This guideline is developed for patient groups that wish to cooperate /collaborate in academic research. The content is easy to understand, both for organizations that are already participating in research and those that plan to do so in the future. We hope that this guideline will help in easing the resistance surrounding cooperation/collaboration in research and help you to adopt the appropriate approach toward participation.

In the 2nd edition, we have also added text for researchers. This information can be directly used by researchers to deepen their understanding of collaborators and coordinators and the patients or related parties who form the subjects of research.

The guideline was developed by members and associates of patient groups for rare and intractable diseases (NANBYO). Therefore, they are primarily designed for such patients and groups, and some sections include information that does not apply to patients and patient groups dealing with other diseases. However, on the whole, we have tried our best to offer general descriptions and content that can be understood by a broad audience.

While designing the guidelines, we conducted a number of interviews with members of research groups and members and associates of a patient group for rare and intractable diseases (NANBYO) that is already involved in research collaborations. We would like to take this opportunity to thank everyone who was involved in this process. In addition, the design team also referred to the results of a questionnaire surveys conducted during academic years 2012 and 2013 (contents can be viewed on the Project Team Introduction page).

The guideline also includes links to sites containing reference materials. We have tried to keep the main body of the guidelines as simple and concise as possible, so please refer to these links for more detailed information.

This guideline was not complete at the time of publication, and we plan to add and compile more important information in the future. We hope that your kind contributions will help to make this guideline even more beneficial in the future. We welcome your opinions. Please also note that the content of this guideline may be revised without notice. We will release an updated version if revisions are made, so please confirm this in advance.

The guidelines are available in both PDF (printable) and HTML (online) formats. The PDF version is available for download here.


You can download as a PDF version.

1-1. About the “research cooperation/coordination” outlined in these guidelines

In these guidelines, “research cooperation/collaboration by patient groups (or individual patients)” is interpreted in the broad sense to mean “activities in which patient groups (or individual patients or their family members) participate in and contribute to academic research conducted by higher education institutions such as universities in any cooperative/collaborative form.” The difference between cooperation and collaboration concerns the nature of the involvement in the research, the former denoting passive involvement wherein patient groups or patients act upon requests from researchers and the latter indicating a more proactive form of involvement. Both cooperation and collaboration play significant roles, therefore, both terms will be retained throughout the guidelines. For a more detailed description, please refer to【2】Types of Involvement.

There are various forms of research cooperation/collaboration. For example, the organization of study meetings by patient groups for the purpose of learning about the content of research can also be regarded as cooperation in the broad sense because such meetings serve to convey the content of research to society and the subjects of the research.

These activities have the following effects:

  • They promote research
  • They convey the research processes and results to society at large
  • They help to confirm whether research is heading in the right direction (thereby providing guidance)

These cooperative/collaborative initiatives not only occur at the request of patients but also at the request of researchers. This implies that it is often not possible to conduct research like those described above based on the efforts of the researcher alone. New forms of cooperation/collaboration are likely to develop in the future. In other words, the diverse nature of cooperation/collaboration indicates that researchers also expect this kind of participation (in the broad sense), and this is synonymous with the fact that patient groups and individual patients have a lot to offer in the way of research.

1-2. Academic research and research teams

The range of meaning conveyed by the term academic research is extremely broad, and in the case of rare and intractable diseases, a wide range of research is being conducted, from basic to applied and clinical research. For example, while many researchers conducting basic research are scientists and engineers, researchers in the field of medicine usually conduct clinical research.

In Japan, support for academic research is generally provided by government bodies, such as the Ministry of Education, Culture, Sports, Science, and Technology (MEXT) ; and the Ministry of Health, Labor, and Welfare (MHLW). Researchers submit application forms describing their research theme and research plan, as well as the novelty and potential for expansion of their research. After the research is approved through a screening process, they conduct research for a period of one or several years.

The number of researchers involved in a research project varies depending on the scale of the research. If multiple researchers are involved, members of the research team usually assume the following roles: (Please also note that the role titles vary slightly depending on the program subsidizing the research.)

  • Principal investigator: Assumes complete responsibility for the execution of the research plan (including the coordination of research results). The role of principal investigator is almost always assigned to a researcher at a higher education institution such as a university.
  • Joint investigator: Shares duties with the principal investigator and conducts research as a part of the team.
  • Cooperating investigator: Cooperates in conducting the research.

