40th Patient Organization Seminar (Online Event) Let’s Think Together and Take a Step Forward: Patient and Citizen Participation in Healthcare (PPI)

Seminar Date: Wednesday, November 30, 2022

Video Streaming Period: Thursday, April 6, 2023 – Friday, May 31, 2024

Opening Remarks

In recent years, PPI (Patient and Public Involvement) has been gaining attention across various institutions and organizations. Through this seminar, we hope to make PPI feel more accessible and provide an opportunity for participants to take that first step toward actively engaging in their own treatment based on their own decisions.I hope this will be an opportunity for us to reflect together on what PPI is, why it is necessary, and what patients and patient organizations can do to contribute.

Lecture

Presentation 1: Latest Trends in PPI—Toward Further Promotion of PPI— Ms. Akiko Yoshida, Principal Researcher,
The Office of Pharmaceutical Industry Research

I understand that there is no clear definition of PPI in Japan.PPI is a concept that was first adopted in the United Kingdom, and in recent years, it has evolved into a philosophy that calls for the participation of patients and citizens in decision-making processes not only in the field of research but also in healthcare policy and other areas more broadly. In Europe, the value of patient participation is increasingly being measured, and the ways in which patients are involved are becoming more diverse.Regarding the value of information provided by patients, it seems that this value is recognized not only in terms of their firsthand experiences with illnesses and treatments but also in their ability to offer different perspectives and raise issues that had not been considered—in other words, “adding concrete insights to scientific opinions and making a difference.” I hope that a day will come in the near future when this value is measured in Japan as well.

It has long been recognized that there are two major challenges to promoting PPI in Japan. On the part of researchers and developers, there are few opportunities to obtain information about the significance and practical methods of PPI. On the part of patients and citizens, there are few educational opportunities regarding PPI, resulting in a shortage of individuals who are both willing and prepared to participate.In recent years, these challenges have been significantly addressed through the efforts of various organizations. Opportunities for researchers and developers to access information have increased, while educational opportunities and avenues for participation have expanded for patients and citizens. The foundation for PPI is now taking shape, and I believe we are entering a period of growth.

Drawing on insights from the UK, I believe that “collaboration”—connecting the information and insights of patient organizations and various stakeholders—is crucial for further promoting PPI. To that end, I believe it is essential, first and foremost, for many people to participate in opportunities to connect—which is the first step toward engagement.

Presentation 2: Learning and Practicing PPI—Adopting a “Just Give It a Try” Mindset
Ms. Noriko Iwaya, Representative, Intractable Diseases Support “familia Yamaguchi”

My son suffers from congenital thrombotic thrombocytopenic purpura (CTTP), a condition affecting only about 60 people in Japan and 200 cases worldwide. Every other Friday, he receives a transfusion of 480 mL of fresh frozen plasma over a period of six hours.

My introduction to PPI came in September 2019 when I obtained the AMED (Japan Agency for Medical Research and Development) PPI guidebook.After learning about PPI online, I had more opportunities to present at events such as online forums and the 21st Conference on the Future of CRCs and Clinical Trials 2021 in Yokohama, and I’ve been fortunate to collaborate with other patient organizations and industry professionals.

The reason I’ve come to study and practice this so deeply is that my son was unable to participate in an international clinical trial. I didn’t understand why he couldn’t participate, nor did I even know the difference between a clinical trial and a clinical study to begin with—and I couldn’t believe it took about 10 years for a drug to become available. That experience strengthened my desire to learn.

Through my learning journey, I’ve met many people and had more opportunities to participate in various projects. However, even when I do participate, I often lack the courage to speak up because I don’t understand the common language. I realized that learning this common language is essential for participating in such projects.

There are various initiatives beginning with the letter “P,” such as “Patient Centricity” and “Patient Advocacy,” but regardless of the name, I believe the most important thing is to view one another as partners and engage in activities that create a win-win situation.

Our partner as patients’ families is the attending physician. When my son was 0 years old, only 80 mL of a 480 mL blood unit was transfused, and I felt it was a tremendous waste to discard the remaining 400 mL—blood donated out of goodwill.Furthermore, since anaphylactic shock frequently occurred due to transfusions from unspecified donors, I consulted with the attending physician to see if there was a solution, and they divided one unit into six 80-mL packs.I’ve since heard that this system of smaller packs is now standard practice, and I’ve come to realize just how important it is to voice my own opinions and perspectives. I consider this to be the beginning of my journey with PPI.

Through my work supporting patients with intractable diseases, I hear about the hardships, concerns, and hopes of many different people. PPI is one way to give shape to these experiences, and I hope you can imagine how this will also benefit future patients.

