Rare Disease Drug Development Scope of Work Template

Planning a rare disease drug development program requires careful coordination across multiple scientific, regulatory, and patient-centered workstreams. This rare disease drug development scope of work template helps pharmaceutical companies, biotech startups, CROs, and research institutions define clear project parameters from discovery through regulatory approval.

Rare disease programs face unique challenges including small patient populations, limited natural history data, and complex regulatory pathways. This template is structured to capture essential details across five critical work streams: natural history study design, biomarker identification and validation, clinical trial protocols, regulatory strategy and orphan drug designation, and patient advocacy partnerships.

Paperform makes it easy to collaborate with cross-functional teams including clinical operations, regulatory affairs, translational medicine, and patient advocacy groups. The conditional logic built into this template adapts questions based on development stage, indication type, and prior data availability, ensuring you collect only the most relevant information for your specific program.

Use this template to establish scope with external partners like academic research centers, patient registries, and regulatory consultants. Once submitted, you can use Stepper (stepper.io) to automate downstream workflows—routing different sections to specialized teams, triggering milestone reviews, updating project management tools like Asana or Monday.com, and creating a clear audit trail for regulatory submissions.

For organizations managing multiple rare disease programs or working with licensing partners, this template can be customized for different therapeutic areas while maintaining a consistent structure that supports portfolio management and strategic decision-making. Whether you're defining a Phase I safety study or planning a comprehensive development program through commercialization, this template provides the framework to align stakeholders and drive rare disease innovation forward.