Some large-scale research projects have secretariat functions that generally serve as principal investigatory organizations.

1-3. Patients (patient groups) in the field of rare and intractable (incurable) diseases

In Japan, there are between 500 and 600 groups of rare and refractory diseases (MHLW Specific Diseases Control Division, 2013), which make up a total of 6500 types of individual diseases (NIH Database, 2014).

Patient groups in Japan are mainly formed for each individual disease or disease group, and those are established mainly for a certain prefecture (Regional NANBYO center), as well as the councils and networks which consolidate them. The majority of members are patients and their families and, depending on the organization, healthcare professionals may also participate.

In rare and refractory diseases, there are a lot of limitations, such as small number of patients, small number of related healthcare professionals and researchers, insufficient understanding of the disease, absence of established treatment modality and diagnosis, and extremely small markets for drug development. Therefore, starting with patient and family associations, which exist for each disease, cooperation and partnership of patient groups with research is expected to be a strong driving force in the field of academic research.

If you wish to learn more regarding the details of patient associations, particularly related to research, please refer to the reports included in the Reference section. 

1-4. Importance of partnerships between researchers and patients (groups)

Most patient groups in Japan are established with the objective of communication and information exchange between patients. Many of these groups are only made up of patients and their families. Therefore, in the past there were many individuals related to certain diseases that thought the plan of being involved in research was too difficult. However, in recent years, the importance of clarifying disease conditions, establishing treatment and diagnosis, and developing medications has been widely recognized, and there are an increasing number of patient organizations that are, or would like to, play an active role in promoting these issues.

Researchers should welcome this trend. Cooperation and coordination between researchers and patients will allow access to information that only the patient has, lead to further understanding of specific diseases, and become a driving force for the research itself.

Patient associations possess a concentration of information from multiple patients and families, integration of internal opinions as a group, and the ability to externally dispatch information. Therefore, it is extremely natural for a researcher (or a research team) to select a patient association for cooperation and coordination on a particular disease.

It is important that the coordination between researchers and patients (groups) involves sharing knowledge in order to head in the same direction and encourage mutual respect. If these are lacking, then such partnerships will not effectively function. The researcher(s) must take on the responsibility of providing adequate explanation to patients, who are not healthcare professionals, and making sure that they understand the information given. In addition, on the patients’ side, instead of extreme attitudes of being allowed to cooperate or a perception that they are doing a favor, it is important to cooperate willingly with an understanding of the purpose of the research.

References

Japan Patients Association
(The sole council in the field of incurable diseases made up of 41 incurable disease federations and 44 individual disease associations (as of March 2015)) 
http://www.nanbyo.jp/
Nanbyo Kodomo Shien Zenkoku Network
(“Nation-wide Support Network for Children with Incurable Diseases”) Authorized Nonprofit Organization (Supports patient association activities and includes 50 member groups (as of March 2015)) 
http://www.nanbyonet.or.jp/

Reference materials (in Japanese)

Types of research:
http://www.mext.go.jp/a_menu/kagaku/kihon/06032816/001/001/006.htm
Information about the different categories of researchers (Grants-in-Aid for Scientific Research Handbook)
http://www.jsps.go.jp/j-grantsinaid/15_hand/data/kakenHB1106A5.pdf

*Please also note that it is generally not possible to apply for government research grants, unless the principal and joint investigators meet the application requirements. It is necessary to confirm the application requirements in advance.

Research for constructing a framework to support NANBYO research, and patient support groups (JPA)
http://www.nanbyo.jp/kenkyu/index.html
Investigation of this current state and conditions of research cooperation between patient associations and reserach teams
http://www.nanbyo.jp/kenkyu/hokoku/h1.html

There are various types of research cooperation/collaboration. The main types are outlined in the table below.

