ISSN 0377-9556 (PRINT)
ISSN 2383-9457 (ONLINE)
ISSN 2383-9457 (ONLINE)
Results of focus group interview: Lists to prepare for utilizing RWD/RWE for regulatory decision making for drugs
| In order to utilize RWD/RWE throughout the entire cycle from drug approval to post-marketing safety management, what do you think the industry and national institutions should prepare for each? | |||
|---|---|---|---|
| Whether the group agreed | |||
| Academia | Industry | Government and medical professional | |
| Regarding RWD/RWE guidelines | |||
| (1) Different guidelines (eg. proper RWD, how to interpret the results, etc.) according to the purpose of RWD/RWE use (eg. new drug application, conditional approval, post market surveillance, etc.) | O | O | O |
| (2) RWD collection | |||
| • Establishing protocol for RWD collection | |||
| - Establishing protocols for RWD collection and RWE generation for approval process | O | O | O |
| - Establishing protocol for RWD collection and RWE generation for post-approval process | X | O | X |
| • Permission of MFDS for established protocol | |||
| - Permission of MFDS for approval process | O | △ |
O |
| - Permission of MFDS for post-approval process | X | X | X |
| • RWD collection at designated institutions where stipulated facilities and equipment are equipped by laws | |||
| - Designation of a RWD collection agency when using RWD for regulatory decisions that require high-level evidence such as approval | - | O | - |
| - Designation of a collection agency when using RWD for other purposes | - | X | - |
| (3) RWD Management | |||
| • Designation of quality evaluation methods to secure validity for each RWD | O | O | O |
| • Evaluate the completeness of data for each RWD | O | O | O |
| • RWD feasibility evaluation by external experts | O | - | - |
| • RWD management at designated institutions where stipulated facilities and equipment are equipped by laws | |||
| - Designation of a management agency when using RWD for regulatory decisions that require high-level evidence such as approval | - | O | - |
| - Designation of management agency when using RWD for other purposes | - | X | - |
| (4) RWE generation | |||
| • Establishing protocol for RWE generation | |||
| - Establishing protocols for RWD collection and RWE generation for approval process | O | O | O |
| - Establishing protocols for RWD collection and RWE generation for post-approval process | X | O | X |
| • Permission of MFDS for established protocol | |||
| - Permission of MFDS for approval process | O | △ |
O |
| - Permission of MFDS for post-approval process | X | X | X |
| • Formatting of research methods and contents description | O | O | O |
| (5) RWE management | |||
| • Monitoring the validity of the RWE analysis method | O | O | O |
| • RWE feasibility evaluation by external experts | O | O | O |
| (6) Overall Preparation for RWD/RWE Utilization | |||
| • Establishing evidence level hierarchy between RWD and between RWE research methodologies | O | - | - |
| Regarding other than RWD/RWE guidelines | |||
| (1) RWD Collection and Utilization | |||
| • Securing personal information linkage between RWD | |||
| - Expanding the possibility of using pseudonymous information | O | - | - |
| - Clarifying the content of sensitive information in personal information protection act | O | - | - |
| • Relaxation of access restrictions on data | |||
| - Expansion of data utilization targets | O | O | - |
| - Expansion of data provision size (eg. expanding the number of provided samples) | O | - | - |
| - Expansion of information to be disclosed (e.g. relaxation of restrictions on disclosure of sensitive diseases) | O | - | - |
| • Expansion of new types of RWD | |||
| - Development of new types of RWD such as patient self-evaluation | O | - | O |
| - Set indicators for use in regulatory decision-making for new RWD | O | - | - |
| (2) RWD management | |||
| • Public data network system development | |||
| - Development of criteria for RWD that can be included in the system | O | - | - |
| - Continuous quality management of RWD in the system | O | - | - |
| • Expansion and education of human resources to manage RWD in the industry. | - | O | O |
| (3) RWE generation | |||
| • Recruitment and education of human resources to create RWE in the industry. | O | O | O |
| (4) RWE management | |||
| • Expansion and training of RWE reviewers within national institutions | O | O | O |
| (5) Overall Preparation for RWD/RWE Utilization | |||
| • Unification of regulations on personal information and sensitive information (Unification of personal information and sensitive | |||
| information in the Personal Information Protection Act, Bioethics and Safety Act, Framework Act on Health and Medical Services, Medical Service Act, etc.) | O | - | - |
| • Relief of regulatory uncertainty in the industry by providing prior review by a government agencies in the RWD/RWE research planning stage | - | O | - |
| • Discovering examples of RWD/RWE use in regulatory decision-making | |||
| - Progressing pilot studies according to data users and usage goals | O | O | - |
| - Reviewing foreign cases using RWD for new drug application and conditional approval, and publishing the case books | O | O | - |
| - Providing forums to share foreign cases of utilizing RWD/RWE and resolving regulatory obstacles | - | O | - |
| • Communication and cooperation among stakeholders | |||
| - Establishing of work linkage and cooperation organization among national institutions | O | - | O |
| - Providing a forum for communication between national institutions, industry, and civic groups | O | - | - |
| • Establishing RWD/RWE related reporting and public relations guidelines | |||
| - Stating it on the drug label(instructions for use) that efficacy and safety information is based on RWD/RWE. | O | - | - |
| - Educating patients and the general public on the difference between RWD/RWE and RCT | O | - | - |
| • Laying the foundation to promote RWD/RWE research (expansion of research funds and infrastructure) | - | O | O |
O: agree, X: disagree, △: have different views, -: not mentioned
*It was also suggested that there are areas in the approval process that can replace the approval of MFDS with post quality assessment.