‘The patient’: at the center of patient-reported outcomes

Abstract

The recent emphasis of including patient reports in their own care management is reviewed in terms of the factors that contributed to its popularity. The role change of patients as being active participants in their own care as a result of the rising consumerism and advocacy has led to increased pressures for including patients in the therapeutic decision-making process. As consumers of clinical services, their perspectives and attitudes towards health and illness acquired more importance. The rising cost of healthcare has added another dimension in cost containment by empowering patients and sharing responsibility in their recovery, which hopefully can improve outcomes. Challenges in the development and implementation of patient-reported outcomes in psychiatry are reviewed and include the still unresolved subjective/objective dichotomy, identification of the most appropriate and relevant patient-reported outcomes. Few outcomes are identified and include: subjective tolerability of medication, self-reported health-related quality of life, preferences, patients’ attitudes towards health and illness, satisfaction with medication and overall satisfaction of quality of care, and functional state, with particular focus on social functioning.

Keywords:

• decision-making
• patient-reported outcomes
• patients
• self-reported

There is increasing recognition of the importance of including patients and their carers in the evaluation and management of their own care. Constructs such as ‘Patient-Centered Care’, ‘Patient-Reported Outcomes (PROs)’, ‘Patient Directed Care’, ‘Personalized Medicine’, and so on have been advanced as new approaches to enhance the quality of care and improve outcomes. Although the underlying concepts behind many of these approaches are not new, their origins go back more than 50 years. Constructs such as ‘Patient-Centered Care’ has its origins in the 1950s, as excellently reviewed in the influential book ‘Through the Patient’s Eyes’, which includes not only a historical review, but also the important recommendations that followed from the Picker/Commonwealth program for patient-centered care [1]. It explored patients’ perspectives and their needs, as they, themselves, define them. Meanwhile, over the last two decades, there has been a resurgence of interest and popularization of a number of such patient-related constructs.

The recent emphasis of eliciting and including patient reports in their own care management owes its popularity to a number of contributing factors. The role change of patients as being active participants in their own care, rather than being considered passive recipients of treatment, has been the product of the rising consumerism and advocacy pressing for more constructive sharing with clinicians in the therapeutic decision-making process. As consumers of clinical services, their perspectives and attitudes toward health and illness has acquired more importance, and a noticeable shift from just symptom improvement to the expectation of improved functioning, as well as feelings of satisfaction and well-being [2]. The rising cost of health care, as well as the recent introduction of a large number of new and relatively expensive medications, has added a pharmacoeconomic perspective, with the expectation of better and safer medications that justify the cost [3]. The recent expression of interest by the US FDA and other European regulatory agencies in PROs, as well as issuing guidelines including PROs in the development and testing of new medications, has also added more impetus [4]. The recent rise in the concept of ‘recovery’ has paved the way for including patients in the decision-making process regarding their own care by empowering patients to be active participants in their own recovery. Recent medical and technological advances have made it possible to tailor treatments to the individual needs and ushered in the beginning of personalized medicine.

Challenges in the development and implementation of PROs in psychiatry, reliability of psychiatric patients’ reports and the subjective/objective dichotomy

A number of serious psychiatric disorders, such as schizophrenia and major affective disorders, can include a spectrum of symptoms that negatively impact on patients’ cognitive abilities, particularly insight and judgment. In essence, how reliable can any information provided by patients under such conditions be? Since many patient-reported experiences are subjective in nature, there has been ongoing doubt about the ability of seriously ill psychiatric patients to accurately evaluate and label their inner feelings and experiences. By now, there has been extensive evidence to dispel such concerns for the majority of psychiatric patients, by demonstrating the consistency of patients’ reports about their inner feelings, as well as the frequent correlation of theirs and their physicians’ reports [5–7]. Obviously, for the group of patients who suffer from severe thinking and cognitive disabilities, the accuracy of their reports is questionable. Additionally, there has been extensive research data elucidating the neurobiological basis for such important PROs, such as ‘subjective tolerability of antipsychotics in schizophrenia’ [8]. Recent data has negatively correlated striatal dopamine functioning with development of negative subjective responses and poor tolerability to medications [9]. Since our initial neuro-imaging data, other studies followed, confirming our findings [10,11]. In other words, subjective tolerability to medications as expressed by patients is largely determined by significant neurobiological alterations in the striatum and not a random response.

