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.

Hwang et al.[i] completed a study looking at 640 phase 3 trials investigating novel therapies and found that 54% of these projects failed in clinical development; 57% of the failures were due to inadequate drug efficacy.  This means that 43% of the projects that failed were due to reasons other than the efficacy of the drug.  It is likely that many of these failures were due to operational deficiencies.  It is important to consider that a Sponsor’s investment in a trial is not only the investment made for the phase in which the project fails, but all investments from discovery leading up to the failure.

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.