POLICY AND ADVOCACY

Background

There are approximately 150 different types of rare kidney diseases, each affecting between 20,000 to 200,000 individuals nationwide. These are the most aggressive forms of kidney diseases that, if left undiagnosed or are not treated properly, often lead to kidney failure.

Unfortunately, the causes of rare kidney disease are poorly understood, and many have no FDA-approved treatments. Rare kidney diseases contribute to the more than $124.5 billion spent on treating Medicare beneficiaries with chronic kidney disease, including the $42.9 billion spent on treating people with end-stage kidney disease (ESKD), yet there has been little to no innovation in diagnostics or upstream treatments for kidney disease patients since the 1940’s and 1950’s when dialysis and immunosuppressants became commonplace. These treatment and diagnostic barriers, coupled with the lack of rare kidney disease awareness and education, often cause a delay in diagnosis that can result in a rapid decline in kidney function and, ultimately, kidney failure. For many rare kidney disease patients, their only options are dialysis, transplant, or death.

Additionally, disproportionately affected communities face a lack of adequate diagnostics and treatment. In fact, Black Americans are up to five times more likely to develop kidney disease than white Americans.

Patients with or at risk for chronic and rare kidney disease are navigating a current system that pushes them to move to Medicare and late-stage coverage to manage their condition in costly ways. Our current incentive structure essentially keeps patients with kidney disease in the waiting room until it is too late. Gaps in early detection programs and insufficient diagnostic measures then lead to higher downstream healthcare costs and worse health outcomes for patients.

Solution

The New Era of Preventing End-Stage Kidney Disease Act was first introduced in the 117th Congress (2022) to respond to the growing need to reshape the way we treat a disease that impacts millions of Americans and costs billions every year. Building on the momentum from the previous Congress, the bill will be reintroduced this Congress by Representatives Gus Bilirakis (R-FL) and Terri Sewell (D-AL).

This critical bill would improve, develop, and deliver health care services to people with rare kidney disease by supporting research, promoting early intervention and diagnostic protocols; addressing health disparities in rural and disproportionately affected communities; improving provider education; and reducing dialysis and transplantation costs.  

(Of particular interest to members of the House and Senate Appropriations Committees)

Background

There are approximately 150 different types of rare kidney diseases, each affecting between 20,000 to 200,000 individuals nationwide. These are the most aggressive forms of kidney diseases that, if left undiagnosed or are not treated properly, often lead to kidney failure.

Unfortunately, the causes of rare kidney disease are poorly understood, and many have no FDA-approved treatments. Rare kidney diseases contribute to the more than $124.5 billion spent on treating Medicare beneficiaries with chronic kidney disease, including the $42.9 billion spent on treating people with end-stage kidney disease (ESKD), yet there has been little to no innovation in diagnostics or upstream treatments for kidney disease patients since the 1940’s and 1950’s when dialysis and immunosuppressants became commonplace. These treatment and diagnostic barriers, coupled with the lack of rare kidney disease awareness and education, often cause a delay in diagnosis that can result in a rapid decline in kidney function and, ultimately, kidney failure. For many rare kidney disease patients, their only options are dialysis, transplant, or death.

Additionally, disproportionately affected communities face a lack of adequate diagnostics and treatment. In fact, Black Americans are up to five times more likely to develop kidney disease than white Americans.

Therefore, it is crucial that the Department of Health and Human Services continues to expand on its critical work on improving diagnostics and treatments for rare kidney disease. Investment into the National Institutes of Health has provided the research that has led to the development of the Kidney Health Gateway Project and the increase in accessible clinical trials.  

Solution

Each year, Congress balances medical research against other federal priorities and decides how much funding will be provided to NIH through the appropriations process. For more money to be spent on Focal Segmental Glomerulosclerosis and nephrotic syndrome, the size of the overall pie needs to grow.

For FY 2024, the community is asking Congress to:

1. Provide $50.924 billion for the National Institutes of Health (NIH)
2. Provide a proportional increase for the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) and the National Institute on Minority Health and Health Disparities (NIMHD) and support the expansion of the RKD research portfolio at NIDDK and NIMHD by funding more research into primary glomerular disease.
3. Provide $11 billion for the Centers for Disease Control and Prevention (CDC) and $6 million for the Chronic Disease Education and Awareness Program.

(Of particular interest to members of the House and Senate Appropriations Committees)

Background

Each year, the Department of Defense Peer Reviewed Medical Research Program (PRMRP) designates a list of conditions that researchers can select from to apply for funding to study. NephCure volunteer advocates have so far successfully secured FSGS on the list of designated diseases each year since 2016 and nephrotic syndrome more recently.

Inclusion on this list allows FSGS and nephrotic syndrome researchers to access up to approx. $360 million in research funding each year, far more than the RKD community could generate on its own. Researchers studying FSGS have successfully competed for over $43 million for FSGS research to better understand the causes, diagnostics, and personalized medicines for FSGS. 

FSGS and nephrotic syndrome are rare diseases that attack the kidney’s filtering units (glomeruli) and can cause serious scarring, leading to permanent kidney damage and kidney failure.

In the U.S., approximately 40,000 patients are living with FSGS, and more than 60% of patients do not have a durable response to current FSGS treatments. Because of this, 50% of patients with FSGS will progress to kidney failure.

There are approximately 20,000 FSGS patients with end-stage kidney disease (ESKD), but only around 1,000 receive kidney transplants every year. Unfortunately, FSGS comes back to attack the new kidney 30-50% of the time.

