4 papers · 1 filter
Semi-parametric efficient estimation of small genetic effects in large-scale population cohorts
Olivier Labayle, Breeshey Roskams-Hieter, Joshua Slaughter +5
Population genetics seeks to quantify DNA variant associations with traits or diseases, as well as interactions among variants and with environmental factors. Computing millions of…
The impact of job stability on monetary poverty in Italy: causal small area estimation
Katarzyna Reluga, Dehan Kong, Setareh Ranjbar +2
Job stability - encompassing secure contracts, adequate wages, social benefits, and career opportunities - is a critical determinant in reducing monetary poverty, as it provides ho…
Use of Real-World Data and Real-World Evidence in Rare Disease Drug Development: A Statistical Perspective
Jie Chen, Susan Gruber, Hana Lee +13
Real-world data (RWD) and real-world evidence (RWE) have been increasingly used in medical product development and regulatory decision-making, especially for rare diseases. After o…
Challenges and Possible Strategies to Address Them in Rare Disease Drug Development: A Statistical Perspective
Jie Chen, Lei Nie, Shiowjen Lee +13
Developing drugs for rare diseases presents unique challenges from a statistical perspective. These challenges may include slowly progressive diseases with unmet medical needs, poo…