The 40th Patient Group Seminar (held online) Thinking Together and Taking the First Step: Patient and Public Involvement in Healthcare (PPI)

Seminar Date : November 30, 2022 (Wednesday)

Video distribution period : April 06, 2023 (Thu) - May 31, 2024 (Fri)

Opening remarks

In recent years, various institutions and organizations have taken up PPI (Patient and Public Involvement: Patient and Public Participation in Healthcare). In this seminar, we hope to make PPI more accessible to patients and encourage them to take the first step toward becoming actively involved in their own treatment. What is PPI, why is it necessary, and what should patients and patient groups do?

Lecture

Subject 1: The Latest Trend of PPI: Toward Further Promotion of PPI
Ms. Akiko Yoshida, Senior Researcher, Pharmaceutical and Industrial Policy Research Institute

I am aware that there is no clear definition of PPI in Japan, but PPI is a concept first adopted in the U.K. In recent years, it has evolved into the idea of involving patients and citizens in decision-making, not only in the field of research but also in healthcare policy and other areas in general. In Europe, the value of patient involvement is increasingly being measured, and there are a wide range of ways to get involved. The value of information provided by patients is not only the actual experience of the disease and its treatment, but also the value of "making a difference by adding something concrete to the scientific opinion," such as offering a different perspective or raising an issue that has not been taken into account. I hope that one day this will be measured in this way in Japan in the near future.

There have been two major challenges to the promotion of PPI in Japan. On the patient/citizen side, there are few educational opportunities on PPI, and therefore, there are few people who are willing and prepared to participate in PPI. These issues have been significantly resolved in recent years through the efforts of various organizations, and there are now more opportunities for researchers and developers to obtain information, and for patients and citizens to receive education and participate in PPI. The foundations for PPI are now being laid, and we can say that we are in a period of growth.

As a suggestion from the UK, "collaboration" to connect the information and knowledge of patient groups and various stakeholders is important for further promotion of PPI. To this end, I believe that, first of all, it is necessary for many people to participate in opportunities for connection (a step toward participation).

Subject 2: Learning and Practice of PPI - Having a mindset to try it anyway
Ms. Noriko Iwaya, Representative, incurable disease support familia Yamaguchi

My son has congenital thrombotic thrombocytopenic purpura, a disease with more than 60 cases in Japan and 200 cases worldwide, and we transfuse 480 mL of fresh frozen plasma every two Fridays for six hours.

My first encounter with PPI was with the AMED (Japan Agency for Medical Research and Development) PPI guidebook that I got in September 2019. I learned about PPI online and have since had the opportunity to present at conferences such as the Online Café, the 21st Conference on CRC and Clinical Trials 2021 in Yokohama, and to collaborate with other patient groups and industry.

The reason why I have come to learn and practice so much is because my son was unable to participate in a global clinical trial. I did not understand why he could not participate, did not understand the difference between a clinical trial and a clinical trial in the first place, and could not believe that it would take approximately 10 years to produce a drug. This experience strengthened my desire to learn.

In my learning, I met many people and had more opportunities to participate in various projects. However, even when I participate, I do not have the courage to speak up because, in the end, I do not understand the common language. I felt it was necessary to know the common language in order to participate in such projects.

There are various activities with the letter P, such as Patient Centricity and Patient Advocacy, but whatever the name, I believe the most important thing is to position each other as partners and practice activities that are win-win.

Our partner for our patients' families is the doctor in charge. When my son was 0 years old, I felt it was very wasteful to transfuse only 80mL of the 480mL per unit and discard the remaining 400mL of blood that was donated in good faith. In addition, anaphylactic shock was frequently occurring due to the transfusion of an unspecified number of people, so we asked his doctor if we could do something about it and asked him to divide one unit of blood into 6 small packs of 80 mL. I have heard that this system of small packs is now in place, and I realize that it is very important to communicate my opinions and ideas. I believe this was the beginning of PPI in my mind.

In our activities to support people with intractable diseases, we hear from various people about their difficulties, worries, and hopes. I would like you to imagine that PPI is one of the ways to transform them into a form, and that this will lead to future patients as well.

