Patient-reported outcomes in epilepsy: a case study exploring their usage and impact

Introduction

In recent years there has been an increasing attention to place people with epilepsy at the centre of clinical care and health research with the aim to improve their experience and ensure that the research is robust and of maximum value for the use of medicines, therapy and health services.

Patient-Reported Outcomes (PROs) are any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by anyone else (1).

The use of PRO measures in clinical practice is gaining interest from different perspectives. Potential benefits include the opportunity to improve and facilitate the communication between patients and clinicians, to provide a comprehensive evaluation of the functional and mental health domains of patients, to monitor more accurately the effects of the interventions and to support the clinical decision-making and patient self-management (2). The adoption of PRO measures has been also suggested to improve the allocation of healthcare resources according to individualized needs and increase the efficiency of patient management and practices (3).

This study aimed to obtain preliminary insights from epilepsy specialists on the use of PRO measures and how they can affect the management of people with epilepsy and improve healthcare resource utilization.

Materials and methods

Statistical analysis

A descriptive summary of the responses given by the participants to each question of the survey was provided.

Results

A summary of the responses to the interview is provided in Table 1. Both respondents stated that people with epilepsy and, when appropriate, family members/caregivers are always involved in the decision-making process on the management of their condition and they are informed about the possibility and the importance of their involvement in the decision-making process.

Both centres assessed patient satisfaction with their care path and treatment process dedicating around 10 minutes to each patient during visits; the epileptologist or both the epileptologist and neuropsychiatrist are the persons in charge for this activity at the adult and paediatric centres, respectively.

Different domains are evaluated at the centres, including the quality of life, emotional state, anxiety, mood, irritability, and limitations at work, school or in other activities; vitality, emotional-behavioural control, subjective assessment of their well-being were reported to be also assessed at the adult epilepsy unit. Paper-based questionnaires and face-to-face interviews were the main modalities used to measure the quality of life of the people with epilepsy at the interviewed centres. Among the scales to identify the PRO for psychopathological and emotional aspects of the disease, the Quality of Life in Epilepsy Inventory-31 (QOLIE-31) and the Adverse Event Profile were reported as the most used at the adult epilepsy centre, and the Generalized Anxiety Disorder-7, Short-Form Health Survey 36, PSY-Flex, SAFA, Child Behavior Checklist at the paediatric one.

The participants to the survey answered that recognized assessment scales to identify the PRO are mainly used within profit and no-profit studies like clinical trials sponsored by pharmaceutical companies, academic/hospital observational studies and research projects at the adulthood site and are administered by clinicians, while they are part of the routine clinical practice and are administered by psychologists at the paediatric centre. The time dedicated to the collection of PRO has been estimated between 20 and 30 minutes at the adult centre and more than 30 minutes at the childhood centre; epileptologists were mainly involved in the collection and analysis of PRO in the adult setting, whereas epileptologists, psychologists, or neuropsychiatrists were in charge of these activities at the childhood centre.

There was a complete consensus among the respondents about the favourable impact that the PRO can have in terms of patient management, disease management and measurement of the success of a treatment, and both considered the PRO as important as other main indicators like the efficacy and tolerability of the treatment. The respondent from the adult epilepsy centre believed that PRO can also contribute to assess the value of a treatment and improve the utilization of healthcare resource. Lack of time, lack of personnel and lack of economic resources were identified as barriers on the use of PRO in clinical practice.

Regarding the future perspectives, the respondents agreed that PRO could reduce the number of visits, exams and treatments, and increase the time spent on each patient and the number of neuropsychological, psychological and rehabilitation services. The implementation of the standardized use of PRO was considered useful at both sites and the increase in human resources was considered a priority to achieve this goal. A digitalization of data collection besides traditional papers from questionnaire could also have an increasing role in the future.

Discussion

This study provided a picture of the attitudes towards the use of PRO measure at two adult and paediatric referral units for epilepsy care in Italy, identified similarities and differences between the centres, and delineated perspectives for the possible advancement of the field in the future.

Both adult and paediatric epilepsy centres measured different domains that may affect the quality of life of people with epilepsy, including emotional state and limitations at work or school. Differences emerged in the tools adopted to assess these domains, with face-to-face interviews being the way preferred in children and adolescents and both face-to-face interviews and paper-based questionnaires adopted in adult outpatients. Despite the increasing digitalization of healthcare services, electronic questionnaires still do not have a significant role in everyday clinical practice and so far, they are utilized almost exclusively within the frame of clinical trials.

In adults, psychopathological and emotional aspect of the disease are mainly explored by means of a short form, rather than comprehensive questionnaire of quality of life, namely the QOLIE-31; in paediatric age, a wider variety of instruments are used to assess the quality of life and evaluate more specifically anxiety disorders, psychological flexibility, psychiatric symptoms, and emotional and behavioural domains.

The PROs are collected during the different phases of patient care, including the patient access to mainly investigate the overall impact of the illness and quality of life, the treatment to mainly monitor the tolerability and define the impact of the therapy on comorbidities, mental health, social functioning and the follow-up to guide and inform decisions on the treatment.

