Effective Patient Engagement Strategies to Develop Therapies and Advance Patient Safety

Last year, I attended a medical conference focused on developing medications for kidney diseases. Unlike previous meetings, this conference was centered on developing upstream interventions, a welcome and very positive development for people with kidney diseases.

A nephrologist stated during a presentation at the conference that dialysis and kidney transplantation are barriers to innovation because they provide a “safe landing” for patients. When I heard this statement, I turned my attention away from the speaker to the audience to gauge the audience members’ nonverbal response. Much to my disbelief, the audience appeared to accept this statement as fact. Internally, I had a much different response.

Growing up, I saw my mom and her two sisters sustain their lives by receiving dialysis but with a diminished quality of life. I saw the constant fatigue and other quality-of-life challenges imposed by dialysis.

Beyond my personal experiences, it is important to look at the data. For dialysis patients, the 5-year survival rate is less than 50%, and there are still no fluid management guidelines. Although kidney transplantation is a superior treatment option compared with dialysis, half of kidney transplants fail within a decade, and some significant quality-of-life issues are associated with kidney transplantation (1). I can say without hesitation that dialysis and kidney transplantation are not a “safe landing” for kidney disease patients.

Shortly after this meeting, I was invited to be a part of the speaker faculty at Kidney Week 2017. My presentation, “Effective Patient Engagement Strategies to Develop Therapies and Advance Patient Safety,” had three learning objectives: to understand the barriers and patient perspectives on kidney disease clinical trials as an impetus for future research, to identify solutions to patient participation in kidney disease clinical trials and offer recommendations, and to create a vision for patient participation in clinical trials.

Understanding barriers and patient perspectives on kidney disease clinical trials: impetus for future research

Although the U.S. Medicare ERSD program was developed with the best of intentions by policymakers and patient advocates, it has created structural barriers to the development of new therapies for kidney diseases. The enduring legacy of the Medicare ESRD program has been unrestricted access to dialysis. However, the rest of the healthcare system in the United States has fallen short of building on this incomparable entitlement program.

Currently, there are no reimbursement incentives that would encourage early detection of chronic kidney disease (CKD). Not surprisingly, fewer than 10% of patients with stage 3 CKD are aware of their kidney function (2). Moreover, there are no incentives to encourage coordination of patient care and early referral to a nephrologist. As a result, 40% of CKD patients “crash” in hospital emergency departments and are transitioned immediately to dialysis (3).

Given that the remedy to structural barriers resides with healthcare policies, it is important to focus on the role of culture in the kidney disease community (4). Until her death in February 2017, Celeste Castillo Lee was an ardent and strong patient advocate for kidney disease patients. She envisioned the role of patients as partners within the kidney disease community. She was very aware of the need to elevate the expectations of all stakeholders, most notably patients. Yet not only have patients accepted a system of care that rewards downstream interventions, but they have accepted a system that has offered minimal innovation in the downstream interventions.

Kidney disease patients and clinical trials

At the suggestion of nephrologist Barbara Gillespie, MD, I conducted a survey of kidney disease patients with two primary objectives:

  • To determine why patients participated in kidney disease clinical trials.
  • To learn why patients did not participate in clinical trials.

The demographic breakdown of those surveyed was as follows:

  • 192 patients: 58% female; 42% male.
  • White, 78%; black, 14%; Hispanic, 5%; Asian, 3%.
  • CKD, 41%; dialysis, 28%; kidney transplantation, 31%.

The survey had one major limitation. The racial composition of the audience did not reflect the US population with kidney diseases.

As no surprise, 70% of the respondents had not participated in a clinical trial. When queried further on their lack of trial participation, these were the responses (Tables 1 and 2):

  • Over two-thirds of patients did not participate in a trial because their doctors never discussed the subject.
  • Almost three-fourths of patients said a physician’s discussing a clinical trial with them would influence their trial participation.



The patients who did participate in clinical trials appeared to be internally motivated:

  • Almost two-thirds of the respondents participated in a trial because they wanted to help fellow patients.
  • Less than 10% of respondents stated compensation as a motivating factor.
  • The number one stated reason for trial participation was the respondent’s communication with their physician.

The survey responses show there is an opportunity to engage patients further in clinical trial participation (Table 2):

  • 36% of patients reported that their trial results were shared with them.
  • Only 17% of patients were recognized for their participation.
  • The majority of patients, 60%, expressed a desire to be recognized for trial participation.


Identifying solutions to patient participation in kidney disease clinical trials: recommendations

Patient-centered policy is the solution to dismantling the structural barriers to CKD patient care. The introduction of H.R. 3867 in September 2017 is the first step in amending Title XVIII of the Social Security Act, which established regulations for the Medicare program. H.R. 3867 would create a demonstration pilot that would focus on early detection of CKD and combine it with coordination of care and engaging patients in their clinical care decisions (5). It is believed that this pilot would improve clinical outcomes while lowering healthcare costs. At long last, there is light on the horizon.