Type of involvement by patients or patient groups Outline Advantages for the patient (or group) Advantages for the researcher (or research team) or doctor
Responding to questionnaires or factual investigations

・ Responding to surveys on aspects of daily life based on a number of diseases and residential area-specific categories

・Factual investigations about patients’ lives

・ Responding to various surveys, including questionnaires about treatment, etc

・By participating in surveys, such as those that examine the lifestyles and community involvement of patients suffering from diseases, patients can directly reflect on their own situations in their responses

・ It is possible to give opinions about things that are normally difficult to talk about

・It is possible to clarify aspects that are normally difficult to talk about and offer suggestions        

・ It is possible to ascertain how patients think and feel

Collection of epidemiological information

・Provision of basic information for use in epidemiological research

・Providing information can help to verify the causes and prevention methods of diseases and the effectiveness of treatments

・It is possible to probe the causes of diseases and verify the effectiveness of prevention and treatment methods

・ It is possible to clarify connections between environment, lifestyle, and health

Provision of biological samples

・ Providing bodily samples, such as blood, urine, and tissue

・By providing samples such as blood and urine, patients can contribute to investigation of the causes of illnesses and benefit from the resulting treatments

・ It is possible to cooperate in an additional step during a regular medical examination

・It is possible to receive specimens from the patient himself/herself, and by analyzing them, investigate the cause of the disease and conduct research aimed at establishing treatments

Provision of data for clinical trials and research

・ Cooperating in or providing personal information for human experiments that examine the effectiveness and safety of new medicines

・By providing personal information, it is possible to contribute to the development of more effective medicines that are also very safe

・It is possible to provide new medicines to the general public by conducting the compulsory experiments required to obtain government approval

・ By collaborating with patient groups, it is possible to recruit more participants for an experiment

Direct participation in research teams (as a practitioner or contributory, etc.)

・Becoming a member of a research team and conducting research, creating research presentations, and reports

・Patients can apply their expertise to part of the research project as a member of the research team

・ It is possible to approach the research (or part of it) from the patient’s perspective, etc.

・ It is possible to intensify interactions with researchers on a regular basis 

・By asking patients to contribute from a position of greater responsibility, it is possible to conduct research from the patient’s perspective

・ It is possible to design questionnaire items and obtain analysis results that can only be achieved through collaboration with patients

Attendance and presentations at research team meetings

・Attend or give presentations at research team meetings

・By directly listening to research presentations and question-and-answer sessions at research team meetings, it is possible to confirm the progress of research

・ It is possible to ask questions to researchers directly

・ By giving presentations, it is possible to provide ideas to researchers directly from the patient’s perspective

・It is possible to directly convey research content to patients, who are also the participants of the research

・ By exchanging opinions on the spot, it is possible to avoid unnecessary misunderstandings and lack of understanding

・ It is possible to discover new research ideas by interacting directly with patients

Glossary

Epidemiological research
Epidemiological research is a form of scientific research that aims to investigate the frequency and distribution of phenomena such as diseases and identify their causes. Epidemiological research is essential for investigating the causes of diseases, verifying methods of prevention and treatment, and identifying connections between environment and lifestyle habits and health. It plays a significant role in developing medicine and preserving and enhancing the health of citizens. Epidemiological research addresses specific information about the physical conditions, surrounding environment, and lifestyles of a large number of research participants. It is also characterized by the involvement of a large number of stakeholders, in addition to doctors.
Clinical research (clinical trials)
Clinical research is a form of medical research wherein researchers enlist the help of patients to decipher the causes of diseases, enhance prevention, diagnosis and treatment, and improve the quality of patients’ lives. This type of research, which aims to collect objective data so that new medicines can receive government approval and be used as regular treatments, is also referred to as “clinical trials.”

Reference materials (in Japanese)

Ethical Guidelines for Epidemiological Research
http://www.mhlw.go.jp/general/seido/kousei/i-kenkyu/ekigaku/0504sisin.html

