“JPMA Media Forum” Held The Diverse Value of Pharmaceuticals: “The Diverse Value Brought by New Modalities—Using Spinal Muscular Atrophy (SMA) as a Case Study”

On March 7, 2025, the “JPMA Media Forum” was held at Muromachi Mitsui Hall & Conference (Chuo Ward, Tokyo).Under the theme “The Diverse Value Brought by New Modalities: The Case of SMA (Spinal Muscular Atrophy),” the event featured a lecture by Yuko Oyama of the SMA Family Association, Reiko Arakawa of the National Center for Global Health and Medicine Hospital,Rei Goto of Keio University, followed by a panel discussion among the three speakers facilitated by Kazuhiko Mori, Executive Director of the JPMA. On the day of the event, more than 120 participants, including media representatives and member companies, attended both in person and via webcast.

Scenes from the Forum

Background of the Forum

In recent years, drugs utilizing new drug development modalities—such as nucleic acid therapeutics and gene therapies—have emerged, making a significant impact on the treatment of conditions that were previously difficult to treat, such as cancer and hereditary diseases.In this forum, we focused on SMA and invited three opinion leaders to speak about the challenges posed by the disease and various examples of the value that pharmaceuticals bring in addressing them. The following is a transcript of their presentations.

 

Opening Remarks

Kazuhiko Mori, Executive Director, JPMA

In recent years, drug discovery has diversified beyond small-molecule chemical synthesis to include biopharmaceuticals and other modalities, expanding treatment options ( Figure 1 ).

“Evaluation” is crucial for fostering such innovation; it is necessary to assess and evaluate drugs from various perspectives. It is precisely because of this evaluation that even better treatments can be developed in the future.In particular, patient feedback is the most important factor and serves as a key motivator for pharmaceutical companies.

While scientific evaluation must be conducted objectively and quantitatively, there are cases where measurement is difficult. Furthermore, since evaluations differ depending on the stakeholder’s perspective, determining how to integrate these perspectives is also a critical challenge.

Figure 1. Diversification of Modalities



In 2025, the JPMA released its “Vision for 2035.” This vision consists of three pillars, and the first pillar—innovation—includes collaborating with patients.We believe it will be increasingly important in the future for patients and various stakeholders to collaborate more closely than ever before to drive new drug discovery innovations ( Figure 2 ).

Figure 2. JPMA’s Vision for 2035


Presentation 1: How Therapeutic Drugs Have Transformed SMA Care

Dr. Reiko Arakawa, Chief, Department of Clinical Genomics, National Center for Global Health and Medicine Hospital

SMA: A Hereditary Disease That Was Once Fatal in Infancy


SMA is a disease caused by the loss of function of the SMN1 gene, leading to the loss of anterior horn cells in the spinal cord and muscle atrophy ( Figure 3 ). It is characterized by severe weakness, particularly in the trunk muscles and muscles close to the body, resulting in extreme weakness in the muscles around the neck, shoulders, and lower back.Consequently, in severe cases, infants are unable to hold their heads up or learn to crawl. With an incidence rate of 1 in 20,000 live births, it is the most common single-gene hereditary disorder with a fatal course in infancy; without treatment, neurodegeneration progresses.SMA is classified into clinical types 0 through IV, with Type I accounting for approximately 30% of patients, Type II for approximately 40%, and Type III for approximately 20%. Patients with Type I experience a decline in respiratory function during infancy, leading to respiratory failure.Consequently, it is generally considered difficult for them to survive past the age of two without the use of a ventilator.

Figure 3. What is SMA?

Treatment That Transforms Lives Beyond Just Improving Symptoms


The history of this disease began with a research paper published in 1980, and the causative gene was identified in 1995. Subsequently, genetic testing was introduced in Japan in 1996, establishing a clear understanding of the disease. With this clearer understanding, the SMA Family Association was founded in 1999 ( Figure 4 ).

Figure 4. From gene identification to the establishment of a disease concept, drug development, and the formation of patient support groups

In the double-blind, controlled trial I participated in, the trial was conducted without participants knowing which child was receiving the actual drug and which was receiving a placebo. While one child’s breathing continued to deteriorate to the point where a ventilator was required, the other child was able to raise their hands and play without needing a ventilator.I was deeply moved not only by the treatment’s efficacy but also by the fact that it had virtually no side effects, improved muscle strength in children with weak muscles, and transformed the lives of these children.