Solution

The eligible conditions list changes each year, and being included one year is not a guarantee of continued participation. Legislators decide what conditions to place on the eligible conditions list based on feedback from constituents. Researchers suggest there are new opportunities for investigating FSGS and nephrotic syndrome in the military population with respect to environmental exposures. An in-depth characterization of military FSGS cases using the Department of Defense serum bank to look at baseline and subsequent exposures could lead to a new area of investigation with immediate relevance to military readiness, troop health and the cost of long-term medical care for CKD.

For FY 2024 we are asking legislators to continue to support the inclusion of “Focal Segmental Glomerulosclerosis (FSGS)” and “nephrotic syndrome” as eligible for study through the PRMRP.  

Use these links to find out if your members of Congress are already listed as co-sponsors. If so, great! Please thank them and move on to the other asks.

 Background

People who donate an organ to save another’s life continue to face discrimination when seeking life, disability and long-term care insurance. Also, they may face discrimination in the workplace when requesting time off to recover from surgery.

About one-third of all kidney transplants performed in the U.S. are living-donor kidney transplants. The other two-thirds involve a kidney from a deceased donor. And while the risks of living-donor kidney transplant are similar to those of deceased-donor kidney transplant, living donors offer increased benefits to rare kidney disease patients, including:

• Significantly reducing the time patients are on the waitlist
• Potentially avoiding dialysis if it has not been initiated
• Increasing short and long-term survival rates.

Giving the gift of life is the most selfless decision anyone can make. When living donors are protected, more people will be encouraged to donate an organ.

Solution

To protect the people who selflessly give the gift of life by donating an organ from discrimination by insurers and employers, the Living Donor Protection Act was introduced in the House by Representative Jerrold Nadler (D-NY-12) and in the Senate by Senator Kirsten Gillibrand (D-NY).

The bill prohibits the denial of coverage or increases in premiums of life or disability insurance for living organ donors. It would also ensure that living donors are protected by the Family & Medical Leave Act (FMLA) which enables them to take time off work to donate a kidney and recover from the surgery without fear of losing their job. The U.S. Department of Labor (DOL) issued an opinion letter in 2018 that stated that organ donation is protected under FMLA. The Living Donor Protection Act would codify the DOL opinion, protecting under federal statute those living donors whose employers are covered under FMLA.

The bill would also require that within six months after enactment, the Secretary of Health and Human Services review and update materials related to living organ donation in order to educate the public on the benefits and risks of living organ donation and the impact of living organ donation on the access of a living organ donor to insurance.

Use these links to find out if your members of Congress are already listed as co-sponsors. If so, great! Please thank them and move on to the other asks.

Background

Health insurers may force a patient to try a different, less expensive medication before they cover a medication they deem to be more costly. This process is called step therapy because the patient may have to go through one or more "steps" to access the medication their doctor prescribed or one the patient has used before with success. 

In many cases, the step therapy medication may not work well and the patient’s symptoms may get worse or take a longer time to heal and in the RKD population, permanent damage could happen during the time it takes to get on the appropriate medication. 

Solution

The Safe Step Act, H.R. 2630/ S. 652. was introduced in the House by Representative Brad Wenstrup (R-OH-02) and in the Senate by Senator Lisa Murkowski (R-AK).

S.652/H.R.2630 requires a group health plan to establish an exception to medication step-therapy protocol in specified cases. A medication step-therapy protocol establishes a specific sequence in which prescription drugs are covered by a group health plan or a health insurance issuer. 

A request for such an exception to the protocol must be granted if: 

• An otherwise required treatment has been ineffective, 
• Such treatment is expected to be ineffective and delaying effective treatment would lead to irreversible consequences, 
• Such treatment will cause or is likely to cause an adverse reaction to the individual, 
• Such treatment is expected to prevent the individual from performing daily activities or occupational responsibilities, 
• The individual is stable based on the prescription drugs already selected, or 
• There are other circumstances as determined by the Employee Benefits Security Administration.

The bill requires a group health plan to implement and make readily available a clear process for an individual to request an exception to the protocol, including required information and criteria for granting an exception. The bill further specifies timelines under which plans must respond to such requests.

Use these links to find out if your members of Congress are already listed as co-sponsors. If so, great! Please thank them and move on to the other asks.

Background

The HELP Copays Act (H.R. 830/S. 1375) led by Congressman Buddy Carter (R-GA) in the House and Senator Roger Marshall (R-KS) in the Senate closes a loophole that allows many employer health plans to deem certain covered drugs as “nonessential,” which means that the insurer will not count any cost-sharing toward the patient’s deductible and out-of-pocket maximum. This bill will require all private plans to count all cost sharing for covered services to accrue to a patient’s deductible and out-of-pocket maximum.

Solution

This bill requires health insurance plans to apply certain payments made “by, or on behalf of” a plan enrollee toward a plan's cost-sharing requirements. Specifically, plans must apply third-party payments, financial assistance, discounts, product vouchers, and other reductions in out-of-pocket expenses toward the requirements. The HELP Copays Act is a two-part solution that:

• Clarifies the ACA definition of cost sharing to ensure payments made “by or on behalf of” patients count towards their deductible and/or out-of-pocket maximum.
• Closes the EHB loophole to ensure that any item or service covered by a health plan is considered part of their EHB package and thus cost sharing for these must be counted towards patients’ annual cost sharing limits.