HDU Board of Directors Responds to the 21st Century Cures Initiative

The Energy and Commerce Committee of the US House of Representatives has begun an initiative focusing on how government can better medical innovation and its utilization. The committee has held a number of meetings and has asked for input from potential stakeholders in this endeavor.  The HDU Board of Directors has written a response letter to the committee.  It is shared below and followed by patients’ comments.  Click here to visit the Energy and Commerce Committee’s webpage about the initiative.  Click here to see the video of the hearing on 21at Century Cures: Examinine the Role of Incentives in Advancing Treatments and Cures for Patients.

21st Century Cures: Examining the Role of Incentives in Advancing Treatments and Cures for Patients

– See more at: http://energycommerce.house.gov/hearing/21st-century-cures-examining-role-incentives-advancing-treatments-and-cures-patients#sthash.6tQb3PgB.dpuf

June 9, 2014

The Honorable Fred Upton, Chairman

Committee on Energy and Commerce

2125 Rayburn House Office Building

Washington, DC 20515

 

The Honorable Diana DeGette

Committee on Energy and Commerce

2125 Rayburn House Office Building

Washington, DC 20515

 

Re: 21st Century Cures Initiative

 

Dear Chairman Upton and Congresswoman DeGette:

 

Home Dialyzors United (HDU) appreciates the opportunity to provide comments to the Energy and Commerce Committee about the 21st Century Cures Initiative. The members and board of directors of HDU share the goals to “accelerate the discovery, development and delivery of new treatments to patients.” We also look forward to assisting the Committee to ensure that these goals are met for home dialysis patients.

 

Home Dialyzors United (HDU), a 501(c)(3) non-profit organization, is the only dialysis patient group dedicated to home dialysis. Our mission is to educate, support, and advocate for home dialysis. We believe that patients and care partners can live a normal life including employment, education, volunteerism, family life and leisure pursuits. We further believe that Congress can help their constituents with chronic kidney failure (ESRD) reach those goals.

 

Both peritoneal dialysis and home hemodialysis offer advantages over in-center treatment. Studies have shown that more frequent dialysis at home means potentially lower risk of death and hospitalization, increased energy, lower cost, fewer medications, and less stress on the heart. In addition, home hemodialysis usually offers more frequent and/or extended time dialysis, which translates into quicker recovery time after treatment, fewer side effects, and a more normal diet. Home patients enjoy more freedom and flexibility to live a normal life.

 

Despite proven benefits, too many patients are unaware of this option largely because Medicare policies are not making home treatment fully accessible to Medicare beneficiaries and new machines designed to be used in the home are often not being approved in a timely manner. Yet, the Social Security Act specifies that it is the intent of Congress that “any patient who is a suitable candidate for home dialysis should be so treated.”   Consider the following statistics:

 

  • Currently, just 10% of U.S. dialysis patients receive treatment at home, a method that is a lower cost option than treatment in traditional centers. Less than 2% of those patients use home hemodialysis treatments.
  • Only 24% of centers are certified to offer home hemodialysis (HHD), which means access to home hemodialysis may be dependent on such arbitrary factors as where the patient lives. Rural areas are traditionally underserved.

 

  • Only 12% of patients report receiving education about HHD despite the fact that Medicare requires education about all options.

 

  • Nephrologists believe that a much higher percentage of patients would be good candidates for HHD but less than 2% are currently using this treatment modality.

 

While HDU believes that well designed studies and the resultant statistical data are a vital component of research, development and delivery of better treatment, we also feel that one aspect is glaringly lacking—that of patient and care partner involvement.While scientific data derived from RCTs contribute to improvements, they do little to assess quality of life and the ability of patients and families to lead a normal life. There is no other chronic disease that, on a daily basis, affects literally every single aspect of life from the kitchen table to the bedroom and even into the broader community. Due to the nature of treatment, chronic kidney failure (ESRD) directly impacts the entire family. Therefore:

 

  • HDU strongly urges all those involved in the 21st Century Cure Initiative to include patients and care partners in every aspect of the process. Who better to offer input than those who live with chronic kidney failure every single day? With the increased emphasis on patient engagement and patient empowerment, the 21st Century Initiative has the chance to be a leader in making a real paradigm shift.

 

  • HDU also requests that the 21st Century Initiative carefully consider the alignment of new discoveries with regulatory policy to facilitate the development and approval of home dialysis friendly innovations. We urge the Initiative to expedite the delivery of new developments. A recent example of this is the FDA fast tracking of research projects and clinical trials, three of which are dialysis related.

