Context and Importance
Patient-reported outcomes (PROs) are indispensable tools in understanding the lived experiences of individuals with health conditions. However, as highlighted in Ramirez et al.'s 2024 paper, generic tools like the EQ-5D often fail to capture the complexities of specific populations, particularly those with rare diseases or underserved conditions At DHT.health, we strive to fill these gaps by addressing limitations in traditional PRO measures and advocating for tools that reflect diverse patient realities.
One critical issue is the disability paradox, where individuals with chronic or severe conditions report better quality of life than anticipated by observers. Studies using methodologies such as a DCETTO (Discrete Choice Experiments Time Trade-Off) have shown how generic measures, such as EQ-5D, can overlook nuanced patient experiences, as seen in haemophilia and achondroplasia. Similarly, PRO comparison studies in rare and underserved populations have highlighted the need for additional evidence to help support the most appropriate PRO measurement.
Challenges: Addressing Systemic Barriers
Ramirez et al. identify several systemic barriers, including biases in research funding, limited representation in clinical trials, and technological access gaps. DHT.health sees additional challenges specific to PRO research:

Addressing these challenges demands systemic change. Advocacy for equity-focused funding, partnerships with patient advocacy groups, and investments in innovative methodologies are critical.
DHT Support This Initiative via:
Showcasing Disability Paradox Studies: Our team has been previously been involved in studies in haemophilia and achondroplasia which highlight where generic tools fall short, advocating for the need for further evidence in other conditions to assist in understanding how generic measures perform. We intend to continue and build upon this work.
Comparative PRO Studies: By comparing tools such as generic and disease-specific tools, we help stakeholders understand performance of the tools in specific populations. One example our team was involved in was comparing the DMD-QOL with the EQ-5D. This form of evidence can inform trial inclusion and relevance in HTA discussions.
Commitment to Innovation: Through continued collection of real-world datasets and patient-centred research, we generate insights and evidence to support such initiatives to ensure health equities are addressed in PRO measurement. Our PIVOT methodology further enhances this by integrating patient and caregiver insights into cohesive and actionable datasets. Insights from our PIVOT-vWD study will be communicated soon.
Achieving health equity in PROs requires collective action from researchers, policymakers, and healthcare providers. DHT.health is proud to lead this charge, leveraging innovative methodologies, community collaboration, and a commitment to inclusivity. Together, we can ensure that PROs truly reflect the diverse voices of all patients, transforming healthcare for the better.
References
1. Ramirez, Lourdes G. et al. Understanding Health Equity in Patient-Reported Outcomes. 2024. The Journal of Allergy and Clinical Immunology: In Practice, Volume 12, Issue 10, 2617 – 2624. https://www.jaci-inpractice.org/article/S2213-2198(24)00407-0/fulltext
2. O’Hara, J., Martin, A.P., Nugent, D., Witkop, M., Buckner, T.W., Skinner, M.W., O’Mahony, B., Mulhern, B., Morgan, G., Li, N. and Sawyer, E.K. (2021), Evidence of a disability paradox in patient-reported outcomes in haemophilia. Haemophilia, 27: 245-252. https://doi.org/10.1111/hae.14278
3. Morgan, G et al. PCR64 Examining the Disability Paradox and Health State Utility Valuation in Achondroplasia. 2024. Value in Health, Volume 27, Issue 6, S307. http://dx.doi.org/10.1016/j.jval.2024.03.1943
4. Morgan, G. et al. PCR65 Assessment of the EQ-5D and DMD-QoL for Duchenne Muscular Dystrophy: A Patient and Caregiver Study. 2023. Value in Health, Volume 26, Issue 12, S461. http://dx.doi.org/10.1016/j.jval.2023.09.2504