As for the “let’s just give it a try” mindset, I didn’t have it to begin with, but as I learned more about PPI, I shifted my thinking to “even if I fail, it’ll be a learning experience, so let’s just give it a try.” At first, I didn’t understand the common language, and I was constantly in a situation where I didn’t know what people were talking about.I used to think it was shameful to admit I didn’t know something, but when I mustered the courage to ask questions, the other person realized that. I believe this is the essence of dialogue, and it’s what allows us to align our perspectives with patients, citizens, researchers, academia, and government officials.

To put it bluntly, I don’t think there’s a “right answer” when it comes to PPI activities; I believe it’s necessary to “just give it a try.” Gathering in settings like this and listening to others’ opinions and perspectives is, I think, the first step toward PPI. Start by sharing your concerns—or anything at all—with your family, your neighbor, or whoever is right in front of you.

Presentation 3: “Nothing without us, with us” ~Don’t talk about us without us~
Ms. Naomi Sakurai, Representative Director, CSR Project (General Incorporated Association)

When I was diagnosed with cancer in 2004, I wondered—even just looking at a single hospital room—why there was such a lack of a user-centered perspective. Furthermore, and this was personally shocking to me, there was absolutely no master plan for cancer control at the time.In 2006, the Basic Act on Cancer Control was enacted, and I believe that’s when patients and citizens began to participate in healthcare policy—starting from a point where there was truly nothing in place.

I first encountered PPI exactly three years into my treatment, when I experienced a recurrence and went to see what kinds of activities patient groups overseas were engaged in. It was truly an eye-opening experience.

A large crowd was staging a petition drive near the National Diet Building in Japan. It was an “Advocate Day,” where we conveyed our views to lawmakers. What I was taught on that occasion was to “use ‘we’ instead of ‘I’” and to “speak with numbers.”Furthermore, regarding the importance of “speaking with one voice”—even though the 200 participating patient organizations had completely different principles and positions—I was taught not to focus on my own organization, but to “talk about what we can do for future patients.”

After that, I participated in a one-week study session on international clinical trials, which required two weeks of preparatory study. The session brought together nearly 40 participants from 24 countries, along with researchers, and we discussed various topics. Through the patients’ diverse activities, I realized that the role of “research advocates” is still lacking.

As for Japan, patient participation had already begun in the field of cancer through healthcare policy, but the Third Basic Plan for the Promotion of Cancer Control went a step further: it stipulated the initiation of patient participation in cancer research, as well as the training of patients capable of participating. Currently, as part of the Ariga Group’s research—in which I am also participating—we are in the process of developing a PPI educational curriculum.

Some of the ways patients’ voices are increasingly being incorporated include participation in ethics committees within academic institutions, making recommendations on pharmaceutical companies’ research, and reviewing informed consent forms. However, what we really want to do is propose research that addresses our specific challenges. I think things are gradually moving forward in the area of supportive care, but the reality is that we haven’t been able to fully engage yet because scientific considerations also come into play.

As a recent initiative, we tried holding a mock clinical trial workshop to discuss the perspectives patients consider when evaluating participation in clinical trials.After some preparatory study, four of us with different patient backgrounds held a discussion. We found that even among just four people, values differed significantly—some prioritized different side effects, others hesitated to participate based on the intervention method, and the way information was presented greatly influenced their impressions. It was a very meaningful discussion. I really hope many companies will adopt this approach.

I believe PPI has various goals, but I think it all starts in the doctor’s office.From the perspective of clinical trials and drug discovery, I believe it is crucial that patients are provided with accurate information so they can make informed choices, thereby promoting better, safer, and more reliable clinical research and medical care. To that end, I believe transparency is essential, as is for patients themselves to carefully consider “Patient Diversity.”

I think coordinating these efforts is extremely important, and I also believe it’s crucial for companies to provide proper feedback.In particular, when coordinating these events, if a company says, “We have this kind of initiative in mind,” I often ask questions such as whether it’s intended only for patients in Tokyo or if those in rural areas also need to be included, as well as about the stage of the disease, family background, and age group. I hope companies will consider the purpose of holding such events, taking all these factors into account.

I believe PPI in Japan is still in its early stages. Many patients have a strong desire to share their experiences. On the other hand, I feel there are still barriers on the researchers’ side, so I hope they will join us in learning more about patient group activities and the patients themselves.

I believe the reason PPI is advancing in Europe and the U.S. is because of HTA (Health Technology Assessment). I think it’s only a matter of time before this happens in Japan as well. On top of that, PROs (Patient-Reported Outcomes) are becoming increasingly important; I hope that once we hear concerns about severe side effects, we’ll respond to them appropriately—not just for the sake of drug discovery. Furthermore, I believe it’s crucial to create opportunities for patients to participate in various settings.

