From President Beverley Yamamoto, HAE Japan (HAEJ):
Icatibant has now been approved for acute attack in Japan and now for the first time patients have access to a therapy that can self-inject. It is making a big difference to patient lives already.
We are still working hard to get self-administration of C1-inhibitor approved and making progress. As we feel authorization may be on the horizon, we have started to consider what training should look like. HAEJ successfully applied for funding for a small research grant from EFPIA to carry out a qualitative study of key stakeholders on the theme of creating a training program for intravenous self-administration for the Japanese setting. We are now partway through the study, and the results are extremely promising.
On 23 February 2019, we hosted the Rare Disease Day event in Kobe again. We had a lot of visitors to our event, both HAE patients and stakeholders and other disease patients and stakeholders. It was a very uplifting event and an important way of sharing capacity and other resources.
HAEJ has been working with the RUDY Japan team in the School of Medicine at Osaka University Hospital. Dr Kassim and his team at the University of Oxford originally created RUDY. The idea of RUDY is to create an IT platform that will enable patient-driven medical research around rare diseases. Osaka University has created RUDY Japan under the leadership of Professor Kazuto Kato. On 31 October 2018 items specifically relating to HAE were added to the platform and launched. There was a big press release event, and national television and newspapers covered the event. HAEJ are working with the RUDY Japan team at Osaka to push forward patient-centric initiatives in rare disease research. On 9 March 2019 four HAEJ members joined a workshop with medical researchers and policymakers to brainstorm about how patients can help set the agenda for rare disease research.