3-1. Patient Action

  • Get acquainted with the research and researchers
    • Investigate the researcher or research team conducting research on your disease. For example, it is possible to search by the disease name using the “internal search” function on the Japan Intractable Diseases Information Center website or search using a list of research teams. It is also a good idea to ask your attending physician.
    • Once you know the name of the researcher or research team, try contacting them. Sending pamphlets or bulletins of your patient group to researchers or informing them about your group’s activities through medium such as the group’s website is also effective. It is also possible to obtain official responses by contacting researchers as a representative of the patient group.
  • Investigate the details of the research cooperation/collaboration
    • Listen to the requests of researchers and consider how you can cooperate/collaborate in the research. If possible, you can better infer about the researchers’ actual intentions by meeting them in person and asking questions.
    • First, ask them to explain the purpose and specific content of their research so that patients and their families can understand. It is also acceptable to ask questions if you do not understand. “Pretending” to understand can affect the subsequent relationship with the researchers. Ask them to explain the research until you completely comprehend the research objectives. 
    • On the other hand, the researchers may not have sufficient knowledge of your patient group. Therefore, it is a good idea to inform them about your activities, the names of the diseases that you are involved in, and the number of members in the group. Similarly, attempt to explain the patients’ situation to the researchers in an easily comprehensible manner.
    • After considering what you wish to obtain from the research and your current situation, discuss the specific ways in which you will be able to cooperate/collaborate. Meanwhile, carefully consider what kind of responsibilities and burdens patients are likely to face.
  • Discuss these aspects within the group and form a conclusion
    • Consider how you can contribute to the research after confirming what kind of cooperation/collaboration is actually required as well as what you wish to obtain from the research. If possible, it is also effective to invite the researchers to your board meeting and have them explain the content of the research.
    • Investigate the advantages of research cooperation/collaboration. For example, consider how the research will contribute to emerging treatments or in improving the daily lives’ of patients. Not all research yields immediate results, so consider what kind of results the current projects will yield in the future from a long-term rather than short-term perspective.
    • You must also carefully examine the disadvantages. Consider specific aspects, such as the human resources, economic impact, and time burden that cooperating will impose on the group and whether your current system can absorb this burden. You should also consider the nature of the burden placed on patients’ cooperating in the research.
    • After carefully considering the advantages and disadvantages, decide whether and how to cooperate/collaborate in the research, and convey this decision to the researchers.

3-2. Researcher Action

  • Connect with patients and patient groups
    • Look for a patient group for the particular disease on which you are working. Most patient groups have a website, which can be easily found by way of a search engine. WINPR website ( see the Reference) can be useful for obtaining information about patient associations overseas.
    • E-mail is the most common way to contact patient groups; however, there will be times when only phones and fax machines are available. There are also circumstances in which replies might be delayed; hence, it is very important to state clearly the researcher’s name and/or team in the subject line of the e-mail.
  • Propose details for cooperation/coordination with research
    • After clearly explaining the details of the researcher (team), simply explain the research proposal. Even if the research is in its early stages, it may be advantageous to hear opinions from the patient’s point of view. While remaining aware that patients are not professional researchers and will better understand information though a presentation rather than being offered printed material, supporting materials will also play a role for consideration of the research, within the patient association.
    • There are many cases where there is anxiety on the patient’s behalf regarding being contacted by researchers; we recommend face-to-face meetings. In this way, one can ask in a straightforward manner regarding the patient association's activities and understand its direction and current status, leading to a deeper mutual understanding.
    • Do not have the attitude that you are doing the patients a favor by researching their disease and avoid creating the atmosphere of a hierarchical relationship.
    • Have a discussion regarding how there can be specific cooperation and coordination on the patient side and clearly explain what responsibilities and burdens will be put on the patient.
    • It may take some time for the patient association to consider the proposal and there may be follow-up questions at a later date; in this case, be sure to clearly respond.

Reference materials

Japan Intractable Diseases Information Center  (in Japanese)
http://www.nanbyou.or.jp/
Japan Patients Assotiation(JPA)(in Japanese)
http://www.nanbyo.jp
NPO ASrid (in English)
http://asrid.org/en

Once a decision to cooperate / collaborate has been made, patient groups should proceed on the basis of the basic process outlined below.

