“What we need to do is to keep working on getting biomarkers more defined to be able to differentiate between the different etiologies. This is going to be the path forward.”
Chronic kidney disease (CKD) is actually 20 times more prevalent and 100 times more lethal than cancer, but it does not get nearly as much attention or funding. In 2017, there were 16 FDA drug approvals for oncology compared with 1 for nephrology.
Wayne Bowden, Biorasi’s VP of Program Development, has overseen the initiation of many CKD trials. He was invited to speak on a panel at a conference last month in Raleigh, North Carolina entitled “Nephrology Clinical Trial Best Practices Master Class.” This conference was unique in that it brought together people from pharma, biotech, CROs, healthcare, and academia.
Here are some take-away messages from the panel discussion:
1. Treat the different diseases differently. CKD is still viewed by many as a single disease with many subtypes, but with constant reclassification and basic science research advances, we are finding that it is inappropriate to lump them together as one. Dr. Barbara Gillespie, who moderated the panel discussion, suggested that we call them “CKDs” instead.
2. Continue to support basic science research. We do not know nearly enough about how to differentiate between these different types of CKD. Right now, physicians often have to look for very small details in histological samples in order to properly diagnose. Biomarkers often present the easiest and clearest path for accurate diagnosis, but we do not yet have enough evidence on biomarker specificity for widespread use. GFR and creatinine are not specific enough. As Mr. Bowden stated at the panel, “What we need to do is to keep working on getting biomarkers more defined to be able to differentiate between the different etiologies. This is going to be the path forward.”
3. Listen to what patients consider relevant health outcomes. Patients do not care what their GFR is. They care about how fatigued they feel and how swollen their ankles are. GFR is easier to measure and more objective, so that’s why it has won out as an outcome, but sponsors must remember that patients won’t commit to a 5-year-long study that doesn’t promise to improve some of the things that they care about.
4. Collaborate with academics to write more effective and feasible study protocols. Academics see the patients and know what they can and will take on, but they don’t talk to stakeholders. Sponsors are very attuned to stakeholders’ needs, which is necessary, but they don’t always know what’s most important to the patient. Isn’t the drug being developed for the patient? Collaboration is the only way to remediate this.
5. Tell patients how severe their renal disease is by using numbers. The point was made that patients with diabetes know what their HbA1c is at any given moment. Why don’t patients with CKD know their GFR? Give them numbers. Tell them that they have stage 2 kidney disease. Get them to know their numbers. Point-of-care devices that measure GFR may be in order. The disease may not have any symptoms early on, but if patients were better aware of the direction they were headed in, they may be more proactive with treatment and may be inclined to participate in clinical trials.
6. Screen for kidney disease. Can you believe that 94% of patients with hypertension and 61% of patients with diabetes are not routinely screened for CKD? These are two of the strongest risk factors for developing CKD. This could be because the tests are costly and time-consuming, but earlier detection and management of CKD may offset this cost if patients are able to take more preventive measures and have more productive years of life.
The CKD community has set some lofty goals for the coming years. There are hopes to enroll 30% of patients with CKD in a clinical trial by 2030. This is important to create a more substantial research base for CKD. On a positive note, we are making advances that will help with this, such as more comprehensive patient registries, so this goal may actually be attainable.
Another important consideration brought up by Dr. Gillespie is that sponsors may have a hard time estimating their market size and return on investment, since most patients are not aware that they have CKD. This makes it difficult for sponsors to rationalize investing in CKD trials. The most simplistic solution for this might be to make the patients better aware of their disease.