COLLEAGUES, as part of the XXV RUSSIAN ONCOLOGICAL CONGRESS, the 12th symposium "Pharmacoeconomics in Oncology" was held on the topic: "Real World Data / Real World Evidence (RWD / RWE): Role in Pharmacoeconomics and Health Technology Assessment" (https://rosoncoweb.ru/news/society/2021/11/15-1/).

Moderators:

• Kolbin Aleksey, Head of the Department of Clinical Pharmacology and Evidence-Based Medicine, I.P. Pavlov
• Pavlysh Andrey, Head of the Department of Pharmacology and Pharmacy, FSBEI HE "North-Western State Medical University named after I.I. Mechnikov", FSBI" N.M. Petrov".

Speakers:

• Gracy Crane, Head of International RWD Policy, Roche;
• Borzova Maria, Counselor of the Trubor Law Office;
• Plavinsky Svyatoslav, Head of the Department of Pedagogy, Philosophy and Law, North-Western State Medical University named after I.I. Mechnikov ";
• Ivanov Alexander, head of the consulting department of Aston Health;
• Musina Nuria, Head of the Department of Development and External Communications of the Federal State Budgetary Institution "TsEKKMP" of the Ministry of Health of Russia;
• Galimov Timur, Executive Director of Data Management 365;
• Zyryanov Sergey, Head of the Department of General and Clinical Pharmacology, FGAOU VO RUDN;
• Rudakova Alla, Professor of the Department of Management and Economics of Pharmacy, Federal State Budgetary Educational Institution of Higher Education "SPKhFU";
• Kurylev Alexey, FSBEI HE I.P. Pavlov, Federal State Budgetary Institution “N.M. Petrov";
• Samsonov Mikhail, director of the medical department of the R-Pharm company.

Discussed:
the main definitions of research data from real clinical practice (RWD, RWD) in the world and the Russian Federation. The current situation is described. The main methods of RWD data collection are highlighted - from clinical trials to database analysis. The directions of practical application of the results of RWD data studies are shown - from registration and amendments to the registration dossier, to pharmacovigilance, and reimbursement. Recommendations and conclusions for the implementation of the RWD/RWE paradigm in Russia are offered.

In particular, a brief description of the US regulatory experience was given. Proposals are presented in terms of organizing and improving the legal framework in the field of RWD/RWE regulation in the EAEU.

The definitions of evidence-based medicine, the apparent conflict between evidence and features of RWD data collection are considered, the possibilities of improving the planning of RWD studies and increasing their evidence are shown, examples from the field of oncology are given.
A detailed description of the clinical register is presented: definition, methods of data collection, methods of their analysis. The possibilities of using clinical registers as a source of RWD are shown.

Practical examples of the use of RWD approaches are given, a systematic experience of conducting research is presented, tools and methodology for application in the context of the COVID-19 pandemic are proposed.

The role of RWE studies in the collection of information on adverse events was discussed, the special value of studies of this kind, the methodology for conducting studies devoted to assessing the safety profile, was emphasized.

An experience of evaluating the available data from randomized clinical trials and RCP based on the results of indirect comparisons of the use of CDK 4/6 inhibitors for combined therapy of mBC is presented.

The current situation with obtaining information from up-to-date databases on the medical care provided to patients with malignant neoplasms is considered. An analysis of information sources is given, methodological approaches are proposed that allow obtaining the most complete information about the treatment of patients with malignant neoplasms.

BY THE RESULTS OF THE SYMPOSIUM “PHARMACOECONOMICS IN ONCOLOGY. DATA FROM REAL CLINICAL PRACTICE: ROLE IN PHARMACOECONOMY AND ASSESSMENT OF HEALTHCARE TECHNOLOGIES "ACCEPTED THE FINAL RESOLUTION:
I. Proposed translation of RWD/RWE terms into Russian.
RWD (Real - World Data) - data of real clinical practice - data related to the patient's state of health and (or) to the process of providing medical care obtained from various sources;
RWE (Real - World Evidence) - evidence obtained from real clinical practice - clinical evidence regarding the use and potential benefits or risks of using a drug, obtained from the collection and analysis of RCP data.
II. The limitations for the implementation of the RWD tools have been identified.

1. In the legal regulation of the Russian Federation:

1.1 There are no legislative definitions of the terms "real clinical practice", "data of real clinical practice", "research of real clinical practice" and "evidence of real clinical practice", as well as the rules governing the procedures for collecting and analyzing RWD.

