Blog-nephrology

Kidney disease is a global epidemic, yet unlike cancer, diabetes or heart disease, the clinical research community is investing few resources to address this healthcare crisis.

As much as 10 percent of the global population are now affected by chronic kidney disease (CKD), and millions die each year because they do not have access to affordable treatments. More than two million people currently receive dialysis or a kidney transplant to stay alive, yet this number may only represent 10 percent of people who actually need treatment, in large part because of the incredible expense of these options. In the US, alone treatment of CKD is expected to exceed $48 billion per year; and end-stage renal disease (ESRD) patients already consume nearly seven percent of the total Medicare budget even though they represent less than one percent of the covered population.

Despite the huge healthcare burden, unsustainable cost, and limited treatment options, research into kidney disease lags virtually every other prominent disease category. There are fewer trials in this field of research and fewer trained investigators, and among trials that are conducted they tend to have smaller numbers of patients and are often unblinded. The dearth in innovation in this space is driven by the fact that kidney diseases disproportionately affects a demographic that is largely disenfranchised, and lack sufficient advocacy, public attention and funding. As a result, the existing infrastructure and guidance documents supporting nephrology research pale in comparison to other internal medicine specialties.

But that’s all beginning to change, thanks to the Kidney Health Initiative (KHI). In 2012 a group of industry stakeholders, including the US Food and Drug Administration (FDA), the American Society of Nephrology (ASN), the American Association of Kidney Patients (AAKP) and several biopharma and healthcare industry organizations formed the KHI. Our goal: to advance the scientific understanding of existing treatments for kidney disease, foster development of new therapies, and create a more collaborative nephrology community. We believe the plight of kidney patients is urgent and they deserve to see new therapies, and we are committed to creating an environment and funding opportunities to make it easier to bring new treatments to market. 

Since we launched this partnership, KHI leaders have been working together to identify the best projects and strategies to move research forward. We have determined that changing the status quo will require projects that address four key areas:

  • Developing new highly prognostic and predictive biomarkers
  • Increasing patient engagement and activism
  • Enhancing clinical trial infrastructure 
  • Improving our investigational clinical trial designs with special attention to improved patient selection (i.e., entry criteria) and endpoints.

In a recent article published in the Journal of the American Society of Nephrology, co-authored by several KHI members, we outlined the barriers to overcoming the obstacles faced in nephrology research, and offered recommendations, some of which are already beginning to be implemented. For example, the nephrology community has a unique opportunity to capitalize on the fact that the vast majority of patients with ESRD are seen several times each week for provision of clinical care by two large dialysis providers. With appropriate informed consent, dialysis unit electronic health records could be used to collect clinical information and generate high-quality clinical data in almost real time, providing a snapshot of current standards of care.

We also identified the value of harnessing resources such as PCORnet, the national health data network developed by the Patient Centered Outcomes Research Institute (PCORI). PCORnet combines data from both clinical data research networks and patient–powered research networks (PPRNs), including several kidney-centered groups such as NephCure Kidney Network International and the Vasculitis Patient Powered Research Network. These networks can help fill an important gap in the current research paradigm. At present, clinical research in nephrology largely consists of observational studies, which may generate important hypotheses but rarely yield definitive answers. Research networks like PCORnet provide the opportunity to conduct randomized, pragmatic trials at far less prohibitive costs so we can begin to answer some of these important questions.

These are just a few of the potential strategies that can help the biopharma industry realize the incredible market opportunities this area of research holds. Kidney disease is a global unmet medical need, and the organizations that can overcome these barriers have the potential to develop desperately needed treatments that could impact millions of patients around the world.

 
Related: Quintiles and DaVita Clinical Research announce strategic alliance to optimize renal disease trials

Topics in this blog post: Healthcare, Biopharma, Clinical Trials, R&D, Nephrology