Pfizer supported the publication of this article as well as the meeting of the experts. Authors of the article received an honorarium from Pfizer in connection with their participation in the meeting of the experts.

Authors: Dr. Elżbieta Senkus-Konefka, Dr. Géczi Lajos, Dr. Karen Drumea, Dr. Adrian Pana, Dr. Ilya Tsimafeyeu, Dr. Yüksel Ürün, Dr. Finek Jindrich, Dr. Borštnar Simona

The importance of Real-World Data (RWD) and Real-World Evidence (RWE) is increasing, especially in oncology. While COVID 19 has accelerated the adoption of RWE, the concept is relatively new in oncology within Central and Eastern Europe (CEE). Although everyone believes there is a place for RWE to complement clinical trials in clinical decision-making, there are still many questions about the value of this type of research in clinical guidelines, decision-making, or regulatory/reimbursement approvals. RWE offers excellent potential to accelerate therapy development and monitor the successes and failures of both newly approved and existing therapies.

Resource constraints are a reality in cancer care in Central and Eastern Europe and reimbursement discussions are based on budget, rather than the value of a treatment. RWE provides insights into the benefits of a treatment in a specific country, the impact of treatment modalities, and the impact on patients. Indeed, often conventional approaches are less efficient but remain the preferred course of treatment in some countries, without demonstrating any tangible benefits. Despite new scientific guidelines, physicians sometimes use old conventional approaches. RWD collection and evidence derived from these instances could also be a good resource to show the real benefit of any treatment modality.

Expert oncologist from the CEE region came together and shared their experience in RWE generation, including how it is perceived and utilised, production challenges, and the action points for stakeholders.

The key challenges in RWE generation, especially in CEE region, are the following:

• Data ownership and ethical approvals: There is not enough clarity on how data privacy and data protection should be the handled when collecting data for RWD. Data governance should be clarified and regulated for future research purposes. In many cases, hospitals do not share data, even if it is anonymised.

• Data standardisation: One of the biggest challenges in most CEE countries is the absence of a central data collection system (each hospital having its data set), and the lack of a standardised data collection approach to meaningfully respond to research questions. Most of the time, even open texts are handwritten and challenging to understand. Although there is data, it can't be processed by any technological methods, such as AI. There are good examples, such as the Czech Republic, which has centralised disease and treatment registries, which produce a lot of evidence, and there is a clear plan on how it should be utilised. There is also a lack of interoperability in many cases, even at the same hospital between departments.

• Clinical patient load and time constraints: to have meaningful data collection, clinicians need support. Because in many cases, clinicians do not have enough support personnel to help with inpatient care. Data collection might seem like an additional burden, without any incentive for physicians in clinical care, but not in academia.

• Transparency: Because most CEE countries have central reimbursement bodies, these institutions collect some form of data. This type of data is reliable as physicians take great care as it has a direct effect on reimbursements of a therapies. Most countries will collect the data but not provide any details due to privacy laws. However, in some countries, such as Hungary, these data sets are transparent and can also answer some research questions. Still, there are points raised by oncologists that data collection must adhere to strict protocols and relevant research methodologies should be applied when conducting research or evaluating the results from these types of databases.

• Lack of knowledge and experience of RWE amongst oncologists: Even though oncologists in CEE countries are experienced in clinical trials, most of them, maybe like in other parts of the world, lack sufficient knowledge or experience to work with RWD or with this type of research.

• Lack of local guidelines to regulate RWD collection/analysis and adaptation of RWE in local regulatory/reimbursement discussions: local guidelines are missing regarding how to collect RW data. It is not clear how collected and analysed data will affect any decision-making process.

Based on the above challenges set forth, there are future recommendations that could be put in place to ensure an increased uptake of RWE in CEE.  Authorities like the FDA (Food and Drug Administration of US), EMA (European Medicines Agency) provide some guidance on RWE, however it is crucial to have local guidelines. Oncology societies, payors and regulatory bodies should play a role in determining how big data, AI, RWD collection, RWE generation will be used. In addition, there is an educational need for AI and big data among oncologists. As technology continues to develop rapidly, clinicians, especially oncologists, need to be educated on the types of technologies available. Oncologists should have the opportunity to modify existing technology to ensure it fits the needed criteria.  Lastly, we should be ready for data standardisation to facilitate meaningful data collection. In most cases, physicians collect some data either for hospital records or payer documents. If this data was standardised, we could learn more about how treatment results are reflected in real life. Data standardisation will also lead to a more focused quality of care and allow us to ensure the highest quality of care and transparency to all patients in the future.

Below you can see the situation of RWE utilisation in regulatory/reimbursement discussion.

NHIF: National Health Insurance Fund
HTA: Health Technology Assessment
RCT: Randomised controlled trials
MoH: Ministry of Health
EAP: Expended Access Programs