Sano blog | protocol design

precision medicine patient recruitment rare disease genetic testing protocol design

FDA draft guidance expands single-trial effectiveness pathway

In June 2026, the FDA released a revised draft guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products, that clarif...

precision medicine rare disease protocol design patient engagement

Split verdicts: What HOPE-3 exposes about rare disease evidence

On 29 July 29, 2026, the same clinical trial data produced opposite verdicts. The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that a...

precision medicine screen failure patient recruitment protocol design

Precision medicine's execution deficit is an accountability problem

A genetically-defined trial can be designed correctly and still stall. The protocol names the right molecular subtype, the endpoints are sound, and the therapeu...

patient recruitment protocol design health equity

Decentralized trials expand access and diversity at Mayo Clinic

On July 20, 2026, Mayo Clinic reported peer-reviewed evidence that decentralization changes who takes part in clinical research. In a study published in JAMA Ne...

precision medicine protocol design population health health equity

The All of Us milestone shows precision medicine’s next infrastructure challenge

The NIH’s All of Us Research Program has become the world’s largest integrated genomic and electronic health record database. Its June 30 data release includes ...

screen failure protocol design

Reducing screen failure starts with better trial design

When a clinical trial misses its enrollment targets, the instinct is often to look at recruitment through more channels, more outreach, and a bigger advertising...

rare disease protocol design

Rethinking drug approval for ultra-rare disease: Inside the UK’s n-of-1 ASO trial

Last week, the rare disease field celebrated another breakthrough. A teenage girl with an ultra-rare genetic neurodegenerative disease was treated with a bespok...

precision medicine protocol design

Lessons from recent FDA actions in precision medicine

Over the past few years, several cell and gene therapies (CGTs) in precision medicine have received FDA Complete Response Letters (CRLs). As the agency increase...

Regulatory signals for precision medicine trial sponsors

rare disease protocol design

Top five regulatory signals sponsors can act on now

The FDA’s activity in 2025 reflected a mix of scientific progress and operational instability. Precision medicine programs benefitted from new draft guidance, r...

patient recruitment protocol design

Expediting study start-up: Best practices for sponsors

Study start-up is a critical stage of the drug development process and can have a major impact on trial timelines, budgets, and overall success. Start-up activi...

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