On March 22 2022 I held this thought-provoking presentation about the use of modern information technologies to aid patient access to orphan drugs at the World EPA congress in Amsterdam.
My conclusion: These technologies could be valuable instruments that help to provide solutions to fill the data gaps in health technology assessments and guide policy decisions. However, since there are many actors involved, to improve patient access to orphan drugs an ecosystem innovation is needed. Unless this is addressed, these modern technologies will not provide solutions that benefit patient access.