August 20, 2021

How are diversity and inclusiveness considered in PROM development and collection?

High-quality PROMs, as well as real-world evidence insights, are becoming crucial for the medical community: reflective of the vibrant, diverse and multicultural societies we live in, they enrich pure clinical assessments to improve research and treatment for the benefit of all patients.

The silent health crises - inequality and prejudice

Regardless of the health status of individuals, external factors such as geography, education, race and income all have considerable impacts on population health. In essence, societal inequality lies at the heart of discrepancies in influencing population health as the German physician, Rudolf Virchow first emphasised in the 19th century. 1

Recent world events have placed issues of injustice, discrimination and societal inequality centre stage once again. Whilst we like to believe that society is moving towards a more diverse, inclusive world, the underbelly of inequality and prejudice remains systemic. How this affects healthcare provisions not only in the United States, but also in Europe is stark. 2 Moreover, how this is reflected unintentionally in the collection of patient-reported outcomes (PROs) is largely unaddressed. With this in mind, this article explores the current state of play of diversity and inclusiveness in the collection and development of patient-reported outcome measures (PROMs).

The role of patient-reported outcomes in informing healthcare

PROs provide the patient voice a platform for individual treatments and for wider population studies and are measured via PROMs. They are used in parallel with clinical evidence to inform decisions made by clinicians, industry, medicines regulators, health technology assessment bodies and other healthcare decision-makers. PROs should aim to assist all endeavours to adopt inclusivity in healthcare to benefit risk evaluations and regulatory decision making.

The true value of PROMs research relies on being conducted in large patient numbers which encompasses all members of diverse patient populations, however recent publications allude to the fact that this logic is not being put into practice adequately.

A lack of ethnic inclusiveness and diversity in drug development

A recent study by Pugh et al. investigating the incorporation of PROs in clinical trials demonstrated that certain patient groups are not being satisfactorily represented. The investigators examined PRO capture across 10 National Clinical Trials Network (NCTN) Oncology clinical trials and found that 24.7% declined to participate in PROs, and that 62.2% declined electronic PRO capture. Through this, it was established that racial or ethnic minorities, those with less education and older patients were less likely to consent to PRO collection. 3

Whilst the conclusions require additional research into the specific causes of this reluctance to participate in PROMs collection, other research has uncovered that ethnic diversity is lacking in oncology clinical trials.4 The National Institute for Health Research (NIHR) Portfolio Cancer clinical trials including PROs (2011-2014) were reviewed and a mere 17% reported any ethnic profile data. Moreover, 13% of the total number of participants were identified as belonging to non-white ethnic groups. Was patient diversity lacking? An additional observation recorded was that none of the trial publications reported using culturally/linguistically validated PROMs, despite the multi-national status of many of the trials.4

Why is this a concern?

If some groups of patients or cultural contexts are not represented at the design stage in a clinical trial, then any PROMs used may not capture their experience, excluding the experience of that community and making findings less generalisable. If patient perspectives from diverse backgrounds are not included in the study of an emerging drug; the effects of certain care management regimes or surgical outcomes, then how can we claim there is true “value” in the results if they are not representative of the “real world”.

Patient-reported outcomes highlights disparities in ethnic diversity relating to back pain

Inclusive PROMs collection can lead to insights between different patient subgroups. Milani et al. explored the associations of self-reported race/ethnicity with back pain PROMs and health care utilisation among older adults experiencing a new episode of back pain over 2 years. The results illustrated that patients from African-American and Hispanic descent displayed less improvement in back pain-related functional limitations over time, and less spine-related health care utilisation, as compared to Whites and non-Hispanics, respectively.5

Increasing efforts for inclusion

Addario et al. aimed to show how the integration of the patient and/or patient advocate at all stages of PROMs development can help to realise the full potential of PROs. Efforts were made to achieve inclusivity in their investigation as patients and patient advocates from low‐ or middle‐income backgrounds or countries appear to be less frequently involved in PROMs development.6

Additionally, patients and patient advocates can make an informed contribution to the language used in questionnaires in order to ensure the use of plain language, the lack of which has been noted in some existing cancer PROMs.7 Supporting the use of plain language in PROMs and ensuring all terms used are understood in the same way across different cultures, and healthcare frameworks can help increase compliance for patients with low literacy levels.8

Barriers exist to include a complete mix of patient voices in healthcare and drug development studies due to the systemic societal problems relating to diversity and inclusion. Nevertheless, fostering an appreciation of inclusion in PROMs design and implementation in studies and clinical practice can only benefit the robustness of such tools and help inform all participants in healthcare of varying needs across all populations and enhance doctor-patient communication. Encouraging patient diversity in studies can only add to the information that can be derived from PROMs. The medical community depends on realistic data of high quality and PROMs as well as Real World Evidence insight have the potential to be reflective of the vibrant, diverse, multi-cultural societies we live in.