A National Shortage of Growth Hormone Is Putting Children at Risk

Growth hormone is prescribed for children with a variety of diseases and conditions that thwart normal growth. Some of these conditions involve the pituitary—a pea-sized gland located at the base of the brain.

Despite its small size, the pituitary plays an outsize role in the endocrine system, controlling how all the other glands function. It also produces growth hormone.

When a child is born with a pituitary that’s too small or nonexistent, or that produces too little growth hormone, a pediatric endocrinologist will treat these issues with a pharmaceutical version of the hormone.

• Children may also experience poor growth as a result of:
• conditions that injure the pituitary gland or hypothalamus such as a tumor, trauma, radiation or surgery to that area of the brain
• genetic disorders such as Turner syndrome or Cushing syndrome
• gastrointestinal diseases that interfere with the ability to absorb nutrients such as Crohn’s disease, ulcerative colitis or celiac disease
• chronic kidney disease
• heart disease
• immune-related diseases

Does the shortage of growth hormone pose a danger to the children who depend on it?

“Most children who take growth hormone are not in immediate medical danger due to the shortage,” says Dr. Zoltan Antal, Chief of Pediatric Endocrinology at Weill Cornell Medicine and an assistant attending pediatrician at NewYork-Presbyterian’s Phyllis and David Komansky Center for Children’s Health. “However, the shortage could be life-threatening in certain situations.”

For example, he explains, children with severe hypoglycemia (low blood sugar) resulting from growth hormone deficiency may experience seizures and even death without access to the pharmaceutical version. And for children who experience a dramatic improvement in growth and other areas of their health due to growth hormone therapy, the shortage puts them at risk of reversing all of these benefits.

That’s why the shortage is so alarming. Read on for Dr. Antal’s insights regarding the various factors at play and what families can do in the meantime.

What is causing the shortage?

It all started in the summer of 2022, he says, with a few calls about a shortage of specific pen sizes and brands. “Calls started coming in from parents, and within a few months these became more frequent. Then, my colleagues started talking about it, and we realized it was a national problem.”

Was the shortage related to COVID-19? Was it caused by supply chain issues? By unrest in the world? It turns out that the explanations are complex and multifactorial.

There are 6 to 7 brands of growth hormone available in the United States, and historically endocrinologists have prescribed them on a rotating basis.

About 10 years ago, says Dr. Antal, the major pharmacy benefit managers (PBMs) in the country decided to authorize preferred brands of growth hormone, specific to the insurance company in question. Health plans, large employers, unions and government entities contract PBMs to manage their prescription drug benefits programs. These contracts may rotate from brand to brand when contracts are renegotiated, but importantly, one growth hormone manufacturer may dominate the insurance market at any given time. If that company then has a medication shortage, the effect on patients is disproportionately large.

As yet, “no one has received an adequate explanation of the shortage,” he says, “but what started with the dominant manufacturer has spread to all the others.”

Parents tried to switch their children from one brand to another. Their insurance carriers would take 48 hours to authorize the switch, and by then, that brand had become unavailable.

Another shortage

As if that merry-go-round weren’t frustrating enough, another shortage came along.

“Some growth hormone doesn’t come ‘pre-prepared.’ It may come as a medication cartridge, but a pen “device” is needed to deliver the medication dose,” says Dr. Antal. “The pen device is considered ‘durable medical equipment,’ while the cartridge is labeled a pharmaceutical. Shortages of both have basically doubled the problem, and solving it will take double the effort.

“There should be natural coordination between these two components,” he continues. “That’s just common sense.” But common sense isn’t the name of the game right now.

How are these shortages affecting the field of pediatric endocrinology?

According to Dr. Antal:

• Specialists are spending a huge amount of time arguing with insurance companies.
• Many offices are devoting more than 75 percent of their efforts to dealing with the growth hormone problem.
• Patients are experiencing long wait times for appointments.
• The future of the specialty is in jeopardy, with both current and in-training endocrinologists seeing these types of pressures as one of many reasons not to stay in or enter the field.

What can parents and caregivers do?

• If your child has a history of low blood sugar due to growth hormone deficiency, make sure to discuss this specific concern with your endocrinologist.
• As difficult as it seems, avoid switching between brands of growth hormone. Sit tight.
• Engage in advocacy. If enough families band together and make their voices heard, that could make a difference.
• Connect with other parents and caregivers online and share information.

In the meantime, Dr. Antal and his colleagues at Weill Cornell Medicine, along with their fellow endocrinologists across the country, are working overtime to address the shortage. “Despite the difficulties we face, we’re hopeful that we’ll resolve the twin shortages of medication and pen devices in 2024—sooner rather than later.”

Make an appointment with a pediatric endocrinologist at Weill Cornell Medicine here.