benefit” is not necessarily the same benefit sought by researchers. All patients want treatments that can reduce the morbidity and mortality associated with their disease; however, many prioritize improved quality of life over simply extending life (3, 4). In the words of William Gahl, M.D., of the National Institutes of Health in a recent TED talk about rare diseases, people “want to be protected, but they don’t want to be protected to death” (5). Patients and
Bartlett, Barnes, and McIvor: Patient Voice/Engagement in Research
families want to be “heard” to make sure the medical/research community understands their plight, their fight, and their desire to contribute meaningful information that may not save their lives but may improve the HRQL of patients who follow them.
Incorporating the Patient Perspective into Research Greater understanding of patient preferences and expectations together with expanded definitions of what constitutes “successful” outcomes are examples of how the patient perspective can ensure that ongoing research offers meaningful results to a broader group of stakeholders. As partners in the research process, patients can help researchers to prioritize what needs to be studied and how to work together effectively to accomplish research goals. Chronic obstructive pulmonary disease (COPD) can serve as a good exemplar. With COPD, a complex systemic illness, the earliest manifestation is airflow obstruction that is only partially reversible. As the disease progresses, patients experience increasingly frequent and severe exacerbations and develop comorbidities that impact multiple aspects of HRQL. For example, impairments in lung function result in changes in day-to-day function, such as activity limitations due to decreased walking capacity, reduced participation, and changes in social and work roles. Traditionally, medical specialists have relied on lung function measures (e.g., FEV1) to both diagnose COPD and gauge the effectiveness of treatments. However, people living with COPD and their families may judge treatment effectiveness by the extent to which it reduces bothersome symptoms and allows them to participate fully and effectively in valued activities at work and at home. Thus, there is a need to include patients in the process of identifying outcomes that resonate with their experiences. As a result, patientreported outcomes (PROs) are increasingly recognized as crucial elements that complement traditional medical data in the comprehensive assessment of COPD, both for determining the impact and burden of the disease itself and also for evaluating the success of therapeutic interventions. The collection of standardized PROs has been S113
ARCH CONFERENCE reflected first though the use of the Chronic Respiratory Questionnaire and then St. George’s Respiratory Questionnaire in COPD research. The growing practice of routinely incorporating outcomes that are valued and reported by patients as well as assessments of the impact on illness and treatment on HRQL reflects an important evolution of priorities in pulmonary, critical care, and sleep research. Comparative effectiveness research (CER) has been defined as “the conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat, and monitor health conditions in ‘real world’ settings” (6). CER has also now evolved to routinely include patient perspectives. The goal of CER is to provide patients, their advocates and caregivers, healthcare professionals, federal officials, policy makers, and payers with evidence-based information to make informed healthcare decisions (7). However, to achieve this goal, research must be conceived, implemented, and disseminated in ways that ensure results are meaningful to patients, caregivers, and other decision makers. Previously, CER studies were designed by researchers with relatively little input from patients, but patient engagement is rapidly gaining recognition as a crucial aspect of successful translation and implementation of CER. However, experiences with patient engagement in research, including community-based participatory research, suggest that success often hinges on patients being interested and emotionally involved in the research question and understanding their role in the CER process. Patients, researchers, and other stakeholders in the healthcare system all want high-quality research that adheres to established ethical standards; this not only supports the objective of improving health and healthcare but also helps ensure continued participation in and support for research. In an era of increasing economic restraints, consensus is growing that the use of public money for medical research should be subject to public consultation and engagement. A growing body of evidence points to the value of including patients throughout the research process from identifying key gaps and priorities to helping interpret and disseminate results. Integrating patients into the research process increases the likelihood of creating S114
successful, cost-effective studies with outcomes that are meaningful to a broad range of stakeholders that include not only medical consumers and research and clinical community but also industry, healthcare administrators, and regulators. Thus, there is a critical need to shift the paradigm of how medical research is conceived and conducted.
A Voice for Patients in the Regulation of Therapeutic Clinical Trials There is also a need to apply a patientcentered approach to the regulation of clinical trials. The current paradigm for regulatory activities related to clinical trials is largely a two-way conversation between regulatory agencies and industry. There are increasing calls from many sectors that patients and patient advocates should also be included in these conversations. Including patients would expand and open dialogue among a triad of key stakeholders— regulatory agencies, industry, and patients (Figure 1). However, to be effective, conversations must begin at the trial design phase, before protocols are established. The U.S. Food and Drug Administration (FDA) is expanding its
efforts to meaningfully engage patients in regulatory decision making. For example, the FDA’s new Patient-Focused Drug Development Program holds face-to-face discussions with patients and patient representatives (family members, caregivers, and sometimes advocates) about their experiences, preferences, and research priorities. This initiative is viewed as a positive step by many patient groups. During a recent webinar, FDA leaders discussed their enthusiasm about “connecting with the patient community,” noting that “effective patient stakeholder engagement is critical to the success of these meetings” and for learning directly from patients about what is important to them (personal observation).
Engaging Patients as Research Partners Despite enthusiasm for patient-centered outcomes research (PCOR), optimal ways to engage patients in all aspects of the research process remain unclear. Significant challenges exist that can undermine efforts to involve patients as active research partners. These include the sometimes discrepant perspectives of experts and patients/public members, imbalances
Figure 1. Schematic illustrating a proposed approach to multi-stakeholder engagement in medical research.
