Pascal JD Goetghebeur

By Helen Brooker & Pascal Goetghebeur In the shift toward decentralized clinical trials and home-based testing, the transition from hardware-bound cognitive testing to flexible, web-based platforms often raises a critical concern: does moving away from specialized equipment compromise data integrity? When measuring reaction times where every millisecond counts, the "noise" introduced by standard consumer hardware could potentially mask subtle treatment effects.

Article | October 21, 2024 By Joan Busner, PhD, Lew Fredane, MD, & Pascal Goetghebeur Prader-Willi syndrome (PWS) is a rare genetic disorder affecting 1 in 20,000-30,000 individuals worldwide. Recombinant human growth hormone is the only FDA-approved treatment for PWS but does not address hyperphagia or other core symptoms. Currently, no approved therapies exist for hyperphagia, behavioral challenges, or sleep issues.

By Helen Brooker and Pascal Goetghebeur Patient-focused drug development is increasingly important to regulators, emphasizing patient-reported outcomes in new drug applications. Phase I clinical trials, the initial step toward regulatory approval, primarily assess safety, tolerability, and dosage responses in healthy volunteers. However, cognitive changes due to disease or treatment are often overlooked despite their significant impact on patients’ quality of life.