  • Discuss details with the researchers in advance
    • Carefully discuss in advance what you will be required to do and when and how you will do this with the researchers. Confirm the schedule for meetings that is due to be held during the research period (briefings, debriefings, etc.), the survey period (if a survey is necessary), and the period for sending research reports. It is possible for both parties to reduce work and expenditure by organizing meetings to coincide with the publication of the group bulletin, general meeting, or exchange meeting, etc.
    • Prepare materials with research details, preferably ahead of time, so that appropriate explanations can easily be given.
    • Discuss in advance who will respond to inquiries, how to respond to questions, and who will be responsible for this.
    • It is also important to consider costs. For example, confirm whether the researchers or patients will bear the necessary costs, such as the cost of posting and returning questionnaires, transportation expenses for meetings and interviews, staff, and the cost of renting meeting spaces. Also confirm the method of payment.
  • Confirm how information will be dealt with
    • Reach an agreement about how to deal with members’ personal information (names, names of diseases, addresses, etc.). Discuss aspects such as whether a register of names will be submitted to the researchers or whether the patient group will manage the register and request cooperation from the research subjects.
    • In addition to any oral agreements, establish a written agreement governing the treatment of information obtained in surveys.
    • Confidentiality issues not only apply to the researchers collecting patient information but also to the patients cooperating / collaborating in the research. Clarify the content and scope of confidentiality in advance and act responsibly when cooperating/collaborating in the research.
  • Explain the research to the participants in the patient group
    • When explaining the research content and requesting the cooperation of group members, it is important to convey information accurately. It is preferable to have the researchers provide a one to two page summary of the research, which can then be used to explain the research to group members and disseminated with the group bulletin or survey sheet.
    • When restricting the scope of research subjects or target of member, also include the reason for doing so in the explanation
    • Consider how to explain the research according to the circumstances of your group. (For example, consider whether to organize a face-to-face briefing, use the patient group mailing list, or explain the research at a group meeting, etc.)
    • Prepare ahead of time so that you can explain, respectfully and smoothly, in cooperation with the researcher (team) in a manner that is appropriate for every situation.
  • Proceed to cooperate/collaborate in the research
    • Proceed to participate in the research in accordance with the pre-arranged schedule. Where necessary, create opportunities for exchanging opinions with the researchers, and try to respond quickly to the doubts, requests, and grievances of group members.
  • Report the results of the research
    • There are various ways to report the research results, including research presentations, journal submissions, presentations at research meetings, and preparation of reports. Decide on how to report the results of the research by discussing this with the researchers.
    • When reporting results via the patient group website, ask the researchers to confirm the content of the report in advance. Please also refer to 【6】Important Points to Consider.

Depending on the content of the research, results may not always be achieved in the short-term. Naturally, patients would prefer to see immediate results; however, better results may be achieved through ongoing, long-term cooperation/collaboration. In this section, we consider the important points to bear in mind when participating in ongoing research cooperation/collaboration.

