Industry Policy Committee Survey on the problems of patients with rare diseases

Patients and their families with intractable and rare diseases face a variety of challenges, including "long diagnosis times," "limited treatment options," and "insufficient social understanding and support. In order to solve these issues, various efforts have been made, including planning and promotion of various projects and policies by the government, daily medical treatment by medical institutions, implementation of basic and clinical research by academia, and support activities for patients by patient groups. In October 2021, the Pharmaceutical Manufacturers Association of Japan (PMAJ) will launch the Intractable and Rare Diseases Task Force to identify issues surrounding patients and their families with intractable and rare diseases and to consider measures that the pharmaceutical industry should take to address them, in order to play a role in efforts to resolve these issues. The task force will identify issues surrounding patients and families with intractable and rare diseases and consider measures that should be taken by the pharmaceutical industry. The Task Force is composed of members from multiple pharmaceutical companies and is working in unison across the organization to examine the issues.

The Task Force conducted a survey of patients and families of patients with rare diseases and related organizations in this field in order to obtain suggestions on issues that should be addressed by the pharmaceutical industry. We also checked to see if there were any differences in perception between these issues and those identified by the Pharmaceutical Manufacturers Association of Japan (PMAJ). Through the results of this systematic survey of rare diseases, we identified the real issues faced by patients and families with rare diseases and set the direction of solutions for the Pharmaceutical Manufacturers Association of Japan (PMAJ). We hope that this publication will lead to the expansion and acceleration of efforts not only by pharmaceutical companies, but also by various stakeholders including government, medical institutions, academia, patient groups, and other industries.