Abstract
Context: Harmonising outcome reporting and definitions in clinical trials and in routine patient records can enable healthcare systems to provide more efficient outcome-driven and patient centred interventions. We report on the work of the IMI Big Data Consortium Prostate Cancer Diagnosis and Treatment Enhancement Through the Power of Big Data in Europe (PIONEER).
Objective: To update and integrate existing prostate cancer (PCa) Core Outcome Set (COS) for the different stages of PCa, while also assessing their applicability and developing standardised definitions of prioritised outcomes.
Evidence acquisition: We followed a four-stage process:(1) systematic reviews;(2)
qualitative interviews;(3) expert group meetings to agree standardised terminologies;(4) recommendations for most appropriate definitions of clinician reported outcomes.
Evidence synthesis: Following four systematic reviews, a multinational interview study and expert group consensus meetings, we defined the most clinically suitable definitions for a) localised and locally advanced, and b) metastatic and non-metastatic castration resistant PCa COS. No new outcomes were identified in our COS for localised/locally advanced PCa. For our metastatic and non-metastatic CRPC COS, nine new core outcomes were identified.
Conclusion: These are the first COS in PCa where the definitions of the prioritised outcomes have been surveyed in a systematic, transparent, and replicable way. Furthermore, this is the first-time recommendations for outcome definitions across all prostate cancer COS have been agreed on by a muldisciplinary expert group and recommended for use in research and clinical
practice settings (see Table 4,5). To limit heterogeneity across research, they should be recommended for future effectiveness trials, systematic reviews, guidelines and clinical practice of localised and metastatic PCa.
Patient summary: PCa patient outcomes after treatment are difficult to compare due to variability. To enable better use of PCa patient data, PIONEER standardised and recommended outcomes (and their definitions) that should be collected as a minimum in all future studies.
Objective: To update and integrate existing prostate cancer (PCa) Core Outcome Set (COS) for the different stages of PCa, while also assessing their applicability and developing standardised definitions of prioritised outcomes.
Evidence acquisition: We followed a four-stage process:(1) systematic reviews;(2)
qualitative interviews;(3) expert group meetings to agree standardised terminologies;(4) recommendations for most appropriate definitions of clinician reported outcomes.
Evidence synthesis: Following four systematic reviews, a multinational interview study and expert group consensus meetings, we defined the most clinically suitable definitions for a) localised and locally advanced, and b) metastatic and non-metastatic castration resistant PCa COS. No new outcomes were identified in our COS for localised/locally advanced PCa. For our metastatic and non-metastatic CRPC COS, nine new core outcomes were identified.
Conclusion: These are the first COS in PCa where the definitions of the prioritised outcomes have been surveyed in a systematic, transparent, and replicable way. Furthermore, this is the first-time recommendations for outcome definitions across all prostate cancer COS have been agreed on by a muldisciplinary expert group and recommended for use in research and clinical
practice settings (see Table 4,5). To limit heterogeneity across research, they should be recommended for future effectiveness trials, systematic reviews, guidelines and clinical practice of localised and metastatic PCa.
Patient summary: PCa patient outcomes after treatment are difficult to compare due to variability. To enable better use of PCa patient data, PIONEER standardised and recommended outcomes (and their definitions) that should be collected as a minimum in all future studies.
| Original language | English |
|---|---|
| Pages (from-to) | 503-514 |
| Number of pages | 12 |
| Journal | European Urology |
| Volume | 81 |
| Issue number | 5 |
| Early online date | 13 Apr 2022 |
| DOIs | |
| Publication status | Published - May 2022 |
Bibliographical note
Acknowledgements: We are thankful to all the patients and health careprofessionals who took part in our interview studies and to the patient
representatives and advocates who provided insight during our various consensus
meetings. We are grateful to Dr. Susie Dodd for her advice in applying the 38-item
outcome taxonomy which she and her COMET colleagues developed, and we
accept responsibility for any (mis)application. PIONEER is funded through the IMI2 Joint Undertaking and is listed under grant agreement No. 777492. This joint under- taking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA.
Data Availability Statement
Supplementary data to this article can be found online athttps://doi.org/10.1016/j.eururo.2022.01.042.
Keywords
- Prostate Cancer
- Patient pathway
- Comparative effectiveness research
- Core outcome sets
- Standardised outcome sets
- Outcomes
- Randomized controlled trials
- Systematic reviews
- Big Data
- Real world Data
Access to Document
- Beyer_etal_EU_Updating_And_Integrating_VoR
This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).