THOUGHT LEADERSHIP

Over the past 15 – 20 years, projected peak sales and return on investment (ROI) of new drugs has been shrinking (source: Statista and Deloitte Centre for Health Solutions). To offset this, pharmaceutical companies are pushing to get more drugs to market and multiple indications for each agent.

Decentralized clinical trials (DCT) seems to have burst on to the scene in just the last few years, so it is easy to get swept up into how we can best implement them into our clinical trials. However, it is worth examining the past of this supposedly “new” method of conducting clinical trials.

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.

Remote interactions—often termed eClinical Technology—are making their way into clinical trials. Precipitated by the COVID-19 pandemic, we have seen an uptick in the adoption of televisits, remote monitoring, and eConsent in clinical trials. 

I recently served as the moderator for MassBio’s forum on “Closing the Real-World Evidence Gap: Pragmatic Clinical Trials & Observational Studies.” This lively and informative discussion was led by a panel of industry leaders, which included Robert Califf, MD, MACC, Head of Medical Strategy and Policy, Verily Life Sciences and Google Health; Jane Liang White, ScD, Senior Director, Statistical Group Lead for Oncology/Hematology Franchise at Pfizer; and Rebecca Miksad, MD, MPH, Senior Medical Director of Flatiron Health.