  • The relationship with the research team: try to build a relationship of mutual trust
    • To facilitate successful long-term cooperation/collaboration, it is not only necessary to help researchers with their research but also to have the researchers understand the activities of the patient group. Therefore, it is also important to invite researchers to general meetings, organize discussions and study groups, and honestly convey your thoughts to each other.
    • Of course, if a patient group enables the general public to better understand the disease, it in turn makes it easier to obtain its support. By actively providing a wide range of information not only to researchers but also to the general public, it is possible to strengthen the relationship with the researchers more efficiently.
    • It is important to have the researchers hold regular meetings to report research results to the patient group. If all decisions are left to the researcher and patients do not, even to some degree, understand how the research is progressing, it is easy to lose sight of the purpose of long-term cooperation/collaboration.
    • It is not possible to build a relationship of mutual trust with researchers simply by strengthening your interpersonal relationships. In research, there are certain social rules. Examples include rules relating to the treatment of research results, accountability, and conflicts of interest. These rules are also introduced in this guideline, so we encourage you to confirm them in advance.
  • Carefully examine the research together with other officials of the patient group
    • It is possible that, in the early stage of cooperation/collaboration, the patient group decides to “give it a try” based on the opinion of the group officials without understanding the specific details of the research. However, as your involvement continues, it becomes necessary to obtain agreement and confirmation from group officials about the necessity for research cooperation/collaboration.
    • So what criteria can be used to determine whether or not to continue cooperating/collaborating in research? Examples include the benefits of the research for group members, the future prospects of the research, return of research results to individual patients, and non-research-related incentives.
    • However, it is difficult to accurately address the benefits and future prospects of research only within the patient groups. In some cases, it is necessary to request explanations from the researchers. Also from the perspective of deepening interpersonal relationships with the researchers, it is necessary to consider asking the researchers to give presentations at the officers’ board meeting or group meeting.
    • As the research cooperation/collaboration progresses, you may also face requests from a large number of researchers. If you are unable to cooperate in all of the studies, you will need to establish selection criteria. However, if it is difficult to individually make these decisions, you may need to consult an advisor or other external specialist.
  • Appeal to members and provide explanation
    • It is necessary to enlist the help of individual group members when cooperating/collaborating in research, for example, to respond to questionnaires and cooperate with factual investigations and clinical trials. There may be a case where some members may not approve of having their own data used for research purposes. Further, to protect personal information, it is also essential to gain the approval of group members with respect to ongoing research cooperation /collaboration.
    • For example, clinical trials play an essential role in the development of medicines. Medical progress cannot be achieved without the cooperation of patients.
    • However, it is certain that patients, while recognizing the necessity of research, do not see their own involvement in research as an immediate concern. Therefore, conveying the significance of research to group members is another job for patient groups.
    • Patient groups can appeal to members through the group bulletin or by mail; however, the ability to select participants and respond quickly also plays an important part in building trust with the researchers. Therefore, you may need to organize information in a database. While facilitating research, the use of a database will also benefit the patient group.
  • Strengthen the foundations of the patient group
    • How the group can contribute to the development of medicine is an extremely important question in terms of the significance of the group’s existence. However, cooperating/collaborating in research are not the only roles of the patient group. If the group fails to consider the human resources, time, and economic burdens of participating in research, ongoing long-term cooperation becomes impossible.
    • For example, even if your group receives questionnaire requests, in reality, it is not possible to deal with these without investing time and effort. In the case of ongoing projects, in particular, it is also necessary to confirm the details of financial assistance with the researchers. While it is necessary to strengthen the foundations of the patient group, it is not possible to bear all the costs in ongoing research collaboration.
    • If the patient group strengthens its foundations, it may be able to take the lead in research projects, such as conducting collaborative research with specialists by using funds obtained for the purpose of meeting the specific needs of the research. For example, conducting surveys of the living conditions of patients in which researchers are also involved can produce more accurate, extensive results.
  • Learn about success stories in your own country and overseas
    • Patient groups outside Japan have witnessed an increase in activities wherein doctors and patients cooperate to create new treatments and diagnostic criteria. Sharing examples of successful research cooperation/collaboration internationally will lead to better results.
    • It is also useful to exchange opinions with other patient groups to overcome problems and issues related to research cooperation/collaboration. The work of other patient groups can provide great insight when considering the specific aspects of research such as questionnaire items. Expanding collaboration among patient groups is also important to multiply the number of success stories.

Rare and intractable diseases are a kind of “illness.” So long as there are researchers working toward a breakthrough, let us recognize those making effort to “cure” and consider together what we can do to “be cured”.

  • Treatment of results
    • While researchers are required to disclose the results of their research to patient groups, it is also important to understand that, in the context of research, there are certain situations in which patient groups must also respect confidentiality. In some cases, the researchers may not be able to disclose everything to the patient group. Clear communication is essential to maximize the potential of the research results while maintaining a relationship of mutual trust.
    • In particular, when publicizing research results, it is important for the patient group to carefully consider what information should and should not be disclosed.
      • The following points should be considered regarding personal information: Examples include what information to provide, who will see this information, what it will be used for (during and after the research period), and will it be possible to request deletion of this information, if necessary. Depending on the nature of cooperation/collaboration, it may be important to discuss these points with the researchers and reach a conclusion before or during the research period.
      • The following points are to be considered while handling information provided by the researchers: Examples include the extent to which you can include the content of discussions at research team meetings and personal opinions (the difference between fact and impression) in patient group reports. Unlike minutes of committee meetings, research content is generally not revealed before its publication in conference presentations and academic papers. In particular, if patient groups publicize the content of privately held meetings at their own discretion, this may later give rise to an extremely difficult situation. Moreover, even if the expected results are achieved, conveying them in a subjective manner is likely to inconvenience the researchers. Try confirming with the researchers which methods of publicizing and introducing research are appropriate. An innocuous way to introduce the research results is to post a link to the content on the researchers’ website or have the researchers create informative materials that you can introduce to patients.
  • The important points to consider are listed below. Try to talk through these points with the researchers in advance