Regarding the "mindset of just trying anyway," I never had one in the first place, but as I learned about PPI, I changed my mindset to one of just trying anyway, because even if I fail, it will be a story. In the beginning, I did not understand the common language and had no idea what I was talking about. I felt ashamed of what I did not know, but by having the courage to ask questions, the other person would realize this. This is what dialogue is all about, and I believe it is through this that we can bring patients and citizens, researchers and academia, and government together to see eye to eye.

There is no right answer to PPI activities, and I think it is necessary to "give it a try for now. I think that the entrance to PPI is to gather in a place like this and listen to other people's opinions and ideas. First of all, please try to speak out to your family members, neighbors, or anyone in front of you, no matter what your problems are.

Lecture 3: Nothing without us, with us ~Don't talk about us without us
Ms. Naomi Sakurai, Representative Director, General Incorporated Association CSR Project

When I was diagnosed with cancer in 2004, I wondered why there was such a lack of a user perspective, even for a single hospital room. In addition, and this also came as a shock to me personally, there was no master plan for cancer control at that time. 2006 saw the enactment of the Basic Act on Cancer Control, and I believe that this was the beginning of the participation of patients and citizens in healthcare policy from a place where there really was none.

I first encountered PPI just three years after my treatment, when I experienced a recurrence of cancer and went to see what patient association activities overseas were doing. This was a real eye-opener.

There were many people lobbying around the Diet building in Japan, and there was an Advocate day where we would tell the legislators what we were thinking, and what I was taught was to use "We" instead of "I" and to "talk in numbers. Furthermore, I was taught to "take it with one voice," and although the principles and arguments of the 200 participating patient groups are completely different, I was taught to talk not about my group, but about what I can do for those who will be patients in the future.

Later, I also participated in a week-long study group on international clinical trials, for which I had two weeks of prior study. The study group included just under 40 people from 24 countries and researchers and we talked about many things, but I felt that the Research Advocate part was missing from the various activities of the patients.

In the area of oncology, patient participation had already begun in the area of medical policy, but the Third Basic Plan for the Promotion of Cancer Control went a step further and stated that patient participation in cancer research should begin and that patients who are able to participate should be trained. Currently, we are in the process of creating a PPI education curriculum as part of the Aruga Group's research in which I am also participating.

Many of the things that patients' voices are being incorporated into include serving on ethics committees within academia, making recommendations to pharmaceutical companies regarding their research, and reviewing consent forms. But where we really want to do is make suggestions for research that would address our issues, etc. I think the supportive care side is moving a little bit, but we are still not able to get into it because of the scientific and other content that comes in.

As a recent effort, we tried a mock clinical trial workshop to discuss from what perspective patients would consider participating in a clinical trial. The discussion was very meaningful, as even just four people have different values, such as the side effects they value, the method of intervention, and the impression they get from the way the trial is explained to them. It was a very meaningful discussion. I would very much like to see this done in many companies.

I think there are various goals for PPI, but I think the starting point is the examination room. In terms of clinical trials and drug discovery, I think it is important that patients are properly informed and can make choices with conviction in order to promote better, safer, and more secure clinical research and medical care. For this purpose, I think it is necessary to have transparency and for patients themselves to examine patient diversity.

I think it is very important to coordinate the process, and it is also important for the company to provide feedback. Especially in this coordination, when someone says, "I have a project like this," we often ask, for example, whether it is only for patients in Tokyo or for those in rural areas, the degree of progression of the disease, family background, age, and so on. I would like the companies to think about what the purpose of the event is, including those aspects.

I believe that PPI in Japan has only just begun. On the patient side, there are many people who are eager to deliver their own experiences. On the other hand, I feel that there are still barriers on the researchers' side, so I would like them to learn more about patient group activities and patients together.

I believe that PPI is progressing in Europe and the U.S. because of HTA (health economic assessment). I think it is just a matter of time in Japan. On top of that, PRO (Patient Reported Outcomes) is also becoming important, not only for drug discovery, but if they have picked up the voices of patients who say that side effects are painful, I hope they will respond to them properly. I also think it is important to create opportunities for patients to participate in various places.

As the title of my lecture says, the basic principle is "Nothing without us, with us. I hope that the main principle will be, "Don't talk about us without us.