As the title of this lecture suggests, I believe the fundamental principle is “Nothing without us, with us.” I hope this principle—“Do not speak about us without us”—will be adopted as a guiding principle.

Panel Discussion

Nishimura (hereinafter referred to without honorifics)

I believe that PPI is perceived, implemented, and approached differently depending on one’s perspective, and that its goals vary accordingly. This is only natural, and fortunately, I believe that in Japan, all stakeholders are on a similar level, so please share your thoughts from your respective perspectives.
First, Mr. Yoshida provided an overview at the beginning—from your perspective as a researcher, what value do you see in PPI?

Yoshida

I believe the true value lies in our ability to develop medications that truly meet patients’ needs. I’m currently researching the structure of the drug pricing system; while drug pricing is one aspect of evaluating a medication’s value, I believe that as long as a drug reaches patients, that evaluation of value must be based on the patient’s perspective.

Nishimura

Mr. Okuda and Mr. Fujita from the Clinical Evaluation Subcommittee: What are your perspectives from your positions at your respective companies?

Okuda

I believe the three concepts of collaboration, learning, and dialogue are crucial for accelerating PPI. As Mr. Iwaya mentioned in his presentation, even simply hearing someone say, “I don’t understand,” provides valuable insights for companies. While learning is important, sharing a common understanding of what we don’t know also gives us an opportunity to identify what we need to do.

Nishimura

In a learning context, there is typically a relationship between teacher and student, but dialogue involves sharing thoughts without a hierarchical structure. I was reminded once again that the true essence of PPI lies in setting aside all positional distinctions to communicate—because that is what leads to collaboration.

Fujita

Earlier, there was discussion about collaboration and dialogue. Recently, with the shift from the term “Patient Centricity” to “patient and citizen participation,” I’ve come to realize that there is an increasing demand for a co-creation mindset—where society as a whole works together to develop better medicines and better healthcare.I also believe that going forward, we will need a system capable of aggregating feedback from patients with diverse backgrounds—such as those from different regions and living environments—and incorporating it into drug development.

Nishimura

Due to the nature of my work, I often have the opportunity to interview patients and their families. When I think about how patients travel long distances and spend a lot of time commuting to the hospital, I realize it’s not enough to simply wait in an office near the station and hold discussions.
Mr. Yoshida mentioned that PPI is advancing in Europe—what are the key differences compared to Japan?

Yoshida

While there are differences in systems and regulatory frameworks, I also believe there are differences in national character.As Mr. Iwaya mentioned, in Japan, even when a child goes to the hospital, it’s often the parent who speaks with the doctor. I hope things will change if we can foster a shift in mindset—one where children, like Mr. Iwaya’s son, can make their own decisions. Of course, I also believe there are challenges related to health literacy.

Nishimura

Was there a specific reason behind Mr. Iwaya’s son’s decision?

Iwaya

When he turned twenty, he declared, “I’m going to be on the supporting side. I’ll make my own decisions.” I think reaching adulthood was the turning point for him. He was supported by those around him, and there were adults who approved of his decision to dive into the world of esports—it seems that these experiences inspired him to want to become a role model for children with cancer.

Nishimura

I also think it’s wonderful that parents understand their children, trust them, and let them go.
Is it common for family members, rather than the patients themselves, to take the lead when it comes to AYA cancer patients?

Sakurai

Yes, there are. I’m part of the AYA generation, but even within that group, patient journeys are diverse. They can vary completely depending on when the diagnosis is disclosed.In pediatric cases, the decision-making authority for medical care lies with the parents. Not only is pediatric cancer treatment extremely grueling, but if long-term side effects arise, even though the parents made the decision, it is the child who must bear the burden. As we’ve come to understand more through data collection, I believe pharmaceutical companies now need to provide support that truly accompanies patients throughout their Patient Journey.

Nishimura

In the field of rare and intractable diseases, there are cases where minors are not told their diagnosis but are instead encouraged to view their condition as part of their individuality. While I think that’s one valid approach, there are also instances where complications arise after the age of twenty, and the person is shocked to learn for the first time that they actually have a disease. This experience reaffirmed my belief that learning and dialogue are important not only in PPI but in a wide variety of contexts.
The theme this time is to view PPI in a broader context, not just in the context of drug discovery. If you have any examples or ideas, please let me know.

Iwaya

I run an “Intractable Disease Café” through my own organization, and while I was wondering how to raise awareness about it, I mustered the courage to reach out to the Intractable Disease Support Center at a university hospital. As a result, they learned about our café and helped connect us with people who had contacted the center for assistance.The person who contacted me was a parent of a child with a rare disease, and we were able to discuss the situation together based on my own experiences.

