Translational Research: The Roadblocks to Reality

By Kevin Hrusovsky, Executive Chairman and CEO, Quanterix

It takes an average of 17 years to bring a scientific discovery into clinical practice. This statistic, which has been validated by research published in Public Health Reports and the Journal of the Royal Society of Medicine, reveals a troubling reality for many patients in desperate need of the new practices, tests, drugs and technologies stuck in the years-long gap between research and implementation into healthcare practices and policies. 

I was fortunate enough to be among the panelists who discussed this pressing issue at last year’s One Mind Summit. This annual event brings together the world’s top scientists, philanthropists, pharmaceutical and healthcare leaders, and government decision makers to discuss the industry at large and how we can work together to move it forward. Our discussion, which has been detailed in a new research paper in the Journal of Neurotrauma, looks at the current state and challenges of translational research in both a broad sense and as it relates to concussion screenings.

The process of adopting, accepting and utilizing a new technique, drug or technology can be highly complex. As such, the path has been broken down into two phases, T1 and T2. T1, referred to as “bench to bedside,” moves discoveries from basic concepts into effective treatments. T2, which is often called “bedside to practice,” and was the focus of the panel, involves the movement of clinical evidence into patient healthcare and policy.

Many factors contribute to the sluggish pace of the T2 phase. Largely, this is due to the action required by groups outside of the research field, including professional healthcare organizations, regulatory agencies, legislators, caregivers and the patients themselves.

As would be expected, moving research into hospitals and doctors’ offices requires an extensive evidence review and the development of clinical guidelines that enable successful implementation of these advancements. This “evidence-based practice,” while vital, creates significant delays as hundreds of government and non-governmental agencies conduct their reviews, creating often-conflicting clinical guidelines. The National Academy of Sciences (NAS) is working to address these concerns with more standardized processes and better dissemination practices, but translational research still faces other bureaucratic hurdles.

For example, while recognized as a key pathway forward and a crucial gatekeeper for untested products and unproven treatments, the FDA can bar the way to clinical application, as the qualification process can be challenging and necessitate additional research or validation studies. Maintaining funding during this process was a key challenge discussed during the panel, drawing attention to an unmet need for organizations caught between initial development and implementation.

Notably, on the other hand are patients, caregivers and the public at large, who are among the most powerful agents of change. Historically, these groups have made great strides in moving scientific research into their own hands. This was the case for polio and HIV research, which both required the support of these groups in order for treatments to reach patients. These groups are also key for mobilizing legislators who can affect and sustain critical healthcare policy. The power of vocal and passionate grassroots efforts should not go unrecognized, especially in today’s political landscape where they face fierce competition for politicians’ time and government funding.  

Despite these challenges, researchers are in agreement that timely implementation and continuous oversight is key to a better understanding of diseases and improved treatment outcomes. A balance must be stuck between the necessary checks warranted for new drugs, technologies and procedures and the need to bring these advances to bear quickly.

Today, as we continue to make strides toward a more personalized healthcare paradigm, we’re seeing an increasing number of institutions and organizations rely on patient surveys, data collections and pragmatic trials to inform real-time adjustments to individualized treatment plans.  These practices are enabling better evaluation of new drugs and screening tools, especially in areas where there has traditionally been a lack of criteria and a limited understanding of causation, such as PTSD.

The ability to interpret and act on patient data in real time is offering great promise for many therapeutic areas. Brain health research is one such area seeing significant benefits from precision healthcare practices that are facilitating the introduction of new technologies and screening techniques. Take concussions for example. Currently, there is no gold standard for concussion screenings in emergency rooms or urgent care centers. This is particularly troubling as there are roughly 2.5 million TBI visits, and an estimated 1.5 million missed diagnoses each year, which could result in serious complications, especially if another hit occurs. We explore this more in a previous blog post, available here.

A standardized screening technique for concussions is yet another example of a vitally important practice that is facing implementation challenges. A key factor in bringing it to patients will be the ability to demonstrate significant patient care benefits. This is why we’ve focused on putting the science first at Quanterix and have been involved in more than 100 peer-reviewed papers that validate Simoa’s abilities across various therapeutic areas, including recent papers published in JAMA and Neurology.

We were honored to be involved in this discussion, which tackled a primary barrier to healthcare advancements. We would not be where we are today without the efforts of researchers around the world who work tirelessly to develop cutting-edge tests and treatments. It now falls on us, as patients, caregivers, healthcare advocates and leaders within the field, to support these developments so they can begin benefitting patients now – not 17 years later.

To learn more, we encourage you to read the full study here: http://online.liebertpub.com/doi/abs/10.1089/neu.2016.4821