1. Measurement methodology in PRO research

1.2. Patient involvement in developing PROMs

Until recently, many PROMs have not been developed with the extensive participation of patients (1) by clinicians. These tended to gather data that is important from the clinician’s perspective rather than patients and were often in a language and format that was a barrier to engagement and participation. This means that PROMs are not necessarily measuring concepts important to patients. This gap has been realised and increasingly efforts are made to include patients in the development of PROMs although barriers remain, notably the necessary time investment and budget impact[1][2]. There are a number of things patient groups can do to address this gap, before, during, and after PROM development:

Identifying the need for PROMs - This is of particular importance to pharmaceutical companies, who must identify measures very early on in medicines development.

Patient input to setting the research question to be addressed, as well as to the forming of outputs, can facilitate the design of tools and the capture of relevant PROs. Patient input into setting the question can enhance payback in a number of ways.

  • On the impact of the research process and on the experience of receiving care and support
  • On the trend to complexity in R&D and the demands this makes on research participants (focusing investigations
  • What (if any) tangible results patients might anticipate seeing as a result of their participation (making the recruitment of subjects easier and more honest).

Evaluating and reviewing PROMs - Patients and patient groups can learn to appraise the quality of PROMs. They can then use the information they gain to inform similar patients regarding which scales are acceptable and which are not. This may be particularly important for patients consenting to participate in clinical trials.

In the following a few aspects of such an evaluation are given (non comprehensive) which assess comprehension of the PROM and its relevance by:

- Ensuring that the items within the PROM cover the aspects of the concept aimed to be measured.

- Verifying the acceptability of the PROM by patients.

- Collecting feedback on each question (item) of the PROM (i.e. perceived patient interpretation of each question, misunderstandings related to any questions and any proposed reformulations of questions.

- Critically reviewing the instructions, questions, response options and recall period of the PROM.

Developing and evaluating conceptual and/or theoretical frameworks - Validating potential tools requires qualitative research with patients. Although in the past patients have been consulted, there is an identified need for collaboration in tool development.

Trigger further patient education and interventions, demonstrating to patients that the data they input is meaningful to their care.

[1] Wiering, B., de Boer, D. & Delnoij, D. Patient involvement in the development of patient-reported outcome measures: The developers’ perspective. BMC Health Serv Res 17, 635 (2017). https://doi.org/10.1186/s12913-017-2582-8

[2] Carlton, J., Peasgood, T., Khan, S. et al. An emerging framework for fully incorporating public involvement (PI) into patient-reported outcome measures (PROMs). J Patient Rep Outcomes 4, 4 (2020). https://doi.org/10.1186/s41687-019-0172-8