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ARCH CONFERENCE related to identity and power, lack of organizational resources, and difficulty engaging a diverse group of patients/public members (8). The lack of familiarity of patients/public members with scientific and medical terminology is also an important barrier to communication (8). For example, a recent McMaster report card study found that less than half of people asked knew what COPD was, and only 17% knew and understood the term “COPD.” There is a clear need to enhance communication between patients and research teams to ensure patients participate fully in their care and manage their chronic health conditions. Providers can use a range of evidence-based health literacy strategies to improve communication, including avoiding the use of jargon and using the patient’s words, asking questions to confirm understanding, presenting key information verbally and in writing, and using consultation tools such as decisions aids, pictograms, leaflets, and videos to reinforce health information (9). Greater understanding of the impact of diverse cultural and social contexts, including access to reliable information, is needed to identify and address hidden obstacles. For example, lingering attitudes that are often expressed quietly in minority communities include the notion that research is of little relevance to the community or that harm will result from participating in research at academic centers. One approach to directly addressing barriers is for researchers to actively partner with patient advocacy groups and voluntary health organizations. For example, in the United States, the American Thoracic Society (ATS) has been at the forefront of bringing patients and patient advocates together with researchers and clinicians for more than a decade. Twelve years ago, ATS created a Public Advisory Roundtable (PAR) involving 15 patient groups in its core, with more than 20 others in its wider Council of Public Representatives. All PAR groups are not-for-profit charitable organizations that are actively involved in patient outreach, advocacy, and support. PAR helps coordinate patient speakers for scientific sessions at the annual meeting. PAR members also have funded more than $6 million in pulmonary research in the last several years. Another approach to integrating patients in research is a relatively new patient engagement model at the Drug
Information Association (DIA), a not-forprofit organization that acts as a neutral ground for industry and regulatory agencies. In the United States, DIA brings 20 fellows to its annual conference (now in its third year of offering such fellowships) to facilitate direct interaction between patients, patient advocates, and drug research efforts. DIA recently created a new Patient Engagement Program that involves current and past fellows and provides guidance, training, and networking for patient groups. DIA Europe implemented similar patient engagement activities a decade ago. The UK has pioneered several initiatives to actively engage patients in research. For example, the National Institute of Health Research offers resources to researchers, private organizations, patients, and other stakeholders to facilitate the active involvement of patients and the public in research. INVOLVE is a national advisory group funded by the National Institute of Health Research that supports greater public involvement in the National Health Service (NHS) as well as public health and social care research (10). The NHS maintains a comprehensive “Acronym Buster,” which is available online and through technology applications. The Acronym Buster defines more than 600 medical terms and abbreviations commonly used in the NHS (11). Patient and public involvement has been a key feature of the “patient-led” NHS since the early 1970s and is one of the standards for audit by the national Healthcare Commission (12). Researchers are expected to incorporate patient and public involvement into their research, from the earliest stages of proposal development and ethics review. The Joint Research Office, operated by the NHS, University College of London, and affiliated hospitals, together with several NHS trusts, provides a Research Support Center for researchers that includes a dedicated patient and public involvement manager, practical guides, and other resources to promote the involvement of patients and the public in research (13). The UK’s National Association for Patient Participation is a unique umbrella body that supports patient participation groups in which individuals work in partnership with their practices to empower patients in health promotion, improve patient– clinician communication, contribute to ongoing quality initiatives, provide practical support, and help implement change (14).
Bartlett, Barnes, and McIvor: Patient Voice/Engagement in Research
New Opportunities for PCOR in Pulmonary, Sleep, and Critical Care Research There are several examples of new opportunities that can be harnessed to expand patient-centered research in pulmonary, sleep, and critical care medicine. Unique Opportunities for Funding PCOR Research
PCORI, the Patient-Centered Outcomes Research Institute, is a new, independent federal initiative in the United States with the mandate of understanding how to meaningfully involve patients in all levels of research. PCORI focuses on supporting research and the development of research methodology that can “help people and their caregivers communicate and make informed health care decisions, allowing their voices to be heard in assessing the value of health care options” (15). PCORI projects mandate that patients and other healthcare stakeholders play a key role in all activities. In 2013, PCORI funding totalled $355 million in research contracts; in 2014, this will increase to $500 million. PCORI studies target interventions at the community, home, and health system levels and assess combinations of patient education tools, medications, team-based approaches, or interventions delivered in homes or other locations outside medical facilities. A recent PCORI initiative will invest up to $17 million in PCOR projects that evaluate comprehensive and coordinated approaches to reducing poor health and other complications related to uncontrolled asthma in minority populations as well as improving both patient and provider adherence to treatment guidelines. There are also new opportunities for PCOR-funded research through other federal agencies. For example, Krishnan and colleagues published the results of a recent workshop specifically designed to facilitate discussion and consensus building among a diverse group of stakeholders to identify CER in COPD (16). The workshop was supported by the Agency for Healthcare Research and National Institutes of Health/ National Heart, Lung, and Blood Institute. Scientific Exchanges Sponsored by Patients and Advocates
Patients and patient advocacy groups also are seeking to increase patient engagement directly by underwriting research efforts and S115