1.2 There are no regulatory approaches for introducing RWD and/or evidence obtained from the analysis of RWD and their use in conjunction with the results of clinical trials when updating data on the safety and efficacy of drugs.

1.3 There are no tools to compare drug data with patient data. Federal register data, clinical trial data do not interact with drug movement monitoring systems.

1.4 There is no procedure for registering RWD studies as a post-authorisation clinical study or other.

2. When introducing innovative models of drug provision:
There is no RWD paradigm in the domestic health care system, which does not allow the introduction of a value-oriented approach to innovative models of drug provision. At the same time, to launch a pilot project on the introduction of innovative models of drug provision, it is necessary, inter alia, to analyze data on the clinical efficacy of a drug for public procurement using innovative models (for example, according to the "risk-sharing" model).
3. When using information systems:
The quality of the data collected by laboratory information systems and medical information systems and their incompatible format does not allow systematic processing and use of RWD when making decisions in the health care system.
4. When creating registers:
4.1 The Federal Register combines data only on certain categories of patients (limited to certain lists of nosologies and categories of persons entitled to receive state social assistance).
4.2 In Russia, there is no unified register of patients receiving medical care within the framework of the state health care system, i.e. an aggregator register that would combine the data of all other registers.
III. Solutions for the introduction of the RWD paradigm into the healthcare system of the Russian Federation are proposed:
1. At the level of legal regulation of the Russian Federation:
1.1 To amend the Federal Law of 12.04.2010 No. 61-FZ "On the Circulation of Medicines": to supplement Art. 4 definitions "real clinical practice", "data of real clinical practice", "research of real clinical practice", "evidence of real clinical practice", synchronized with the terminological apparatus of the Eurasian Economic Union.
1.2 Develop normative acts on the procedure for processing and analyzing RWD. To fix in them the requirements: to the methods of collecting and analyzing RWD for further assessment of their quality and transparency; to methods of dealing with possible medical errors and data entry errors; to sources of information that can be used for research purposes.
1.3 Include the RWD, evidence obtained from the analysis of RWD, together with the results of clinical trials, in the scientific justification when making changes to the instructions for medical use in relation to the safety and efficacy of previously registered medicinal products, decisions to suspend the use of a medicinal product, inclusion / exclusion of a medicinal product from restrictive lists, as well as confirmation of the validity of the results of a clinical trial when making regulatory decisions.
1.4 Create a national standard of requirements and ethical principles for the collection, storage and processing of RWD.
1.5 Create a specialized structure to control the quality of tools for collecting and processing RWD.
2. When introducing innovative models of drug provision:
2.1 Optimize antitrust legislation to eliminate the risks of unjustified application of existing regulatory norms to innovative models of drug provision.
2.2 Introduce a mechanism for recording RWD when analyzing data on the clinical efficacy of a medicinal product for public procurement "according to innovative models".
2.3 Introduce a mechanism for recording RWD when evaluating treatment outcomes in a model of a payment based on treatment agreement.
3. When using information systems:
3.1 Introduce a set of measures to control the quality of data collected / entered within the framework of MIS and LIS.
3.2 Introduce a set of measures for the integration of MIS and LIS, specializing in the collection of structured medical data.
4. When creating registers:
4.1 Review approaches to maintaining patient registers in order to transform registers into sources of reliable scientific information about the patient population, the prescribed and applied therapy, treatment outcomes, etc., and provide for the maintenance of a single register for all nosologies.
4.2 Develop a procedure for interdepartmental interaction with the aim of maintaining and processing the data of such a register, as well as the possibility of providing limited access for the formation of RWD from the medical and scientific community.
4.3 Establish the obligatory use of registers in clinical practice, as well as in the framework of health technology assessment (HTA).
4.4 Provide for the organization of a system of access to the unified state information system in the field of health care (Unified State Health Information System), incl. to the data of the RWD (and not only to its individual subsystems) for medical specialists, experts, scientists, representatives of non-state companies (the IT sector, the pharmaceutical industry, manufacturers of medical and diagnostic equipment).
5. In organization of interdepartmental and expert interaction:
Create working groups to address the above issues in order to implement effective approaches to collecting, analyzing and evaluating RWD.

The text of the resolution was approved by the leadership of the Association of Health Technology Assessment Experts, the Association of Clinical Pharmacologists, the St. Petersburg office of the International Society for Pharmacoeconomic Research and Scientific Outcome Analysis (St. Petersburg branch of ISPOR)

11/9/2021