Topics Toward the Utilization of Health Care Data and Real World Data The Next Generation Medical Infrastructure Act and the efforts of the Pharmaceutical Manufacturers Association of Japan (PMAJ)
Data related to health and medical care that occurs in daily life is also called Real World Data (RWD) in contrast to clinical trial data in the pharmaceutical industry RWD is utilized in many stages of the pharmaceutical industry, including drug discovery, development, post-marketing safety, and clinical research. For example, RWD is expected to facilitate efficient research and development. In addition, RWD is used to generate evidence (scientific basis) for the safety and efficacy of drugs that match the various characteristics of each individual, thereby contributing to more appropriate use of drugs. The utilization of health and medical data and the development of an environment for this purpose are important issues not only for the pharmaceutical industry but also for the national government, and one of the representative efforts of the national government is the revision of the Next Generation Medical Infrastructure Act. The Pharmaceutical Manufacturers Association of Japan (PMAJ) is also involved in efforts to improve public understanding of the utilization of health and medical data. This paper introduces these two efforts.
Revision of the Next Generation Medical Infrastructure Act
Establishment of "Pseudonymized Medical Information" to expand the scope of utilization
The official name of the Next Generation Medical Infrastructure Act is "Act on Anonymously Processed Medical Information for the Purpose of Contributing to Research and Development in the Medical Field. The law was enacted in May 2018 to facilitate the utilization of medical information by pharmaceutical companies and others, with the aim of realizing a society of health and longevity through the promotion of research and development in the medical field. This law will be amended at the Ordinary Diet session in May 2023 and will come into effect within a year. Amendments have been made to meet the needs of pharmaceutical companies, including a new definition of "pseudonymized processed medical information" and the ability to provide data to the Pharmaceuticals and Medical Devices Agency (PMDA), etc., for the purpose of pharmaceutical applications.
Issues before the amendment of the law
Under this law, it is now possible for a business entity authorized by the government to provide medical information that has been anonymously processed so that the patient cannot be identified (anonymously processed medical information) to pharmaceutical companies, universities, etc. However, this process of anonymizing the information makes it impossible to identify the patient. However, this process of anonymizing medical information has resulted in the following problems, and the value of the data has been greatly impaired by categorizing the data, shifting numerical values, and deleting data with small numbers so that it cannot be used to identify individuals.
| The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations. | We have to delete a small number of cases, and cannot obtain useful data for medical research. |
| The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations. | When the authenticity of individual sample data needs to be verified, it is not possible to go back to the original data, such as medical records. |
| The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations. | Once extracted data is no longer linked to patients, it is not possible to track changes in patients over time by adding subsequently obtained data. |
| The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations. | When further research is desired on individual case data, it is not possible to obtain additional data from other sources such as medical records. |
In addition, since the provision of anonymized processed medical information to third parties is prohibited, for example, pharmaceutical companies cannot submit data to PMDA or other pharmaceutical regulatory authorities for pharmaceutical use.
Against this backdrop, although five years have passed since the law went into effect, the number of cases of use has remained at around 20 or so.
Discussions on the Amendment of the Law
Upon the revision of this law, the Cabinet Office established the "Working Group to Study the Next Generation Medical Infrastructure Act" in December 2021. As a member of the group, Mitsuhiro Kondo, Chairman of the Pharmaceutical Evaluation Committee of the Pharmaceutical Manufacturers Association of Japan (title at that time, current Vice Chairman), participated in the discussion and made the following proposals.
| The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations. | Establishment of the concept of pseudonymized medical information (names and contact information are only deleted, but medical data are not processed). |
| The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations. | Introduction of a system to ensure the reliability of data required by domestic and foreign pharmaceutical regulations. |
| The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations. | Allow provision of pseudonymized medical information to third parties as required for pharmaceutical purposes in Japan and abroad. |
| The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations. | Linkage with public databases such as Receipt Information, Database of Information on Specified Health Checkups, etc. (NationalDatabase, NDB), and Death Certificates |
These proposals were endorsed by the other members and fully reflected in the revised law.
Establishment of "Pseudonymous Processed Medical Information
The revised law newly defines "Pseudonymous Processed Medical Information" ( Figure 1 ). Pseudonymized processed medical information requires the deletion of information that can identify a specific individual by itself, such as a name, but unlike anonymized processed medical information, it does not require the deletion or alteration of even specific test values or disease names. Therefore, the reliability of data is improved and more precise research can be conducted. Also, since unique data will not be deleted or altered, research on rare diseases and rare side effects will be possible, greatly expanding the scope of utilization.
Figure 1 Image of Pseudonymous Processed Medical Information
Source: Prime Minister's Office website
https://www.kantei.go.jp/jp/singi/kenkouiryou/data_rikatsuyou/jisedai_iryokiban_wg/dai8/siryou1.pdf
Mechanism for utilization of processed medical information under pseudonyms
Unlike anonymized processed medical information, the use of pseudonymized processed medical information requires prior government approval ( Figure 2 ). Certain safety management measures are expected to be required, but the criteria are currently under consideration and will be stipulated in guidelines to be issued in the future.