    • Establish specific rules about the content of agreements, including rules governing the treatment of research results
    • Patient groups should also follow these rules when dealing with research results
    • Confirm the ownership and confidentiality status of research results in advance
    • Group members must behave appropriately based on the awareness that the patient group is also participating in the research
    • Be aware of whether you are participating in the research as a patient group or as individuals
      • When participating as a patient group, your group will be cooperating/ collaborating as an organization, so it is only natural that your organization will bear certain responsibilities. You will also need to explain the organizational direction when selecting participants, so consider this in advance. In addition, when giving comments, be fully aware that your words represent the organization. Even after determining which member of the patient group will be responsible, do not leave everything to this person. Instead, pay attention as an organization to the details of the cooperation and the content of comments and statements.
      • When participating as individuals, there is the possibility that an individual member will be solely responsible, so please exercise caution in this respect. If there is anything that you do not understand, you should ask the researchers about it in advance. If you are going to be involved as an individual although the patient group already exists, consider whether it might be more appropriate to participate as an organization. Confirm matters with the group where necessary.
    • Does the patient him/herself give consent, or does his/her family do so?
      • If the patient is a child or is unable to make his/her own decisions, the family of the patient may wish to cooperate in the research on behalf of the patient. Try to talk to the patient, keeping in mind that their own opinion may differ from that of their family.
    • Completely understand that research is time-consuming and is not always successful
      • New drugs can only be released if the researchers obtain government approval after confirming the effectiveness and safety of the new ingredients through long-term research and development. The period since commencing the research to the release of a drug is said to be 10–15 years, a considerably long period of time.
      • Specialists have observed that out of 100 possible ingredients tested in clinical trials involving humans, only 8 ingredients will be used as a “drug.” Moreover, since a large number of pre-trials ( in vivo experiments on animals and in vitro test tube experiments) must be conducted before the main trial begins, the probability that a certain ingredient will be selected for a drug is as low as 1/3000. Although pharmaceutical development is conducted with the backing of a variety of data, in reality, only 8% of projects succeed.

    (This section will be added later)

This guideline was created over a two-year period by a project group comprising patients and associates who had sensed their importance and necessity while investigating and analyzing patient groups.

 This guideline is not complete. Some parts may also be incorrect. However, by going through this guideline, results can be improved considerably by simply understanding in advance the difficulties that you may face when contending with research groups in pursuit of your group’s mission. Rather than getting involved without knowing anything, you may achieve smoother communication and greater understanding with the researchers by simply indicating what should be considered beforehand, albeit just concepts. As a result, mutual feeling of trust will improve considerably and the possibility of achieving effective and positive cooperation/collaboration will also increase.

 We will continue to revise and supplement these guidelines in the future. We would also like to provide explanations that are easier to understand— for example, by including diagrams. We intend to create a set of guidelines based on the opinions of the readers, so we hope that readers will help us by contributing in the future.

These guidelines were designed as part of the research projects outlined below:

  • First Edition:“Constructing a Framework to Promote the Active Support of Patient Groups in Research on Intractable Diseases,” ( Kanja shien dantai tō ga shutai teki ni NANBYO kenkyū shien o jisshi suru tame no taisei kōchiku ni muketa kenkyū;) MHLW Research Grant, 2012-2013
  • Second Edition:“Support Systems for Patients with Intractable Diseases,” ( NANBYO kanja e no shien taisei ni kansuru kenkyū) MHLW Research Grant, 2014

Guideline Design Team (in alphabetical order)

  • Yukiko MORI
    General incorporated association Japan Patients’ Association, general incorporated association Japan Collagen Disease Patients’ Group
  • Shiori NAGAMORI
    General incorporated association Japan Patients’ Association, specified nonprofit corporation Hokkaido Network for Patients with Intractable Diseases
  • Yukiko NISHIMURA *Principal organizer
    General incorporated association Japan Patients’ Association, specified nonprofit corporation ASrid (Advocacy Service for Rare and Intractable Diseases’ Stakeholders in Japan), University of Tokyo
  • Hiroshi OGURO
    General incorporated association Japan Patients’ Association, general incorporated association Japan Collagen Disease Patients’ Group
  • Special thanks to Masatoshi Iwasaki (ASrid) an Crimson Interactive Pvt. Ltd.

Contact Email Address: contact@asrid.org