Washington Summary May 2018

Tolvaptan (brand name JYNARQUE™)

In April 2018, the FDA approved the first drug available in the US to treat ADPKD.

Tolvaptan is a selective vasopressin V2-receptor antagonist. This means it blocks vasopressin, an anti-diuretic hormone secreted by the pituitary gland, which plays a key role in the fluid balance of the kidneys. Until now, the only treatment for people with PKD was dialysis or a kidney transplant.

PKDF held a webinar on April 30 that provides important information on this drug and its availability. For more information, go to pkdfoundation.staging.wpengine.com/jynarque.

PKDF PKD Research Funding Priorities

PKD Foundation has submitted its FY19 funding requests to the House and Senate Appropriations Committees.

PKDF has asked Congress to return ADPKD to the list of eligible research programs under the Department of Defense’s Peer Review Medical Research Program (PRMRP). In addition, we have requested $20 million specifically for PKD research through the Congressionally Directed Medical Research Program’s (CDMRP). Since 2006, PKD researchers have received $25 million in medical research grants, including funding for the ongoing metformin and statin clinical trials.

With regard to NIH, PKDF is part of the Friends of NIDDK coalition that is requesting $2.165 billion for NIDDK, an increase of $194 million from the final FY18 level. On its own, PKDF supported this proposed increase and also has advised the two Appropriations Committees of its support for having the NIH advise Congress of NIH’s future PKD research plans.

Affordable Care Act (ACA) Cost Sharing

Congress has been unable to find a way to stabilize the ACA individual insurance market by providing cost-sharing reduction (CSR) funds. Sen. Susan Collins (R-ME) still hopes that she will get the Senate vote that she was promised last year. However, Chairman Lamar Alexander (R-TN) of the Senate HELP Committee, who had been working on the issue with Sen. Patty Murray (D-WA), reportedly now is working with just the Trump administration on proposals to stabilize the individual insurance market place.

Insurance Coverage for Essential Health Benefits and Pre-Existing Conditions

The administration has announced additional proposals that would revise or replace major ACA provisions. In general, the recent April rules give states more authority over ACA-compliant insurance plans. The rules retain essential health benefits; but states could limit some benefits such as office visits or prescription drugs.

In April, PKDF joined more than 100 other patient advocacy groups in expressing to Congress our concerns about the impact of the new short-term insurance plan rules on patients. The groups believe that these rules would “limit access to quality and affordable health insurance coverage for all Americans, and disproportionately harm individuals with pre-existing conditions and people with disabilities. “

PKDF also signed a letter to CMS on short-term plans.  The letter expressed concerns similar to the ones mentioned above.

Additionally, some states are taking their own actions. In late January, Idaho announced that it would permit the sale of non-ACA compliant policies to its residents. Under these policies, insurers could charge higher premiums for people with preexisting conditions. On March 8, CMS advised Idaho officials that the state’s plan fails to enforce essential ACA provisions. Idaho officials still believe that their proposal can be modified to comply with ACA.

North Dakota officials are studying Idaho’s proposal and other options for reducing insurance costs in that state.

Elsewhere, Iowa has a new law that would allow the Iowa Farm Bureau and Wellmark to sell non-ACA-compliant health benefit policies. The plans would not be considered insurance and would not have to cover people with pre-existing health conditions.

The House Agriculture Committee’s farm bill (HR 2) contains language that would fund rural associations that offer health insurance to their members. The farm bill plan is similar to ACA’s cooperative plans, which suffered financial losses. Whether rural co-op plans would provide more insurance options and lower premium costs cannot be determined at this time. The House has not yet considered HR 2.

If you receive your health insurance through a non-ACA plan, you still should monitor future activities in your home state.

Bills of Importance to the PKD Community

  • Living Donor Protection Act (HR 1270/no Senate bill yet) would remove barriers to living organ donation. Rep. Jerrold Nadler (D-NY) and Rep. Jaime Herrera Beutler (R-WA) introduced the bill. PKD and several other patient groups have signed a letter urging House Members to cosponsor HR 1270.
  • The OPEN Act (HR 1223/ S 1509) would make it easier for companies to repurpose approved drugs for treating rare diseases. Reps. Gus Bilirakis (R-FL), GK Butterfield (D-NC), and Mike McCaul (R-TX) introduced HR 1223.  Sens. Orrin Hatch (R-UT) and Robert Menendez (D-NJ) introduced S 1509.

Say Thanks to Supporters

The following Senators and Members of Congress have cosponsored either the OPEN Act or the Living Donor Protection Act since the previous newsletter.   If any of them represent you, please say “thank you” the next time that you contact them.

HR 1223, the OPEN Act

  • Rep. John Culberson (R-TX)

HR 1270, Living Donor Protection Act

  • Rep. Andre Carson (D-IN)
  • Rep. John Duncan, Jr. (R-TN)

Stay Alert

When the time comes, we will ask PKD advocates to immediately contact their elected officials to protect your interests. Your voice needs to be heard.

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UPDATED:  May 8, 2018