THOUGHT LEADERSHIP

Home visits as part of clinical research have accelerated in recent years as we strive to find the right balance between facilitating study participation while accommodating participant’s busy everyday lives. Clinical trials in rare diseases are even more challenging than trials in other diseases due to a number of factors:

• Small number of eligible trial participants
• Complicated by heterogeneity among rare disease patients
• Most have no cure and manifest at a young age
• Less than 10% of rare diseases have a specific treatment
• Many have other debilitating conditions / physical limitations making it difficult to attend frequent study visits

The combination of home study visits and the right technology removes barriers to optimal patient recruitment, compliance and retention.

Emerging biotech companies must master many challenges in bringing a product to market. Even the acronyms common to early development, like IND (Investigational New Drug Application), SAD (single ascending dose), and MAD (multiple ascending dose) imply that a company must know how their treatment impacts the body, how to analyze study results, and how to express their findings to regulatory authorities. The expertise of medical writers is valuable in helping an emerging biotech reach its drug development goals.

AICROS, the Association of International Clinical Research Organizations, is a consortium of small to mid-size CROs that collaborate to provide clinical research services to the biotechnology, pharmaceutical and device industries worldwide. PROMETRIKA joined AICROS in 2018 and on May 29 -30th, PROMETRIKA’s Head of Clinical Operations, Heather Paden, attended the AICROS Annual meeting, which took place in Kaunas, Lithuania.

Recently, our own Nicole LaVallee, PhD (Director of Biostatistics) and Miganush Stepanians, PhD (President & CEO and senior advisor in Biostatistics) collaborated with the scientists at Boston Children’s Hospital on analyses of the Angelman Syndrome Natural History Study. The results of this collaborative effort were published in the American Journal of Medical Genetics.