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.

As a Clinical Project Manager (CPM), one of my primary roles on a project is to ensure that the clinical and data management stakeholders have the tools necessary to streamline safety review and data quality review on a project. The CPM’s first action should be to complete a Stakeholder Analysis. According to the Project Management Body of Knowledge (PMBOK), Fifth Edition, released by the Project Management Institute (PMI), the key processes in Stakeholder Management are:     
                                                     1. Identify Stakeholders
                                                     2. Plan Stakeholder Management
                                                     3. Manage Stakeholder Engagement
                                                     4. Control Stakeholder Engagement
Knowing who the stakeholders are, and how and when to engage them in the development process of a clinical application, can lead to major improvements in product usability and ultimate project success. 

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.