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Children with medical complexity are high users of acute health care, but little is known about their service use across the continuum of care services and in the context of overall health care expenditures.
Although accounting for ,1% of the child population, children with medical complexity use almost one-third of all pediatric health care expenditures and make multiple transitions across providers and health care settings.
To say our health care system is in flux would be timid; a better descriptive term may be tumultuous. During times such as these the medically complex are at risk. No one can be more fragile and at risk than a child who is totally dependent and unable to advocate for themselves. They in turn are totally reliant on their parents and caregiver support system. Yet, in the case of a medically fragile child with extremely complex disorders that frighten even physicians, they find themselves dependent upon parents who are also fragile and dependent. The parent/caregiver is, more often than not is stricken by unrelenting grief that is attendant to the loss of the perfect child; and they are intensely aware that the complexity and chronicity of the child’s condition has forever changed their lives and that they face years or decades of care, or a lifetime of the complex childcare.
They are not alone in this odyssey, doctors and other pediatric providers are facing these pressures:
These pressures are not without their emotional consequences for medical providers; we too often think of our providers as all knowing and invincible and neglect that knowledge has limits, even today. Many of the causative factors of these medically complex disorders are genetic. Here there are no cures; it may take eons to get a diagnosis; and then therapies are few. Often the multi organ involvement requires teams of doctors practicing in a system designed for conditions that existed decades ago, and a system incapable of responding to the complexities we face today. Anyone truly interested in this problem should at least review the stunning clinical variation in this emerging population. Anyone with passing knowledge of this clinical population will concur that a new clinical care model is mandatory for this population.
The Affordable Care Act does contain a provision that was intended to bring attention to this very problem:
With little or no attention or funding this provision has not fulfilled its goals. However the cause is being pursued. The Children’s Hospital Association has taken up the cause and is pursuing a reform effort. In its efforts at reform it reminds us that this effort can reap fiscal and economic benefits as it is estimated that although these children are only 6% of the Medicaid population they are 40% of the cost.
Here in my home State of New York the Health Homes planning for the pediatric population has just commenced and it provides a unique opportunity to create a truly unique leading edge design for the delivery system to children with medical complexity that could serve not only the Medicaid population but all medically complex children as well. The Health Homes concept already allows for the complexities and service model needs for special populations through tiered reimbursement schedules. It goes without saying that a population representing up to 40% of pediatric Medicaid costs require an accountably model all its own. The only question is where the infrastructure to shoulder that burden is: “who could lead the way, if you will”?
The answer is clear. We need only to acknowledge that the majority of pediatric assets are employed and controlled by the pediatric units of regional hospitals. In upstate New York that would include but not be limited to: Albany Medical Center, Crouse (Syracuse), Upstate Medical Center (Syracuse), Strong (Rochester), Women’s and Children (Buffalo). The next step is up to our State Legislative representatives and policy makers in the NYS Department of Health to take the lead and build the systems that unite these and other major pediatric resources. New York State could be the first in the nation to design such a system.
And there is no better use of the $8.0 billion waiver funds: On April 14, 2014 Governor Andrew M. Cuomo announced that New York has finalized terms and conditions with the federal government for a groundbreaking waiver that will allow the state to reinvest $8 billion in federal savings generated by Medicaid Redesign Team (MRT) reforms.
It is widely recognized that when we speak of orphan disease there are 7000-8000 acknowledged; collectively it is accepted that 7-8% of us may be affected at any given time. Yet it is rarely acknowledged that most of these syndromes have multiple markers. If you download the rare disease data base from Orphanet you find over 65,000 different flavors of 7000-8000 syndromes. If you check the list of orphan drugs approved you may find 400 or so drugs; if you eliminate duplications, you may fall below 300. Furthermore, given the pace of discovery of new drugs versus the discovery of new markers, I don’t think you would want to bet against markers topping 100,000 before orphan drugs get to 450.
I accept the reality that there has not been, is not now, and in the near term may not be cures and therapeutics for the vast majority of rare diseases. I also recognize that pursuing therapeutic treatments for the growing list of 65,000 + rare disease flavors, which may cost $10-15K per month is not a viable economic model upon which I can put my hope.
I think about the larger picture of rare diseases a lot. I am a rare disease advocate. I care for two grandchildren with rare diseases, they are my motivation. One would not be alive today without the changes of modern medicine and one, just 50 years ago, could have been a castaway to an institution were it not for sweeping cultural changes.
I am happy to collaborate with industry in advancing access to their inventions and therapeutics; but I want them to realize that on the average it does not put a dent in the aggregate problems posed by rare disease.
We are overwhelmingly a population without cures and standardized care. Our homes are often a hospitals or clinics unto themselves relying on practioners who often don’t know, being funded by insurance plans that are often hostile to our needs, and living in an era of decreased funding for research.
The industries who court us for our support are among the world’s best funded and most profitable, and need to recognize our needs span the year, every day, 24 hour a day, our needs are not limited to federal and state legislative sessions. Yet, the needs of the consumer and industry are broader and more collateral than we think. Barriers to care are not just legislative in nature. The acquisition and delivery of medical services is a greater barrier. And keeping up with its changing table of organization is just as much a barrier to a company marketing and producing its products as it is to the patient attempting to acquire medical services.
The Affordable Care Act has given more access to many people, but it has balkanized the market with each state having its own adaptions. Simultaneously, much of industry has trimmed its staff at local levels and it is ill prepared for battles being raged. It may well be that patient advocates, who must survive at the local level, are now seeing their day of power and influence ascending at the local level. Industry should broaden its dialogue with local patient advocates, it would behoove us both.
Ed Fennell
efennell43@gmail.com
Last year, I set up a new charity called Findacure in order to help patients with what we are calling fundamental diseases.
This came out of my personal experience of trying to find a cure for Black Bone Disease (also called Alkaptonuria), the rare genetic disease affecting my children. Ten years of fighting to raise funds, setting up an international research consortium and organising trials made me acutely aware of the uphill struggle facing all patients with such neglected diseases.
If so, please contribute here.
With best wishes and kind regards,
Nick
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Dr Nicolas Sireau