ARCH CONFERENCE sponsoring scientific exchanges. In 2012, ATS and PAR convened a meeting that brought together a diverse group of researchers across specialties to discuss commonalities and differences among fibrotic diseases including the heart, lung, liver, kidney, and skin. The concept was developed by a patient advocate. Currently, efforts are underway to organize Fibrosis Across Species in 2014, a meeting which aims to bring patient advocates together with human and veterinary researchers to look at fibrosis occurring in horses, dogs, and cats. Other patient advocacy organizations (e.g., Lymphangioleiomyomatosis Foundation, Scleroderma Society of Canada) also sponsor conferences with the specific goal of bringing patients and medical experts together. Most offer separate sessions for patients and health professionals but also include combined sessions. Harnessing New Technologies
New technologies can now be used to enhance PCOR. For example, mobile (including smartphone) data collection can now capture PROs in real time and do so less intrusively in real-world settings, which may yield more valid and reliable data (17). “Push technologies” can ensure that collected data are automatically transmitted to the research team in real time without any effort required from the patient. Tracking COPD exacerbations is another example wherein new technology can dramatically increase both the quality and the quantity of data collected. Traditionally, exacerbations have been difficult to assess and compare across studies, as data collection was not standardized and often relied on retrospectively collected reports (e.g., patient interviews, diaries, or medical/ hospital chart data). Johnston and colleagues recently examined whether use of a daily symptom e-diary (completed on a smartphone) could be used to detect exacerbations (18). Alerts were triggered when symptom changes, missed diary transmissions, or medical care for a respiratory problem occurred, and clinic visits were initiated when exacerbations were suspected. Individuals transmitted 99.9% of 28,514 possible daily diaries, despite the fact that many older participants reported they had never turned on a computer or owned a smartphone. Thus, smartphone-based collection of COPD symptoms appears to be feasible, acceptable to patients, and can help identify S116
clinically meaningful increases in disease activity in real time. Another example is the EXACT-PRO initiative, a unique collaboration between research and clinical specialists in COPD, instrument development experts, and FDA representatives (http:// www.exactproinitiative.com) (19). EXACTPRO offers a standardized e-diary, available in 42 languages, that can be loaded onto various devices to help patients precisely and reliably record the severity, frequency, and duration of acute exacerbations of COPD and chronic bronchitis. The collection of more ecologically valid data may offer new insights into symptom patterns, patient behavior, acceptability of therapies, and the patient experience. Recently, a number of wellness monitors have become available that can track a range of health information in real time. For example, phones equipped with accelerometers can automatically upload data to a website, which can then be used to monitor, map, and characterize patient activity patterns. Recently, researchers compared the reliability and validity of the Fitbit, a popular and inexpensive activitymonitoring device about the size of an AA battery, with results obtained from both polysomnography (PSG) and actigraphy (20). Although PSG is the gold standard for sleep measurement, it is intrusive, time intensive, and expensive, and actigraphy is often used as an alternative method to estimate sleep time and efficiency. The investigators concluded that Fitbit data showed acceptable reliability, with high sensitivity (accurate identification of sleep epochs) but low specificity (accurate identification of wake epochs). Results were similar to those obtained via actigraphy (though both devices overestimated sleep efficiency and total sleep time as compared with PSG). In some cases, research and clinical data can be transmitted directly into a patient’s electronic health record. At UMass Memorial, patients with diabetes upload glucometer data directly into a patient portal that analyses the data and provides actionable alerts to the healthcare team. Indeed, the application of new technology opens many new avenues of investigation. Mobile phones equipped with geo-positioning tools can be used to estimate the effect of a patient’s geographical location on their health, such as linking health events and outcomes to air pollution, proximity to industry, and proximity to motorways. Such efforts can
further elucidate the role of potential triggers such as weather, air pollution levels, etc., to serious health events, such as acute asthma and COPD exacerbations. Web-based interactive and engaging patient education tools developed using new platforms such as Raspberry Pi can be integrated into studies to standardize and enhance key elements of research, such as patient education and spirometry.
Outstanding Challenges to Patient Integration There are recognized challenges to integrating patients (and other stakeholders) into the research process, and additional work will be needed to ensure the active and meaningful participation of patients in the research process. PCORI, for example, uses several strategies to involve patients as equal partners including: (1) involving patients with identifying and prioritizing research questions; (2) integrating patients into each step of the research cycle from conception through dissemination of results; (3) providing specialized training to enable patients to have productive conversations with research partners; and (4) using structured activities (e.g., moderated group discussion) to facilitate the active participation of all participants, while ensuring that one or several voices do not dominate (21). Since 2002, patients have been collaborative partners in biannual OMERACT (Outcomes Measures in Rheumatology) conferences (22). A recent review documented many positive aspects, including the identification of new areas important to patients that had been previously overlooked by providers (e.g., fatigue) as well as a larger cultural shifts in recognition of the patient voice within the international rheumatology community. However, the authors concluded that full use of the patient perspective is still challenging. Better understanding of factors such as how to select patients, optimal ways to train both patients and researchers, and how to document the impact of patient involvement in research are needed.
Conclusions The shift to incorporate patient perspectives of disease burden, treatments, and
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ARCH CONFERENCE healthcare interactions began some time ago with the development of HRQL tools. Now, growing recognition of the need to complement classical RCTs with data from real-world studies provides a fresh opportunity to highlight the value of integrating patients’ preferences, expectations, and experiences of living with chronic diseases to gain insight into what constitutes meaningful treatment outcomes. The growing cadre of patientfocused organizations and initiatives (e.g., PCORI and ATS PAR, INVOLVE and National Association for Patient Participation in the UK) are testament to the evolving role of actively integrating patients into all aspects of research in recent years.
