REPRISE: The Patient Perspective

Tolvaptan in Later-Stage Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Patient Point of View 


Our family has seen the devastating impact of ADPKD on at least four generations.  Starting with my maternal great grandmother, we have had multiple family members die prematurely.  I grew up listening to my mom tell stories about how her father died of renal failure at the age of 59 in 1958.  My mom also had ADPKD and died at 52. I was diagnosed at 39, and had a pre-emptive kidney transplant at 43.  After speaking with nephrologists we have decided not to test our children, at this time.

Growing up and seeing my mom, Maryanne, suffer from ADPKD left a deep imprint. I saw her suffer greatly.  She had the constant back pain, fatigue, and nausea.  She suffered from these symptoms while she was raising three children. I never once heard my mom complain. Her children were her priority.

I grew up in the 1960’s and 1970’s when there was little known about ADPKD. My mom faced the uncertainly with an optimistic outlook, and served as an example for me to emulate later in life.  I remain grateful for her leadership.

Against this background, I grew up with the fear of having ADPKD. I chose to bury my head in the sand and not determine whether I had this genetic disease .  In many ways, it made sense because there were no treatments for ADPKD.  I knew that if I was diagnosed, I would have difficulty getting both health and life insurance   Moreover, I saw my mom suffer on dialysis, and the prospect of going on dialysis scared me to my core.  I literally saw my mom die slowly over four and a half years.

Hopefully, we are entering a new area for ADPKD patients.  If tolvaptan is approved by the FDA, US ADPKD patients will have their first treatment.  This is significant for several reasons:

  1. It offers patients hope which is very powerful.  When I was diagnosed with ADPKD, I was at CKD stage 3-B.  Upon initial diagnosis, my nephrologist anticipated that I would experience renal failure in 5-7 years.  Unfortunately, I progressed to renal failure in three years.  I would have loved to have the option of having a treatment that could have possibly delayed my renal failure.  The value of extended time creates the opportunity to plan for a living donor rather than dialysis.
  2. Ostsuka investment in ADPKD has had several strategic benefits for patients:
  • Through their investment in ADPKD, Otsuka has collaborated with the FDA on (hopefully) a path to approval.  These collaborative discussions offer a wealth of lessons and instruction for future drugs.
  • It has spurred further investment by pharma into ADPKD.  There are now several pharma companies developing treatments for ADPKD
  • For the ADPKD patient experiencing renal decline, the treatment options are no longer binary, dialysis or kidney transplant.  If Tolvaptan is approved, it provides an opportunity for nephrologists to have a risk-benefit conversation on a treatment option. 

If approved by the FDA, Tolvaptan will be valuable for the US nephrology community.  It will challenge the US Kidney Care ecosystem which rewards renal failure.  It provides the chance to have a risk-benefit conversation on a treatment intervention.  This is a skill area that nephrologists need to develop in anticipation of the multiple upstream interventions rather than downstream treatments being developed.  This is a much welcomed and needed change for kidney disease patients.

 Kevin Fowler