Citizens, Patients and Musubu The 39th Pharmaceutical Manufacturers Association of Japan (PMAJ) Patient Group Seminar" 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 Pharmaceutical Manufacturers Association of Japan (PMAJ) Patient Group Collaboration Promotion Committee held the "39th PMAJ 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 was attended by approximately 70 people and provided a meaningful opportunity for many patient groups to receive information.

The seminar The seminar

Provision of information

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

The Clinical Evaluation Subcommittee of the Pharmaceutical Evaluation Committee of the Pharmaceutical Manufacturers Association of Japan (PMAJ) provided information on drug development based on patients' voices.

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 not only to participate in clinical trials, but also to share their opinions based on their own experiences in creating a clinical trial plan, thereby creating a clinical trial together.

Efforts for patient involvement in drug development at pharmaceutical companies 4.

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. Therefore, the Pharmaceutical Manufacturers Association of Japan (PMAJ) 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 pharmaceutical products (Pharmaceuticals and Medical Devices Agency, PMDA) is also promoting patient involvement: a working group to study 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 (Japan Research 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

In the panel discussion, facilitated by Yoko Matsumoto, Vice President of the National Federation of Cancer Patients' Associations, Kimie Sakurai, Vice President of NPO GISTERS, Noriko Komazawa, Vice President and Secretary General of the Japan Narcolepsy Society, and two members of the Clinical Evaluation Subcommittee of the Pharmaceuticals Evaluation Committee of the Pharmaceutical Manufacturers' Association of Japan participated as panelists. 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 very 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 participating in clinical trials
  2. A:
    Yes" was selected by 5.4% of respondents.
  3. Q:
    Reasons for participation in clinical trials
  4. A:
    Necessity" and "No other choice" were the majority.
  5. Q:
    Image of Clinical Trials
  6. A:
    While "hope," "process of new drug development," and "option after standard treatment" were the most common responses, "human experimentation" was also mentioned.
  7. Q:
    Concerns
  8. A:
    Unexpected side effects and physical effects" was the most common response. Financial concerns including transportation and accommodation costs," "Relationship with attending physician," and "Concerns about moving to a new hospital" were also cited as concerns.

(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 site for clinical trial recruitment on our website (currently closed to 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 groups. Some said, "I searched on portals, etc., but couldn't find it" or "I couldn't identify it.

Mr. Matsumoto: On the website of the Foundation for Cancer Research, there is 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.
Pharmaceutical Cooperative We believe that it is important to provide clinical trial information to patients and the public.

  1. i. Efforts to communicate accurately and clearly
  2. The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations.
    Administrative Efforts: It was 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 the Pharmaceutical Manufacturers Association of Japan (PMAJ) also believes that further efforts are needed.
  3. The event concluded with a speech by Mr. Takafumi Adachi, Director of Public Relations.
    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, the Pharmaceutical Manufacturers Association of Japan (PMAJ) revised its clinical trial awareness pamphlet entitled "'Drugs' and 'Clinical Trials'" and made it available on its website. We will actively disseminate this information, including at events such as today's seminar.

(3) Examples of collaboration between patient groups and pharmaceutical companies on clinical trials

Pharmaceutical Cooperative Association There are examples of efforts to dispel concerns about clinical trials, such as asking patients' opinions at the planning, implementation, and post-implementation stages of a clinical trial to consider which trials are easy to participate in, and having employees of pharmaceutical companies 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 from the Early Stages of Clinical Trials
Pharmaceutical Cooperative Pharmaceutical companies are professionals in medicine, but the professionals in disease are patients. We believe that the greatest significance 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 our daily lives, could express 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 adjust our lives and livelihood to treatment, but I still thought it would be good if we could adjust treatment and drugs to our lives.
Recruitment of Participants for Clinical Trials
Pharmaceutical Cooperative Basically, we ask doctors at medical institutions conducting clinical trials to find patients who match the clinical trial. 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 pharmaceutical companies other than clinical trials

Pharmaceutical Cooperative We have been working on mindset building by inviting patients to lectures and dialogue meetings, 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?
Pharmaceutical Cooperative I think it is important to have a relationship where patients, pharmaceutical companies, 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 if there are opportunities to meet face to face with medical professionals and government agencies in addition to pharmaceutical companies, so that we can 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, we have talked about opening the door to the future together. Clinical trials were the entry point, but I think we have reconfirmed it 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.

Announcements

In the news from the Pharmaceutical Manufacturers Association of Japan, the General Policy Subcommittee of the Industrial Policy Committee of the Pharmaceutical Manufacturers Association of Japan gave a presentation on the "Pharmaceutical Manufacturers Association of Japan Industrial Vision 2025 Supplementary Edition (for patients and their families)" and the activities of the Advocacy Group of the Committee, which was newly established by the Pharmaceutical Manufacturers Association of Japan.

The Pharmaceutical Manufacturers Association of Japan Industry Vision 2025 Supplement (for patients and their families)" 2.

The Pharmaceutical Manufacturers Association of Japan (PMAJ) established the "PMAJ Industry Vision 2025" in January 2016 as a vision for the future in order to deliver innovative new medicines to patients as quickly as possible. We are using artificial intelligence and other technologies to improve the quality of life of our 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 that it is important for patients and their families to be well informed about diseases and pharmaceuticals in order to live longer while undergoing treatment. The Pharmaceutical Manufacturers' Association 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.

Activities of the Pharmaceutical Manufacturers Association of Japan (Advocacy Group)

We believe that in order to realize a healthy and comfortable society, it is important to gain the understanding and support of various people in society, so we have established a new Advocacy Group in the General Policy Subcommittee of the Industrial Policy Committee of the Pharmaceutical Manufacturers' Association of Japan. The word "advocacy" means to express one's thoughts and intentions, and we hope to communicate our aspirations as a pharmaceutical company as expressed in the "Pharmaceutical Manufacturers' Association 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, Executive Director of the Pharmaceutical Manufacturers Association of Japan (PMAJ), 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 website of the Pharmaceutical Manufacturers Association of Japan (PMAJ) (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 Organization Seminar TF, Patient Organization Collaboration Promotion Committee)

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