How to START? Four pillars to optimally begin your orphan drug development

Abstract

Drug development is a complex, resource intensive and long process in any disease area, and developing medicines to treat rare diseases presents even more challenges due to the small patient populations, often limited disease knowledge, heterogeneous clinical manifestations, and disease progression. However, common to all drug development programs is the need to gather as much information as possible on both the disease and the patients' needs ahead of the development path definition. Here, we propose a checklist named START, a tool that provides an overview of the key pillars to be considered when starting an orphan drug development: STakeholder mapping, Available information on the disease, Resources, and Target patient value profile. This tool helps to build solid foundations of a successful patient-centered medicines development program and guides different types of developers through a set of questions to ask for guidance through the starting phase of a rare disease therapeutic pathway.

Keywords: Data gathering; Drug development; Orphan drugs; Patients’ needs; Rare diseases; Stakeholder analysis and engagement.

Fig. 1

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