Sakurai

Cancer treatment is a long process, and in many cases, side effects persist for a long time.Through our “Patient Shoes” program, we give corporate employees the opportunity to experience firsthand just how difficult it is to retrieve medication when your fingertips are numb due to peripheral neuropathy. We do this with the hope that the feeling you have when opening that single pill will spark something new.

Nishimura

If it’s just one pill, you can manage even if the packaging is hard to open, but some people have to take 20 pills every time. If the packaging isn’t designed appropriately, it can prevent patients from taking their medication.
It’s tough when all the medication is in powder form, and it’s really tough when all the pills are large, so I’d really like them to take these factors into consideration.

Sakurai

It may seem like a small thing, but I want you to imagine how these small things become part of our daily lives.
The “3 doses, 1 day off” schedule is also hard to understand. I think it would be great if you could start by learning about things like that. The same goes for children—I imagine it’s incredibly difficult for a child to take medication.

Nishimura

That’s right. Since it’s the parents who have to give the medicine, some find it stressful every time. Even when parents and children usually get along well, things can become tense just when it’s time to take the medicine, and this can sometimes lead parents to feel reluctant to give it to their children.

Okuda

I have also heard that when developing pediatric medications, companies are actively seeking children’s opinions on the size and taste of the medicine, and I believe that in the future, such efforts to listen to children’s voices will expand even further in the pediatric field.

Sakurai

If it’s good for children, it must be good for the elderly, too.

Nishimura

That’s right. Since older adults are likely taking some kind of medication, I think that expertise will come in handy.
Returning to the topic of children, I think it’s important that PPI initiatives do not rely solely on language that only adults can understand. If children feel their voices are being heard, they’ll be more likely to speak up; and if they see that their input leads to change, they’ll be more willing to share their thoughts further. So even though this market may not be particularly large, I believe it’s important to treat their voices as equally significant as those of adults.
In the field of cancer, I believe not only patients but also researchers, physicians, and allied health professionals understand the importance of PPI and are engaged in various activities. In that context, could you tell us about any achievements or challenges you’ve observed, Ms. Sakurai?

Sakurai

Breast cancer is also influenced by the gender balance—there are many male doctors and many female patients—and our values are completely different. There have been instances where, during the research design phase, I said, “That’s not right,” and we ended up changing the trial design.
The power imbalance is certainly a challenge. It’s not easy to say, “Doctor, that’s wrong.” Patients also have to steel themselves and persevere in certain situations. Especially when I participate in settings like committees reviewing guidelines, I feel an immense sense of responsibility and keep everyone’s faces in mind as I work.

Nishimura

We believe the voices of patients are valuable and strive to convey that. At the same time, since this comes with responsibility, it goes without saying that we cannot simply say whatever we want; at times, evidence and objectivity are also necessary. The patient community must also learn to strike that balance.
As Mr. Iwaya mentioned, there are many terms that start with “P,” but I believe taking action is the most important thing. What I’d especially like to ask of companies is that they don’t just stop at learning or having a dialogue—I hope they’ll turn these efforts into concrete actions.

Information Session

Introduction to the Guidelines for Collaboration with Patient Organizations and the Transparency Guideline

The “Guidelines on Collaboration with Patient Organizations” were established in 2013 and revised in May 2022 to ensure that, in all forms of collaboration, we act with integrity and high ethical standards, respect the independence of patient organizations, and strive to achieve a thorough mutual understanding regarding the purpose and content of such collaboration.
The “Transparency Guideline for the Relationship Between Corporate Activities and Patient Organizations” was formulated in 2012 and revised in May 2022 to ensure greater transparency by disclosing information regarding financial support and other forms of assistance provided to patient organizations under specific rules, and to foster broad understanding that such activities are conducted in good faith, contributing to the activities and development of patient organizations while upholding high ethical standards.They were established in 2012 and revised in May 2022.
Member companies of the JPMA are required to establish their own guidelines and collaborate in accordance with them.

Key Takeaways and Closing Remarks

Mr. Yoshiyuki Ishida, Executive Director of the Japan Pharmaceutical Manufacturers Association, expressed his gratitude, noting that it was a valuable opportunity to deepen his understanding of how crucial PPIs are for building a better future.

A Space for Conversation in Place of a Tea Party

After the main program concluded, we arranged an online session where speakers and patient organization representatives in the audience could freely converse—as a replacement for the informal tea gatherings typically held during in-person events. There was a lively exchange of opinions on topics that hadn’t been fully discussed or that participants wanted to hear more about, making it a very fulfilling experience for everyone who took part.

(Atsushi Nomura, Leader of the Patient Organization Seminar Task Force, Patient Cooperation Committee)

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