Principles of Precision Medicine: Treating the Right Patient at the Right Time
Precision medicine is changing how we think about healthcare, clinical trials, and drug development. For…
Navigating Global Complexity: Applying ICH E17 to Modern Clinical Trial Biometrics
Clinical trials are no longer local experiments with global ambitions. Increasingly, they are global evidence…
Rare Disease Drug Approvals Accelerate as FDA Clears Three New Orphan Therapies
May 2026 — The U.S. Food and Drug Administration has approved three therapies in May…
Proteins Technologies: Expanding the Rare Disease Discovery Layer
ncORFs and “Peptideins”: Expanding the Human Proteome Deutsch et al.’s Nature paper expands the map of the human…
The Heterogeneity Problem: Can Math Models Make Clinical Trials Smarter?
Rare diseases, especially lysosomal storage disorders, remain among the hardest areas of clinical research. Why? Small patient…
𝗙𝗿𝗼𝗺 𝗦𝗺𝗮𝗹𝗹 𝗣𝗼𝗽𝘂𝗹𝗮𝘁𝗶𝗼𝗻𝘀 𝘁𝗼 𝗦𝗺𝗮𝗿𝘁𝗲𝗿 𝗧𝗿𝗶𝗮𝗹𝘀
𝗧𝗵𝗲 𝗘𝘃𝗼𝗹𝘃𝗶𝗻𝗴 𝗥𝗮𝗿𝗲 𝗢𝗻𝗰𝗼𝗹𝗼𝗴𝘆 𝗟𝗮𝗻𝗱𝘀𝗰𝗮𝗽𝗲 Rare and orphan oncology is experiencing a transformation driven by…
7 Opportunities for a Biostatistics-Focused CRO in Rare Diseases
Rare disease trials are changing fast. Many studies now involve very small patient groups, single-arm…
𝗙𝗗𝗔’𝘀 𝗘𝘃𝗼𝗹𝘃𝗶𝗻𝗴 𝗦𝘁𝗮𝗻𝗰𝗲 𝗼𝗻 𝗕𝗮𝘆𝗲𝘀𝗶𝗮𝗻 𝗠𝗲𝘁𝗵𝗼𝗱𝘀: 𝗪𝗵𝗮𝘁 𝗦𝗽𝗼𝗻𝘀𝗼𝗿𝘀 𝗡𝗲𝗲𝗱 𝘁𝗼 𝗞𝗻𝗼𝘄 𝗶𝗻 𝟮𝟬𝟮𝟲
What we see at the January 2026 draft guidance, which formalizes a major shift: Bayesian…
BRINGING an ORPHAN DRUG to MARKET?
Rare disease programs cannot afford statistical missteps.If you need experienced biostatistical leadership that understands small…
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