Connecting with Citizens and Patients The 39th JPMA Patient Group Seminar" to be held online Let's Think Together, Future Drug Development (Clinical Trials) - Let's Open the Door to the Future Together

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The JPMA Patient Cooperation Committee held the "39th JPMA Patient Group Seminar" online on November 24, 2021. Under the theme of "Let's Think Together, Future Drug Development (Clinical Trials) - Let's Open the Door to the Future Together," the seminar attracted approximately 70 participants on the day and provided a meaningful opportunity for many patient groups to provide information.

Scenes from the seminar Scenes from the seminar

Provision of information

Drug Development Based on Patients' Voices: Pharmaceutical Companies' Efforts and Future Expectations

The Clinical Evaluation Subcommittee of the JPMA Drug Evaluation Committee provided information on drug development based on the patient's voice.

1. how a drug is made (flow of drug development)

  1. 　Basic research (2-3 years)
  2. 　Research using animals (3-5 years)
  3. 　Clinical trials (3-7 years)
  4. 　Application and review for approval (1 year)
  5. 　Approval
  6. 　Marketing Bulletins

2. patient participation in drug development (PPI)

PPI stands for "Patient and Public Involvement," which means that medical care and research are promoted by incorporating the opinions of patients and citizens. In the development of pharmaceuticals, pharmaceutical companies and experts such as physicians have been discussing and evaluating drug candidates and preparing clinical trial plans. Recently, it has become important for patients to not only participate in clinical trials, but also to share their opinions based on their own experiences in creating the clinical trial plan, thereby creating the clinical trial together.

3) Efforts for patient involvement in pharmaceutical companies 4) Government agencies (PMDA)

One of the reasons why patient participation in drug development (clinical trials) has not progressed is that pharmaceutical companies do not know how to communicate with patients. In response, JPMA has compiled a guidebook on how pharmaceutical companies can communicate with patients, based on the opinions of patient groups. In addition, we were able to revise a pamphlet on clinical trials for patients, "'Drugs' and 'Clinical Trials'", for the first time in 14 years, in cooperation with patient groups.

4. initiatives for patient involvement at administrative agencies (PMDA) 5.

The government agency primarily responsible for the review of pharmaceuticals (Pharmaceuticals and Medical Devices Agency, PMDA) is also promoting patient involvement: a working group to examine patient involvement was established in 2019, and guidance announcing patient involvement was published on September 7, 2021.

In addition, registration and publication of clinical trial information became mandatory in 2018. Furthermore, from September 1, 2020, it became mandatory to disclose the names of clinical trial sites. The registration of clinical trial information has also been centralized, and all clinical trials in Japan can now be searched in Japanese on the jRCT (Japanese Registration Center for Clinical Trial Information) website, which is a public system for clinical research implementation plans and research summaries.

Expected effects of patient involvement

The development of drugs that reflect the voices of patients is expected to lead to "faster delivery of valuable drugs to patients," which is a common desire of patients and pharmaceutical companies. We will continue our activities to promote drug development that listens to the patient's voice more than ever.

Panel Discussion

The panel discussion was facilitated by Ms. Yoko Matsumoto, Vice Chairperson of the National Federation of Cancer Patients' Associations, with the participation of panelists Kimie Sakurai, Vice Chairperson of the NPO GISTERS, Ms. Noriko Komazawa, Vice Chairperson and Secretary General of the NPO Japan Narcolepsy Society, and two members of the Clinical Evaluation Committee of the JPMA's Drug Evaluation Committee. The two participants expressed their thoughts on drug development (clinical trials) from their respective standpoints.

1. introduction of organizations

(1) Japan Narcolepsy Association (NPO)

This is a patient group for hypersomnia-related disorders with a focus on narcolepsy. Narcolepsy is a type of sleep disorder and a chronic rare disease of juvenile onset. Founded in 1967, the association has approximately 400 members.

(2) NPO GISTERS

Gastrointestinal stromal tumor (GIST) is a rare submucosal tumor of the gastrointestinal tract with an estimated incidence of 1 in 100,000 people. Despite the rarity of the disease, interest in the disease is high, and there are currently fewer than 550 registered users on SNS.

2. issues in clinical trials

(1) Anxiety, resistance, etc. to participation in clinical trials

Mr. Komazawa: When participating in a clinical trial, a major hurdle is controlling sleepiness through abstinence. In narcolepsy, there is a phenomenon of muscle weakness when expressing feelings of joy, anger, sadness, or pleasure, and abstinence from medication further restricts behavior. For this reason, I believe that very few people who work in companies take part in clinical trials, and participation in clinical trials is mainly for students and housewives.
Sakurai: For us, clinical trials are a step beyond standard treatment. Clinical trial participation is also indicated in the medical practice guidelines. Our association regularly conducts a survey of our members. I will share the results of 2020, especially regarding clinical trials (222 respondents).

