Monitoring patient-reported outcomes is becoming the new norm in clinical trials
The journal Value in Healthpublished an analysis of the use of patient-reported outcomes (PROs) in clinical trials from 2008 to 2023. The authors analyzed more than 475,000 registered studies and showed a steady increase in the inclusion of PROs in clinical trial design.
What the study showed
In other words, by 2023, almost one in four studies in adults already included an assessment of outcomes reported by the patients themselves.
What exactly is meant by PROs?
We are talking about questionnaires and scales that patients complete themselves to assess:
Such data do not replace clinical endpoints, but allow us to understand how the patient actually tolerates treatment and what effect the therapy has on their daily life.
Which instruments are used most often?
A published analysis showed that among universal questionnaires, the leading positions are took:
At the same time, it was the EQ-5D that became the most widely used universal instrument: it overtook SF after 2017 and by 2023 was used in 1023 studies per year, while SF instruments were used in 781.
Separately, the authors note the very rapid growth of the PROMIS family: it is the fastest-growing class of instruments over the entire observation period.
What is happening in Pediatrics
In pediatric studies, the PedsQL was most frequently used, and the use of:
However, the authors emphasize that pediatric PROs are still being implemented more slowly than in adult studies, despite the clear growth.
There are also regional differences
The analysis showed that PRO use also depends on the region where the studies are conducted:
This suggests that the choice of questionnaires in clinical trials is gradually becoming standardized, but a globally unified approach has not yet been established.
In what areas are PROs particularly important?
The authors note that PROs are most often used in studies of:
This is particularly noticeable for oncology: specialized questionnaires such as the EORTC QLQ-C30 and FACIT are widely used here.
The article shows that monitoring patient-reported outcomes is no longer an optional element clinical trial. It is becoming part of the standard approach to assessing treatment outcomes, along with traditional clinical measures.
The EQ-5D occupies a special place as one of the most common and practically applicable questionnaires for assessing patient quality of life in studies.
Source:
Patient-Reported Outcome Measures in Clinical Trials: An Analysis of Trends From 2008 to 2023
Value in Health
journal
What the study showed
- Among adult studies, the share of protocols with PROs increased from 2.7% in 2008 to 23.5% in 2023
- In pediatric studies, from 8.0% to 17.7%
- A total of 88,484 studies included PROs out of 475,844 analyzed
In other words, by 2023, almost one in four studies in adults already included an assessment of outcomes reported by the patients themselves.
What exactly is meant by PROs?
We are talking about questionnaires and scales that patients complete themselves to assess:
- quality of life
- symptoms
- physical, emotional, and social functioning
- the impact of therapy on daily life
Such data do not replace clinical endpoints, but allow us to understand how the patient actually tolerates treatment and what effect the therapy has on their daily life.
Which instruments are used most often?
A published analysis showed that among universal questionnaires, the leading positions are took:
- EQ-5D
- SF-36 / SF-12 / SF-8 / SF-6D
- PROMIS
At the same time, it was the EQ-5D that became the most widely used universal instrument: it overtook SF after 2017 and by 2023 was used in 1023 studies per year, while SF instruments were used in 781.
Separately, the authors note the very rapid growth of the PROMIS family: it is the fastest-growing class of instruments over the entire observation period.
What is happening in Pediatrics
In pediatric studies, the PedsQL was most frequently used, and the use of:
- EQ-5D-Y
- PROMIS Pediatric
However, the authors emphasize that pediatric PROs are still being implemented more slowly than in adult studies, despite the clear growth.
There are also regional differences
The analysis showed that PRO use also depends on the region where the studies are conducted:
- In Europe, the EQ-5D is most frequently used.
- In North America, the SF is stronger. and PROMIS
- PRO implementation is less uniform in other regions.
This suggests that the choice of questionnaires in clinical trials is gradually becoming standardized, but a globally unified approach has not yet been established.
In what areas are PROs particularly important?
The authors note that PROs are most often used in studies of:
- oncological diseases
- nervous system diseases
This is particularly noticeable for oncology: specialized questionnaires such as the EORTC QLQ-C30 and FACIT are widely used here.
The article shows that monitoring patient-reported outcomes is no longer an optional element clinical trial. It is becoming part of the standard approach to assessing treatment outcomes, along with traditional clinical measures.
The EQ-5D occupies a special place as one of the most common and practically applicable questionnaires for assessing patient quality of life in studies.
Source:
Patient-Reported Outcome Measures in Clinical Trials: An Analysis of Trends From 2008 to 2023
Value in Health
journal
