Tuesday, February 21, 2017


Nathaniel Rankin
RANKIN FAMILYNathaniel Rankin in 2013.

Nathaniel Rankin
RANKIN FAMILYA photo of Nathaniel the Rankins were given before they adopted him. He was identified as “Baby S.”

Nathaniel Rankin and his family puts a human face on the issues of funding cuts in Federal/State benefit programs. It provides you with the raw emotions, consequences, and inhuman suffering that many Americans will and are contending with. 

Too often victims are hidden, lost in the noise of politics, and so many other daily distractions. These are victims whose silence or inability to be heard over all the other media "noise", barely making it through each day and are now looking at a future where there is no answer for them as to how they, their loved ones, will survive. 

Proposed Federal/State inhuman changes to Medicaid would result in forcing States to cover fewer people and provide fewer services. Similar horrific affects will happen if ACA/Obamacare,Medicare, Social Security, etc. benefits programs are changed.

The Rankins know they are not the poster family for Medicaid, which is often described as a safety net for the poor. Except for Nathaniel, the family has had private health insurance through Rich’s work as a carpenter.
Yet their case underscores how Medicaid covers a far more diverse population than is typically recognized; with more than 70 million beneficiaries, it is the largest insurer in the country. It covers some women who might not typically qualify if they are diagnosed with breast or cervical cancer. It is the main provider of long-term services for seniors and people with disabilities and pays for one-fourth of all mental health and substance abuse treatments. Forty percent of children rely on Medicaid.
The cost of the program is split between state and federal governments, with the federal government picking up a greater proportion in poorer states. If expenses grow, because a recession causes more people to enroll or the cost of expensive new drugs, for example, the federal government doles out more money. (The federal government pays for the large majority of the optional Medicaid expansion under the Affordable Care Act, which Missouri did not pursue.)
president trump and leading republicans, including House Speaker paul ryan and new Health and Human Services Secretary tom price have proposed capping the amount of federal tax dollars delivered to each state. The plan could take the form of a block grant or a per capita allotment, but the goal is to cut spending and give states more authority to reshape their Medicaid programs.
“Caring for the most vulnerable patients must include not only ensuring they receive the care they need, but it must include ensuring programs like Medicaid that provide such care are sustainable,” House republicans wrote last week outlining their health policy plans.
Some states, with permission from the Obama administration, have already launched Medicaid experiments, adopting cost-sharing provisions and pushing beneficiaries to consider how they get care. In Indiana, for example, some enrollees have to pay what are essentially premiums and can get penalized for using the emergency department for a non-emergency.
But supporters of block grants want states to be able to go further in their reforms, without being delayed by a federal review. Some republicans, for example, have proposed letting states require able-bodied adults to work to remain eligible.
seema verma, trump’s nominee to lead the Centers for Medicare and Medicaid Services, said at her confirmation hearing last week that block grants and per capita caps should be on the table.
“This is about putting states in a leadership role so that they can manage their programs better,” said verma, who helped design the Indiana plan. “States are closer to the people that they serve than the federal government and they have a better understanding of what can work in their state.”
With limits on federal contributions, though, states would need to account for the lost funding as expenses rise. They could increase their contributions, but advocates and experts say they are more likely to charge beneficiaries, cut the amount they pay to clinicians, slash the services they provide, or limit who gets care.
“It’s a very effective policy if you want to reduce spending, but the question is, at what cost?” said Sayeh Nikpay, a Vanderbilt University health economist who recently wrote about Medicaid caps in the New England Journal of Medicine. “You end up reducing costs by covering very few people and covering very few benefits.”
             Nathaniel Rankin
               Nathaniel Rankin waits for lunch at his home.