Currently, there are three types of SMA treatments: gene therapies, nucleic acid therapeutics, and small-molecule drugs. Each treatment has its own unique characteristics and plays a significant role in SMA treatment.

Newborn Screening—Aiming for Earlier Treatment


Newborn screening has demonstrated the benefits of early treatment for SMA. If treatment is not started until the patient requires a ventilator, it becomes difficult to wean them off the ventilator later. In Type I SMA in particular, neurodegeneration progresses rapidly, leading to a significant decline in motor function.According to data from the United States, if treatment begins within 14 days of birth, most children can learn to walk, which dramatically changes their lives. While this requires efforts from both the medical community and society at large, against this backdrop, the family association’s dedicated efforts are driving progress toward making newborn screening a publicly funded program.

Furthermore, in the United States, newborn screening will be established as a national program starting in 2024.The benefits of early treatment through newborn screening are clear: even among patients with two copies of the SMN2 gene—who are considered to have severe disease—76% can learn to walk if treated before symptom onset. After symptom onset, this rate drops to just 19%. This has a significant impact on patients’ lives (Figure 5.

Figure 5. The Effects of Early Treatment Through Newborn Screening

Meanwhile, in Japan, the Agency for Children and Families has launched a pilot program for newborn mass screening.While expanded newborn screening covers nearly all prefectures, not all children are eligible to receive it, and in some regions, it must be paid for out-of-pocket. As a result, there are still many children whose conditions go undetected and who develop symptoms. It is hoped that this program will be implemented uniformly throughout Japan as a public health initiative ( Figure 6 ).

Figure 6. Status of Expanded Screening Implementation in Japan

Ongoing Challenges


If treatment can be started within 14 days of birth, it can significantly change these children’s lives; however, conducting examinations, making diagnoses, and explaining treatment options within this timeframe is difficult, and the limited number of medical institutions capable of providing this care poses a major challenge.

Furthermore, medical care is not unnecessary even after treatment. Children treated for Type I SMA often try their hardest to sit up despite their frail bodies, which can lead to the progression of scoliosis and joint dislocations. While surgery involving the insertion of screws is performed in the United States, stainless steel screws are not available in Japan; consequently, dislocations frequently occur even after the most expensive treatments.Furthermore, support for families is essential. The shock of learning that a child they believed was born healthy has been diagnosed with SMA is immense. It is also crucial to strengthen genetic counseling systems to clearly convey to parents that the availability of a treatment and the ability to diagnose the condition are positive developments, and to help them come to terms with the situation ( Figure 7 ).

Figure 7. Team-Based Care for SMA

The integration of cybernetic therapies represents a new challenge aimed at improving motor function and enabling walking. Combining devices such as robotic suits with medications can help reduce the burden on caregivers and support patients’ independence. Therefore, there is a need to develop compact devices suitable for children.Furthermore, the inclusion of SMA in newborn screening programs and the widespread adoption of genomic analysis have expanded treatment options. It is crucial to explore appropriate treatment methods through dialogue with society, including the appropriate use of high-cost medications.

 

Topic 2: Hope Brought by New Modalities—From the Perspective of Families of SMA Patients—

Ms. Yuko Oyama, President, SMA (Spinal Muscular Atrophy) Family Association

SMA and Treatment


The earlier SMA manifests, the more severe it is. Type I, which manifests by 6 months of age, is said to result in the death of 90% of patients by age 2 without respiratory support.For a long time, SMA was considered an incurable disease, but with the introduction of three innovative treatments since 2017 ( Figure 8 ), it has become a curable—and preventable—disease.

Dramatic Improvement in Symptoms

In the era before treatments were available, children with SMA required respiratory support within a few weeks of birth and were unable to sit or stand for the rest of their lives, leading to a bedridden existence.However, there are cases where children—even those with such severe disease that they required respiratory support as early as two weeks after birth and were expected to die within a few months—began treatment at six weeks of age and have since recovered to the point where, at age five, they are now off the ventilator and practicing walking with a walker.This is a phenomenon that would have been absolutely unthinkable with SMA in the past, and such a dramatic change has brought immense hope to families with children who have SMA.

Figure 8. SMA Treatments

Below, we present the case studies of patients and their families introduced during the lecture.

Positive Energy and Unwavering Motivation

We have received a message from parents who have two sons with SMA. Their older son received a definitive diagnosis at 10 months of age and began treatment at 11 months. Their younger son, on the other hand, underwent prenatal diagnosis and began treatment on his fifth day of life.