Patients are the ultimate healthcare stakeholder—the ultimate “end user”—and their perspectives on treatment goals, valued outcomes, preferences, and healthcare experiences are all determinants of adherence and outcomes. Patients can provide important insights into whether potential intervention or care pathways will be feasible and worthwhile to implement. Yet there are important challenges of integrating patients into the research process that must be addressed. Just as research attitudes and methods are evolving, so is technology. The introduction of new smartphone and push technologies offers unparalleled
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opportunities to collect patient-centered data in real time, less intrusively, and in a way that may yield more valid and reliable data than ever before. Thus, a new era is dawning in which patients can and will speak for themselves. Actively integrating patients, family members, and caregivers directly into all aspects of clinical research is critical to ensuring that studies are feasible and realistic to implement and that the results obtained are meaningful not only to patients but also to a broad range of stakeholders in healthcare systems. n Author disclosures are available with the text of this article at www.atsjournals.org.
Publications/nhs-handbook/Acronym/Pages/Acronym-buster. aspx Hogg CNL. Patient and public involvement: what next for the NHS? Health Expect 2007;10:129–138. Joint Research Office. Joint Research Office Practical Guides on Patient and Public Involvement. London, UK: University College London [accessed 2013 Sep 5]. Available from: http://www.ucl.ac. uk/jro/patient-public-involvement/practical_guide National Association for Patient Participation (NAPP). NAPP: an introduction. UK: NAPP [accessed 2013 Sep 5]. Available from: http://www.napp.org.uk/index.html PCORI. Patient-Centered Outcomes Research Institute [Internet] [accessed 2013 Sep 4]. Available from: www.pcori.org Krishnan JA, Lindenauer PK, Au DH, Carson SS, Lee TA, McBurnie MA, Naureckas ET, Vollmer WM, Mularski RA. Stakeholder priorities for comparative effectiveness research in chronic obstructive pulmonary disease: a workshop report. Am J Respir Crit Care Med 2013;187:320–326. Stone AA, Shiffman S, Atienza AA, Nebeling L. The science of realtime data capture: self reports in health research. Oxford, UK: Oxford University Press; 2007. Johnston NW, Lambert K, Hussack P, Gerhardsson de Verdier M, Higenbottam T, Lewis J, Newbold P, Jenkins M, Norman GR, Coyle PV, et al. Detection of COPD exacerbations and compliance with patient-reported daily symptom diaries. Chest 2013;144: 507–514. Leidy NK, Murray LT. Patient-reported outcome (PRO) measures for clinical trials of COPD: the EXACT and E-RS. COPD 2013;10: 393–398. Montgomery-Downs HE, Insana SP, Bond JA. Movement toward a novel activity monitoring device. Sleep Breath 2012;16: 913–917. Fleurence R, Selby JV, Odom-Walker K, Hunt G, Meltzer D, Slutsky JR, Yancy C. How the Patient-Centered Outcomes Research Institute is engaging patients and others in shaping its research agenda. Health Aff (Millwood) 2013;32:393–400. de Wit M, Abma T, Koelewijn-van Loon M, Collins S, Kirwan J. Involving patient research partners has a significant impact on outcomes research: a responsive evaluation of the international OMERACT conferences. BMJ Open 2013;3.
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Department of Medicine, McGill University, Respiratory Epidemiology and Clinical Research Unit, and Division of Clinical Epidemiology, Royal Victoria Hospital, Montreal, Quebec, Canada; 2American Thoracic Society Public Advisory Round Table and Vice President of the Coalition for Pulmonary Fibrosis, Culver City, California; and 3Department of Medicine, Firestone Institute for Respiratory Health, McMaster University, Hamilton, Ontario, Canada
Abstract Research in respiratory, sleep, and critical care medicine has historically been the domain of scientists and clinicians attempting to understand pathophysiological mechanisms and consequences of disease in an effort to develop effective treatments. This traditional approach of placing scientific rigor before the patient’s reality is changing. There is growing recognition of the importance of integrating patient perspectives (e.g., preferences, expectations, and expanded definitions of what constitutes “successful” outcomes) into clinical research to achieve meaningful results for a broader group of stakeholders. This evolution is reflected in the growth of patientcentered organizations and patient advocacy groups that seek to meaningfully integrate patients into the process of prioritizing research needs and creating alliances wherein patients and
researchers can partner together to accomplish research goals. In tandem, a growing number of real-world trials (i.e., those with broader, more representative patient populations and routine care pathways) now complement findings from traditional randomized controlled trials and offer new opportunities to design studies that better reflect patients’ healthcare preferences and experiences. Patients’ perspectives are key determinants of treatment adherence and outcomes, as well as the feasibility and likely value of implementing care pathways. The advent of smartphone and push technologies offer new opportunities for the collection of more patient-centered and ecologically valid patient data, thereby adding new dimensions to meaningfully integrate patients into real-world research. Keywords: patient-centered research; patient-centered outcomes; patient engagement
(Received in original form September 26, 2013; accepted in final form December 5, 2013 ) Supported by the Respiratory Effectiveness Group, which paid the publication costs. Author Contributions: All authors contributed to conception, critical revision, and final approval of the manuscript. S.J.B. integrated sections drafted by each author to develop the first draft. Correspondence and requests for reprints should be addressed to Susan J. Bartlett, Ph.D., Associate Professor of Medicine, McGill University/Royal Victoria Hospital, 687 av des Pins Ouest, Ross 4.31, Montreal, PQ, H3A 1A1 Canada. E-mail: [email protected] Ann Am Thorac Soc Vol 11, Supplement 2, pp S112–S117, Feb 2014 Copyright © 2014 by the American Thoracic Society DOI: 10.1513/AnnalsATS.201309-327RM Internet address: www.atsjournals.org
Chronic pulmonary conditions, critical illness, and sleep disorders impact multiple aspects of an individual’s physical, emotional, and mental health. Beyond reducing symptoms, clinical interventions are increasingly judged not only on their ability to alleviate symptoms but also on their ability to reduce impairments to each of these health domains. This shift toward incorporating a more patient-centered view of health and wellbeing began many years ago with the development of specific health-related quality of life (HRQL) tools. Although the growing trend of moving beyond classically designed randomized controlled trials (RCTs) to developing studies S112
that better capture “real life” experiences and outcomes valued by patients is a necessary step, there is also a need for proactive patient engagement to ensure the patient perspective is integrated early in the conception of a study to ensure that outcomes are meaningful and relevant to patients and other healthcare stakeholders.
