Patient Organization Collaboration Promotion Committee The 28th and 29th Patient Group Seminars

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Activities of Overseas Patients' Associations

History of Intractable Disease Patient Associations and Councils in the U.S.

In January 1983, the Orphan Drug Act was passed in the United States to support the development of products for the treatment, prevention and diagnosis of rare diseases. Drug Act was passed to support the development of orphan drugs. NORD also provides policy support and recommendations for legislation related to research and development and healthcare funding, and has a much broader target audience than Japanese patient associations. NORD also provides policy support and advocacy for research and development as well as legislation related to health care funding, thus targeting a much broader area than Japanese patient associations. This is not a matter of which is better or worse, but rather a matter of the fact that NORD's starting point is "to create drugs to cure diseases," which is different from that of Japanese patient associations, and therefore the awareness of the people involved toward patient associations and councils is also different between Japan and the United States.
The situation is basically the same in Japan and the U.S., where there are few (no) people involved, few (no) information, and few (no) treatments, diagnoses, and drugs.

Importance of Council Activities and Significance of International Collaboration

While the activities of individual patient associations are of course important, in light of the recent severe economic situation, I believe that it will be important to work not only with individual patient associations but also, in some cases, in cooperation and coordination with other organizations and parties concerned, with the aim of disseminating information to society. In fact, in the past few years, several councils have emerged around the world.
To cite an example of collaboration among patient councils, Rare Disease International (RDI) is an organization of patient associations and councils in 30 to 60 countries, which makes policy recommendations to the World Health Organization (WHO) and the United Nations. In 2013, JPA signed a Memorandum of Understanding with NORD and the European Organization for Rare Diseases (EURORDIS) to ensure that JPA has access to information from around the world. Currently, JPA serves as the Japanese contact point and ASrid as the support organization.
Many Japanese patient associations may think that Japan has nothing to offer, but there are many things to promote to the world, such as the concept of "NANBYO" (intractable disease), domestic researchers and their research results, and the universal health insurance system.
However, since it is difficult for all patient associations to implement international collaboration, we believe it is important for the councils (intermediary organizations) to play a part in this role.

Similarities and differences between domestic and overseas patient associations/councils

Comparing domestic councils and overseas patient associations/councils, there are no major differences in their basic activities. However, the major difference between Japan and other countries lies in the collaboration with external stakeholders and "non-patient-related" parties. Specifically, overseas patient groups and councils are composed of professionals from the medical, business, and IT fields, in addition to patient-related personnel. In particular, IT technicians are regarded as indispensable for the operation of the councils. Other examples of overseas patient groups and councils include those that organize their own events to raise funds and support clinical research using the funds, as well as those where the council sponsors academic conferences. Of course, the backgrounds of Japan and other countries, including laws and regulations, are different, but more than that, I believe that the major difference between Japan and other countries is the awareness from patient associations that "patient associations are only possible with the understanding of external stakeholders" and the awareness from external stakeholders that "patient associations are necessary. In this regard, it can be said that patient associations and companies in Japan are truly in a transitional stage.

Directions and suggestions for future activities of domestic patient associations

Japan is rich in examples of experiences of "confronting" illness, which I believe is a great strength. In addition to "facing" the disease, the goal from now on is to "cure" the disease. Although the enactment of the Intractable Disease Control Law has come to an end, I believe that the existence of patient associations will be important as the law is put into operation. From there, it will be important to gradually develop activities from "only" individual diseases to "beyond" individual diseases, and from "only" patients to "mainly" patients. There are various problems and shortcomings surrounding intractable and rare diseases, which cannot be solved by individual people or organizations alone. Therefore, as a mechanism for connecting, we are conducting various activities such as holding Rare Disease Day, building a patient-centered database "J-RARE," establishing guidelines for research cooperation and collaboration for patients, and creating a global index of patient associations.

Closing Remarks

I believe that activities in the area of rare and intractable diseases are daunting, with no clear goal in sight. However, it is definitely more important than you may think, and there are many people who need it. Therefore, we would like to continue to discuss with you how to "continue," "nurture," "find," and "communicate" our activities.

Questions and Answers

During the Q&A session, many questions were raised at both the Tokyo and Osaka venues.
In response to questions about the specific activities of overseas patient associations, examples of clinical trial support and a charity marathon project in the name of a patient were introduced.
In addition, there were many questions about the differences between Japan and other countries, particularly with regard to the activities of patient associations and councils. I believe that even a discussion among those gathered today will generate some new ideas. In addition, regarding the direction of domestic patient associations, he said, "I think that the emotional satisfaction, sense of security, and solidarity gained by facing illnesses are extremely important. Furthermore, I think it is important to think about what to do in order to cure the disease.
Following Dr. Nishimura's lecture, Naoko Kajiwara, Vice Chairperson of the Pharmaceutical Manufacturers Association of Japan (PMAJ), gave an explanation of the relevant laws and regulations on advertising at the Tokyo meeting place and Naoyo Uesugi, Vice Chairperson of the PMAJ at the Osaka meeting place. She explained that pharmaceutical companies work with patients and patient groups under various laws and advertising regulations, and that the Pharmaceutical Manufacturers Association of Japan (PMAJ) has issued Guidelines for Collaboration (*1) for better collaboration with patient groups and Transparency Guidelines (*2) to enhance transparency in mutual relations. The guidelines are posted on the website of the Pharmaceutical Manufacturers Association of Japan (PMAJ).
Finally, Mr. Tokuo Tanaka, Executive Director of the Pharmaceutical Manufacturers Association of Japan (PMAJ), introduced the results of the "Survey on Sei-katsu-sha Attitudes toward Drugs and the Pharmaceutical Industry" conducted by the PMAJ in FY2015, showing that more than 85% of respondents had positive opinions in the "Impression and Evaluation of the Pharmaceutical Industry" item. The seminar was closed on a high note with a promise to continue to strive for even greater transparency and trustworthiness in the industry without being satisfied with the results.

Hiroki Shiba, Patient Organization Collaboration Promotion Committee

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