Prepared statement from the American Association of Kidney Patients (AAKP) to the FDA's Cardiovascular and Renal Drugs Advisory Committee given by AAKP's Vice President Paul T. Conway. 

October 18, 2010

My name is Paul Conway and I appear before you today as a patient who has managed kidney disease failure for over 30 years, as a taxpayer and as the Vice President of the American Association of Kidney Patients. I appreciate the opportunity and privilege to participate in this stage of the public policy process and to provide comments on Erythropoiesis-Stimulating Agents or ESAs.

As a kidney patient, my personal journey has been marked by end stage renal disease, long periods of anemia, ESA therapy, nearly two years of peritoneal dialysis, organ transplantation, ongoing immuno-suppression and disease management. Along the way, I have dealt with a multitude of other conditions that stem from this disease. At each stage in the journey, my life has been preserved and extended by skilled teams of specialists, nephrologists, surgeons and nurses who utilized their expertise and knowledge of my health conditions to custom-tailor life sustaining treatments and medication regimens. I am deeply grateful to these selfless heroes. I am also respectful of the untold number of other professionals whose pioneering research, breakthrough medical procedures and pharmaceuticals aided their efforts.

Based on my direct experience, I know the impact of anemia on mental and physical stamina, and the beneficial impact of ESA’s. My views are supported by the experiences of countless other patients from all walks of life whose paths have intersected with mine and through conversations with medical experts across the county.

I respectfully offer three key points to your deliberations as an FDA Advisory Committee. First, we must avoid blood transfusions. Second, dialysis and chronic kidney disease (CKD) patients are different. Third, the decision on what therapy to use must remain between the patient and their doctor, and not usurped by disinterested parties who know nothing about the person who suffers or their makeup as an individual.

Blood Transfusions

Stated simply, ESA therapy has been a breakthrough in dialysis patient management. It has reduced the need for blood transfusions significantly. Transfusions lead to transplantation antibodies, hepatitis, increase hospitalizations, patient and health care costs and loss of personal productivity. A patient with radiation colitis and renal failure would have been hospitalized every week in 1978 for a transfusion. Now, that patient can simply receive ESA injections and maintain a target hemoglobin value without a blood transfusion. The number of transfusions dropped from nearly five hundred per thousand patient years to a little over two hundred between 1992 and 2005 – a change due directly to ESAs.

Dialysis VS Non-Dialysis

Through the years, ESA therapy has evolved as a result of randomized controlled trials, but mainly in the non-dialysis population. It is critically important to match the scientific evidence to the population sampled. When a person is perfectly healthy, he or she may lose around 0.83 cc of blood per day. A CKD patient may lose around 3.15 cc of blood per day and a hemodialysis patient may lose 6.27 cc of blood per day. Thus, patients on dialysis are constantly losing blood, are even more prone to anemia and have a definite need for ESAs.

Individualized Care

Future policies or potential policy modifications regarding anemia should assure that drugs are used safely and that ESA therapy be individualized to the needs of the patient by their medical team. Anemia causes tremendous fatigue in patients with kidney disease.

In my case, before my nephrologist and I agreed to start ESA therapy, it was a challenge to simply get out of bed in the morning, and remaining fully engaged an entire day required tremendous personal discipline. At the time, I served as the Deputy Secretary of Health and Human Services for the Commonwealth of Virginia and both my work and schedule were demanding. Had it not been for ESA therapy and the unwavering support of Governor George Allen and his team, it would have been extremely difficult to continue my duties. ESA therapy allowed me to pursue my commitment to public service and afforded me the ability to contribute to society as a taxpayer instead of tapping assistance that would have rightfully gone to those in greater medical need. Take it from me – the fog and exhaustion caused by anemia can, to paraphrase President Theodore Roosevelt, remove a person from the arena and change them from doers to mere observers.

Studies have shown that raising the hemoglobin from 10 to 12 g/dL increased the Kidney Disease Quality of Life domains for physical activity and for feeling good. It was also shown that the patients in the TREAT trial that had the higher mortality responded poorly to darbepoetin and had the highest incidence of heart disease, implantable defibrillators, strokes and heart attacks.

Thus, patients who are active and healthy with minimal kidney disease may wish to have their hemoglobin around 12 g/dL. Those patients who are sicker, and are resistant to ESAs should have lower hemoglobin. Once again, this decision should be made by the doctor and the patient.

I would like to thank the Advisory Committee for the opportunity to appear here today. On behalf of patients like me and the American Association of Kidney Patients, I would respectfully request that you carefully weigh comments offered by the patients whose lives, families and livelihoods have been positively impacted through the effective use of ESAs.