Nathaniel almost died the first night he spent in his new home.
A glob of mucus plugged his trach tube, robbing him of the ability to breathe. At the time, he was eight months old. His oxygen levels plummeted. His body turned gray, his lips blue. Finally, Kim was able to insert a new tube, and Rich pumped air into his lungs.
Kim and Rich couldn’t legally adopt him until after a six-month interim period, but that night, he became their son. Kim, 50, calls it their and Nathaniel’s birth story.
“You’re holding him in your hands, and life and death is right there,” Rich, 56, said. “That’s when I felt like I would grieve just as much for this child as I would any of my biological children.”
(Rich had two children from a previous marriage and he and Kim had five biological children; their other children are now between 17 and 34 years old.)
As his parents, Kim and Rich have fought to get Nathaniel the best care available. Last year, he underwent an operation at Cincinnati Children’s Hospital Medical Center that stopped him from aspirating food and liquids into his lungs. It also helped stabilize his airway so it’s not such an urgent emergency if his trach tube comes out.
But the surgery also made it impossible for him to ever be able to speak.
When Nathaniel cries, tears roll from his eyes and a silent strain is etched on his face, but he does not wail or whimper. The only sound he makes is a wet wheeze from the hole at the base of his neck, which grows louder and faster as he gets more upset. And when he cries or laughs, or eats, he brings up phlegm into his airway that needs to be suctioned.
“God is our refuge and strength, a very present help in trouble. Therefore we will not fear,” reads a mural in the Rankins’ home, quoting Psalm 46.
Adopting Nathaniel has brought plenty of challenges for the Rankins and reshaped their lives in unanticipated ways. Kim thought she would be able to return to graduate school after a year of taking care of Nathaniel, but she is still his full-time caretaker.
“The simple answer is, ‘God gives the grace to accept changes,’” she said.
But there have been countless gifts from falling in love with Nathaniel. Kim and Rich have seen their other children become more compassionate since they’ve had their new little brother.
Raising Nathaniel has also altered how they view politics.
“It’s been a wrestle,” Kim said. “Some of the things we believe are upheld by the republican party and yet we have this child that we dearly love who is likely going to have services cut by people in the republican party. It’s hard.”
In the end, despite the disagreements he had with her, Rich said he voted for Hillary Clinton in the 2016 election because he felt Nathaniel would be better served by her policies. (Kim didn’t want to say whom she voted for.)
The Rankins are not sure what Nathaniel’s future holds and are weighing what kind of school he should, or could, attend. But their goals also include enabling him to experience life like any 4-year-old boy would want to, maybe even joining the Boy Scouts one day.
Still, there are risks. He loves to play in water, but any water that gets into his trach tube goes straight into his lungs. During one airway emergency, when Nathaniel aspirated vomit into his lungs, the hospital treated him as a drowning victim.
“I just want to go backpacking with him,” Rich said. “I want to take him up a mountain.”
The big fear for the Rankins is that Nathaniel could lose access to his care or have it interrupted with the potential Medicaid changes. It’s taken plenty of phone calls from them, and letters from their doctors, but with his coverage, Nathaniel has been able to get what he needs: the operation by the airway specialists in Cincinnati, visits to his team of doctors in St. Louis, regular appointments with occupational and speech therapists.
There are other changes the Rankins worry about as well. Nathaniel used to be in a managed care plan, which is designed to coordinate care at lower costs. But Kim said it was harder to get the nursing support the Rankins felt Nathaniel required and took more involvement from his doctors, so he moved to a fee-for-service plan. If the state cuts Medicaid services, would he have to go back to managed care?
Nathaniel’s providers have said he should stay on Medicaid so his care isn’t disrupted, but if needed, the Rankins could add Nathaniel to their private insurance plan. But there are hitches there, too. The ACA prohibits insurance companies from setting limits on the expenses they will cover for individuals, but if the law is repealed and that provision is lost, the cost of Nathaniel’s care could quickly surpass any newly imposed limit.
“There is a lot of advocating we have to do for his care, so to add advocating for his insurance, that would just compound it,” Kim said.
In Missouri, state tax dollars account for 17 percent of the Medicaid budget, with 51 percent coming from the federal government and 31 percent coming from a tax on health providers. And some parts of the program are already facing cuts. In his recent budget plan, Governor eric greitens, a republican who took office last month, proposed trimming Medicaid reimbursement rates.
Timothy McBride, a health economist at Washington University in St. Louis, said Missouri already had some of the strictest Medicaid eligibility requirements in the country, so if it lost some share of federal funding, it would have limited options.
“It would have to be dropping services and cutting back on provider payments,” said McBride, who chairs the state’s Medicaid oversight committee. “There’s really nothing else they could do.”
Nathaniel and his speech pathologist, Jane Keegan Quarles, were making Valentine’s cards on a recent day at St. Louis Children’s Hospital, and she was showing him how to get his augmented communication device, a special app on an iPad, to say that word. She tapped a few symbols on the iPad and a computerized voice echoed: “Fold.” Quarles creased the card and handed it to Nathaniel, who doodled on it.
As they have thought about Nathaniel’s coverage, Kim and Rich have also wondered if potential cuts to Medicaid would discourage people from adopting children, especially those with intense medical needs. That would mean fewer kids like Nathaniel might find families like the Rankins.
“We took a risk in falling in love with Nathaniel, because there was no guarantee he was going to live,” Kim Rankin said one night at home. “I think so much of our culture, we want to guard against risk taking, we want to be certain that not only are we going to be OK, but that the people we love are going to be OK.
“And every day I wake up and I don’t know if Nathaniel is going to be OK,” she continued. “There are things that could happen to him that even now, after his airway surgery, could take his life that a normal child could breathe right through. But I think the rewards of taking risks for the sake of love has really been the most valuable lesson to me.”
Quarles and Nathaniel finished making their card and handed it to Kim. 
Quarles held up Nathaniel’s device and coached him to “use your words” to tell his mom something. She pointed, and he jabbed his finger at two symbols. “Love,” the voice said. Two more jabs: “You.”
“Ah, I love you too, Nathaniel!” Kim said.
Nathaniel stepped to his mother, who swallowed him in a hug.
Most people who don't experience or see this suffering will never understand the true horrific situations that these human beings experience now and how much worse it will become if these benefits programs have funding cuts. 

