The 39th Patient Group Seminar (held online) Let's Think Together, Future Drug Development (Clinical Trials) - Let's Open the Door to the Future Together

Seminar Date : November 24, 2021 (Wednesday)

Video streaming period: Thursday, December 23, 2021 - Wednesday, May 31, 2023

Links to materials marked with an asterisk (*) are provided at the end of the text.

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

The following information was provided by the Clinical Evaluation Subcommittee of the Pharmaceutical Evaluation Committee of the Pharmaceutical Manufacturers Association of Japan (PMAJ) as information on drug development.

1. How drugs are made (flow of drug development)

2. Patient Participation in Drug Development (PPI)

PPI stands for " Patient and Public Involvement " and refers to the process of advancing medical care and research by incorporating the opinions of patients and the public. In the development of drugs, pharmaceutical companies and experts such as physicians have been consulting with each other to evaluate drug candidates and create clinical trial plans. Recently, it has become important not only for patients to participate in clinical trials, but also for them to share their opinions based on their own real-life experiences when creating the clinical trial plan, thereby creating the clinical trial together.

3. Status of efforts for patient involvement in drug development at pharmaceutical companies

One of the reasons behind the lack of progress in patient participation from the pharmaceutical company side is that they do not know how to communicate with patients. For this reason, the Pharmaceutical Manufacturers Association of Japan (PMAJ) has compiled a guidebook (*) on how pharmaceutical companies should 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. Efforts for patient involvement by the government (PMDA)

The government agency (Pharmaceuticals and Medical Devices Agency: PMDA), which is mainly responsible for the review of pharmaceutical products, 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 this year.
In addition, registration and publication of clinical trial information became mandatory in 2018. Furthermore, from September 1, 2020, it became obligatory to disclose the names of the medical institutions conducting clinical trials. The registration of clinical trial information has also been centralized, and all domestic clinical trials can now be searched in Japanese on the jRCT (Japanese Registration Center for Clinical Trial Information) Clinical Research Implementation Plan and Research Outline Disclosure System (*) website.

5. Expected Effects of Patient Involvement

The development of pharmaceuticals 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 patients' voices more than ever.

Panel Discussion

1. Introduction of the organization

(1)The Japan Narcolepsy Association (*)

The Narcolepsy Association is an association of patients with hypersomnia and 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 400 members.

(2)NPO GISTERS(*)

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, there are currently fewer than 550 people registered on SNS.

2. Issues in Clinical Trials

(1)Anxiety and resistance to participating in clinical trials

Mr. Komazawa

A major hurdle in participating in clinical trials is controlling sleepiness by abstaining from medication. In narcolepsy, there is a phenomenon of muscle weakness when expressing feelings of joy, anger, sadness, or pleasure, and abstinence 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.

Mr. Sakurai

For us, clinical trials are a step beyond standard treatment. Clinical trial participation is also indicated in the guidelines. Our association regularly conducts a survey of its members. We will share the results in 2020, especially for the clinical trial part. (222 respondents)

(2) Is information on clinical trials reaching patients?

Mr. Komazawa

A great number of patients were aware that clinical trials are not available unless they are referred by their primary physician. Especially in rural areas where there were no specialist doctors, clinical trial information did not reach them, so they could not take a clinical trial even if they wanted to. Under such circumstances, a pharmaceutical company asked us for advice on how to proceed with patient enrollment for clinical trials, and we created a website for clinical trial recruitment on our website. (Recruitment is now closed.)

Mr. Sakurai

Collecting clinical trial information has been very difficult. Here are some of the responses to the aforementioned questionnaire regarding clinical trial information.

Mr. Matsumoto

The Cancer Research Foundation has a "Patient-oriented 'Cancer Information Site'" (*) on its website. The site provides reliable and up-to-date cancer information related to clinical trials and clinical studies. However, since the site has just been created, we feel that the search method is a little difficult, and we are considering ways to make it easier to understand.

Pharmaceutical Manufacturers Association of Japan

We believe it is important to provide clinical trial information to patients and the public.

(1) Efforts to communicate accurately and clearly

2) Need to raise awareness of clinical trials

This year, the Pharmaceutical Manufacturers Association of Japan (PMAJ) created a pamphlet on "Drugs and Clinical Trials" to raise awareness of clinical trials and made it available on our website. We feel the need to actively disseminate this information, including through opportunities such as today's seminar.

