About "Survey on the problems of healthcare professionals in rare diseases
November 07, 2024
The Japan Pharmaceutical Manufacturers Association (JPMA), in collaboration with the Initiative on Rare and Undiagnosed Diseases (IRUD), the Rare Disease Consortium Japan (RDCJ) The survey was conducted in collaboration with IRUD, the Rare Disease Consortium Japan (RDCJ), and EY Strategy & Consulting K.K. (planning and management support).
The survey consists of a quantitative survey (Survey 1) and a qualitative survey (Survey 2), and identifies issues in the five areas of rare diseases (1. basic and applied research, 2. development and clinical trials, 3. diagnosis, 4. treatment and prognosis management, 5. disease awareness) from the perspective of healthcare professionals. The report also outlines the expectations of stakeholders (pharmaceutical industry, academic societies, patient groups, and government) with regard to these issues and the direction of solutions to these issues.
Survey on Problems of Healthcare Professionals in Rare Diseases
Challenges in rare diseases and the direction of problem solving
The Pharmaceutical Manufacturers Association of Japan (PMAJ) is committed to working with stakeholders to resolve the various issues identified in this survey that affect healthcare professionals, with the aim of realizing a society in which patients and families living with rare diseases can live more comfortably.
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Summary of the survey on the problems of healthcare professionals in rare diseases
Survey 1: Quantitative survey (Web-based questionnaire survey)
Survey period] Thursday, July 25, 2024 to Friday, August 23, 2024
[Number of valid responses] 327
[Target] Healthcare professionals who belong to the IRUD Diagnostic Committee, healthcare professionals referred by the Committee, and healthcare professionals participating in RDCJ
Survey 2: Qualitative survey (interview survey)
Survey period] Monday, September 2, 2024 to Friday, September 13, 2024
[Number of valid responses] 15 respondents
[Target] 327 participants in the quantitative survey who agreed to cooperate.
What is a rare disease?
Although the number of patients per disease is small, more than 7,000 rare diseases have been identified worldwide, and the total number of patients is estimated to be 300 million. Although definitions differ from country to country, according to the "Orphan Drug, Orphan Medical Device, and Orphan Regenerative Medicine Product Designation System" based on the Law Concerning Quality, Efficacy, and Safety Assurance of Pharmaceuticals, Medical Devices, etc. (Pharmaceutical Affairs Law), the requirements for designation are that "the number of eligible patients is less than 50,000 in Japan The requirements for designation are: "the number of target patients is less than 50,000 in Japan," "there is a high medical need for the product," and "the product has a high potential for development. In the U.S., the number of patients is less than 200,000, and in Europe, the number of patients per 10,000 population is less than 5. In addition to the small number of patients, the disease is characterized by its high severity and the fact that the mechanism of pathogenesis is unknown, making the development of treatment methods difficult and limiting effective therapies.
That is all.
For further information, please contact
Public Relations Department, Japan Pharmaceutical Manufacturers Association, Inc.
- Phone: +81-3-3241-0374
- Phone: +81-3-3241-0374