 

  • HDU urges movement in the direction of real patient-centric outcome measures. For example, collecting data on patients who are employed and incentivizing facilities to keep those patients employed would have a nearly immediate effect on care and innovation. Not only would such a measure help meet the psychosocial needs of patients, but it would also save government dollars. According to 2010 USRDS statistics, only about 20% of chronic kidney failure patients, ages 18-54, are currently employed.

 

  • In order to foster patient and care partner engagement, HDU employs a “Buddy System” of peer mentoring. HDU also requests that peer mentoring be included in the delivery of new developments.

 

  • Lastly, we ask that the 21st Century Initiative support a “home first” culture by fostering not only new developments but also the education of all patients about the advantages of home dialysis therapies.

 

Home Dialyzors United is a voice for many patients and care partners who are living a normal life despite chronic kidney failure. In the process of developing these comments, HDU has solicited opinions from a cross section of our individual members through our social media sites. A sampling of these opinions is attached in the addendum.

 

HDU thanks the committee for the opportunity to comment on the 21st Century Cures Initiative.

 

Sincerely,

Home Dialyzors United Board of Directors

Jim Smith

Denise Eilers, RN, BSN

Benjamin Ruback

Patricia Colongione

Melissa Sondergaard

 

21st Century Cures: Examining the Role of Incentives in Advancing Treatments and Cures for Patients

– See more at: http://energycommerce.house.gov/hearing/21st-century-cures-examining-role-incentives-advancing-treatments-and-cures-patients#sthash.6tQb3PgB.dpuf

21st Century Cures: Examining the Role of Incentives in Advancing Treatments and Cures for Patients

– See more at: http://energycommerce.house.gov/hearing/21st-century-cures-examining-role-incentives-advancing-treatments-and-cures-patients#sthash.6tQb3PgB.dpuf

 

 

21ST CENTURY CURES INITIATIVE

HDU ENERGY & COMMERCE LETTER

PATIENT COMMENTS

 

What is the state of discovery of cures and treatments for your disease? Are there cures and treatments now or on the horizon?

 

Dialysis machines have evolved extremely slowly for about 50 years. I usually say that if the evolution in consumer electronics had followed that of dialysis machines, my laptop would be the size of Manhattan.

 

Then NxStage came along and changed the game. They made a machine that was (somewhat) portable so people could enjoy just a modicum of freedom. Now we are 1000’s of patients enjoying the freedom this machine gives us.

 

But many of us are waiting for truly portable dialysis machines, just like we’re waiting for devices that can be worn and/or implanted to help us overcome our kidney disease.

Henning Sondergaard

 

At this time, there is no cure for end-stage renal disease, only treatments. There are two treatment options, transplantation or dialysis. Not everyone is eligible for transplantation, many people cannot afford the expense of medication after the period of time when they become in-eligible for Medicare coverage, and furthermore, there are simply not available organs to meet the need of those waiting.

                                   

The other treatment option currently available is dialysis. The majority of patients dialyze in dialysis units, generally three times a week. Kidneys function twenty-four hours a day, seven days a week; most dialysis patients get twelve hours a week of dialysis to clean their blood. Between these intervals, toxins and fluids build up in their bodies, and systemic long-term health effects commonly occur. Immediately post dialysis, most patients who dialyze in this manner, have severe intra-cellular fluid shifts, resulting in low blood pressure that gives rise to cramping, dizziness, and a generalized feeling of exhaustion.

                                   

A small percentage of patients perform dialysis at home, either doing peritoneal or hemo-dialysis. Peritoneal dialysis uses the body’s own peritoneal membrane within the abdominal cavity to act as a filter. It is a continuous treatment and provides a reasonable standard of dialysis. However, its effectiveness tends to rely on a person have residual urine output, again, not everyone is a candidate, and after a time, the peritoneal membrane tends to lose its capability to filter effectively.

                                   

The remaining number of home patients do hemo dialysis using either a standard full-size machine, or a smaller, portable (still very heavy) machine. Home dialysis allows patients to do more frequent treatments in the comfort of their own home. This gives better long-term outcomes, lower mortality rates and a much better quality of life. The best outcomes are achieved from overnight treatments. This is currently no machine on the market that is FDA approved for home nocturnal use.

                                   

There are facilities who are endeavoring to make wearable and implantable artificial kidneys, and although on the horizon, probably within the next few years, at this time are either in the design and/or trial phase.

 

I personally do home hemo, using the more portable machine. Although listed for a transplant, a high antibody level means a transplant is an unlikely option. In the past I have done peritoneal dialysis, but this no longer works for me. I elected to do home hemo, because although onerous on time, it gives me the greatest flexibility, I feel well doing it, and it allows me greater control and independence over my life. In addition, get to spend more time with my family and it is also relatively easy to travel.

