Fig. 1
From: How to START? Four pillars to optimally begin your orphan drug development
START to drug development. The different questions for each rare disease drug development project to ask are on Stakeholder mapping, thereby providing information on actors and fundamental infrastructure in the development process, Available information on the diseases, gathering information non the disease, financial Resources, gaining an overview on the financial means for the development and Target Patient Value Profile, before a stakeholder starts drug development. for Stakeholder mapping, this means looking at all stakeholders with a vested interest in the development process, such as patients, clinicians, and supporting contacts with platforms and development networks. For Available information on the disease, this entails searching for data and tools. For financial Resources, this means searching for a combination of public and private funding and setting out a fundraising strategy for the different steps in the development pathway. For the Target patient value profile, it means making an overview of the opinion and perspective of the patient regarding disease profile being investigated and the expected outcomes of a therapeutic development