From JPMA Message from the Executive Director

My name is Kazuhiko Mori, and I was appointed Executive Director of the Japan Pharmaceutical Manufacturers Association (JPMA) on October 1, 2020. I have been physically weak since childhood, and although fortunately I have not had any serious illnesses, I have grown up with a lot of "pharmaceuticals" in my life, and have always had an interest in pharmaceuticals. I went on to study pharmacy at university and began my career at the former Ministry of Health and Welfare (now the Ministry of Health, Labour and Welfare), where I worked in the pharmaceutical review process, and so on.

Within a few years of my employment, I was involved in the approval review of recombinant human growth hormone and recombinant human insulin, which were introduced in the 1980s as a result of biotechnology developed in the 1970s. From my experience in the new drug approval review process in the late 1980s, I have come to understand that no matter how remarkable the progress of science and technology, the path to application and practical use is never easy. When we reviewed the application materials for new drugs that had gone through the trouble of filing an application, we found inconsistencies with the original data supporting the application materials, problems with the design of the clinical trials, and disappointing results that were insufficient to demonstrate efficacy and safety, which was very frustrating. One day, I had an opportunity to talk with people who had been involved in clinical development at companies for many years, and I asked them about the problems they were aware of and the questions they were simply feeling as a practitioner in charge of new drug review. As I was explaining that I often wonder why the clinical trial results of new drug applications are so poor, something occurred to me. The idea was that instead of the reviewers issuing a negative recommendation after an application is submitted, it would be better to explain in advance what kind of quality and content of clinical trial results they expect from the company developing the drug, and to "consult" with them to see if this is feasible, so that development can proceed smoothly and the review process can be facilitated. I still remember one of the companies I was talking with saying, "I see, 'consultation' - that's very interesting.

The reason why the regulatory authorities and pharmaceutical companies consult with each other on clinical development is not only to ensure that their work proceeds smoothly and smoothly, but also to ensure that excellent new drugs are developed and delivered to patients suffering from illnesses in an appropriate and timely manner. When the Pharmaceutical Affairs Law was revised in 1996, the pharmaceutical industry requested that clinical trials be improved, and the Clinical Trial Guidance Department of the former Pharmaceutical Affairs Organization of Japan (PAL) began providing clinical trial consultation services in 1997. At that time, the U.S. Food and Drug Administration (FDA) and regulatory authorities in European countries were also providing various guidance and advice to pharmaceutical companies on clinical development at meetings of the International Conference on Harmonization of Pharmaceutical Regulations (ICH), which was promoting international harmonization of pharmaceutical regulations, and at the Drug Information Association (DIA) annual meetings in the United States and Europe. I learned that the U.S. Food and Drug Administration (FDA) and regulatory authorities in European countries also provide guidance and advice to pharmaceutical companies on clinical development. In 2002, I traveled to the European Medicines Agency (EMEA, now EMA) to introduce Japan's clinical trial consultation system and observe how the EMA receives consultations from pharmaceutical companies. It was very instructive for me to understand how important and beneficial it is for various parties with different positions and expertise to communicate well with each other, which is common across countries.

At the same time, the human genome project was almost completed, and expectations for the realization of epoch-making treatments through advances in life sciences were growing, such as the appearance of a series of molecular-targeted therapies for cancer, etc. The 21st century will usher in the era of biopharmaceuticals, represented by antibody drugs, and with the results of the human whole genome analysis project, etc., it is expected that epoch-making new drugs will become available in the near future. The 21st century has ushered in the era of biopharmaceuticals, represented by antibody drugs, and the results of the whole human genome analysis project, among others, have led to a succession of epoch-making new drugs. In addition to surgery, radiotherapy, and chemotherapy, cancer immunotherapies such as anti-PD-1 antibodies and CAR-T have emerged as the fourth cancer treatment, and gene therapy drugs that can cure serious genetic diseases with a single administration have appeared.

On the other hand, the development of new drugs remains extremely difficult, and the success rate from the basic research stage is said to be 1 in 30,000. Naturally, the more development failures there are, the higher the cost of development becomes. Moreover, the production cost of biopharmaceuticals is still higher than that of conventional small-molecule chemical syntheses, and there is strong concern that the impact of extremely expensive new drugs on national healthcare costs could be devastating, even though they have revolutionary therapeutic effects. Various solutions have been proposed to address the soaring costs of new drug development. In particular, improvements are being considered that would greatly streamline the clinical development process and provide greater benefits to patients. For example, ideas to utilize real-world data, such as clinical trials using patient registries rather than the randomized controlled trials that have been the golden standard for the past 70 years, and study designs called "master protocols" that test multiple candidate drugs in multiple diseases, are being considered for clinical development and post-marketing surveillance. and others are now being applied to clinical development and post-marketing surveillance. There is also a rapid movement to apply information technology, digital devices, and rapidly advancing artificial intelligence (AI) to clinical trials. Clinical development methods continue to evolve on a scale and at a pace previously unimaginable. Today, I am keenly aware that Japan's clinical development environment also needs to evolve further to keep up with global changes.

In the past, and even today, many researchers, medical professionals, and pharmaceutical companies are tirelessly working to bring innovative products into practical use to provide the treatments that patients suffering from illnesses have been waiting for with great anticipation. I have had the opportunity to speak with various domestic and international pharmaceutical companies involved in the clinical development of new drugs, and they all want to deliver products that meet the expectations of patients as quickly as possible. The results of important clinical trials of breakthrough new drugs are often published in international medical journals such as The New England Journal of Medicine (NEJM), and the global medical community is engaged in lively discussion and commentary on them. New drugs are now global products that patients and healthcare professionals around the world are keenly interested in and expect to use as soon as possible.

There are still many diseases in need of superior treatment, and patients around the world are waiting for the arrival of new breakthrough drugs. As a person who shares the same aspiration, I would like to do my utmost to bring epoch-making new drugs from the Japanese pharmaceutical industry to patients as quickly as possible through rational development.