A New Year with a Plan for Digging Deep into the Gaps and Impediments of the Clinical Trials System

Happy new 2016 to everyone! As the new year rolls around, we are moving into Phase 2 of our project— idea generation and synthesis— with an intensive focus on the gap and impediments identified during Phase 1. We are seizing an opportunity— the ongoing conversation about the time and amount of money it takes new treatments to come to market in the US that has seemingly reached a fevered pitch in the mainstream media and on social media.

Over the past year, we have classified each gap and impediment as belonging primarily to one of five categories— clinical trials system structure and clinical trial design, information and data exchange, culture, market forces, and legal and regulatory issues.

• First, the issues categorized as clinical trial system structure and clinical trial design concern sub-optimal structure and function occurring in each phase of the clinical trials system from pre-clinical development through postmarketing studies (Phase IV). A series of factors contribute to lack of innovation as well as failure pre and post-market; we will examine alternative system structures and trial designs.

• Second, a variety of gaps and impediments in information and data exchange drive inefficiency and error in preclinical research and clinical trials despite the availability of relevant technological advances. We will examine how technology can be harnessed for progress and why system stakeholders are currently resistant to adoption.

• Third, the generally-accepted narrow assumptions of how preclinical research and clinical trials must occur have become embedded cultural issues that seriously limit research and innovation; we will explore creative approaches to address these narrow assumptions.

• Fourth, issues categorized as market forces may be the most publically-discussed aspect of the clinical trials system. The role of transnational pharmaceutical and biotech companies in the US economy, the rise of for-profit research, and drug pricing methodologies regularly make headlines must be part of any productive conversation about the clinical trials system no matter how controversial.

• Fifth and finally, law and regulation form the framework that dramatically affects the availability, cost, and reward for drug, biologic, and device development; as a result, legal and regulatory issues are at the heart of the some of the most difficult problems facing the clinical trials system. We will examine the intended and unintended consequences of law and regulation on innovation, research, and the practice of medicine.

We invite everyone to join us in the search for proactive, creative strategies. During Phase 2, our goal is to work only on those gaps and impediments that have not been addressed by other groups working to reform the clinical trials system and that have a major impact on the clinical trials system as a whole. We will continue to collaborate with a broad range of expert and non-expert stakeholders. Experts from other clinical trials initiatives as well as renowned strategic advisors from a wide range of disciplines bring fresh insights and broader perspectives to the discussion. Healthy and unwell individuals and their families who interact with the clinical trials system bring personal experiences to inform our work and keep us focused on the project mission. Through a collaborative and iterative process, we will continue to engage with this diverse group of stakeholders as we generate new ideas and take the first steps towards Phase 3 and developing a set of relevant, systems based recommendations.

Jen Bernstein and Sauleh Siddiqui