It is already a "living death" for many and will become real death in the future if these programs are eliminated or reduced in the benefits provided.

Volunteers and faith based resource will never be able to cope with the waiting disaster of this type of genocide. 

Even "good" caring people are and will be in denial, hiding in their bubbles feeling that it  is always about "someone else". 

Well "someone else" used to think the same denial until in a nano second their lives changed forever when they or some loved one became ill.                     

Sunday, February 12, 2017



Last week, the Food and Drug Administration announced that a drug previously not officially available to patients in the United States had been approved. Deflazacort, a corticosteroid, has been shown to be useful and life-prolonging for patients with Duchenne muscular dystrophy, a rare and fatal disease. Its U.S. launch has been delayed, however, after lawmakers questioned the dramatic price hike that came with its debut here.
How much of a price increase? That depends on who you ask. The drug will be marketed under the name Emflaza, and its sticker price will be $89,000 for a year’s worth of pills. The CEO of Marathon Pharmaceuticals, the company marketing the drug, said that the typical cost per patient after discounts and insurance rebates would be $54,000 per year.
The Wall Street Journal reports that families currently pay an average of $1,200 to import deflazacort from countries where it’s available. The price varies: One mother reports paying $800 to $1,000, and other families pay up to $1,600.
Marathon Pharmaceuticals didn’t invent or even perform its own clinical trials on deflazacort, but bought the rights to data from clinical trials performed on Duchenne muscular dystrophy patients in the 1990s, using that to submit an application to the FDA. The clinical trial data showed that the drug helped patients keep more muscle strength, and patients taking it retained their ability to walk for longer.
The company can do this because of the Orphan Drug Act, which gives pharmaceutical companies exclusive patent rights for a few years after developing a new drug or gaining approval for a different use for an existing one. It’s an important program when used as intended, but this isn’t really its intended use.
There are only around 12,000 patients with the disease in this country, and the Washington Post reports that an estimated 7-9% of them even have access deflazacort. In theory, the cost to patients will be close to zero between insurance and discounts, but the high price to insurers is still frustrating.
Lawmakers have even taken up the cause: Sen. Bernie Sanders (VT) and Rep. Elijah Cummings (MD) sent Marathon a letter asking for “detailed information” about the drug’s development, including how much it actually cost Marathon to bring it to market.
Rep. Robert Aderholt of Alabama, the chairman of the committee that funds the FDA, also questioned the drug’s price hike.
“It makes me question whether the current construct of how FDA approves orphan drugs does more harm than good if companies have found a way to game the system,” Aderholt said in a statement yesterday.
Coincidentally, yesterday Marathon announced that it plans to delay the Emflaza launch to discuss the pricing scheme with caregivers and other stakeholders. It was originally supposed to go on the market last month.
As of February 12, 2017:

I have previously written about Duchenne muscular dystrophy, a rare and fatal, genetic muscle disease that affects children  (CLICK HERE FOR PREVIOUS POST OF SEPTEMBER 19,2016) and (CLICK HERE FOR PREVIOUS POSTS PRIOR TO SEPTEMBER 19, 2016)

Also check for further information- Cure Duchenne Organization website-(Click Here).

What Is Duchenne Muscular Dystrophy?

DMD is a degenerative neuromuscular disorder causing severe progressive muscle loss and even premature death. It is one of the nine types of muscular dystrophy and it is mainly caused by the absence of dystrophin, an important protein that helps keep the muscle cells intact.
Symptoms of this disease first appear during childhood, usually between ages 3 and 5. It primarily affects boys, but can also affect girls in rare cases.
Patients affected with DMD usually do not survive beyond their teen years, but thanks to advancement in medicine and cardiac care, the life expectancy of boys affected by the condition is increasing dramatically. Some of the patients have reached young adulthood, with some having families of their own and others living into their 40s and 50s.

                       UPDATED FEBRUARY 12, 2017

A first corticosteroid treatment for Duchenne muscular dystrophy (DMD) regardless of mutation, Emflaza  (Generic name is deflazacort), has been approved for use in patients ages 5 and older by U.S. Food and Drug Administration (FDA) as of February 9, 2017.
“We are in a new era in the treatment of Duchenne muscular dystrophy. For the first time, patients in the U.S. with Duchenne will have widespread access to an FDA approved medicine that is indicated for all genetic forms of the condition,” Timothy M. Cunniff, an executive vice president at Marathon Pharmaceuticals, the drug’s developer, said in a company press release.
Corticosteroids, including deflazacort, are widely used in many countries to slow the decline in muscle strength in boys with DMD. They work by reducing inflammation and suppressing the immune system. 
“This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy,” said Billy Dunn, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in an FDA agency press release. “We hope that this treatment option will benefit many patients with DMD.”
Emflaza will be available in tablet form and as an oral suspension. Its effectiveness in delaying DMD symptoms has been demonstrated in several clinical trials.
One Phase 3 study involved 196 DMD patients, ages 5 to 15, all with a documented mutation in the dystrophin gene and evidence of muscular weakness before 5 years old. The 12-week trial compared the effectiveness of two corticosteroids —  deflazacort (0.9 mg/kg/day) and prednisone (1.2 mg/kg/day) — against placebo.  

