Industry Policy Committee Survey on the problems of healthcare professionals in rare diseases
Patients and their families with rare diseases face a variety of 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, the Pharmaceutical Manufacturers Association of Japan (PMAJ) conducted the "Survey on Challenges Facing Patients with Rare Diseases (released in February 2023)" and identified particularly important issues that pharmaceutical companies should address. In response to these issues, we 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 once again that many issues for patients and families affected by rare diseases have yet to be resolved, and it was necessary to take up the voices of healthcare professionals who support rare disease medicine in Japan and accelerate more concrete efforts to address them.
The Initiative on Rare and Undiagnosed Diseases, the Japan Rare Diseases Initiative, and the Japan Rare Diseases Association have been established with the aim of "identifying issues faced by healthcare professionals involved in rare diseases, proposing and implementing solutions, thereby contributing to improving the quality of rare disease care and research and making a contribution to patients and their families. Diseases, the Rare Disease Consortium Japan, and EY Strategy & Consulting K.K. (planning and management support), we conducted the "Survey of Healthcare Professionals' Concerns in Rare Diseases" with the aim of "contributing to the quality of healthcare and research related to rare diseases, and to patients and their families.
This document summarizes the direction of problem solving in rare disease medicine and the actions required by each stakeholder in response to the issues identified in this survey. The Pharmaceutical Manufacturers Association of Japan (PMAJ) is committed to working together with various stakeholders to resolve issues surrounding rare diseases so that many people are aware of the challenges they face and to realize a society in which patients and families living with rare diseases can live with peace of mind.
Abstract, Background, Objectives, Overview of Rare Diseases, Survey Methodology
Abstract, Background, Purpose, Outline of Rare Diseases, and Survey Methodology
Survey Results
Sense of Challenges in Rare Diseases in Japan
Results】 【Conclusion
Conclusion, References, List of related persons, Acknowledgements
Survey on the problems of healthcare professionals in rare diseases [Materials] Full text (9. 33MB)
Reference
Survey on problems of healthcare professionals in rare diseases (English version)