At the adult epilepsy centre, the collection, analysis and use of the PRO take place mostly within the context of clinical trials or specific projects, whereas at the paediatric epilepsy centre PRO are part of everyday clinical activity. This difference may be explained not only by a different attitude between the epilepsy specialists and the differences in the populations seen but also by differences in the organization of the centres interviewed. The adult centre, indeed, is more commonly involved in clinical studies and the PRO assessment is entrusted to the clinicians without the availability of other healthcare professionals (e.g., nurses and psychologists), while the paediatric centre has a minor involvement in research and clinical investigations and can count on the resource of psychologists and neuropsychologists. Of note, the lack of time and lack of human resources were acknowledged as the main barriers for the implementation of the PRO in routine practice at the adult centre; the lack of economic resources was recognized by the paediatric unit as a limit for the purchase of the copyright for specific online tests and questionnaires and, hence, the further improvement of PRO assessment in clinical practice. This is consistent with the available evidence suggesting that several barriers still exist for the widespread use of PROs in everyday practice (4).

Despite the heterogeneity in the actual use of the PRO in clinical practice, there was a uniform perception throughout the two centres involved that PRO are important as other main indicators like the efficacy and tolerability of a treatment and their use can be beneficial to optimize the care of people with epilepsy. The belief that the PRO can be included among the indicators of the value of a treatment and the information obtained from their use can also affect the utilization of resources emerged. In this regard, the respondents to the questionnaire considered the reduction of clinical visits as a potential consequence of a more widespread utilization of PRO, together with a decrease in the burden of antiseizure medication and an increase in psychological/neuropsychological services and treatments. These changes may be interpreted as a more rational utilization of healthcare resource, an increase in the value of the services provided and a better management of both the epilepsy, with the avoidance of unnecessary and potentially harmful medications, and the associated comorbidities.

Finally, both the interviewees considered useful to implement the collection of the PRO in clinical practice in a standardized manner and allocate more resources for this purpose. Digitalization might also play an increasing role in the collection of PRO in the future, and the possibility of identifying populations more inclined to use digital tools could be considered.

This study was an explorative attempt to provide a brief overview of the current and future usage and impact of PRO in epilepsy. The contrast between adult and paediatric units represented a plus in the study. Several shortcomings need to be acknowledged. Only two Italian centres were involved in the initiative; in this regard, however, it is worth noticing that respondents to the survey worked at a tertiary, academic hospital, and both centres were accredited by the Italian League Against Epilepsy and represent crucial referral points for a vast territory in the country. In addition, the questionnaire was not piloted before administration to the two respondents, although framing the question in one way or another can have significant influence on the answer gathered.

While the respondents gave their perceptions on how PROs could reduce the number of visits, exams and treatments, and increase the time spent on each patient and the number of neuropsychological, psychological and rehabilitation services, no actual data were available to triangulate these self-reported perspective. Although the perspectives of the respondents did not necessarily represent the national scenario and could not inform about the situation in other countries, this pilot study can pave the way for similar experiences to get insights and identify geographical differences about the role and contribution of PRO measures in epilepsy across care centres representative of the Italian context and worldwide.

In summary, the perspective revealed by the survey indicated that clinicians are well aware that epilepsy does not represent just a question of reducing seizures, and the disease burden extends into many psychological and social domains that ultimately impact the quality of life. The time dedicated to “listen to” the person with epilepsy is crucial and a more robust inclusion of his/her view in the management of his/her condition is of paramount importance. The assessment of the seizure frequency is not sufficient, and the PRO are thought to provide a more reliable assessment of treatment effectiveness and tolerability, to contribute to inform and guide clinical decisions, to have a favourable impact on clinical outcomes and satisfaction of people with epilepsy, and to improve the utilization of healthcare resources. Initiatives to identify measurement instrument sets for people with epilepsy are currently on the road (5,6). An active plan involving the key stakeholders would be needed to harmonize the adoption of PRO and ensure they can actually have favourable impacts for people with epilepsy and health services (7).

Acknowledgements

Ethos S.r.l. gave assistance to develop the survey. The survey was conducted, and the participants were selected by Ethos S.r.l. with the support of Angelini. This has not impacted the content of the manuscript. We express our gratitude to Dr. Giovanni Falcicchio and Dr. Teresa Francavilla from the Epilepsy Centre of Bari Hospital, Italy, for their dedicated time during the preparation phase of the investigation. A special acknowledgement goes to Dr. Vittorio Sciruicchio from the UOSVD Epilepsy Centre and EEG of the Developmental Age at Bari Hospital, Italy, for actively contributing to the survey in the paediatric area through his valuable participation.

Disclosures

Conflict of interest: Simona Lattanzi has received speaker’s or consultancy fees from Angelini Pharma, Eisai, GW Pharmaceuticals, Jazz Pharmaceuticals, Medscape and UCB Pharma and has served on advisory boards for Angelini Pharma, Arvelle Therapeutics, BIAL, Eisai, GW Pharmaceuticals and Rapport Therapeutics outside the submitted work; she has received research grant support from the Italian Ministry of Health and Ministry of University and Research. Angela La Neve has received speaker’s or consultancy fees from Eisai, Mylan, Sanofi, Bial, GW, Arvelle Therapeutics, Angelini Pharma and UCB Pharma outside the submitted work. Both authors reported personal fees from Ethos S.r.l. in compensation of time for the development of the survey.

Financial support: This publication was supported by an unrestricted grant from Angelini Pharma S.p.A. The sponsor did not play a role in the design, execution, interpretation, or writing of this article.

Authors’ contributions: All authors contributed to interpreting the data, drafting, and revising the manuscript. All authors approved the final submitted version.

Data Availability: Anonymized data will be shared upon reasonable request of any qualified investigator.

Supplementary appendix. Structured survey about Patient-Reported Outcomes.