  1. Q:
    Experience of participating in clinical trials
  2. A:
    Yes" was selected by 5.4% of the respondents.
  3. Q:
    Reasons for participation in clinical trials
  4. A:
    The majority of the respondents answered "need," and "no other options."
  5. Q:
    Image of Clinical Trials
  6. A:
    While "hope," "process of new drug development," and "options after standard treatment" were the most common responses, "human experimentation" was also mentioned.
  7. Q:
    Concerns
  8. A:
    Unexpected side effects/impact on the body" was the most common response. Financial concerns including transportation and accommodation costs," "Relationship with primary doctor," and "Anxiety about moving to a new hospital" were also cited.

(2) Is information on clinical trials reaching patients?

Mr. Komazawa: A great number of patients were aware that they cannot receive clinical trials unless they are referred by their primary physician. Especially in rural areas where there were no specialist physicians, clinical trial information did not reach them, so they were unable to undergo a clinical trial even if they wanted to. Under such circumstances, a pharmaceutical company asked us for advice on how to proceed with patient registration for clinical trials, and we created a website for clinical trial recruitment on our website (currently closed for recruitment).
Mr. Sakurai: Collecting information on clinical trials has been very difficult. Here are some of the responses to the aforementioned questionnaire regarding clinical trial information.

  1. Q:
    Do you have sufficient information on clinical trials?
  2. A:
    Yes" was selected by 5.4% of the respondents.
  3. Q:
    Where do you look for clinical trial information?
  4. A:
    The majority of respondents answered "websites of patient organizations. Some said, "I searched for it on portals, etc., but couldn't find it" or "I can't identify it.

Mr. Matsumoto: The Cancer Research Foundation website has a "Patient-oriented 'Cancer Information Site'". Here, you can look up reliable and up-to-date cancer information related to clinical trials and clinical studies. Since we have just created the site, we feel that the search method is a little difficult, and we are considering how to make it easier to understand.
JPMA We believe it is important to provide clinical trial information to patients and the public.

  1. i. Efforts to communicate accurately and clearly
  2. •
    Administrative Efforts: It has been decided that all clinical trial information in Japan will be posted on jRCT. However, we feel that there is a high hurdle in terms of whether patients and the public can reach the site and obtain the information they want, and JPMA believes that further efforts are needed.
  3. •
    Efforts by pharmaceutical companies: Some companies are disclosing clinical trial information on their websites in accordance with their own policies. Some companies are actively disclosing information, while others are still in the process of doing so, but all companies are beginning to take a positive attitude toward information disclosure.
  1. ii. Necessity of clinical trial awareness
  2. 　
    In March 2021, JPMA revised a pamphlet on "Drugs and Clinical Trials" to raise awareness of clinical trials and made it available on the website. We will actively disseminate the information including events such as today's seminar. iii.

(3) Examples of collaboration between patient groups and JPMA regarding clinical trials

JPMA There are examples of efforts to dispel concerns about clinical trials, such as asking patients' opinions at the planning stage, during the trial, and after the trial, and considering which trials are easy to participate in, and having pharmaceutical company employees actually check the procedures at the planning stage of a clinical trial.
Mr. Sakurai: We often check the protocol and consent documents. I feel that this will increase further in the future.
Mr. Komazawa: When you are hospitalized for a clinical trial, the only thing you can look forward to is a meal, so we felt that we would like to hear those opinions. The recruitment of clinical trials on the website made it easier to introduce clinical trials when consulting not only members but also patients who do not belong to a patient group.

[Questions received in advance from seminar audience members

The value of patient participation in clinical trials from the early stages:
JPMA The pharmaceutical companies are professionals in medicine, but the professionals in disease are the patients. We believe that the most significant thing for drug development is to know in advance the viewpoints of patients when clinical trials enter their daily lives.
Mr. Sakurai: If we could get patients to really think about their opinions, and if we, as people in their daily lives, could voice our opinions from the beginning, I think many things would change, such as the need for fake drugs.
Mr. Komazawa: I was really happy when a pharmaceutical company asked me, "Is there anything you wish there was such a drug?" I was really happy when they asked me.
Mr. Matsumoto: We may be at a stage where we have to adapt our lives and livelihood to treatment, but I still thought it would be good if we could adapt treatment and medicine to our lives.
Recruitment of Participants for Clinical Trials
JPMA Basically, we ask the doctors at the medical institutions conducting the clinical trials to find patients who match the clinical trials. There is also recruitment through newspaper ads, radio, and the Internet, and some recruitment through patient groups. However, we feel that clinical trial information is not being disseminated in a way that is easy to understand, so we will continue to work on this issue further in the future.
Matsumoto: We have not yet reached a point where we can say "Hey, hey, hey" to pharmaceutical companies. I thought it would be good if we could build a relationship of trust by exchanging various information closely on a daily basis, rather than by suddenly getting along with them when we start a clinical trial.

