5th EpiCom Workshop: Patient-reported outcomes: The value of measuring PROs and how to implement them in daily practice and research

The opening presentation was given by Behrooz Z. Alizadeh, a leading scientist and lecturer at UMCG, including in the field of Inflammatory Bowel Disease (IBD), who explained what PROs are, outlined the steps involved in developing patient-reported outcome measures (PROMs) and described the characteristics of PRO instruments, including their validity, reliability, reproducibility, informativeness and applicability in research and routine clinical practice. He pointed out that PROs refer to any report on the status of a patient's health condition that comes directly from the patient, without interpretation of the patient's response by a clinician or anyone else. They provide the patient with an opportunity to express what is important to him or her, and are not to be confused with an observer reported outcomes (ORO), when the clinician is asking for a description of the symptoms and complaints. He discussed the ideal properties of a PRO instrument (i.e. a standardised, validated questionnaire to measure PROs), which is intended to be completed by patients in order to measure their perceptions of functional status, and examined how PRO instruments are gaining weight in routine practice and research to measure clinically and personally relevant endpoints in the management of patients, including those with IBD. In his view, despite extended initiatives to develop various PRO instruments, no validated PRO instruments are available for the vast majority of (para)clinical evaluations. The decision of the FDA to enforce use of PROs in RCTs represents a step forward, but there is an ongoing dilemma over the need for and implementation of validated PRO instruments in routine clinical guidelines. For the time being, with the aim of measuring “co-primary” outcomes, PROs will be integrated into currently non-PRO evaluations of ORO, physical examinations, biochemical tests, imaging techniques, and interventional procedures (e.g. endoscopy) in the management of diseases such as IBD; an example is the IBD disability index, a newly developed and validated PRO instrument for IBD patients. Collectively, this will empower the IBD team to make effective decisions not only in management of the disease itself, but also in treating the patient as a person.

The workshop continued with the very emotional testimony of Kirsty Gibson, a mental health nurse who has suffered from Ulcerative Colitis since the age of 7 years and who has concurrent Primary Sclerosing Cholangitis. Kirsty explained how health care quality could be improved by using PROs. During a consultation, PROs allow measurement of the ability to perform functional tasks, which would not be routinely assessed by standard clinical evaluation. PROs support patient engagement and fuel conversations between patients and care providers that facilitate shared decision making and enhance safe and effective delivery of health care. Kirsty reminded us that quality of life encompasses a variety of important outcomes for patients, such as body image, emotional health, work/social life balance, bowel and urinary function, sexual function and fertility. Despite the time constraints in clinical practice, emphasis should be placed on working in a multidisciplinary fashion with relevant professionals such as social workers, psychologists and nurses in order to provide patients with comprehensive care.

Jean-Frédéric Colombel, an internationally renowned clinician and researcher in IBD and gastroenterology from Mount Sinai in New York, USA, continued by discussing the outcomes meaningful to patients in IBD drug development. He explained that in recent IBD clinical trials there has been a shift towards histology and endoscopy as treatment targets, without involving PROs. Additionally, clinical data reported by physicians are not objective and fail to capture the impact of disease on the patient. In fact, however, the recent literature indicates that there is a moderate correlation between PROs and objective evaluation of inflammatory activity in IBD; therefore, for the purpose of drug licensing, PROs should indeed be a major endpoint along with objective markers of inflammatory activity. Unfortunately, despite the FDA recommendations that both objective assessment of inflammation and symptom control (PROs) should be used for drug licensing, the PROs used in the recent clinical trials are of a heterogeneous nature and do not meet FDA recommendations. Furthermore, as highlighted in a post hoc analysis of the EXTEND trial, there is a need to determine the cut-off values for remission in the different PROs and for assessment of the long-term impact of drugs in IBD on the potential progression of damage and disability.

In the second part of the workshop, an interactive session focussed on the implementation of PROs in routine practice and research. Participants were asked by Naila Arebi to work on three scenarios in order to understand and define the role of PROs in clinical practice, in health policy and treatment reimbursement decisions, and in clinical trials and regulatory approval. With regard to clinical practice, it has been suggested that the questionnaires should be administered at various time intervals using different modalities, including paper and app-based technology, in order to enhance patient participation, which will allow clinicians to better evaluate any discrepancies in disease management. Factors to be evaluated should include classic PROMs, along with additional factors such as fatigue and appetite. A similar approach should be considered during development of health policy and reimbursement decisions, as these factors provide additional assessments of indirect health costs. Patient representation in clinical trial designs may also be beneficial.

The workshop concluded with a tandem talk between Pia Munkholm from Copenhagen and Marieke Pierik on the “PROmise” of remote monitoring tools in IBD daily care, which provided a brilliant example of how technology and PROs can be used to assist in patient care. The digitalisation of PRO assessment along with the use of calprotectin allowed trained IBD nurses to prioritise health access for high-risk patients and efficiency of health care delivery. The main challenge is how to adapt these digital platforms to local hospital software systems so that they can be used in different jurisdictions.  

Workshop evaluation questionnaire:

Before the start of the workshop, Marieke Pierik asked participants to vote on the following statements:

1. I know what PROs are
2. I think that PROs increase the administrative burden for health care professionals
3. I think that PROs should be used in research studies
4. I think that PROs should be used in clinical practice
5. I think that PROs should be used in clinical drug trials
6. I am willing to change my practice to include PROs!

Naila Arebi reviewed the results of the answers to these statements and asked the participants to vote again at the end of the workshop. Comparison of the answers provided at the beginning and the end of the workshop showed a clear increase in awareness and understanding among the workshop participants of the importance of PROs in daily practice and research (Table 1).

Table 1. Results of the evaluation questionnaire from the workshop

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