  • A Message from My Father

    Thanks to gene therapy, my second son—who couldn’t even roll over—is now able to sit up and propel his wheelchair. He works hard at rehabilitation and spends happy days with friends at preschool and the developmental support center. He loves cars and has big dreams for the future. Being able to move his body on his own has boosted his confidence, and he has started trying various sports, including baseball.Thanks to early treatment, my third son is developing at a level appropriate for his age, including in terms of motor skills. I feel that the healthy days we enjoy are made possible by this treatment. For our family as well, seeing our second and third sons grow up healthy after undergoing treatment seems to give us the energy to approach our daily lives with a positive outlook.

  • A Message from a Mother

    We realized later that, before treatment, our second son had been suffering because he couldn’t move his body freely. While we were shocked when we learned of the disease, we were even more relieved to have a clear diagnosis. It happened to be the year gene therapy was approved, and we feel grateful that he was born in an era where such treatment is available.Over the four years since his treatment, the things he can do have gradually increased, though new challenges—such as scoliosis—will likely arise in the future. However, I feel that neither he nor we as parents will ever give up on standing on his own two feet and walking—and that is what keeps us motivated in our rehabilitation efforts.Our third son is growing up to be a truly carefree and cheerful child. As parents, we constantly worry and feel anxious about the fact that our child will have to live with SMA for the rest of his life, but we hope that, with the support of those around us, we can stay positive and truly feel him growing as he matures.

No Longer Needing Medical Care: A New Dream—“I Want to Walk!”

The introduction of pharmaceuticals has been of great value not only to infants and young children but also to patients whose symptoms progressed some time after the onset of the disease. The middle school girl who courageously sent us a message is one such patient.

  • Messages from Middle School Students

    I started treatment seven years ago, and now—albeit for short periods—I can sit up on my own and lift my legs slightly. I’m improving little by little, but there are still many things I want to do and skills I want to master. In the future, I hope to be able to walk, jump rope, and do pull-ups.

She used to need a ventilator at night, but thanks to treatment, she is now able to spend more time without it. Additionally, while she previously received nutrition through a gastrostomy tube, she is now able to eat almost entirely on her own. She has also developed a new dream of walking and is working very hard on her rehabilitation, including practicing walking with a robotic suit.

Expanding Opportunities for Education and Employment

This high school student began treatment 10 years after the onset of his condition. He operates a communication device using a switch on his fingertip; while he could previously press the switch only 10 times per minute, treatment has enabled him to press it up to 33 times per minute. This has allowed him to operate the device freely, and he has now started working at a café, where he operates a robot.He operates the switch with the fingers of both hands, using two iPads and a computer simultaneously to guide customers via a robot installed in the café. Even if the visible changes are small, these small changes have enabled him to gain opportunities for education and employment, opening up vast possibilities for his future.

Slowing Disease Progression

Therapeutic drugs are also improving the quality of life for adult patients. Here is a message from a woman with Type II SMA. Thanks to the treatment’s effects, her symptoms are progressing more slowly, and she reports experiencing improvements in and maintenance of her functional abilities.

  • Message from an Adult Woman #1

    I have Type II ALS. I use a ventilator when I sleep and an electric wheelchair when I go out. I require 24-hour assistance from my family and caregivers, and at home, I lie in bed and draw pictures on my computer. I started taking my medication in January 2022, and it has now been three years.Before taking the medication, my hand would get tired very quickly just from holding a spoon, but two weeks after starting it, I was able to finish a whole cup of yogurt on my own. When writing, I used to run out of strength after just one line, but now I can write about ten lines—I was so happy when I was able to write a letter to a friend.There haven’t been any major changes since then, but I feel like the progression of my symptoms has slowed. Even if there aren’t any major physical changes, simply slowing the progression of the disease is a huge benefit for me. Slowing the progression means I have more time to pursue my dreams and goals. I plan to keep doing my best every day, with the help of my medication and the support of those around me.

In this way, she works drawing illustrations on her computer while operating the mouse from her hospital bed.

Promoting Social Participation

This is another woman who began treatment several decades after the onset of her condition.

