2025 年 42 巻 3 号 p. 290-294
Patients and families affected with intractable and rare diseases face numerous challenges, and healthcare professionals also encounter significant difficulties in diagnosis and treatment. In response, Japan Pharmaceutical Manufacturers Association (JPMA) established the Intractable and Rare Diseases Task Force in October 2021. The Task Force has been actively working on resolving the associated challenges. This paper presents three key initiatives undertaken by the Task Force : I. Survey on the challenges faced by patients with rare diseases, II. Policy Recommendations on intractable and rare diseases, and III. Survey on the challenges faced by healthcare professionals dealing with rare diseases.
To overcome the challenges identified in these surveys, all stakeholders need to work together. In addition to improving diagnostic techniques, developing new treatments, and strengthening patient support, raising awareness about intractable and rare diseases is equally important to enhance public understanding of these challenges. JPMA remains committed to working alongside various stakeholders to address the issues surrounding intractable and rare diseases and hopes that collaboration on intractable and rare diseases will expand to include more stakeholders.