The Need for Patient-centered and “Real World” Research Research in pulmonary medicine, critical illness, and sleep has historically been driven by questions identified by scientists and
clinicians who focused largely on understanding pathophysiological mechanisms and consequences of disease in an effort to develop effective therapeutics. This failure to engage patients in the design of traditional research may have, inadvertently, placed scientific rigor before the patient’s reality. Indeed, the research contributions of individuals living with chronic diseases has been largely relegated to whether or not they wish to participate as “subjects” in clinical trials. There has been little opportunity for patients to help identify important research questions, let alone participate in the design, analysis, interpretation, or dissemination of study
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ARCH CONFERENCE results. Furthermore, when the evidence base used to develop treatment guidelines relies on findings from RCTs that enroll highly selected participants, the result is often interventions and care pathways that are challenging or, at times, unrealistic to implement. In an era of healthcare reform, patients are increasingly acknowledged as having unique and important perspectives about treatment goals, preferences, and healthcare experiences—all of which are key determinants of adherence and real-life outcomes. Observational studies and pragmatic trials represent an alternative approach to gathering information across a broader group of patients. Health decision makers increasingly seek “real world” data from observational trials to complement those generated by RCTs (1). (We acknowledge the term “real world” is disputed by some. Just as the strong internal validity of RCTs also reduces generalizability of results, observational studies, too, have important limitations, including selection bias and confounding, which often remain unmeasured but have the potential to significantly influence outcomes observed. All studies, too, are vulnerable to the Hawthorne effect.) Mixed method approaches now add qualitative elements (e.g., individual interviews, focus groups) to traditional RCTs to gain insight into participants’ expectations, interpretation of symptoms, treatment, healthcare experiences, and other factors that influence persistence with therapeutic regimens. However, to create research that reflects the lived experiences of patients and offers results that are meaningful results to patients and other healthcare stakeholders, it is necessary not only to incorporate the patient voice but also to actively engage patients as partners and key stakeholders in the research process.
Understanding What Matters Most to Patients To have a true appreciation for the perspective of medical consumers (patients, families, caregivers), it is important to recognize that they are not only stakeholders in healthcare and the healthcare system but that they are the ultimate stakeholder—and, arguably, the ones with the most at stake. Decisions that patients make about whether to initiate and adhere to prescribed
treatments reflect a complex and evolving process that often begins well before scripts are provided at clinic visits or participants enroll in clinical trials. For many, decisions about the necessity and potential benefits of treatment emerge around the time of diagnosis and continue to evolve in relation to ongoing treatment experiences. Historically, patients have been expected to “comply” with medical advice, even if their understanding of their disease has been limited and no attempt has been made to engage them in decision making about their treatment. There has been longstanding concern that it may be unrealistic and potentially burdensome to expect patients to understand the myriad of considerations of complex diseases and multimorbidity to participate in medical decision making. Rather than asking patients directly about treatment preferences and needs, the medical community has traditionally examined indirect evidence to gain insight into patient perspectives and preferences. For example, factors such as recruitment rates and study withdrawals in clinical trials have been interpreted as evidence of the acceptability, tolerability, and relative value and burden of treatments. Treatment preferences among patients are often surmised by examining adherence, for example, to oral versus inhaled therapies as well as to specific types of inhalation devices. Up to half of all medicines prescribed for chronic conditions are not taken as prescribed (2). Often suboptimal adherence, or even treatment refusal, is viewed as evidence of patient aversion to treatmentassociated risks. However, patients are generally willing to accept risk if they also perceive the trade-off is the opportunity for benefits they value. There is an unwritten understanding among patients, especially those suffering from rare diseases or lifethreatening conditions with few or no lifesaving options, that “benefit” is not necessarily the same benefit sought by researchers. All patients want treatments that can reduce the morbidity and mortality associated with their disease; however, many prioritize improved quality of life over simply extending life (3, 4). In the words of William Gahl, M.D., of the National Institutes of Health in a recent TED talk about rare diseases, people “want to be protected, but they don’t want to be protected to death” (5). Patients and
Bartlett, Barnes, and McIvor: Patient Voice/Engagement in Research
families want to be “heard” to make sure the medical/research community understands their plight, their fight, and their desire to contribute meaningful information that may not save their lives but may improve the HRQL of patients who follow them.
