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.

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.

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.

Last Thursday saw the 12th annual celebration of Rare Disease Day – an international day of recognition when hundreds of events take place around the world.