Real-world Evidence in Onco-Hematological Patients

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About this Research Topic

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Background

Clinical practice, particularly in onco-hematological settings, often relies on randomised controlled trials (RCTs) because they often provide the highest level of scientific evidence. Generalising trial results to everyday clinical practice is not straightforward because of low overall trial accrual (<5% of all newly diagnosed cancer patients) and under-representation of patients frailties, such as older age, advanced disease stage, comorbidities and lower socio-economic status. RCTs provide evidence concerning the best achievable responses under controlled experimental conditions and are essential for clinical research, particularly in pharmaco-epidemiological field; real-world studies generate evidence concerning the actual advantage achieved in real-life setting, which is indispensable for public health research aimed at assessing (and improving) the impact of daily life treatment. In addition, real world studies can provide information on pattern and quality of care as well as access to adequate health care, obtaining relevant information for the health care organisation.

For example, FABIO (Biologic Drugs in Oncology, the Italian acronym being Farmaci Biologici in Oncologia) was an Italian multiregional research project that evaluated safety, effectiveness, and cost-effectiveness of biologic drugs approved for treating cancer in a real world setting, including a study on multiple myeloma in non-transplant patients.

The EUROCARE project (www.eurocare.it), based on population cancer registries (CRs) data, analysed and interpreted the difference of prognosis and survival for oncological disorders by different European countries thanks also to the high resolution (HR) studies (www.hrstudies.eu) involving European CRs but collecting more detailed clinical data (stage at diagnosis, relapse, diagnostic procedure, molecular profile and treatment).
Since nowadays it is possible to realise real-world studies that achieve relevant scientific evidence, we devote this Special Issue to this type of studies to evaluate onco-hematological patients' characteristics, pattern and quality of care and the impact of treatments in the real-world setting. Real-world studies can collect a huge number of cases, which is an added value to analyse rare hematological malignancies.

Specific topics of interest are, but not limited to, the following areas:
- Observational registry-based studies
- Administrative flow studies
- Hospital-based studies
- All non-RCT studies.

Keywords: Hematological malignancies, clinical practice, real-world evidence, onco-hematology, cancer registries

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