Japan is creating access to new treatments for neglected diseases
June 9, 2024
By Mandeep Dhaliwal and Osamu Kunii for Nikkei Asia
Dr. Mandeep Dhaliwal is director of the HIV and health group at the U.N. Development Program. Dr. Osamu Kunii is chief executive of the Tokyo-based Global Health Innovative Technology Fund.
Too often, a new, game-changing medicine emerges from rigorous research and clinical development, secures regulatory approval and then encounters significant implementation delays before reaching the people and communities who need it most.
This is a global health story that we have heard time and again, and each time it can cost lives.
Fortunately, an innovative approach of targeted investment and collaboration, championed by the government of Japan and its global partners, is beginning to turn this narrative around by accelerating access to medicines for neglected diseases.
A key turning point is the development and pending introduction of a new pediatric treatment for preschool-aged children with schistosomiasis, a neglected tropical disease, also known as bilharzia, that is spread by contact with fresh water contaminated with parasitic flatworms and which can disable and kill.
The medicine is urgently needed to close a treatment gap for an estimated 50 million preschool-aged children at risk of infection by the second-most devastating human parasitic disease after malaria.
Policymakers and technical partners, including the Japanese ministries of Foreign Affairs and Health, Labour and Welfare, recently met in Tokyo to reach consensus on the key steps needed to support the rapid, successful and equitable introduction of the new medicine.
This came on the heels of the European Medicines Agency giving a positive scientific opinion on the medicine and just days after the World Health Organization also signed off on its safety and effectiveness.
The quick turnaround was made possible by focused access planning that began in the early stages of the development of the drug. The process was driven by a group of partners, many anchored in Japan, and included efforts to prepare health systems to roll out the new medicine and to engage with local communities to promote acceptance and generate demand while work was still underway in the lab.
Because of this, the new pediatric treatment could become a success story in global health and an example for introducing future innovations.
Biopholaria Glabrata snails, which host the worms that cause schistosomiasis: An estimated 50 million preschool-aged children are at risk of infection. © AP
The Pediatric Praziquantel Consortium, an international partnership led by the pharmaceutical company Merck, drove the development of the new medicine, aiming to achieve the WHO's goal of eliminating schistosomiasis as a public health problem by 2030.
The work, though, began in Japan, where Astellas Pharma, a founding member of the Praziquantel Consortium, utilized proprietary technology to lead initial development before passing the formulation it created to Merck for further optimization.
The Japan-based Global Health Innovative Technology Fund (GHIT Fund), which leverages Japanese innovation and leadership in the fight against neglected diseases, supported the development with a 1.85 billion yen ($11.86 million) investment. Additional support came from the European & Developing Countries Clinical Trials Partnership (EDCTP) and the Bill & Melinda Gates Foundation.
Access planning proceeded in parallel, driven by the Praziquantel Consortium and the U.N. Development Program-led Access and Delivery Partnership (ADP), the GHIT Fund and Uniting Efforts for Innovation, Access and Delivery, a joint initiative between the partnership, the government of Japan and GHIT.
Together, these partners, working on both product development and access, have been collaborating with health ministries and disease control program officials from countries impacted by schistosomiasis to help strengthen health systems in anticipation of the new treatment becoming available.
In February, the partners convened to share country-level initiatives and lessons on implementation with the aim of optimizing delivery, strengthening monitoring systems and engaging and supporting community health workers to ensure that national health systems are ready as soon as the treatment becomes available.
Communities need to be ready too, which is why work has been underway to help countries lay the groundwork for the rapid deployment of the new treatment. The Praziquantel Consortium, supported by the GHIT Fund and EDCTP, is collaborating with ministries of health in Kenya, Ivory Coast and Uganda on implementation research programs aimed at understanding some of the key social dynamics that can influence how prepared communities are to accept health innovations.
These programs aim for efficiency by carrying out pilot programs that build on existing mechanisms, rather than developing new ones. They also have a human effect, helping raise awareness of schistosomiasis infection and treatment in young children, and bridge gaps between new science and innovation, health systems and affected communities.
This kind of preparation can help generate demand and foster transparency, acceptance and uptake. Other programs like Strengthening Capacity for Delivery and Uptake of Paediatric Praziquantel for Schistosomiasis, led by the National Institute for Medical Research in Tanzania with support from ADP, have also been working with community leaders and health authorities to raise awareness and promote treatment acceptance and demand.
By connecting and amplifying these efforts, we can take an important step forward in global health. Japan's vision to invest in both R&D and access and the commitment of all partners to work together toward a common goal will be vital to this effort. But more than that, this project also signals recognition that the typical model of R&D followed by lengthy implementation must be improved.
By starting early and deliberately to help countries get ready to deliver health innovations to people in need, we can advance a model that prioritizes science, access and equity. This model can be adapted for many more new health solutions and technologies as we begin to rewrite the global health story for the better. Many lives are depending on this.