What is the pathway to patient-centric ethical drug development?

Pathway to patient-centric drug development

Shift from patient engagement to public engagement

Healthcare in Japan is transitioning from care that patients passively receive, to one that they independently choose.
This trend is becoming increasingly important, not only in terms of patients’ conducting their own research and openly discussing information with healthcare professionals, but also within drug research and development. Why is collaboration between pharmaceutical researchers and patients or citizens, which was minimal in the past, needed more now? How can this be achieved?
To answer this question and more, we interviewed Dr. Imamura, who is taking on the challenge of creating a place where researchers and patients can learn together.

Dr. Kyoko Imamura

President of the Japan Institute for Public Engagement
Project Professor, IT Healthcare, Graduate School of Pharmaceutical Sciences, The University of Tokyo.


Patient and Public Involvement (PPI) in healthcare has been gaining momentum, but can you tell us what is currently meant by ‘patient-centric drug development’?

In the past, patients had no choice but to wait for drugs to be developed, but now it’s about patients, citizens, and researchers making joint efforts to develop therapies that are in need. For example, when developing therapies for diseases that impair the ability to walk, a successful therapy’s value is not simply about enabling the patient to walk. Listening carefully to patients may reveal that in addition to not being able to walk, they also have difficulty moving their arms. In the past, such ‘patient voice’ information would only reach the family or perhaps the primary physician, but not researchers. In such case, developing a therapy that could improve arm function would still be of enormous value to patients and their families, even if leg symptoms did not improve. As drug developers, we want to both learn more about what patients are having trouble with, and at the same time communicate various circumstances within drug development that we would like the public to understand. Unless both drug developers and patients understand each other and unite, we will not be able to develop valuable medicines that truly meet the needs of patients. Various discussions and efforts to engage patients and citizens in drug development are now underway.

Which disease areas have particularly active patient engagement?

Patient engagement is particularly active in central nervous system diseases, such as Alzheimer’s disease, and in areas that are considered to be intractable or rare diseases. These diseases have diverse symptoms that can be difficult to understand for anyone other than the patients themselves, requiring greater communication. For example, with conditions such as hypertension and high cholesterol, animal models can be used for studies, but studies with neurological, muscular, and mental diseases are difficult to conduct, and thus, in many cases the symptoms are not quantified. So, even if we try to collect data internationally, the standards and methods for collecting data differ, creating a challenge that is quite different from hypertension therapy development.


Is Japan’s drug development involving patients making progress?

Unfortunately, I don’t think so. If you compare Japan’s drug development with that of the West, Japan is quite behind in engaging patients and the public. Japan is a very fortunate country in that it has a tightly regulated insurance and medical administration that allows patients access to a certain level of medical care without having to actively call for it, but in terms of changing the processes that contribute to developing new therapies that solve patient’s problems, it struggles to keep up with the West. Drug development is a science so data is always the most important factor in the process, however patients’ opinions also represent important data. Unless patients are able to express their needs clearly, and their voices are well quantified and aggregated as data, their opinions will not be reflected in the development plans of pharmaceutical companies and medical device manufacturers. Within the global competition for drug development, the Japanese market is limited in size. Unless you sufficiently call out specific needs, it will be difficult to develop drugs that meet needs of Japanese patients. On the other hand, even when patient numbers are small, if we unite efforts to produce high-quality data, we may be able to make a statement to the world from this country. As Japanese, we tend not to be very good at asserting ourselves, but I think this is an area where we really need to step up our efforts.


Why is public involvement emphasized in addition to patient engagement?

We can’t draw a line between patients and citizens since everyone will be sick or injured at some point in their lives. The same is true for health and disease, and it is also difficult to draw a line between them. If we are to have patients’ opinions successfully reflected in drug development, it’s essential that we also win the understanding and interest of the general public that surround patients. Moreover, the development of drugs nowadays costs more money and time than ever before, raising unavoidable questions on the allocation of social resources. Since these resources are necessary to curing diseases, and healthcare always prompts the discussion of how the society will cover the costs, I hope that the general public – not just patients – will participate in this important discussion. We want the general public to take interest in these issues, but it is very difficult to have healthy people take ownership of this issue. I think a better place to start this conversation is by involving family members and neighbors of patients who are suffering from the disease and wondering what to do about it.


Is this why you’ve created a place where people from different backgrounds can learn together?

Yes. In the beginning, I held study sessions for physicians working at pharmaceutical companies. However, as the number of physicians involved in the development process increased, the scope of the project gradually expanded. I’ve now started to connect with people from EUPATI, a European organization that promotes patient and public participation, as well as with people in Japan. This spring, a general incorporated association, PPI Japan, was established in Japan, with Japanese researchers and patient groups taking the lead in introducing EUPATI’s excellent tools to Japan, and promoting PPI (Patient and Public Involvement) in Japan. I would like to contribute to PPI in Japan by drawing on the insights gained from my field of work at the University of Tokyo’s Social Collaboration Course and the recently established Japanese Institute for Public Engagement. I hope that we will all work together to shape therapeutic development for patient-centric drug development that suits the needs of Japanese patients.


What is the key to achieving patient and public participation in Japanese healthcare?

The key is not limited to drug development, but I think it is important to foster leadership that can find clues to solve problems in the field and respond to them. Even in a small group presentation, there are people who naturally act as leaders by listening to what everyone has to say, assigning people to take charge, and presenting a summary. I would like to create a place where we can grow together by sharing our experiences and discussions with people who are in leadership roles in various fields. I believe that this will eventually lead to the creation of a social foundation that will make Japan’s medical care even better for patients by giving them a stakeholder voice that cannot be ignored.
It is with this in mind that we have developed an online learning program with courses that allow people to learn about drug development in a systematic manner that captures processes from entry to exit.


(Source: National Institute of Health Sciences,


We are launching three courses, beginning in October this year, that will help patients, industry and researchers to learn about drug development systematically from beginning to end *.
This holistic approach is designed to address the bias we see when those with specific knowledge and interest in one area tend to apply this to all areas. Participants will be able to cover for things outside of their field of expertise and quickly gain a balanced perspective. The detailed syllabus is open to the public, so people can follow their interests, starting with areas that hold immediate interest and taking related classes in the future. Since it is an online program, people can join from anywhere. Patients, people associated with patients and healthcare, and anyone else who wants to learn together, are welcome to join us.

*Each course will be conducted in Japanese.


We thank Dr. Imamura, for her easy-to-understand explanation on patient participation and insights into its trends in Japan.
COSMO will continue to pursue innovative ways of helping patients’ voices reach the public and wider audiences. We hope that you, our newsletter readers, will join us in spreading the word of Dr. Imamura’s efforts and this online learning program to acquaintances.

(Reiko Hino, Japanese version published on September 10, 2020, English version published on September 25)

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