Incorporating the Patient Perspective into Research Greater understanding of patient preferences and expectations together with expanded definitions of what constitutes “successful” outcomes are examples of how the patient perspective can ensure that ongoing research offers meaningful results to a broader group of stakeholders. As partners in the research process, patients can help researchers to prioritize what needs to be studied and how to work together effectively to accomplish research goals. Chronic obstructive pulmonary disease (COPD) can serve as a good exemplar. With COPD, a complex systemic illness, the earliest manifestation is airflow obstruction that is only partially reversible. As the disease progresses, patients experience increasingly frequent and severe exacerbations and develop comorbidities that impact multiple aspects of HRQL. For example, impairments in lung function result in changes in day-to-day function, such as activity limitations due to decreased walking capacity, reduced participation, and changes in social and work roles. Traditionally, medical specialists have relied on lung function measures (e.g., FEV1) to both diagnose COPD and gauge the effectiveness of treatments. However, people living with COPD and their families may judge treatment effectiveness by the extent to which it reduces bothersome symptoms and allows them to participate fully and effectively in valued activities at work and at home. Thus, there is a need to include patients in the process of identifying outcomes that resonate with their experiences. As a result, patientreported outcomes (PROs) are increasingly recognized as crucial elements that complement traditional medical data in the comprehensive assessment of COPD, both for determining the impact and burden of the disease itself and also for evaluating the success of therapeutic interventions. The collection of standardized PROs has been S113
ARCH CONFERENCE reflected first though the use of the Chronic Respiratory Questionnaire and then St. George’s Respiratory Questionnaire in COPD research. The growing practice of routinely incorporating outcomes that are valued and reported by patients as well as assessments of the impact on illness and treatment on HRQL reflects an important evolution of priorities in pulmonary, critical care, and sleep research. Comparative effectiveness research (CER) has been defined as “the conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat, and monitor health conditions in ‘real world’ settings” (6). CER has also now evolved to routinely include patient perspectives. The goal of CER is to provide patients, their advocates and caregivers, healthcare professionals, federal officials, policy makers, and payers with evidence-based information to make informed healthcare decisions (7). However, to achieve this goal, research must be conceived, implemented, and disseminated in ways that ensure results are meaningful to patients, caregivers, and other decision makers. Previously, CER studies were designed by researchers with relatively little input from patients, but patient engagement is rapidly gaining recognition as a crucial aspect of successful translation and implementation of CER. However, experiences with patient engagement in research, including community-based participatory research, suggest that success often hinges on patients being interested and emotionally involved in the research question and understanding their role in the CER process. Patients, researchers, and other stakeholders in the healthcare system all want high-quality research that adheres to established ethical standards; this not only supports the objective of improving health and healthcare but also helps ensure continued participation in and support for research. In an era of increasing economic restraints, consensus is growing that the use of public money for medical research should be subject to public consultation and engagement. A growing body of evidence points to the value of including patients throughout the research process from identifying key gaps and priorities to helping interpret and disseminate results. Integrating patients into the research process increases the likelihood of creating S114
successful, cost-effective studies with outcomes that are meaningful to a broad range of stakeholders that include not only medical consumers and research and clinical community but also industry, healthcare administrators, and regulators. Thus, there is a critical need to shift the paradigm of how medical research is conceived and conducted.
A Voice for Patients in the Regulation of Therapeutic Clinical Trials There is also a need to apply a patientcentered approach to the regulation of clinical trials. The current paradigm for regulatory activities related to clinical trials is largely a two-way conversation between regulatory agencies and industry. There are increasing calls from many sectors that patients and patient advocates should also be included in these conversations. Including patients would expand and open dialogue among a triad of key stakeholders— regulatory agencies, industry, and patients (Figure 1). However, to be effective, conversations must begin at the trial design phase, before protocols are established. The U.S. Food and Drug Administration (FDA) is expanding its
efforts to meaningfully engage patients in regulatory decision making. For example, the FDA’s new Patient-Focused Drug Development Program holds face-to-face discussions with patients and patient representatives (family members, caregivers, and sometimes advocates) about their experiences, preferences, and research priorities. This initiative is viewed as a positive step by many patient groups. During a recent webinar, FDA leaders discussed their enthusiasm about “connecting with the patient community,” noting that “effective patient stakeholder engagement is critical to the success of these meetings” and for learning directly from patients about what is important to them (personal observation).
Engaging Patients as Research Partners Despite enthusiasm for patient-centered outcomes research (PCOR), optimal ways to engage patients in all aspects of the research process remain unclear. Significant challenges exist that can undermine efforts to involve patients as active research partners. These include the sometimes discrepant perspectives of experts and patients/public members, imbalances
Figure 1. Schematic illustrating a proposed approach to multi-stakeholder engagement in medical research.