Amanda HYPERLINK “https://www.facebook.com/profile.php?id=677651841&fref=ufi”KirkbyHYPERLINK “https://www.facebook.com/profile.php?id=677651841&fref=ufi” WilsonHYPERLINK “https://www.facebook.com/profile.php?id=677651841&fref=ufi”HYPERLINK “https://www.facebook.com/profile.php?id=677651841&fref=ufi”HYPERLINK “https://www.facebook.com/profile.php?id=677651841&fref=ufi”

 

 

More money and emphasis needs to be put on WAK and the small implantable kidney. In this day and age it shouldn’t be taking so long for this big improvement.

Unfortunately we are still in the dark ages when it comes a cure, a real cure, for kidney failure, either the hereditary type (PKD) or from high blood pressure and diabetes. The best near-term hope seems to be a wearable or implantable device using sorbent technology, which may be a decade or less off and offer round-the-clock dialysis for many waiting for a transplant or those for whom a transplant is not feasible.

 

Renal failure is fast approaching epidemic proportions, worldwide. Yet, the emphasis and funding continues to be placed on treatment and not basic research into why kidneys fail. It’s a very sad comment that dialysis treatment has focused on an out-moded modality and an unproven treatment standard (3x/wk.), while too little has been spent on original research. The reason for this, I believe, is the heavy reliance on a for-profit treatment system in the U.S., which offers little incentive to innovate and seek a cure. The current dialysis system is too lucrative. Unlike other diseases, like some forms of cancer, where huge gains have been made in the last forty years, dialysis is still back in the 1970s.

 

 

 

What programs or policies have you utilized to support and foster research, such as patient registries, public-private partnerships, and venture philanthropy?

 

 

In the last year I have participated in two research programs trying to better dialysis machines. One was by a Swedish designer who wanted to make the home hemo machines more ‘humane’ the other one was by two Danish engineer students who were very keen on advancing home hemo dialysis to the next level – whatever that is.

                                   

I have also closely followed the development of the WAK and talked to its designer a few times. And lastly I keep a very keen interest in all the other technologies out there so we can get better treatment.

                                   

Unfortunately it seems like most researchers in nephrology are afraid of patient voices. There is way too little exchange between doctors and patients in our field. Patients have so much experience from their own bodies that many of us are better specialists than our physicians. Now we just need those physicians to stop being afraid of us and listen to the experts – their very own patients!

 

 

I believe we need more accurate patient attitude research on rehabilitation – on how patients feel about the present dialysis system’s limitations on lifestyle and quality of life issues. We have tons of rather irrelevant medical data, which divorces the person from their medical histories. I have just finished a project with the public policy school at USC, and am seeking financial support for a very extensive kidney patient attitude study in Southern California.    

                

Some doctors might say there are plenty ‘quality of life’ (QOL) studies. But they all are focused on irrelevant medical data that have been determined by medical staff to reflect QOL but that are really just poorly disguised data on how well people hold up to in-center treatment. We need real life data, a.k.a. qualitative data that is based on what patients find is important for them, and NOT what doctors and nurses have pre-determined should be researched about us.

Henning HYPERLINK “https://www.facebook.com/henning.sondergaard?fref=ufi”SondergaardHYPERLINK “https://www.facebook.com/henning.sondergaard?fref=ufi”HYPERLINK “https://www.facebook.com/henning.sondergaard?fref=ufi”HYPERLINK “https://www.facebook.com/henning.sondergaard?fref=ufi”

 

How can Congress incentivize, coordinate, and accelerate basic research for diseases we know relatively little about?

 

The most major issue I have with dialysis and renal disease in general is that the questions that they are all trying to ask are so obviously made for doctors by doctors. Nobody ever asks what is important for patients.

 

Nobody ever looks at quality of life (QOL) issues. There are 1000’s of research papers that claim to focus on QOL but they are all looking at medical issues that doctors have determined must be QOL issues – NOT what patients really think about their life and their situation as dialysis patients.

 

Then, of course, there’s the issue of home dialysis. When is somebody going to take a good hard look at the benefits of it over in-center treatment? We all know how much better it is so why is it only 1.3% on HHD and 7.3% who are on PD at home? This is appalling numbers and something serious needs to be done to make this (all the more humane) treatment option available to EVERYONE who is eligible and willing. And it needs to be done yesterday!

 

 

Shoulda been done yesterday!