  • Message from an Adult Woman #2

    He has Type II SMA and uses a ventilator 24 hours a day. I usually work remotely as a public relations representative for the SMA Family Association. We started SMA therapy in the fall of 2021, so this is our fourth year. Shortly after starting the treatment, his abdomen began to move, and his post-meal stomachaches disappeared as if by magic.Regarding my speech, a university professor who studies the voice told me that my speech has become smoother than before and that the clarity of my voice has improved. Additionally, the joints in my fingers and toes have become more flexible, and I don’t get tired as easily.The benefits of starting the SMA treatment include being able to enjoy delicious meals with friends, participate in university research, use a computer smoothly, and have more opportunities to engage in society. I would like to continue taking the SMA treatment going forward.


Although she is bedridden, her health has stabilized thanks to treatment, and she is actively participating in society—for example, by operating a computer with her fingertips. She has become an indispensable member of our organization.

We have also received numerous messages of gratitude regarding the treatment (Figure 9 ).

Figure 9. Voices of Patients and Their Families

Future Challenges

While the value of this treatment is immeasurable, challenges remain. The top priorities include establishing systems—such as newborn screening—to enable early diagnosis and treatment, as well as standardizing SMA treatment nationwide and eliminating regional disparities. Furthermore, quantitative assessments based on actual conditions are necessary.Currently, physical function is assessed using the World Health Organization (WHO) Movement Milestones; however, new assessment criteria are needed to accurately evaluate the impact that even subtle changes have on patients’ lives.

Furthermore, it is necessary to calculate an appropriate cost-effectiveness ratio that takes into account not only medical expenses but also costs related to education and welfare, as well as lifetime income for both the patient and their family. Quality of life—including reductions in the need for medical care and daily living assistance, as well as improvements in productivity resulting from treatment—must also be included in the evaluation. Additionally, since patients and their families are seeking further enhancements in effectiveness, we look forward to future developments.

In Conclusion


Our Association (Figure 10 ) was established in 1999 and celebrated its 25th anniversary in 2025. The organization currently has approximately 300 member families, ranging in age from 0 to 70 years old, and is characterized by its diverse membership—from those requiring ventilators to those with no symptoms at all.The association’s activities include organizing social gatherings and study sessions, participating in academic conferences, publishing a newsletter, and providing consultation services to members. Furthermore, over the past decade or so, the association has been focusing on advocacy efforts, collaborating with members of the Diet and others to continuously submit requests to the government—such as for the approval of new drugs and the nationwide implementation of free newborn screening.

Figure 10. About the SMA Family Association


Presentation 3: The Value of Pharmaceuticals and Their Evaluation

Professor Ryo Goto, Graduate School of Business Administration, Keio University

Rising healthcare costs, particularly those associated with high-cost pharmaceuticals, are drawing attention

The rise in medical expenses is a social issue, and high-cost pharmaceuticals and medical technologies are sometimes viewed as problematic. According to data from the Federation of Health Insurance Societies (Kenporen), the number of claims exceeding 10 million yen per month is increasing; however, such high-cost claims represent only a small fraction of the total.The total amount is approximately 40 billion yen, which represents less than 1% of the National Federation of Health Insurance Societies’ total annual medical expenses (approximately 5 trillion yen). While attention is focused on the high cost per claim, the fiscal impact on overall medical expenses is not particularly significant.

Trends in Japan’s Medical Expenses


Regarding social security benefits, Figure 11 shows the trends in the ratio of medical expenses and pensions to GDP. The definition of “medical expenses” here differs slightly from that of national medical expenses; it includes benefits funded by taxes and other sources outside of insured medical care, but does not include out-of-pocket costs borne by patients.Pension expenditures have remained stable since 2010 and have recently shown a downward trend. Healthcare expenditures increased until around 2010 but, excluding the COVID-19 period, did not grow significantly in the latter half of the 2010s. From a fiscal perspective, it is important that benefit expenditures remain within the GDP growth rate, as tax revenues and insurance premium revenues also increase in tandem with GDP growth.

Figure 11. Trends in Medical Expenses (Based on Social Security Benefits)

Looking at the growth rates of healthcare costs, pensions, and GDP over time, both healthcare costs and pensions exceeded GDP growth during the 2000s. This indicates a period of low economic growth.In the 2010s, while the growth rate of pension expenditures stabilized due to measures such as the application of the macro-slide mechanism, the growth in healthcare costs was notable. Subsequently, healthcare costs increased further due to the impact of the COVID-19 pandemic.

Going forward, nominal GDP (GDP not adjusted for inflation) is projected to grow by 2–3%. If healthcare costs increase by only about 1%, this would be less than the growth in nominal GDP, meaning that, in real terms, healthcare costs would be reduced. Furthermore, the number of inpatients and outpatients has been on a long-term downward trend, so the demand for medical care is not expected to increase significantly.

