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

Area of Science:

• Drug development
• Rare diseases
• Orphan drug development

Background:

• Drug development is complex and resource-intensive, especially for rare diseases.
• Challenges in rare disease drug development include small patient populations and limited disease knowledge.
• Gathering comprehensive disease and patient needs information is crucial before defining the development path.

Purpose of the Study:

• To introduce the START checklist as a tool for initiating orphan drug development.
• To provide a structured approach for developers navigating the early stages of rare disease therapeutics.

Main Methods:

• The START checklist is proposed, encompassing four key pillars: STakeholder mapping, Available information on the disease, Resources, and Target patient value profile.
• The tool guides developers through essential questions for the initial phase of rare disease therapeutic pathway development.

Main Results:

• The START checklist offers an overview of critical considerations for starting orphan drug development.
• It facilitates the establishment of a solid foundation for patient-centered medicine development programs.

Conclusions:

• The START checklist is a valuable tool for guiding developers through the complexities of rare disease drug development.
• Implementing the START checklist promotes a patient-centered approach, enhancing the likelihood of successful therapeutic pathway development.