JPMA Media Forum" Held The Diverse Value Offered by Innovative Medicines: A Case Study of Acute Lymphoblastic Leukemia (ALL) in Children
On May 18, 2026, the “JPMA Media Forum” was held. Under the theme “What Are the Diverse Values Brought by Innovative Medicines—Using Pediatric ALL as a Case Study—,” the event featured presentations by Professor Motohiro Kato of the Department of Pediatrics at the University of Tokyo Hospital, Deputy Head Nurse Yuko Osuki, and patient Mitsuki Sasaki, followed by a panel discussion among the three speakers facilitated by Yasunori Yoshida, Executive Director of the JPMA.On the day of the event, more than 90 participants, including media representatives and member companies, attended both in person and via webcast.
Opening Remarks
Yasunori Yoshida, Executive Director, JPMA
Regarding the current state of the rapidly changing environment surrounding drug discovery, while the primary focus used to be on diseases with a large patient population, such as lifestyle-related diseases, the scope has now expanded to include intractable and rare diseases, and the very concept of treatment is undergoing significant change.Moving beyond an era centered on small-molecule drugs, new modalities—such as antibody therapeutics, bispecific antibodies, antibody-drug conjugates (ADCs), nucleic acid therapeutics, and cell therapy—have emerged one after another, making personalized medicine tailored to the specific pathology of each disease and patient a reality.
It is no longer sufficient to evaluate the value of these innovative medicines solely based on efficacy and safety, as was done in the past. This is because new drugs are beginning to offer value in a broader sense—not only by improving the disease itself, but also by enhancing patients’ quality of life (QOL), enabling them to maintain their daily lives during treatment, improving the quality of care provided by healthcare professionals, and generating ripple effects throughout the entire healthcare system.We believe that to further accelerate drug discovery innovation in Japan, it is essential to establish mechanisms for visualizing, socially sharing, and appropriately evaluating these multifaceted values.
Presentation 1: How Therapeutic Drugs Have Transformed the Management of Pediatric ALL
Prof. Motohiro Kato, Department of Pediatrics, The University of Tokyo Hospital
Acute lymphoblastic leukemia (ALL) is the most common form of childhood cancer and was considered virtually incurable in the 1950s and 1960s.However, thanks to the subsequent establishment of multi-agent chemotherapy, advances in stratified treatment that adjusts treatment intensity according to the disease stage, and the enhancement of supportive care to prevent and alleviate side effects and other discomforts, the disease has undergone a significant transformation and is now one for which a high cure rate can be expected with standard treatment.
Figure 1: Dramatic Improvement in Treatment Outcomes for Pediatric ALL
However, alongside these advances, the challenges posed by conventional anticancer drug therapy have become increasingly apparent. The intensity of chemotherapy has reached its limits, and simply increasing its intensity is no longer sufficient to reduce the remaining incidence of recurrence. Furthermore, chemotherapy often requires prolonged hospitalization and is frequently associated with severe side effects such as mouth sores, infections, hair loss, and organ damage.In pediatric medicine, moreover, because patients have a long life ahead of them after treatment, it is necessary to focus on late complications such as growth, fertility, and secondary cancers. We are shifting from an era where “curing the disease” was the top priority to an era where the goal is to “cure the patient and ensure they live a healthy life.”
Against this backdrop, new treatment modalities such as targeted therapies and immunotherapies have emerged. Targeted therapies, designed based on the molecular mechanisms of the disease, have dramatically improved the prognosis for leukemias with specific genetic abnormalities.Immunotherapies, such as CAR-T cell therapy and bispecific antibodies, tend to have lower recurrence rates and fewer side effects, and are viewed with great promise as new treatment options.
Bispecific antibodies, in particular, are a treatment that harnesses the patient’s own immune system to attack cancer cells by linking leukemia cells to T cells. Unlike conventional cytotoxic anticancer drugs, their impact on normal cells is limited, resulting in fewer side effects such as infections, stomatitis, and hair loss; they have also demonstrated efficacy against recurrent and refractory cases.Overseas, these therapies are being adopted not only for recurrent cases but also for newly diagnosed cases, beginning to transform the traditional chemotherapy-centered treatment paradigm.
Furthermore, the emergence of these new drugs is significant not only for improving survival rates but also for expanding the possibilities for patients to undergo treatment while maintaining the quality of their school and home lives. With the increase in treatment options, it has become increasingly important for healthcare providers not to unilaterally impose what they consider to be the “best” treatment, but rather to work together with patients and their families to plan treatment based on what matters most to them.
