Sunday, December 23, 2018

PKD Treatment: Concern for New Medicare Policy, Lixivaptan in Phase 2 Testing

PKD Treatment Policy

From Commercial Appeal, Karyn Waxman, Guest columnist

New Medicare policy will harm patients like me | Opinion


Every day, 12.5 million Americans struggle with polycystic kidney disease, a disease that causes numerous cysts to grow in the kidneys, slowly reducing kidney functionality and possibly leading to kidney failure. I am one of them.

As the Memphis chapter founder and coordinator for the PKD Foundation, I work to help provide a forum for patients who want to find treatments and cure this disease.

That’s why I was alarmed by the Centers for Medicare and Medicaid Services’ new Medicare Advantage Part B step therapy policy, which would begin implementation next year.

Under step therapy, or “fail first,” health insurers require patients to use more cost-effective drugs before using more complex drugs, if the first drug doesn’t work. While this method may work for other parts of Medicare where generic and name brand drugs are indistinguishable, Medicare Part B drugs are not compatible with step therapy drugs.

Part B covers treatment for many serious conditions like PKD and relies on treatment that is tailored to the biology of each patient.

If health insurers forced doctors to prescribe less effective Part B treatments in the name of cost cutting, their zeal for cutting corners may backfire, leading to an increase in visits to the emergency room by Medicare Part B patients who respond poorly to the less effective treatment. As a result, the cost of treatment would rise exponentially, impacting the entire health care system and increasing costs for consumers.

This method also violates the trusted relationship between a doctor and patient. Doctors who treat patients with PKD often forge relationships with those patients that last years, forming a partnership to find the right combination of drugs that will allow each patient to manage their condition and retain a normal quality of life.


When an insurance company steps in on this years-long process in order to dictate which drugs it will allow, it throws years of hard work and collaboration out the window, jeopardizing both the doctor-patient relationship and the health of the patient.

This issue is incredibly personal for me. As someone who struggles with PKD, I anticipate undergoing a kidney transplant from a living donor in the next year. In order to make sure my body doesn’t reject the kidney, I’ll need to undergo a specific regimen of medications, tailored to my own body, for the rest of my life. I’ll be immunosuppressed, meaning I’ll be more susceptible to infections and certain types of cancer.

I can’t afford to “fail first,” or to gamble with drugs that an insurance company might choose for me based off of their own bottom line. This backwards approach takes away the rights of the doctor and the patient, and too often leads to increase in costs on the entire healthcare system.

Patients who battle PKD require timely care and should be permitted to opt out of step therapy, if their health or financial situations change. We simply can’t afford to try a cheaper, riskier treatment for a prolonged period with little success when there is an existing treatment that works. While health insurance companies want to pinch pennies, ultimately, we are the ones who may pay the price.

Sen. Lamar Alexander has been a champion for improving access to health care in this country. He understands that patients deserve to be taken seriously, and a doctor’s expertise should not be overrun by the will of an insurance company. That is why I urge Sen. Alexander to stand up for patients and doctors’ rights and reverse this harmful policy.

Karyn Waxman is the Memphis chapter founder and coordinator for the PKD Foundation.




From Business Wire

Palladio Biosciences Announces Dosing of First Patients with Lixivaptan in ELiSA, a Phase 2 Clinical Study in Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Palladio Biosciences, Inc. (Palladio) http://palladiobio.com/, a privately held biopharmaceutical company founded to develop medicines that make a meaningful impact on the lives of patients with orphan diseases of the kidney, today announces the dosing of the first patients with lixivaptan in ELiSA, a phase 2 clinical study in patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD).

The ELiSA study (Evaluation of Lixivaptan in Subjects with ADPKD), which is being conducted at several clinical sites in the United States, will look at how well two different doses of lixivaptan work in preserving kidney function. It will also look at how well adult subjects with ADPKD tolerate the study drug, which will be given by mouth for 7 days. The study is enrolling male or female ADPKD patients between 18 and 60 years of age and estimated Glomerular Filtration Rate (eGFR) greater than 30 mL/min/1.73m2. Completion of the ELiSA study will pave the way for the initiation of a Phase 3 registration study in 2019.

“Dosing the first ADPKD patients with lixivaptan is a major milestone for Palladio Biosciences,” said Lorenzo Pellegrini, CEO of Palladio. “The ELiSA Study is a key component of our clinical development program and will be used to inform the design of our upcoming pivotal Phase 3 study. We believe that lixivaptan has the potential to meet some of the many unmet medical needs of ADPKD patients.”

David Baron, Chief Scientific Officer of the PKD Foundation, said, “PKD is part of America’s under-recognized kidney disease epidemic. We at the PKD Foundation continue to support the development of new drug therapies for patients with PKD, in part by increasing awareness of PKD clinical studies. We look forward to seeing results of the ELiSA trial and the continued development of lixivaptan for ADPKD.”

To learn more about the trial, visit the Clinicaltrials.gov website at https://www.clinicaltrials.gov/ct2/show/study/NCT03487913.Additional details can also be found at http://palladiobio.com/clinical-trials/ and https://pkdcure.org/clinical-study/elisa-evaluation-lixivaptan-subjects-adpkd/.

About Lixivaptan:

Lixivaptan was granted Investigational New Drug (IND) clearance to proceed with a Phase 2 clinical trial of lixivaptan capsules in patients with ADPKD in the first half of 2018. It had previously received orphan designation by FDA for the treatment of ADPKD. It is a potent, selective vasopressin V2 receptor antagonist, a mechanism of action that has clinical proof of concept to slow kidney function decline in adults at risk of rapidly progressing ADPKD. Lixivaptan was previously administered to more than 1,600 subjects across 36 clinical studies as part of a prior clinical development program for the treatment of hyponatremia. Palladio expects to leverage lixivaptan’s large body of data generated in the hyponatremia clinical program to accelerate the development of lixivaptan for the treatment of ADPKD.

About Polycystic Kidney Disease (PKD) – Key Facts and Figures:

PKD is an inherited genetic disease that affects thousands of people in the United States and millions globally. ADPKD is the most common type of PKD. A person with ADPKD has a 50 percent chance of passing the disease on to each of his or her children. The disease is characterized by uncontrolled growth of fluid-filled cysts in the kidney, which can each grow to be as large as a football. Symptoms often include kidney infections and chronic pain. The continued enlargement of cysts and replacement of normal kidney tissue causes irreversible loss of renal function. In the United States, approximately 2,500 new people with PKD require dialysis or a kidney transplant every year, making PKD the 4th leading cause of kidney failure. There is no cure for PKD.

About Palladio Biosciences, Inc.:

Palladio Biosciences is a privately-owned, clinical stage biopharmaceutical company developing medicines for orphan diseases of the kidney and is located in Newtown, PA. For more information, please visit www.palladiobio.com.

About the PKD Foundation:

The PKD Foundation is the only organization in the U.S. solely dedicated to finding treatments and a cure for PKD to improve the lives of those it affects.

Our vision is to #endPKD. To learn more about PKD, please visit the PKD Foundation website: https://pkdcure.org/resources/category/advocacy/#

Please note that lixivaptan is for investigational use only.

Contacts
Palladio Biosciences, Inc.
Linda Hogan, +1 908-294-8728
lhogan@palladiobio.com

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