Panel Discussion

Nishimura (titles below omitted)

I believe that the PPI is perceived and promoted in different ways and with different goals depending on each individual's position. That is rather natural, and fortunately, I think each stakeholder is at a similar level in Japan, so please talk about what you think from your respective standpoints.
First of all, I asked Mr. Yoshida to give an over view at the beginning, but what do you think is the value of PPI from your standpoint as a researcher?

Yoshida

I believe that the value lies in the fact that we can create pharmaceuticals that are close to patients. I am researching the ideal drug price system, and I believe that drug prices are one way to evaluate the value of drugs, and as long as drugs reach patients, the evaluation of value should also be from the patient's perspective.

Nishimura

Mr. Okuda and Mr. Fujita of the Clinical Evaluation Subcommittee, what do you think from the standpoint of working for an individual company?

Okuda

I think that collaboration, learning, and dialogue are three very important words to accelerate PPI. As Mr. Iwaya mentioned in his presentation, just having someone say, "I don't understand," can bring awareness to the company side. I think learning is important, but having a shared awareness of what we don't know gives us the opportunity to know what we should do as well.

Nishimura

When learning, there is a relationship between those who teach and those who learn, but dialogue is about sharing what we think of each other, regardless of hierarchy. In the sense that communication by taking away all of our positions leads to collaboration, I was reminded that this is really the essence of PPI.

Fujita

I was reminded of what was said earlier about collaboration and dialogue, and I also felt that the recent term Patient Centricity, which refers to patient and citizen participation, is increasingly calling for an attitude of co-creation to create better medicine and better medical care for society as a whole. I also felt that it is necessary in the future to have a system that can reflect the opinions of patients from different backgrounds, such as regions and living environments, in drug development.

Nishimura

In my profession, I have many opportunities to interview patients and their families, and when I think that patients take time to come to the hospital from faraway places, I feel that it is not enough to just wait in an office close to the station and discuss.
Mr. Yoshida mentioned that PPI is progressing in Europe. What are the differences between Europe and Japan?

Yoshida

There are differences in the systems and the way the regulator regulates, but I believe there are also differences in the national character. As Mr. Iwaya mentioned, in Japan, even if you go to a hospital with your child, in many cases it is the parents who talk to the doctor. If people can change their mindset to make decisions on their own, as Mr. Iwaya's son did, I expect that will change. Of course, there is also the issue of health literacy.

Nishimura

What triggered your son, Mr. Iwaya?

Iwaya

When he turned 20, he said, "I will be on the support side. I will make my own decisions. I think that his coming of age turned him on. He had support from those around him and adults who agreed with his decision to jump into the world of e-sports.

Nishimura

I think it is also wonderful that the parents understood and trusted their children and sent them off.
Do family members often come to the forefront of the AYA generation of cancer patients, rather than the patients themselves?

Sakurai

Yes, there are. I am an AYA, and even among AYAs, the Patient Journey is diverse. It depends on when the patient is informed of the disease. In the case of children, parents have the right to make medical decisions. Treatment for childhood cancer can be very tough, and if there are late effects, it is the child who bears the burden, even if it was the parents who made the decision. Now that we have learned some things through data collection, I think it will be necessary for the pharmaceutical companies to provide support that will help the patient journey.

Nishimura

In the area of rare or intractable diseases, there are some cases where minors are not told the name of the disease, but are told it as an individuality. I think that is one approach, but on the other hand, they may be shocked to learn that they have a disease when complications, etc. appear after the age of 20, and that is when they learn that they have a disease. I was reminded that learning and dialogue are important not only in PPI but also in many other situations.
The theme of this year's meeting is to take a broader view of PPI than just drug discovery. Do you have any examples or thoughts on this?

Iwaya

When I was thinking about how to publicize the intractable disease café that my organization is holding, I first gathered up the courage to knock on the university hospital's intractable disease countermeasures center. I asked them to let people know that we were holding a café for intractable diseases and to act as a bridge between the café and people who inquired at the Center for Intractable Disease Control. The people who inquired were parents with sick children, and I was able to think together with them based on my experience.