Considering these points, the rise in healthcare costs is not necessarily a crisis; rather, it is important to take the time to discuss how those resources are allocated. Of course, using healthcare funds efficiently is crucial, so we need to consider what constitutes a truly valuable pharmaceutical product.

Evaluating the Value of Pharmaceuticals

The process of evaluating the value of health technologies, such as pharmaceuticals, and utilizing that evaluation to inform policy and other decision-making is known as “Health Technology Assessment (HTA),” and the scope of health technology values considered in HTA is expanding. I would like to emphasize that cost-effectiveness is merely one evaluation criterion in HTA—it is not the only factor.In HTA, the evaluation of pharmaceuticals is based primarily on three value dimensions: “health improvement,” “economic value,” and “other values.” A current challenge is that these values are not being sufficiently taken into account.

How Should We Evaluate Diverse Forms of Value?

Regarding the evaluation of diverse values, the first step is to identify the elements of value and measure them. Next, by actually applying the measured indicators to health technology assessment and subsequently reviewing those indicators, a more appropriate evaluation can be achieved. This is a process that should be carried out collaboratively by all stakeholders, not just health economics experts. However, this remains a challenge in Japan today.The evaluation of diverse values is an ongoing process; the fact that evaluation and quantification are not yet fully developed does not mean we should ignore these considerations.

On the value of improved health, economic value, and other forms of value

Regarding the value of health improvements, easily measurable indicators such as efficacy and safety, as well as life expectancy and certain aspects of quality of life (QOL), are currently used in Health Technology Assessment (HTA). However, it has been pointed out that these alone are insufficient, and efforts are now underway to incorporate assessments of well-being and capabilities as well.

Economic value includes not only the medical costs of treatment and public long-term care expenses but also the value of the time (productivity) of patients and their families. The impact on the broader economy—not just on patients and their families—is also an important aspect of economic value.

Regarding other forms of value, various concepts have been proposed, and efforts to clarify these concepts are underway. Many forms of value remain unquantifiable. For example, the “dissemination of science” effect—where treatments may emerge in the future as medical technology advances, even if the immediate health benefits are limited—and the hope derived from even a small possibility have been proposed as value categories.

Evaluation Criteria for Pharmaceuticals in HTA: A Summary of the Current Situation

Cost-effectiveness analysis currently evaluates only a portion of the value derived from health improvements and economic value; quantitative assessment of other forms of value has not yet been established.

Regarding the “consideration” of diverse values in Health Technology Assessment (HTA), it is widely recognized that “cost-effectiveness” is only one part of these diverse values. For values that are difficult to measure at this time, decisions are made after qualitatively “considering” them.Furthermore, case-by-case considerations are made, and in some cases, consensus is gradually reached among the parties involved, leading to the establishment of “rules for consideration.” While cost-effectiveness can be quantified, I believe that recognizing its limitations and engaging in collective deliberation will lead to more appropriate decision-making and improved quality.

Regarding SMA Gene Therapy Drugs

In the United Kingdom, this gene therapy was recommended by the HTA agency approximately one year after its approval. At that time, its cost-effectiveness was uncertain and had not been calculated, but it was recommended due to its significant benefits and value. Subsequently, the cost-effectiveness analysis was reviewed, and a new evaluation was conducted.Although it could not be said to be highly cost-effective, it was still determined to have significant value that could not be measured by cost-effectiveness alone, and it was recommended as a technology for rare diseases.

Are there other elements that are being evaluated as part of a broader value framework?

Values other than cost-effectiveness that can be quantified are gradually being evaluated, and qualitative aspects that cannot be quantified are also being actively taken into consideration.

Recently, the impact on family members’ health (spillover effects) has been recognized as a broad value component. Specifically, the impact of having to give up work due to nursing or caregiving responsibilities (impact on productivity) and the direct impact of nursing and caregiving on health (impact on quality of life) have been highlighted, and there is a growing movement to actively evaluate these factors.