(4) Examples of collaboration between patient groups and JPMA other than clinical trials

JPMA We have been working to foster a mindset by inviting patients to lectures and dialogue sessions, visiting medical institutions to see and feel the treatment sites, etc. More and more companies are implementing initiatives that make us regularly think about the reasons we work, our motivation, and our perspectives and viewpoints.
Mr. Sakurai We help with lectures and new employee training, and plan and cooperate with support pages and booklets for patients.
Mr. Komazawa: There is a place for people to talk about their problems and situations regarding their diseases. I think it would be good if many patients connect with pharmaceutical companies.
Mr. Matsumoto What would you like to do to go one step further and open the door to the future together?
JPMA I think it is important to have a relationship where patients, JPMA, medical professionals, government officials, and professionals from each side can have a closer dialogue. I would be happy if we can have continuous communication while ensuring appropriate relationships and transparency.
Mr. Sakurai: For those of us who have been able to live longer because of the creation of medicine, we have a strong sense of gratitude toward medicine. I hope that our voices will continue to be heard. We would like to think together.
Mr. Komazawa I think it would be good to have opportunities to meet face to face with medical professionals and government agencies other than pharmaceutical companies to gain a larger perspective.
In addition, patient associations are often run by the parties involved, which is exhausting. We would like to break out of this situation. I would like to ask for your help.

(5) Summary

Mr. Matsumoto Today, I have spoken about opening the door to the future together. Clinical trials were the entry point, but I think we have reaffirmed this as one of the ways to support our "living" and "life". I would like to continue to work hand in hand with pharmaceutical companies and other related organizations to open the door to support our lives and livelihood. I hope that today will be a step toward that end.

Announcement

In the news from JPMA, the General Policy Subcommittee of the Pharmaceutical Industrial Policy Committee gave a presentation on the "JPMA Industrial Vision 2025 Supplement (For Patients and Families)" and the activities of the newly established Advocacy Group of the JPMA Industrial Policy Committee.

JPMA Industry Vision 2025 Supplement (for patients and their families)

JPMA established the "JPMA Industry Vision 2025" in January 2016 as a vision for the future in order to deliver groundbreaking new drugs to patients as quickly as possible. The following items are of relevance to patients.

(Item 1) Creating new medicines using digital technology

We are challenging to create drugs with superior efficacy and safety in a short period of time by utilizing artificial intelligence and other technologies in our research. Clinical research, including clinical trials, is usually conducted in hospitals, but new information and communication technologies are being explored to allow people to participate at home.

(Item 2) Realizing a society that is easy to live in through the use of "Big Data

Information (big data) on the health, illness, and treatment of thousands or tens of thousands of people, while protecting their personal information, is collected and analyzed using the latest computers, which is expected to lead to new tests to detect illness earlier and the development of highly effective drugs.

(Item 4) Provide easy-to-understand information on medicines

We believe it is important for patients and their families to be well informed about diseases and pharmaceuticals in order to live longer while undergoing treatment. JPMA will collect information for patients held by each member company and create a system where information on diseases and medicines can be obtained from websites and other sources.

JPMA's activities (Advocacy Group)

We believe that in order to realize a healthy and comfortable society, it is important to have the understanding and support of various people in society, so we have established a new Advocacy Group in the General Policy Subcommittee of the JPMA's Industrial Policy Committee. The word "advocacy" means to express one's thoughts and intentions, and we hope to communicate our aspirations as a JPMA company, as expressed in the "JPMA Industrial Vision 2025," through dialogue and collaboration with society.

In order to create new drugs that meet the expectations of patients, it is essential to collaborate with patient groups, for example, by listening to patients' concerns about diseases and treatments.

We will work together with patient groups to realize a society that is healthy and easy to live in, while expanding opportunities for dialogue and collaboration not only in drug development but also in a variety of other areas.

Summary

Mr. Tokuo Tanaka, JPMA's Executive Director, thanked the participants for their past experiences and valuable information on drug development from the standpoint of people with intractable diseases and cancer. The seminar provided meaningful information to the participating patient groups and an opportunity for pharmaceutical companies to think together about drug development for the future of patients.

In addition, a video of the seminar is available on demand on the JPMA website (until the end of November, 2022) so that more patients can be informed of the seminar's contents.
https://www.jpma.or.jp/information/patient/seminar/seminar_39.html

( Miwako Watanabe, Patient Cooperation Committee, Patient Cooperation Seminar TF)

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