Figure 2: ALL Treatment: Summary
Presentation 2: Patients’ and Families’ Expectations for Pharmaceuticals
Ms. Mitsuki Sasa, a patient with Ph+ALL*
- *Ph+ALL=Philadelphia chromosome-positive acute lymphoblastic leukemia
I was diagnosed with leukemia in the third grade of elementary school and was discharged after about a year of inpatient treatment. Although I went into remission afterward, the disease relapsed in May of my senior year of high school, and I had to face another long course of treatment. During that relapse treatment, I received therapy with a bispecific antibody.
The greatest benefit was “being able to go outside during a time when I normally wouldn’t have been able to.”During my final summer of high school—a time I would normally have had to spend in the hospital—I was able to make the bench for the baseball team’s summer tournament and serve as the base coach. I was also able to participate in preparations for the school festival and hang out with friends. Each of these seemingly ordinary moments served as a great source of encouragement as I prepared to face the grueling bone marrow transplant that lay ahead.
Figure 3: Positive Developments
Furthermore, treatment with bispecific antibodies resulted in fewer side effects, and it was a major benefit that I was able to proceed to the next phase of treatment without experiencing a significant decline in physical strength. It was also impressive that, compared to conventional IV drips, I could move around relatively freely while carrying the device, which expanded the range of my activities both inside and outside the hospital.Now, several months after being discharged from the hospital, I continue to visit the hospital once every few months for follow-up appointments. I am studying sports and health science at university and have recovered enough to go to the gym again, just as I did before.
On the other hand, as a patient, I did experience some inconveniences, such as the size and operating noise of the treatment device, the need to visit the hospital whenever there was a device malfunction or to refill the medication, and the fact that the long tube tended to get caught on things in my daily life.Even for me, with a commute of about an hour from home to the hospital, it felt like a burden, so I thought it must be an even bigger problem for patients living farther away. With that in mind, I hope to see the devices become even smaller and quieter, the establishment of systems that allow local medical institutions to provide care, the development of mechanisms that enable patients to receive treatment with peace of mind at home or while out and about, and the development of medications that further reduce side effects and long-term complications.
Figure 4: Expectations for Medical Products
During treatment, I sometimes looked up information online out of anxiety about side effects and my future prognosis, which only made me more frightened. However, what helped me overcome that anxiety was being able to talk about my feelings with my doctors, nurses, childcare workers, friends, and family.Looking back, I realize that talking to others helped me sort through my feelings and look forward, and I am grateful that I am able to live a healthy life today thanks to the support of so many people.
Figure 5: In Conclusion
Topic 3: The Value of Pharmaceuticals from the Perspective of Nursing Practice
Ms. Yuko Osuki, Assistant Head Nurse, Department of Pediatrics, The University of Tokyo Hospital
In 2006, pediatric leukemia treatment typically required patients to be hospitalized for a long period—about one to one and a half years—and overnight stays outside the hospital were generally permitted only for a few days during the New Year’s holiday period.Radiation therapy was often part of the treatment, so it was necessary to keep an eye on other challenges associated with the treatment, such as skin problems and effects on cognitive development. Furthermore, the physical and emotional burden was extremely heavy, not only for the patients themselves but also for their accompanying family members.
In contrast, thanks to the introduction of stratified treatment and new modalities, while hospitalization still lasts about one year, patients now have the opportunity for temporary discharge approximately two months after admission. Furthermore, between treatment sessions, opportunities for overnight stays at home can be arranged while monitoring their condition.In other words, the setting for treatment has expanded beyond the hospital to include the home and school. However, patients and their families must now take on responsibilities such as managing the central venous (CV) catheter, operating the portable pump, preventing infections, and responding to complications on their own, which means that nursing care supporting home-based treatment now requires a new level of expertise.
Figure 6: Changes Brought About by the Introduction of New Modalities
There are various forms of detailed support to facilitate continued home care, such as hands-on instruction using pamphlets and dolls, information sharing between inpatient and outpatient units, assistance with showering, battery replacement, heparin locks, and IV line protection. We provide tailored support for each individual while carefully assessing whether the patient can manage these tasks independently or if family members can provide the necessary assistance.
Furthermore, new treatment modalities require new approaches to managing side effects. With bispecific antibodies and CAR-T cell therapy, there is a risk of sudden deterioration, such as cytokine release syndrome (CRS); therefore, enhanced monitoring and the establishment of emergency response systems are essential during the initial administration.In short, as medications evolve, nursing care becomes increasingly sophisticated, shifting from mere routine care to a comprehensive role that includes education, managing side effects, lifestyle support, and family support.
