Download the free whitepaper: Unravelling the complexities of genomics-driven drug discovery

Genomics-driven drug discovery holds immense promise in developing targeted therapies by leveraging genetic and biomarker information to understand the root causes of diseases. However, the field faces significant hurdles.

In this whitepaper, we examine the obstacles of limited recontactability, scarcity of multi-omics data sets, lack of diversity in data, and the challenge of scaling research for rare diseases. By addressing these challenges and proposing strategies to address them, this whitepaper aims to chart a path by which we can harness the full potential of genomics-driven drug discovery.

 

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Practical criteria for selecting sites in rare and genetic trials

Site selection is not where rare disease feasibility should begin

In rare and genetic trials, feasibility is often built on site-reported patient counts. Those estimates shape enrollment models, activation strategy, and timelines. When those assumptions prove inaccurate, the consequences surface as delayed enrollment, endpoint variability, protocol amendments, and portfolio slippage.

In small populations, a single underperforming site can materially affect statistical assumptions and extend development timelines. The stakes are operational, financial, and regulatory.

This report reframes site selection as the execution layer of a broader precision patient strategy. It outlines how to move from site-first feasibility to patient-first feasibility, and how to evaluate sites against the factors that truly determine performance.

Inside, you will learn how to:

✅ Assess real patient availability rather than historical counts
✅ Evaluate diagnostic pathways and genomic confirmation workflows
✅ Reduce endpoint variability in small populations
✅ Identify infrastructure risks in advanced therapy programs
✅ Align site strategy with long-term follow-up and retention requirements

Download the full report now.

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