Patient-Reported Outcomes — What They Are — and Why They Matter

In today’s healthcare environment, it is more important than ever to hear and integrate patient perspectives into medicine research and development processes. Patient-reported outcomes, or PROs, are reports directly from patients regarding their health status, free from any influence by healthcare professionals. Clinical trials that prioritise PROs as primary endpoints or use them to complement other primary outcomes, such as survival rate, gain a strategic advantage: they ensure research aligns with the outcomes that matter most to patients. (1)

Ultimately, only the patient can comprehend the true impact of their condition.

PROs can capture a wide range of patient insights, from simple ratings of pain severity or treatment satisfaction to broader considerations, such as how their condition affects their quality of life, the symptoms they experience, or any challenges they encounter with their care. (1) Therefore, adopting both quantitative and qualitative approaches to measure these metrics is crucial for gaining a comprehensive understanding of diverse patient needs and feelings.

Patient-reported outcome measures (PROMs) are tools used to determine patients’ perspectives on their health, primarily through self-assessment questionnaires. Some of these measures are universally applicable across various conditions, such as the EQ‐5D, which includes dimensions like anxiety and depression. Others are more disease-specific, for example, the PRO-CTCAE questionnaire for cancer patients. Together, these self-report methods enable clinicians to gather patient insights within specific therapy areas and compare these data across conditions. (2,3)

So, what is the value of PROs, and why should their outputs be disseminated across the industry?

The primary benefit of PROs is that they support true patient-centricity. 

With our robust drug discovery and development pathways, countless clinically effective drugs are now available for patients. However, many of these treatments are not always patient-friendly. Let’s consider the combined pill, for example. This has proven to be over 99% effective in preventing pregnancy in sexually active women and people with periods when used correctly, according to the NHS. (4) Despite the efficacy of this contraceptive, many individuals switch pills or discontinue the pill altogether due to its adverse effects on their body and mind, with mood changes being a common reason. (5)

This area of women’s health would, therefore, benefit significantly from PROs to raise awareness of the challenges women face when taking the combined pill. As healthcare professionals, we must go beyond sharing these learnings within the industry and incorporate them into decision-making processes to shape the future development of similar contraceptives that better align with patient needs, empowering them to have a healthy sex life with fewer adversities.

In addition to amplifying patient voices to drive patient-focused drug development, PROs play a crucial role in enhancing the safety and tolerability of novel therapies. This is evident in the field of oncology, particularly with the emerging treatments in malignant haematology and oncology. Patient-reported symptomatic adverse effects will likely aid in the development of these cancer therapies, strengthening the validity of existing safety reports conducted by clinicians and providing longitudinal evidence of toxicities in real-world settings. (6)

With patient safety at the forefront of our healthcare system, PROs are a vital tool in modern healthcare. Moreover, if patient-reported outcomes are central to our medicine development lifecycle, pharmaceutical companies stand to gain financially by delivering drugs tailored to patient interests, benefiting all stakeholders.

At Synthesis, we understand that every patient's experience, priorities, and needs are unique and personal. That is why our team of specialist medical writers is dedicated to circulating their diverse insights throughout the industry to improve health outcomes inclusively. Contact us at info@synthesishealth.co.uk to learn how we can support your partnerships with patients and their advocates throughout the entire drug development process.

References:

1. Weldring T, Smith SM. Patient-Reported Outcomes (PROs) and Patient-Reported Outcome Measures (PROMs). Health Serv Insights. 2013;6:61-68. Published 2013 Aug 4. doi:10.4137/HSI.S11093

2. Herdman M, Gudex C, Lloyd A, et al. Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L). Qual Life Res. 2011;20(10):1727-1736. doi:10.1007/s11136-011-9903-x

3. Di Maio M, Basch E, Denis F, et al. The role of patient-reported outcome measures in the continuum of cancer clinical care: ESMO Clinical Practice Guideline. Annals of Oncology. 2022;33(9):878-892. doi:10.1016/j.annonc.2022.04.007

4. What is the combined pill? https://www.nhs.uk/contraception/methods-of-contraception/combined-pill/what-is-it/

5. Johansson T, Vinther Larsen S, Bui M, Ek WE, Karlsson T, Johansson Å. Population-based cohort study of oral contraceptive use and risk of depression. Epidemiology and Psychiatric Sciences. 2023;32:e39. doi:10.1017/S2045796023000525

6. Kluetz PG, Chingos DT, Basch EM, Mitchell SA. Patient-Reported Outcomes in Cancer Clinical Trials: Measuring Symptomatic Adverse Events With the National Cancer Institute's Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE). Am Soc Clin Oncol Educ Book. 2016;35:67-73. doi:10.1200/EDBK_159514