Panel Discussion

Facilitator: Kazuhiko Mori, Executive Director
Panelists: Ms. Reiko Arakawa, Ms. Yuko Oyama, Mr. Ryo Goto


Q.SMA治療の進歩の中、新たに生まれてきた課題について改めて教えてください。

  • Mr. Arakawa

    From the perspective of healthcare professionals, until now, treatment for SMA was limited to monitoring the progression of the disease, and options such as rehabilitation were limited.However, the range of interventions available for SMA has now expanded significantly. Consequently, if there is a shortage of healthcare providers, even the best treatments will not reach patients. The burden may be particularly heavy in rural areas where there is a shortage of pediatric neurologists, making it crucial to secure sufficient personnel. Furthermore, I believe this issue requires a nationwide effort of mutual support, along with new forms of collaboration and systems unlike anything seen before.

  • Mr. Oyama

    There are indeed many challenges. Since the nature of these challenges is completely different before and after a treatment becomes available, it is important to set priorities and resolve them one by one in a systematic manner.The top priority is to ensure early diagnosis and early treatment, connect every single patient to treatment without leaving anyone behind, and save lives. To achieve this, collaboration among various healthcare professionals is essential. Additionally, the additional medical care and rehabilitation necessary for children who have received treatment to grow up healthy and vibrant, as well as an understanding of the growth and development of both the children and their families, are also high priorities.

Q.早期診断とタイムリーな治療の重要性は理解しました。診断を迅速に行い、早期に最適な治療を行うことで効果が最大化される点について、価値評価の観点からポイントを教えてください。

  • Mr. Goto

    The value of early diagnosis and early treatment is extremely high. This is also recognized in the fields of health economics and health technology assessment (HTA).In our research, we investigated the relationship between school urine screening and the early detection of IGA nephropathy, and reported on its value, including its cost-effectiveness. With regard to SMA as well, the importance of early diagnosis and early treatment is clear, and their value is likely to be high. In general, it is possible to evaluate the value of early diagnosis and early treatment, and this is a field that is advancing.

Q.SMAにおける早期診断と早期治療の重要性は理解しました。しかし、早期診断・早期治療を行った後でもケアが必要だというお話もありました。特に成人の患者さんや発症時期が遅れる方に対して、どのような点が重要だと考えているか教えてください。

  • Mr. Arakawa

    Of course, early treatment is extremely important. However, it is also vital to help children currently living with SMA lead better lives. While early treatment may lead to more patients with milder symptoms in the future, the reality is that many patients currently require significant care.We need to strike a balance between advancing early treatment and providing solid support for the daily lives of patients currently undergoing treatment. Even within patient support groups, I believe there are differences in perspective between those who have undergone newborn screening and those whose symptoms have already manifested.

  • Mr. Oyama

    It feels as if I have a completely different disease now compared to before I started treatment. That’s how remarkable the treatment’s effects are. However, if I were to point out one challenge, it would be the importance of rehabilitation.Our members range in age from 0 to 70, but one challenge they all share is the need to continue rehabilitation in addition to treatment. Without rehabilitation, it is difficult to see improvement, and it is also difficult to enhance quality of life (QOL).In particular, parents of patients who received early treatment may be reluctant to accept that their child has a genetic disease, and parents of children who are asymptomatic or have only mild symptoms may not be as proactive about rehabilitation. It is crucial to fully acknowledge that this is a rare and intractable disease and to understand what kind of disease it is and what symptoms may arise. It is also important to be mindful of rehabilitation in a broad sense, even as a preventive measure.

Q.リハビリの重要性についてはよく理解できました。価値評価の観点からはいかがでしょうか。

  • Mr. Goto

    The value of improved health extends beyond mere survival and functional capacity; it is equally important to consider how it supports daily life and one’s overall well-being. More specifically, research is evaluating the positive impact that social support and care have on health improvement. Furthermore, to properly assess these values, it is essential to identify them in collaboration with healthcare professionals and patients’ families.

Scene from the panel discussion

In Conclusion

At this forum, we were able to share how the emergence of new drugs for SMA—a condition that was once difficult to treat—and the growing adoption of newborn mass screening in Japan are bringing great hope to patients and their families. However, these developments have also given rise to new challenges. To further advance these efforts, we would like to take this opportunity to ask for your understanding and support.

The “intention of Co-Creation” outlined in the JPMA’s Vision for 2035 is that the key to solving complex social issues around the world lies in everyone working together. It is important for people from various backgrounds to have opportunities to discuss and share ideas around the same table.I hope that all of you will also reflect on the meaning and value of having diverse stakeholders participate, and use these insights to drive future progress.

(Pharmaceutical Industrial Policy Committee, Industrial Promotion Subcommittee: Masaaki Kozaki, Tomohiro Murakami, Takuro Matsuda)

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