“We’re not scared,” Whitbeck said, but “when you’re a parent of a child with a chronic illness, you’re always walking on a tight rope … Every day we have to give [Luke] the once-over and make sure everything is good. If it’s a good day, everything is like a normal family.”
Honestly after reading this article it is heartbreaking to see this family go through so much stress, and put in a position that can mean the death of their child.
Under the law, companies with orphan drugs win some of the richest financial incentives in the regulatory world: a 50 percent tax credit on research and development in the U.S., fee waivers and access to federal grants. In a 2009 webinar, an FDA official referred to the incentive package as “our ‘basket of goodies.'”
Interesting how this is under the law, and is referred to as a "basket of goodies", and in contrast families like Luke's are struggling to pay or even get approved by their insurance company to be covered.
A drugmaker had stopped producing medicine for the disorder, saying there weren’t enough patients to justify the cost.
This just proves more that pharmaceutical companies are just in it for the profit.
Some believe that amounts to rationing. “Nobody wants to talk about rationing, but there is already some of that anyway because of [the] levels of insurance that we have,” said Christopher-Paul Milne, director of research at Tufts Center for the Study of Drug Development.
I do think that this does put many people in America at a disadvantage because only the people that are able to afford that level of insurance will more likely have the chance to purchase these drugs at a constant rate for how long they will need it. Like a lot of things in America if you don't have money it's hard to get the quality care that you need.
Insurers, including UnitedHealthcare, try to mitigate the costs of orphans and other high-cost drugs by requiring patients to pay a larger share, setting quantity limits and asking patients to try other less expensive drugs first, a process known as step therapy.
We talked about this in my PH 162 class when we watched a documentary that compares the healthcare systems of 5 other countries to the US. In the documentary it is mentioned that in Britain they have something where there is a "gatekeeper doctor" that determines if you need an actual doctor or can just take something over the counter. This doctor usually tries to persuade the people to choose the cheaper option. Therefore this idea of the step therapy in my opinion is very similar to Britain's idea of this "gatekeeper doctor".
In an email, company spokeswoman Lisa Clemence said the company has raised the price of Cerezyme slightly since it launched and — relative to inflation, it’s 33 percent less expensive today than 22 years ago. Asked how drug prices are set, Clemence emailed that it is “determined by the clinical value they provide to patients and the rarity of the disease they treat.”
Now we kind of see an insight to how they price the drug which is by the rare disease it treats and the clinical value.
While she appreciates the support, Meg Whitbeck is daunted by the uncertainty of what the family has to come up with. “We’re not going to not treat Luke, but we’re also never going to be able to pay these bills,” she said. “It was almost laughable.”
Because Luke's parents were not able to pay for the medicine right away, and were waiting for the insurance company to approve them, they paid $17,000 out of pocket, and were also doing social media fundraisers that was still not enough. This quote from Luke's mom, Meg, I think represents how a lot of people in America feel when it comes to the healthcare system.
But Lempner stated in an email that “specialty medications like these are among the greatest drivers of pharmacy benefit costs for all employers, individuals, insurers and the government.”
Tracey Lempner is the spokeswomen for UnitedHealthcare, which is the company that covers the drug that Luke needs, Cerezyme.
In late April, the Whitbecks received a letter from the insurer asking for additional medical information to process the payments for Luke’s medicine. It was the first time, Meg said that she became scared about how the drug would be paid for. She recalled wondering: “How can something that’s going to keep my baby alive not be covered by insurance?”
Reading this over and over and thinking that it is strange that you have to get approval from your insurance company to see if they will cover your medical needs from minor to major injures that can possibility lead to death. It then brings up the question of what is even the purpose of insurance if they pick and choose on what they want to pay for even in an emergency.
Luke was diagnosed with Gaucher disease — a genetic condition that affects only about 6,000 people in the U.S. Luke’s body lacked the glucocerebrosidase enzyme, and every cell in his tiny frame was becoming compromised. Luke’s liver and spleen were swollen and without treatment his bones would eventually become fragile. Brain damage could follow. Some babies die before the age of two, if they aren’t treated. Patients can live decades with enzyme replacement therapy.
This shows how dangerous Luke's condition is and how fragile his health is. Luke's parents must have been in constant worry and stress that their baby can pass away any day, so if I was in that position why wouldn't I want to spend any money I had to save my child's life.
Bernard Munos, a former Eli Lilly executive now at the nonprofit Milken Institute, said orphan drugs cost $50 million to $200 million to develop at a small company. And regardless of the development costs, he said drugmakers will charge whatever they want “because they can get away with it.”
I thought that this was an interesting point because when a doctor tells you that you are sick and need this medicine that will relieve or cure you of your illness most people will pay whatever just to get better. It's like these drug companies really put you in a position where you have no other choice but to buy their drug in a time of vulnerability, especially if you have a rare disease that apparently that can be helped by this orphan drug.
For many drugmakers, orphan drugs look so profitable that they’re drawing attention from mass-market drugs.
Reading this part really shows how healthcare in the US is really based off making profit instead of actually helping those in need.
The Orphan Drug Act has clearly spurred the creation of rare disease drugs. And more are needed: The National Institutes of Health estimates that one of every 10 Americans suffers from a rare disease. All told, there are about 7,000 of them.
This is very important to know that rare diseases are common within the US, so it is interesting to see why the government would make it difficult to access these medicines and treatments.
The explosion has burdened insurers as well as government programs like Medicare and Medicaid with drugs that cost up to $840,000 a year that they have almost no choice but to pay for.
$840,000 a year for medicine that some people need to live this is more than what most people make from a regular job.
For the Whitbecks, finding a way to pay for the drug has been a nerve-wracking struggle. Their family business and its insurer cough up hundreds of thousands of dollars per year, spreading the cost across the people it insures.
As I have mentioned before and learned in PH 162 so many people in the US struggle to pay for quality healthcare and in a time of an emergency have no other choice but to get the help they need even if it's going to put them in debt.
Cerezyme is an “orphan drug” which means it was created to treat a rare disease, one that affects fewer than 200,000 people in the U.S. The orphan drug program overseen by the Food and Drug Administration is loaded with government incentives and has helped hundreds of thousands of patients like Luke feel better or even stay alive.
The fact that this medicine that Luke takes is considered "a drug that treats a rare disease that affects fewer than 200,000 people just in the US" (Tribble & Lupkin, 2017) , that probably means that it is even more expensive and difficult to get a hold of it.
$300,000-a-year drug
Already in the beginning of this article we are shown how healthcare works here in America. I just learned more about how the American healthcare system works in my PH 162 class, and how costly it is to get quality care. We also talked about that most people here in America are in debt because of medical bills.