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ARCH CONFERENCE related to identity and power, lack of organizational resources, and difficulty engaging a diverse group of patients/public members (8). The lack of familiarity of patients/public members with scientific and medical terminology is also an important barrier to communication (8). For example, a recent McMaster report card study found that less than half of people asked knew what COPD was, and only 17% knew and understood the term “COPD.” There is a clear need to enhance communication between patients and research teams to ensure patients participate fully in their care and manage their chronic health conditions. Providers can use a range of evidence-based health literacy strategies to improve communication, including avoiding the use of jargon and using the patient’s words, asking questions to confirm understanding, presenting key information verbally and in writing, and using consultation tools such as decisions aids, pictograms, leaflets, and videos to reinforce health information (9). Greater understanding of the impact of diverse cultural and social contexts, including access to reliable information, is needed to identify and address hidden obstacles. For example, lingering attitudes that are often expressed quietly in minority communities include the notion that research is of little relevance to the community or that harm will result from participating in research at academic centers. One approach to directly addressing barriers is for researchers to actively partner with patient advocacy groups and voluntary health organizations. For example, in the United States, the American Thoracic Society (ATS) has been at the forefront of bringing patients and patient advocates together with researchers and clinicians for more than a decade. Twelve years ago, ATS created a Public Advisory Roundtable (PAR) involving 15 patient groups in its core, with more than 20 others in its wider Council of Public Representatives. All PAR groups are not-for-profit charitable organizations that are actively involved in patient outreach, advocacy, and support. PAR helps coordinate patient speakers for scientific sessions at the annual meeting. PAR members also have funded more than $6 million in pulmonary research in the last several years. Another approach to integrating patients in research is a relatively new patient engagement model at the Drug
Information Association (DIA), a not-forprofit organization that acts as a neutral ground for industry and regulatory agencies. In the United States, DIA brings 20 fellows to its annual conference (now in its third year of offering such fellowships) to facilitate direct interaction between patients, patient advocates, and drug research efforts. DIA recently created a new Patient Engagement Program that involves current and past fellows and provides guidance, training, and networking for patient groups. DIA Europe implemented similar patient engagement activities a decade ago. The UK has pioneered several initiatives to actively engage patients in research. For example, the National Institute of Health Research offers resources to researchers, private organizations, patients, and other stakeholders to facilitate the active involvement of patients and the public in research. INVOLVE is a national advisory group funded by the National Institute of Health Research that supports greater public involvement in the National Health Service (NHS) as well as public health and social care research (10). The NHS maintains a comprehensive “Acronym Buster,” which is available online and through technology applications. The Acronym Buster defines more than 600 medical terms and abbreviations commonly used in the NHS (11). Patient and public involvement has been a key feature of the “patient-led” NHS since the early 1970s and is one of the standards for audit by the national Healthcare Commission (12). Researchers are expected to incorporate patient and public involvement into their research, from the earliest stages of proposal development and ethics review. The Joint Research Office, operated by the NHS, University College of London, and affiliated hospitals, together with several NHS trusts, provides a Research Support Center for researchers that includes a dedicated patient and public involvement manager, practical guides, and other resources to promote the involvement of patients and the public in research (13). The UK’s National Association for Patient Participation is a unique umbrella body that supports patient participation groups in which individuals work in partnership with their practices to empower patients in health promotion, improve patient– clinician communication, contribute to ongoing quality initiatives, provide practical support, and help implement change (14).
Bartlett, Barnes, and McIvor: Patient Voice/Engagement in Research
New Opportunities for PCOR in Pulmonary, Sleep, and Critical Care Research There are several examples of new opportunities that can be harnessed to expand patient-centered research in pulmonary, sleep, and critical care medicine. Unique Opportunities for Funding PCOR Research
PCORI, the Patient-Centered Outcomes Research Institute, is a new, independent federal initiative in the United States with the mandate of understanding how to meaningfully involve patients in all levels of research. PCORI focuses on supporting research and the development of research methodology that can “help people and their caregivers communicate and make informed health care decisions, allowing their voices to be heard in assessing the value of health care options” (15). PCORI projects mandate that patients and other healthcare stakeholders play a key role in all activities. In 2013, PCORI funding totalled $355 million in research contracts; in 2014, this will increase to $500 million. PCORI studies target interventions at the community, home, and health system levels and assess combinations of patient education tools, medications, team-based approaches, or interventions delivered in homes or other locations outside medical facilities. A recent PCORI initiative will invest up to $17 million in PCOR projects that evaluate comprehensive and coordinated approaches to reducing poor health and other complications related to uncontrolled asthma in minority populations as well as improving both patient and provider adherence to treatment guidelines. There are also new opportunities for PCOR-funded research through other federal agencies. For example, Krishnan and colleagues published the results of a recent workshop specifically designed to facilitate discussion and consensus building among a diverse group of stakeholders to identify CER in COPD (16). The workshop was supported by the Agency for Healthcare Research and National Institutes of Health/ National Heart, Lung, and Blood Institute. Scientific Exchanges Sponsored by Patients and Advocates
Patients and patient advocacy groups also are seeking to increase patient engagement directly by underwriting research efforts and S115
ARCH CONFERENCE sponsoring scientific exchanges. In 2012, ATS and PAR convened a meeting that brought together a diverse group of researchers across specialties to discuss commonalities and differences among fibrotic diseases including the heart, lung, liver, kidney, and skin. The concept was developed by a patient advocate. Currently, efforts are underway to organize Fibrosis Across Species in 2014, a meeting which aims to bring patient advocates together with human and veterinary researchers to look at fibrosis occurring in horses, dogs, and cats. Other patient advocacy organizations (e.g., Lymphangioleiomyomatosis Foundation, Scleroderma Society of Canada) also sponsor conferences with the specific goal of bringing patients and medical experts together. Most offer separate sessions for patients and health professionals but also include combined sessions. Harnessing New Technologies
