Pharmaceutical Industrial Policy Committee Proposals on Intractable and Rare Diseases Based on a Survey of Healthcare Professionals' Concerns
Patients and their families with rare diseases face various challenges from the onset of the disease, through testing/confirmed diagnosis, to the start of treatment. In order to understand the background and factors behind these challenges, JPMA conducted the "Survey on Challenges of Patients with Rare Diseases (released in February 2023)" and identified particularly important issues that should be addressed by pharmaceutical companies. In response to these issues, we have compiled the "Proposal on Intractable and Rare Diseases (released in July 2023)" and have been working to resolve issues surrounding rare diseases.
On the other hand, it was brought to light that many issues for patients and families involved in rare diseases remain unresolved, and it was necessary to accelerate more concrete efforts by addressing the voices of healthcare professionals who support rare disease medicine in Japan. Therefore, the Initiative on Rare and Undiagnosed Diseases (IRUD), Rare Disease Consortium Japan (RDCJ), EY Strategy & Consulting K.K. (planning and management support), we conducted a survey on the problems of healthcare professionals in rare diseases (released in November 2024).
Based on the results of the survey, we have now prepared "Proposals on Intractable and Rare Diseases Based on the Survey on Problems of Healthcare Professionals" in collaboration with IRUD and RDCJ.
JPMA will continue to work with various stakeholders to resolve issues surrounding rare diseases in order to realize a society in which many people are aware of the problems associated with rare diseases and patients and families living with rare diseases can live with peace of mind.