WP17 – Non-interventional study and collection of Real-world-data (RWD)
Impact/relevance
This WP is a prospective, non-interventional umbrella real world study (RWS) that aims to describe the safety and efficacy of allocating patients to standard of care or available clinical trials of targeted anti-cancer drugs in a consecutive group of patients with brain cancer. Known comorbidities for brain cancer include social status, age, gender XYZ. The primary endpoint will be to generate real world data (RWD), which will be analysed and compared with data from the clinical DNOG databases, to produce real world evidence (RWE).
Background
There is a gap between the data from the narrowly focused low-quantity, high-quality interventional studies and lower-quality, high-quantity RWD. Several important issues are missing: (1) precise classification of broad molecular signatures; (2) standardized clinical elements longitudinally tied to molecular data; (3) sufficient quantity of quality data to allow scientific comparison between groups; (4) the ability to analyze and review complex datasets; and (5) the mechanism to evolve interventions as new information is learned. Therefore, prospective, observational trials that include all patients independent of biomarkers and collects comprehensive data on each are needed.1
Access to detailed socio-economic information of each patient in combination with a complete medical history with recording of prescribed medications, history of all hospital admissions and out-patient visits as well as the history of psychiatric diseases will create a core of data, which has no comparison worldwide.
Aims
-To collect RWD by a complete set of clinical, socio-psychological, medico-economics data and biospecimens, including WGS of all patients with brain cancer
-To record the treatment-related adverse events and late effects experienced by patients based on Patient Reported Outcome tools.
-To perform refined biomarker analyses, including (but not limited to) whole genome sequencing, on a fresh tumour biopsy specimen at baseline and at progression, in order to tailor subsequent therapy.
Methods
Eligible patients will have high-grade glioma and acceptable performance status and organ function. This is a non-interventional study and treatment will be standard of care or an available clinical trial based on informed consent, independent of this study.
The study will collect clinical, socio-psychological, medico-economics data and biospecimens for all patients with high-grade glioma treated in Denmark from 2021-2025 (N=2000-2500). The researchers and clinicians at the departments have long-term experiences with access and use of the Danish nationwide and population-based health and disease registries. By using these data sources, we hope to capture additional information about demographics, comorbidities, medications and treatment trajectories for patients with brain cancer in Denmark
– all factors, which we anticipate may play a role in the outcome of the treatment of the disease. Ultimately, we aim at achieving transformative knowledge, which in our perspective may improve patient care and outcome of the treatment by adding this information into the overall data set.
Patient Reported Outcomes has been known in the clinic for decades. First as anamnestic information in the medical record, then as quality of life information and lately as the subjective information concerning experience of somatic and psychological symptoms.
Adding genomics, clinical data, register based data and patient reported outcomes together makes up the most comprehensive data structure needed for a successful implementation.
Expected outcome
To improve outcome for brain tumour patients. Tumour genome analyses may identify new and better treatment options offered to patients in Umbrella trials as part of the program. Identification of patterns and causes of toxicity and the importance of comorbidity will be implemented after validation.
In addition, preventive measures will be implemented upon identification of important host factors for development of failure as well as acute and late effects.
References
Dickson D, Johnson J, Bergan R, Owens R, Subbiah V, Kurzrock R. The Master Observational Trial: A New Class of Master Protocol to Advance Precision Medicine. Cell. 2020 Jan 9;180(1):9-14.