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.

Most clinical researchers are motivated by the desire to improve patient health and drive innovation in healthcare. To optimize health system performance, Bodenheimer and Sinsky, building on the work of Donald Berwick, proposed the quadruple aim as a means to improve health system performance. The quadruple aim focuses on (1) enhancing the patient experience, (2) improving population health, (3) reducing costs, and (4) improving the work life of health care providers, including clinicians and staff. Clinical research should follow the quadruple aim to improve the design and conduct of clinical trials.

In recent years, efforts to streamline clinical research activities while improving subject safety and data integrity have included risk based monitoring (RBM). As recently as May 2019, the International Council for Harmonisation (ICH) proposed revisions to its General Considerations for Clinical Studies (E8) guideline. Section 3 of E8 (R1) stresses the importance of managing the quality of safety measures and data collection as part of an entire Quality Plan in a clinical program and its studies. This effort is called Risk-Based Quality Management (RBQM).