2025 年 42 巻 3 号 p. 158-160
At Alnylam, we believe that patients are experts in their own disease and the experiences that come with it, and that they are the key decision makers in their own treatment and care. Patients are at the centre of everything we do and every decision we make.
Patient Advocacy & Engagement team works with patient advocacy groups around the world, including in Japan, to raise awareness of the patient communities we serve and to ensure that their needs are met. Through collaboration and partnership, we provide information and support to patients and families living with rare genetic diseases. We also actively listen to patients and their families, incorporating their perspectives into all aspects of our business.
To understand the unmet needs of a disease, it is necessary to understand what it means to live with a disease and to find out where and what gaps exist in the current medical environment. For this purpose, anecdotes and data generated from the experiences of patients and their families, such as the path to diagnosis, the burden of the disease, and issues with access to treatment, become important. This is the essence of Evidence Based Advocacy, and collaboration and cooperation between multi–stakeholders, including patient advocacy groups and healthcare professionals, makes it possible to collect, interpret and understand such anecdotes and data.
Based on these beliefs, we have conducted a survey of patients and families with hereditary ATTR (ATTRv) amyloidosis in Japan in collaboration and cooperation with stakeholders from different backgrounds, including patient advocacy groups, and would like to introduce it as an example of Evidence Based Advocacy.