 

 

As you said the majority of the machines are made so health personnel can STAND and control the machine. The way I see it machines need to be able to be controlled by patients lying or sitting down….we have a long way to go….now we are not just surviving….but actually live good lives. That is surely the new goal…bringing patients into the game.

Malene Madsen

 

 

 

Addressing education about treatment choices: When and how are these choices presented?

                 

 

  •          “My husband was never told of any options. He started hemo in the hospital last April and was never given any option of hemo or PD. Then once he was out and in center he was never given any other options. I was the one who did all of the research online to figure out his best options and to find places that did home hemo and sit down to talk with them about the training process, etc.”
  •  
  •          “We found it [home HD] on our own and pursued the option on our own with a little help from our PCP. They may have mentioned PD at first, but more or less pushed for hemo. PD was something they later told us was an option but I don’t know of anyone who went to the center who ever actually did it…even now I only know of two people in our area who do PD and one of them travels an hour away to the same center that we go to. IMO if what they offer is the best option for you that’s fine but if there’s another option, especially a better option, they should at least give you the opportunity to make your own choice even if they don’t offer it themselves!”
  •  
  •          “We were told about PD in our initial nephrology visit but in-center hemo seemed to be their main push. I think they anticipated that my Mom would be on dialysis almost immediately after her first visit based on numbers. But, at 81, she still wants to have all her questions answered before making a decision. So we looked for answers to her questions and ended up on PD after three years of clinic visits. I believe that part of the challenge in our case was that their clinic is segmented. You have in-center dialysis nurses and then you have home dialysis nurses.”
  •  
  •              “My center gave me options: in-center, PD, or no treatment! They had not heard of Buttonholes or told me, ‘They are not safe or effective.’ I had a VERY difficult access AND a very real needle phobia. My Neph agreed HH would be better for me. My clinic REFUSED to allow me to do a Buttonhole b/c they had no techs trained in it! After 8 years the clinic got a new director. She was appalled that I had NEVER been told my HH options AND that when I asked, I was routinely told it is waaaaaaay more dangerous to do HH! As I look back, my heart is sad that I stayed in that stupid center 9 years total!”
  •  
  •          “I was offered in center only, PD was mentioned briefly, but only in a passing manner while I was in the middle of a treatment. I was never told about NxStage. I researched it myself and switched centers to get it. I think I found out about it from ihatedialysis.com. I also got a load of garbage from my old center about how I was going to die if I did home hemo with NxStage. I know it’s because I was still in the middle of my 30 month coordination period and they were raking in the cash from my insurance company.”

 

 

I don’t remember exactly when the options were introduced (I was in center just under a year and remember having a first real conversation about home hemo briefly early on, and re-visited towards the end), but I was told initially that I would not be able to do home hemo because I was single and did not have a “caregiver”. Within a year, I wanted to get out of the center environment so badly, I proved them wrong and completed the training! I just celebrated a year of doing home hemo solo. My home team is learned, accessible, and supportive. It is like night and day, and although there were trade-offs (more work, more time on the machine, etc….), at least I can concentrate on my health and my sanity. I have not infiltrated or bruised myself and my fistula is thankful for it. I am extremely grateful for home hemo, and wishful for better solutions for all kidney patients in the future.

 

Two years ago, I was told there was no hope of a cure, my disease was progressive, and my expected average mortality rate was 3 years. I’ve been told a transplant has a 5 year wait time for my blood type with a high probability that the disease will return to the new kidney.

Robin Franzi

 

 

 

On behavioral psychology and measuring the benefits of dialysis.

 

I am all about promoting and educating people on the BENEFITS of proper dialysis treatment. This section of a lecture from Harvard on US Health Policy had some examples about Organ Donation and how many more organs are available with an OPT OUT system, and some behavioral psychology notes that I think have everything to do with the problems involving the current treatment models of dialysis. 1) One is that we know that human beings have trouble processing large numbers of options. So that would include both physicians and patients. If you give them a lot of choices they may actually make worse choices than if you give them a few choices. 2) it’s also true that people have trouble making choices that have different consequences over time. 3) Another bias is that people really like what they have, in particular, and they are particularly averse to losses, in a way that’s not symmetric with the way they look forward to potential gains, in terms of things that they don’t have. 4) Here’s another important phenomenon called the status quo bias that people are likely to avoid a choice where they can. And so they favor wherever they are now versus any other option to a degree that is not optimal. 5) We know one rule of thumb that people use is pick the top plan or pick the top choice. It’s easy. It’s at the top of the list so organization and presentation of information matter

These behavioral situations totally scream KIDNEY & DIALYSIS to me, and how it is currently presented to anyone dealing with it.

Mikey Hann