Sakurai

Cancer has a long treatment period, and there are quite a few cases where side effects remain for a long time. We have a program called Patient Shoes, in which we simulate for the people in the company how difficult it is to take out a medicine while the fingertips are numb due to terminal neuropathy. We do this with the hope that the feeling they get when they open one of the pills will be the start of something new.

Nishimura

If they only take one pill, they may find it difficult to open it, but some people take 20 pills every time they take a pill. If the packaging is not appropriate, it may not lead to taking the medication.
It is hard if all the pills are in powder form, and it is also really hard if they are all large pills.

Sakurai

It may be a small thing, but please imagine that such a small thing is part of our daily life.
It is hard to understand "3 pitches and 1 rest". I think it would be great if you could start from such a point. It is the same for children. I think it is very difficult for children to take medicine.

Nishimura

Yes, it is. It is the parents who make them take the medicine, and some people find it stressful every time. Usually (parents and children) get along well with each other, but only when they take the medicine do things get rough, and sometimes they don't want their children to take the medicine because of that.

Okuda

I have heard that when developing medicines for children, there are activities to ask children's opinions about the size and taste of medicines, and I believe that activities to listen to children's voices will expand more in the pediatric field in the future.

Sakurai

Surely if it is good for children, it is also good for the elderly.

Nishimura

Yes, I think so. I believe that the elderly are taking some medications, so I think their expertise will come in handy.
Going back to children, I think it is important not to use language that only adults can understand in PPIs. If they feel that their voices will be heard, they will speak up, and if some things change because of that, I think we will see an expansion of trying to talk a little more. In the area of oncology, I think it is important to look at the patient side only.
In the field of oncology, I think not only the patient side but also researchers, doctors, and comedics understand the importance of PPI and are engaged in various activities. In this context, please tell us about any achievements or challenges that you feel have been encountered by Mr. Sakurai.

Sakurai

Breast cancer is affected by the gender balance in which doctors are mostly men and patients are mostly women, and they have very different values from each other. There were times when we had to change the design of a study because we were told "that's not right" at the research design stage.
The power balance is still an issue. It is difficult to say, "Doctor, that's not right. Patients also have to be prepared to do their best. Especially when I am on a committee that is reviewing guidelines, I do so with a sense of responsibility and with everyone's face in my mind.

Nishimura

We believe that the voice of the patient side is valuable. On the other hand, it is also a responsibility, so of course we cannot just say whatever we want. The patient side also needs to learn to strike a balance between the two.
As Mr. Iwaya said, there are many words beginning with "P," but I think the most important thing is to take action anyway. In particular, I would like to ask companies not to stop at learning and talking, but to take concrete action.

Provision of information

Introduction of Guidelines for Collaboration with Patient Groups and Transparency Guidelines

The "Guidelines for Working with Patient Groups" were established in 2013 and revised in May 2022 to ensure that, in any collaboration, we act with high ethical standards and integrity, respect the independence of patient groups, and strive to fully understand each other's objectives and the content of collaboration with patient groups.
The "Transparency Guidelines for Relationships between Corporate Activities and Patient Groups" were formulated in 2012 to ensure transparency by disclosing information on financial support provided to patient groups under certain rules, and to gain widespread understanding that such activities are sincere activities that contribute to the activities and development of patient groups while ensuring a high level of ethical standards. The guidelines were established in 2012 and revised in May 2022 in order to gain a wider understanding of the activities of the association.
Pharmaceutical Association member companies are expected to formulate their own guidelines and work together accordingly.

Closing Remarks

Mr. Yoshiyuki Ishida, Managing Director of the Japan Pharmaceutical Manufacturers Association (JPMA), expressed his appreciation for the valuable time spent at the conference, which deepened the understanding of the importance of PPI in building a better future for the industry.

In lieu of a tea party, a place to talk

After the main program, we held a time for the speakers and audience members of the patient groups to talk freely with each other on the web as an alternative to the tea party that was held at the previous venue. The participants actively exchanged opinions about what they could not talk about and what they wanted to hear more about, making it a very fulfilling time for everyone who attended.

(Atsushi Nomura, Patient Organization Seminar TF Leader, Patient Organization Collaboration Promotion Committee)

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