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

Pharmaceutical Manufacturers Association of Japan

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 a clinical trial that is easy to participate in, and having employees of pharmaceutical companies actually check the procedures at the planning stage of a clinical trial.

Mr. Sakurai

It is common to check the protocol and consent documents. I feel that this will increase in the future.

Mr. Komazawa

When hospitalized for a clinical trial, the only thing to look forward to is the food, so we felt that we would like to hear such opinions. The call for 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.

Mr. Matsumoto

I would like to address two questions we received in advance from the seminar audience.

(1) The value of patient participation in clinical trials from the initial stage

Pharmaceutical Manufacturers Association of Japan

Pharmaceutical companies are professionals of drugs, but patients are professionals of diseases. I think 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 point in our lives where we have to adapt our lives and livelihood to treatment, but I still wish we could adapt treatment and medicine to our lives.

(2) Recruitment of clinical trial participants

Pharmaceutical Manufacturers Association of Japan

Basically, we ask the doctors at the medical institutions conducting the clinical trials to find patients who might be a good fit for the trials. There are also newspaper advertisements, radio, and the Internet, and some patients are recruited through patient groups. However, we feel that clinical trial information is not being disseminated in a way that is easy to understand, and we will continue to work on this issue further in the future.

Mr. Matsumoto

We have not yet reached a point where we can say, "Hey, hey. I thought it would be good if we could build a relationship of trust by exchanging information closely on a daily basis, rather than by suddenly getting along with each other when we start a clinical trial.

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

Pharmaceutical Manufacturers Association of Japan

We have been working on mind-building by inviting patients to lectures and dialogue meetings, visiting medical institutions to see and feel the treatment sites, and so on. More and more companies are making efforts to regularly reflect on the reasons why we work, our motivation, and our perspectives and viewpoints.

Mr. Sakurai

We help with lectures and new employee training, and we plan and collaborate on patient support pages and booklets.

Mr. Komazawa

There is a place for people to talk about their problems and situations regarding their illnesses. I hope many patients will 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 Manufacturers Association of Japan

I think it is important for patients, pharmaceutical companies, medical professionals, government officials, and professionals from each side to have a closer dialogue. I would be happy if we can maintain 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 for medicine. We hope that our voices will continue to be heard. We would like to think together with you.

Mr. Komazawa

In addition to pharmaceutical companies, I think it would be good if we have opportunities to meet face to face with medical professionals and government agencies together and see things from a larger perspective. In addition, patient associations are often run by the parties involved, which is exhausting. I would like to break out of this situation. I hope that I can ask for your help.

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 it as one of the ways to support our lives and livelihood. 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's meeting will be a step toward that end.

Information from the Pharmaceutical Manufacturers Association of Japan: "The Pharmaceutical Manufacturers Association of Japan Industry Vision 2025 Supplement for Patients and Families

The Pharmaceutical Manufacturers Association of Japan (PAPJ) Industry Policy Committee General Policy Subcommittee introduced the "PAPJ Industry Vision 2025 Supplement" and the newly established Advocacy Group of PAPJ.

1. "Pharmaceutical Cooperative Industry Vision 2025 Supplement for Patients and Families

The Pharmaceutical Manufacturers Association of Japan (PAPJ) has set forth its "PAPJ Industry Vision 2025" in January 2016, and in light of various changes in the business environment, the PAPJ has prepared a supplemental version (*) of the Vision 2025.

(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) Utilize "Big Data" to create a society that is easy to live in.

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 for early detection of illness 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 Cooperative will collect information for patients that each member company has and create a system where information on diseases and medicines can be obtained from their websites and other sources.

2. Activities of the Pharmaceutical Cooperative Association (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 (PAL). The word "advocacy" means to express one's thoughts and intentions, and we would like to communicate our aspirations as a pharmaceutical company, as stated 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 continue to expand opportunities for dialogue and collaboration not only in drug development, but also in a variety of other areas, as we work together with patient groups to realize a society that is healthy and easy to live in.

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 perspective of people with intractable diseases and cancer. The seminar provided useful information to the participating patient groups, and was also an opportunity for pharmaceutical companies to think together about drug development for the future of patients.

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