In addition, it will be possible to submit pseudonymized processed medical information to PMDA and other regulatory authorities for regulatory approval applications. We have requested to be able to submit this information to overseas regulatory authorities as well, but the government is currently considering which countries will be allowed to do so.
Fig. 2 Mechanism for utilization of processed pseudonymous medical information
Source: Prime Minister's Office website
https://www.kantei.go.jp/jp/singi/kenkouiryou/data_rikatsuyou/jisedai_iryokiban_wg/dai8/siryou1.pdf
Linkage with public databases such as NDB
In addition, it will be possible to link with public databases such as NDB ( Figure 3 ). Although linkage will be limited to anonymized processed medical information and will focus on receipt data, this will improve data traceability. In particular, since the NDB plans to collect mortality information in the future, it will be possible to conduct research that traces data up to the time of death, which has been difficult to do in the past.
Fig. 3 Linkage with public databases such as NDB
Source: Prime Minister's Office website
https://www.kantei.go.jp/jp/singi/kenkouiryou/data_rikatsuyou/jisedai_iryokiban_wg/dai8/siryou1.pdf
Closing Remarks
The Next Generation Medical Infrastructure Act has been revised to reflect the requests of the Pharmaceutical Manufacturers Association, and will greatly expand the scope of utilization. The ultimate goal of this law is to create a healthy society with longevity. It is expected that pharmaceutical companies will safely and effectively utilize medical information within the framework of this law and contribute to medical care by creating many medicines and evidence.
Publication of "Health Medical Data and Our Lives
Educational activities by the Pharmaceutical Manufacturers Association to foster public understanding
Background and purpose of creation
So far, we have introduced the growing importance of the utilization of health and medical data and the revision of the Next Generation Medical Infrastructure Act, one of the country's representative efforts to address this issue. On the other hand, a survey by the Ministry of Internal Affairs and Communications reported that in Japan, a higher percentage of people are concerned about "not knowing" the details of data utilization, such as who will use the data and for what purpose, than in other countries. This indicates that the utilization of health and medical data is not familiar to the public, and that understanding of what can be achieved through further utilization is not sufficiently widespread.
Based on the above background, the Pharmaceutical Manufacturers Association of Japan (PMAJ) has published "Health Medical Data and Our Lives" (hereinafter referred to as "this booklet") as an educational booklet for the public, with the aim of promoting more progressive utilization of health medical data throughout society ( Figure 4 ). The booklet was created using plain expressions and illustrations in order to interest and inform as many people as possible about what health and medical data are and how they are used. In addition to the booklet, it is also available as an eBook on the Pharmaceutical Manufacturers Association of Japan (PMAJ) website.
Fig. 4 "Health Medical Data and Our Lives" QR code for the eBook version on the right
Message to be conveyed through "Health and Medical Data and Our Lives
Through this booklet, we hope to convey the message that the utilization of health and medical data will lead to the improvement of health and the realization of high-quality medical care for as many people as possible. We then explain how health and medical data is being used "now" and how we aim to use it "in the future. We especially want to emphasize that "your healthcare data can contribute to the health not only of yourself but also of many others".
For example, the utilization of health and medical data will enable you to obtain information useful for maintaining your health and to use new medicines and treatments more quickly. For medical institutions, it will enable them to identify areas for which effective treatments have not yet been established, to provide medicines and treatments tailored to individual patients, and to provide prompt and accurate medical care in the event of an emergency or disaster. Furthermore, for pharmaceutical companies, it will enable them to speed up the provision of groundbreaking new drugs through more efficient and sophisticated clinical trials, to build evidence, and to efficiently identify health risks and implement countermeasures. For governments, it can contribute to the formulation of healthcare policies to address health problems and to the determination of the appropriateness of healthcare resource allocation through cost-effectiveness analysis.
Through such a wide-ranging virtuous circle, we believe that we can not only provide better medical care, but also promote the health of the people, extend life expectancy and healthy life expectancy, stimulate innovation, and realize a wellbeing society ( Fig. 5 ).
Fig. 5 Excerpt from "Health Medical Data and Our Lives"
Future awareness-raising activities
We plan to continue educational activities to eliminate public concerns about "ignorance" of the significance of the utilization of health and medical data and to improve understanding of the utilization of health and medical data. We aim to realize a social environment that can continue to provide diverse values to the public through the utilization of health and medical data.
Currently, we are working with related stakeholders to organize a "glossary" of terms related to health and medical data so that people who are not familiar with medical care can become familiar with them. We ask for your understanding of the activities of the Pharmaceutical Manufacturers Association described in this article and your understanding and cooperation in the utilization of health and medical data.
( Seigo Akari, Ryosuke Annaka, Kanae Togo, Task Force for Promotion of Utilization of Medical Information Database, Pharmaceutical Evaluation Committee)