Thanks to these advances in treatment, school-age patients are finding it easier to maintain connections with friends and school. We believe it is of great significance that life beyond treatment—such as earning credits and advancing to higher education for high school students, or spending time with siblings for young children—is being safeguarded.On the other hand, there are still aspects of long-term late complications and the impact on fertility that are not yet fully understood; therefore, we believe that follow-up systems and information sharing that take into account the long life ahead after treatment are necessary.
Figure 7: Expectations for Pharmaceutical Companies
Panel Discussion
The discussion explored the value of treatment for patients, its impact on families, the role of healthcare providers, and challenges within the social welfare system from a multifaceted perspective.Reflecting on the presence of Dr. Kato and Dr. Osuki, Mr. Sasa said, “When they told me, ‘It’ll be okay,’ I felt a sense of reassurance that made me truly believe it would be okay,” explaining that it was not just the healthcare providers’ words but their very presence that served as a major source of support.
In response, Dr. Kato emphasized that when interacting with patients and their families, his focus is not simply on telling them, “It’ll be okay.” He stated that it is important to clarify what they do not need to worry about and what they should be cautious of, and to explain these points carefully, clearly, and repeatedly until they are fully understood.He also pointed out that truly effective treatment cannot be achieved without understanding not only the illness itself but also what matters most to the patient and their family.
Mr. Osuki also explained that the first step is to determine whether the root of the patient’s anxiety lies in the illness itself or in school and daily life.He added that they provide support in collaboration with teachers from the hospital-based school and physicians. In Mr. Sasa’s case, the treatment schedule was adjusted in advance to accommodate his wish to “participate in his final competition.” These efforts demonstrated that the medical team shared not only a focus on treatment outcomes but also an understanding of what was important in the patient’s life.
The discussion further confirmed that the value provided by new medications extends beyond the patients themselves to include alleviating the psychological burden and improving the quality of life for their families.The ability to continue treatment on an outpatient basis or at home is considered to have a significant positive impact on patients’ overall quality of life, such as making it easier for families to spend time with other children. On the other hand, new burdens have also emerged, such as increased frequency of hospital visits, the management of medical devices, and travel from distant locations; how to address these challenges was identified as a key issue for the future.
Furthermore, the expansion of treatment options is changing the nature of medical decision-making. Mr. Kato summarized that while discussions used to tend to focus on whether a treatment “works or doesn’t work” or “has side effects or doesn’t,” it is now necessary to consider quality of life, the patient’s wishes, and even the time and experiences they do not want to lose during treatment.It was emphasized that rather than imposing what healthcare professionals consider to be the “best” course of action, there is a need for an approach that selects treatments in line with the values of patients and their families.
It was also pointed out that drug development for rare pediatric diseases remains a challenge due to difficulties in verifying efficacy and safety, as well as the need to develop dosages and formulations suitable for children; as a result, “drug lag/loss”—where drugs approved overseas cannot be used domestically—remains a persistent issue.Furthermore, the development of new drugs with fewer side effects, the establishment of long-term post-treatment follow-up systems, and the enhancement of platforms for information sharing are also crucial. To resolve these challenges, it is essential to create a system that ensures patients receive the medications they need by strengthening development and delivery frameworks through collaboration among industry, academia, and government.
Concluding Remarks
What became clear throughout the forum was that the value of innovative pharmaceuticals cannot be measured solely by reducing the severity of disease, preventing recurrence, or increasing survival rates.Through the accounts of doctors, nurses, and patients alike, it was concretely demonstrated that this value extends into the realm of daily life—such as patients being able to attend school, spend time with friends, share everyday moments with their families at home, and look forward to a positive future beyond treatment.
Society’s collective understanding and appropriate evaluation of these multifaceted values will form the foundation for the next wave of innovation. The discussions surrounding pediatric ALL have prompted a reexamination of the very nature of pharmaceutical value assessment and have once again highlighted the need to consider drug discovery, medical care, nursing, patient support, and policy design as an integrated whole.
Innovative pharmaceuticals are no longer merely tools for curing diseases; they are evolving into pillars that support patients’ lives. Society is now faced with the critical question of how to recognize this value and use it to drive the next wave of innovation.
From left: Mr. Kato, Mr. Osuki, Mr. Sasaki, Mr. Sasaki’s mother, and Executive Director Yoshida
(Pharmaceutical Industrial Policy Committee, Industrial Promotion Subcommittee: Takuro Matsuda, Noriko Tomita, Tomohiro Murakami)