There exists a plethora of measuring tools for PROs, but many of them are not psychometrically robust, as well as not sensitive enough to pick the expected changes, particularly in relatively short-term clinical trials. Also, many of them are not suitable for use in various stages of the illness and lack a common metrics to make it possible to compare data.

The most significant patient-related outcomes, besides symptom improvement, includes subjective tolerability of medications, self-reported health-related quality of life, preferences, patients’ attitudes toward health and illness, satisfaction with medication and overall satisfaction of quality of care and functional state, with particular focus on social functioning.

Although there has been more research exploration and evaluation of subjective tolerability and health-related quality of life, unfortunately, the field of quality of life continues to be somewhat fuzzy with the inconsistent methodologies and limitations of measuring tools, as well as lack of conceptual frameworks [12]. Other emerging areas such as the importance of subjective tolerability and its impact on outcomes, quality of life, adherence behavior as well as the recently demonstrated relevance to development of comorbid addictive states are all well recognized, but continue to require more attention and development [8]. Areas such as satisfaction and preferences, by and large, are not well developed and require more research emphasis [13]. Issues related to functioning, particularly evaluation of social functioning, which is one of the relevant and important outcomes in psychiatric management, awaits more informative and applied research approaches [14].

How to reconcile conflicts between patient-reported needs and outcomes, and their clinicians’ assessment continues to be an open and unresolved issue. Patients’ needs and preferences can at times conflict with what is considered to be the best practices. Restrictions placed at times on persons with psychiatric conditions, in the interest of their own safety and the safety of others, and being hospitalized against their wish, may significantly color these patients’ preferences and needs. Similarly, conflicts between patients and their families about management issues can complicate the clinical decision-making process. Although these particular circumstances require particular approaches, for resolution of such conflicts, in general, there needs to be an appropriate approach for resolving conflicts between patients’ expectations and preferences and their clinicians’ recommendations that can be applicable to the vast majority of such situations.

Recommendations for inclusion of PROs in clinical and pharmacoeconomic studies

It is important to define the purpose of PRO measurement. PRO measurement needs to be integrated in the clinical trial protocol, as well as in clinical practice. The population under study, as well as the desired outcomes relevant to the purpose of the study and the stage of the illness, has to be clearly defined a priori. Outcomes for acute presentations are different than those expected in the long-term chronic phase. A recent report by the International Society of Pharmacoeconomics and Outcome Research provides important information about good practices for outcomes research [15].

The most commonly desired PRO in pharmacoeconomic studies includes health-related quality of life, subjective tolerability of medications, patient preferences and satisfaction with medications, as well as social functioning.

Notwithstanding the limitations of currently available measuring instruments, special attention has to be given for choosing not only practical, but instruments that possess adequate psychometric properties. In a recent review, we advocated the need for development of new scales based on modern approaches, such as item response, information theory, item banks and computer-adaptive testing, which can also provide meaningful metrics, useful for the purpose of data comparisons. More details about such a new approach for scale development can be found in the NIH initiative PROMIS (PROs Measurement Information System) [16].

Conclusions

Although the inclusion of psychiatric patients in the evaluation and management of their psychiatric care has a long historical background, what is meant these days by PROs is not only that more emphasis is given to the inclusion of PROs in clinical care and clinical trials, but also emphasizing the conceptual shift in taking patients’ own reports seriously as partners in care. There needs to be mechanisms for further educating our patients, not only in terms of information about therapeutic approaches, but also about their ability to elicit and express their own perspectives regarding care, and also about their inner feelings and their progress. Equally important is the research attention that needs to be given to measuring tools that are more appropriate and sensitive in evaluating a number of the important PROs. The field of psychiatry has been slow in adapting new technological and electronic advances in developing methodologies that can provide important evaluative information in real time. It is clear that adopting the PRO approach can generate an enormous amount of data that requires adequate storage capacity, as well as appropriate analysis. If we wish to improve outcomes, then we need to empower our patients to become real partners, as well as take some responsibility in the process of their own recovery. Improving outcomes is not just a benefit for the patient and their carers, but also adds an economic perspective by getting a bigger bang for the money [17].

Financial & competing interests disclosure

The author has no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.