Results showed that both drugs significantly improved muscle strength in treated patients compared to placebo, but the lower dose of deflazacort caused fewer side effects, including weight gain, while demonstrating efficacy. Significant improvements in scores of muscle strength were also maintained for a one year, the study reported.
A two-year trial involving 29 DMD patients also showed a “numerical advantage” in average muscle strength among deflazacort treated boys compared to placebo, the FDA reported in its release. Treated patients also appeared to retain an ability to walk for a longer time than those in the placebo group. 
Common side effects associated with Emflaza included facial puffiness, weight gain, upper respiratory tract infections or coughing, and increased daytime urination.
“EMFLAZA is an important new drug with proven benefit in boys with Duchenne muscular dystrophy, increasing muscle strength and physical function,” Robert C. Griggs, MD, with the University of Rochester Medical Center and an early investigator of deflazacort, said in the Marathon release. “By undertaking the research needed to secure FDA approval … we now know more about the drug, its dosing and possible interactions. These are all advances in care for patients with Duchenne.”
The FDA previously designated Emflaza an Orphan Drug as a treatment for a rare disease with a high unmet need, and approved its use under priority review.
Marathon announced it plans further studies of Emflaza in DMD patients, including a trial examining the safety and effectiveness of different dose regimens in younger patients, and a safety and efficacy study in non-ambulatory patients.
The company will also continue to work on other DMD treatments it is developing, Cunniff said.
“We are pleased to learn that the FDA has approved EMFLAZA,” added Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy, the largest U.S. nonprofit group for Duchenne MD. “While steroids are considered standard of care for Duchenne patients, there has been no steroid specifically approved for Duchenne. The FDA approval … provides options for our families when making crucial decisions about care with their providers. We hope that this approval gives more families access to this important medication.”

Deflazacort has been available outside the United States for decades.
The big issue facing Marathon will be how to price the drug. Marathon did not invent deflazacort and patients have been able to import it inexpensively for personal use for years because there was not an approved U.S. treatment.
That will now change. Patients will no longer be allowed to import the drug now that an FDA-approved product is available. Northbrook, Illinois-based Marathon said the drug will carry a list price of $89,000 a year but that the company will provide a "robust" assistance program for patients who are not covered by insurance.

Yes $89,000 a year. That's the price tag although It's been available in other countries for years and patients in this country were allowed to import it. Now they'll be able to get it in the U.S. but there are mixed reactions that that news.
Gabe Griffin's family has been fighting for years to help Gabe live longer. He was diagnosed with Duchenne muscular dystrophy at the age of three. ABC33/40 has spent time with him in his backyard and at charity events. His father Scott Griffin says Gabe has been taking deflazacort for six years. When they tried to take him off the drug it didn't go well. "We did notice that he started to get weaker. We have stairs coming up our front door he was having trouble navigating those."
Griffin says they've been paying out of pocket $418 a year to have the drug shipped here from Europe. When he heard the FDA approved it in the U.S. "my initial thought was wow won't have to go through the headache of coming through customs and then you get to the price tag."
MDA Executive Director Terri Wilson says having the drug in the U.S. will make it more readily available for families. MDA says based on the patient assistant programs it's estimated the co-pay for the drug will be less than what families spent out of pocket to import it from other countries.
Griffin says he's skeptical because even with vouchers and assistant programs the drug still may cost a lot more. "I have a $3,000 deductible and so my insurance company doesn't pay anything until my $3,000 deductible is met so I went from paying $418 a year to essentially $3,000 a year."
The MDA says this is the second drug approved by the FDA in the past six months to treat patients with Duchenne muscular dystrophy and that is unprecedented. They are celebrating that fact.
The FDA approval of Emflaza marks a third major proof point of MDA’s research program in recent months. Exondys 51 in September 2016 became the first disease-modifying drug approved by the FDA to treat DMD. 

But Griffin says there are still a lot of questions about that $89,000 price tag like how much will insurance cover and how much will get passed onto the patient. 

He is waiting to see what happens.

What happens to those victims of this horrific disease who cannot afford the enormously expensive cost of this medication, do they not get access to this drug, do they continue to suffer in agony, do they stay alive or die because a pharmaceutical company cares more about profit than human lives?