I recommend three ways to change the culture of the kidney disease community, thereby elevating the community’s expectations:

Patient engagement: understanding patient attitudes and beliefs about clinical trials

Based on my pharmaceutical and consulting experience, I see many assumptions made about people with kidney diseases and their participation in clinical trials. To balance the assumptions with evidence, I would like to see the following:

  • Baseline understanding of kidney disease patients’ viewpoints about clinical trials.
  • Identification of the biggest gaps in understanding patients’ perspectives.
  • Based on gap analysis, identification of research priorities to improve understanding.

Nephrology communication skills training

Recognizing a deficit in skills training for nephrology fellows, Robert Cohen, MD, initiated a quality improvement program to aid nephrology fellows when they have conversations with their patients, including discussions about ending dialysis and preparing for the end of life. Dr. Cohen conducted a 1-day workshop resulting in the improvement of the fellows’ communication skills (6).

I would like to see this training expanded across the spectrum of CKD to include conversations about clinical trials. Given that the Centers for Medicare & Medicaid Services Quality Improvement Program will evaluate nephrologists’ communication skills, this investment would serve several interests.

Changing the conversation

When my mom started hemodialysis in August 1981, the risk of treatment was never fully explained to her or to our family. For example, we were never told that half of hemodialysis patients are not alive at 5 years. At that time, we faced an uncertain future with no options.

Since that time, options have expanded for patients. They include peritoneal dialysis, pre-emptive transplantation, extended criteria organs, and clinical trials that provide upstream interventions for certain kidney diseases. There need to be risk-to-benefit conversations about the various options and full explanations of the risks associated with dialysis. This must change now.

Creating a vision for patient participation in clinical trials

We are in the very early stages of a transformative time in nephrology patient care, in which an environment is being established to create upstream treatments for kidney diseases. I am very excited about the future, based on three developments.

Kidney Health Initiative, Patient Family Partnership Council

One of the legacies of kidney disease advocate Celeste Castillo Lee is the creation of the Patient Family Partnership Council, whose charge is to provide strategic guidance to the Kidney Health Initiative (KHI) about how to activate and include patients, their families, and their care partners in KHI activities, including but not limited to these activities:

  • Advise KHI members regarding patient involvement in their project proposals.
  • Outline opportunities for patients to serve once a project has been endorsed.
  • Identify patients to serve on project work groups.
  • Collaborate on developing patient-centered project(s) to submit for KHI endorsement (7).


The senior leadership of the American Society of Nephrology has demonstrated a willingness not only to listen to patients like myself but also to act on patient feedback. One recent example is the formation of a work group to overcome barriers in upstream innovation.


Kidney Precision Medicine Project

The National Institutes of Health (NIH) investment in the Kidney Precision Medicine Project signals a strategic shift toward upstream innovation and interventions. This project aims to ethically obtain and evaluate human kidney biopsy specimens from participants with acute kidney injury or CKD, create a kidney tissue atlas, define disease subgroups, and identify critical cells, pathways, and targets for novel therapies. This project will pave the way for pharmaceutical investment in novel therapies.

Treatments on the horizon

There are various candidates in phase II and phase III trials for several rare and autoimmune diseases, including Alport syndrome, focal segmental glomerulosclerosis, IgA nephropathy, lupus nephritis, and autosomal dominant polycystic kidney disease.

All of these potential treatments represent upstream innovation and hope for thousands of patients and families.

At the 2016 KHI stakeholder meeting, Eric Dishman, director of the NIH All of US Research Program, drew on his Intel professional career and his patient perspective to make suggestions to KHI stakeholders: Once a vision is clear and visible to a stakeholder community, it becomes easier for a group of stakeholders to invest their dollars and their emotions.

The kidney disease community is now in a transition phase. Now is the time to gather all the stakeholders, including patients, into defining the future state of the kidney disease ecosystem.

April 2018 (Vol. 10, Number 4)


1. 2016 USRDS Annual Report 2. The Voice of the Patient-Organ Transplant, FDA.gov, September 27, 2016.

2. United States Renal Data System Annual Report 2017: Chapter 1-CKD Awareness, Figure 1.13.

3. Pierre Antoine Brown, et al. Factors associated with unplanned dialysis starts in patients followed by nephrologists: A retropective cohort study. PLOS https://doi.org/10.1371/journal.pone.0130080.

4. Peter G Linde, et al. Overcoming Barriers in Kidney Health—Forging a Platform for Innovation. J Am Soc Nephrol 2016; 27:1902–1910.

5. Fact Sheet: CKD Early Detection and Management H.R. 3867 Source: www.kidney.org.

6. RobertCohen. A nephrology fellows’ communication skills course: An educational quality improvement report. Am J Kidney Dis 68; 2:203–211.

7. ASN-online.org, KHI Patient and Family Partnership Council.