New technologies can now be used to enhance PCOR. For example, mobile (including smartphone) data collection can now capture PROs in real time and do so less intrusively in real-world settings, which may yield more valid and reliable data (17). “Push technologies” can ensure that collected data are automatically transmitted to the research team in real time without any effort required from the patient. Tracking COPD exacerbations is another example wherein new technology can dramatically increase both the quality and the quantity of data collected. Traditionally, exacerbations have been difficult to assess and compare across studies, as data collection was not standardized and often relied on retrospectively collected reports (e.g., patient interviews, diaries, or medical/ hospital chart data). Johnston and colleagues recently examined whether use of a daily symptom e-diary (completed on a smartphone) could be used to detect exacerbations (18). Alerts were triggered when symptom changes, missed diary transmissions, or medical care for a respiratory problem occurred, and clinic visits were initiated when exacerbations were suspected. Individuals transmitted 99.9% of 28,514 possible daily diaries, despite the fact that many older participants reported they had never turned on a computer or owned a smartphone. Thus, smartphone-based collection of COPD symptoms appears to be feasible, acceptable to patients, and can help identify S116
clinically meaningful increases in disease activity in real time. Another example is the EXACT-PRO initiative, a unique collaboration between research and clinical specialists in COPD, instrument development experts, and FDA representatives (http:// www.exactproinitiative.com) (19). EXACTPRO offers a standardized e-diary, available in 42 languages, that can be loaded onto various devices to help patients precisely and reliably record the severity, frequency, and duration of acute exacerbations of COPD and chronic bronchitis. The collection of more ecologically valid data may offer new insights into symptom patterns, patient behavior, acceptability of therapies, and the patient experience. Recently, a number of wellness monitors have become available that can track a range of health information in real time. For example, phones equipped with accelerometers can automatically upload data to a website, which can then be used to monitor, map, and characterize patient activity patterns. Recently, researchers compared the reliability and validity of the Fitbit, a popular and inexpensive activitymonitoring device about the size of an AA battery, with results obtained from both polysomnography (PSG) and actigraphy (20). Although PSG is the gold standard for sleep measurement, it is intrusive, time intensive, and expensive, and actigraphy is often used as an alternative method to estimate sleep time and efficiency. The investigators concluded that Fitbit data showed acceptable reliability, with high sensitivity (accurate identification of sleep epochs) but low specificity (accurate identification of wake epochs). Results were similar to those obtained via actigraphy (though both devices overestimated sleep efficiency and total sleep time as compared with PSG). In some cases, research and clinical data can be transmitted directly into a patient’s electronic health record. At UMass Memorial, patients with diabetes upload glucometer data directly into a patient portal that analyses the data and provides actionable alerts to the healthcare team. Indeed, the application of new technology opens many new avenues of investigation. Mobile phones equipped with geo-positioning tools can be used to estimate the effect of a patient’s geographical location on their health, such as linking health events and outcomes to air pollution, proximity to industry, and proximity to motorways. Such efforts can
further elucidate the role of potential triggers such as weather, air pollution levels, etc., to serious health events, such as acute asthma and COPD exacerbations. Web-based interactive and engaging patient education tools developed using new platforms such as Raspberry Pi can be integrated into studies to standardize and enhance key elements of research, such as patient education and spirometry.
Outstanding Challenges to Patient Integration There are recognized challenges to integrating patients (and other stakeholders) into the research process, and additional work will be needed to ensure the active and meaningful participation of patients in the research process. PCORI, for example, uses several strategies to involve patients as equal partners including: (1) involving patients with identifying and prioritizing research questions; (2) integrating patients into each step of the research cycle from conception through dissemination of results; (3) providing specialized training to enable patients to have productive conversations with research partners; and (4) using structured activities (e.g., moderated group discussion) to facilitate the active participation of all participants, while ensuring that one or several voices do not dominate (21). Since 2002, patients have been collaborative partners in biannual OMERACT (Outcomes Measures in Rheumatology) conferences (22). A recent review documented many positive aspects, including the identification of new areas important to patients that had been previously overlooked by providers (e.g., fatigue) as well as a larger cultural shifts in recognition of the patient voice within the international rheumatology community. However, the authors concluded that full use of the patient perspective is still challenging. Better understanding of factors such as how to select patients, optimal ways to train both patients and researchers, and how to document the impact of patient involvement in research are needed.
Conclusions The shift to incorporate patient perspectives of disease burden, treatments, and
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ARCH CONFERENCE healthcare interactions began some time ago with the development of HRQL tools. Now, growing recognition of the need to complement classical RCTs with data from real-world studies provides a fresh opportunity to highlight the value of integrating patients’ preferences, expectations, and experiences of living with chronic diseases to gain insight into what constitutes meaningful treatment outcomes. The growing cadre of patientfocused organizations and initiatives (e.g., PCORI and ATS PAR, INVOLVE and National Association for Patient Participation in the UK) are testament to the evolving role of actively integrating patients into all aspects of research in recent years.
Patients are the ultimate healthcare stakeholder—the ultimate “end user”—and their perspectives on treatment goals, valued outcomes, preferences, and healthcare experiences are all determinants of adherence and outcomes. Patients can provide important insights into whether potential intervention or care pathways will be feasible and worthwhile to implement. Yet there are important challenges of integrating patients into the research process that must be addressed. Just as research attitudes and methods are evolving, so is technology. The introduction of new smartphone and push technologies offers unparalleled
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opportunities to collect patient-centered data in real time, less intrusively, and in a way that may yield more valid and reliable data than ever before. Thus, a new era is dawning in which patients can and will speak for themselves. Actively integrating patients, family members, and caregivers directly into all aspects of clinical research is critical to ensuring that studies are feasible and realistic to implement and that the results obtained are meaningful not only to patients but also to a broad range of stakeholders in healthcare systems. n Author disclosures are available with the text of this article at www.atsjournals.org.
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