‘Short bowel syndrome patients prioritize stable IV therapy over reimbursement for expensive new drugs’

Short bowel syndrome (SBS) is a disease characterized by a short intestine that prevents the body from digesting and absorbing food properly, leading to physiological death.

As with any rare disease, the spectrum of SBS symptoms is vast, with some patients becoming fatally ill due to hypoglycemia-electrolyte deficiency if they do not receive nutrients through daily intravenous (IV) fluids around the clock. Others may require fluid therapy for the first few days but then adapt to the short bowel condition, increasing their food intake and improving to the point where they can survive without fluid therapy.

SBS is divided into two main categories: congenital SBS, which is caused by poor intestinal blood flow or lack of intestinal development in the womb, and acquired short bowel syndrome, which is caused by abnormalities in intestinal blood flow, intestinal rotation, or surgical removal of the intestine.

However, congenital or acquired short bowel syndrome is not a determining factor in the symptom spectrum of people with SBS. For this reason, medical professionals specializing in SBS say that regardless of whether the condition is congenital or acquired, people with SBS have the same symptoms, diagnosis, treatment, natural history, and disease burden.

The problem is that people with SBS are now treated differently depending on whether they are congenital or acquired.

Congenital short bowel syndrome, which is much rarer than acquired short bowel syndrome, is a nationally designated rare disease and receives a “special calculation” benefit that allows patients to pay only 10 percent of the cost when they undergo tests and treatments. So, patients with congenital short bowel syndrome are less affected by economic conditions and can receive stable fluid therapy. However, acquired short bowel syndrome is not a nationally recognized rare disease and is not eligible for this special calculation.

IV fluid therapy, a lifeline for patients with short bowel syndrome, costs 2 million won ($1,359) to 3 million won per month, and congenital SBS patients pay only 10 percent of it, acquired SBS patients pay 100 percent of it, 10 times more than the former group. There has been a movement to improve this unreasonable situation in recent years. SBS patients and their families have talked in online cafes, knocked on the doors of the Ministry of Health and Welfare and the National Assembly, and publicized the reality by sending articles everywhere.

As an old saying goes, heaven seems to help those who help themselves.

In 2023, the Korean Organization for Rare Diseases contacted patients and their families individually and offered to improve the unreasonable situation. This led to the first online meeting of people with short bowel syndrome and their families in February 2024, the official launch of the Short Bowel Syndrome Patients’ Association. In July, the association joined the Korean Organization for Rare Diseases and began its activities earnestly.

“SBS patients and their families were discussing in online cafes about posting writings, making phone calls to the Ministry of Health and Welfare, and sending emails to lawmakers to find a solution to improve the reality that patients are treated differently depending on whether they are congenital or acquired,” said Huh Moon-young, 41, the first president of the SBS patient group. “When we were talking about the need to gather more opinions, the rare disease organization contacted us, and this led to the creation of our association, which is actively promoting the reality of short bowel syndrome treatment through parliamentary debates and media interviews.”

The association does not say that all SBS patients, whether congenital or acquired, require a rare disease designation.

Instead, it emphasizes that people with SBS, congenital or acquired, who require life-saving fluid therapy for a prolonged period should be granted a rare disease designation and be eligible for special calculation benefits. The original intention of the special exemption—to provide essential medical support to the health-compromised—is to ensure that SBS patients, the most vulnerable group whose lives are at risk without long-term fluid therapy, can receive stable treatment.

The government has also been sympathetic to this reality of short bowel syndrome treatment for quite some time, allowing children who were not born with congenital short bowel syndrome but acquired it within the first month of life to be recognized as congenital short bowel syndrome and receive special benefits.

A case in point is Huh’s son, born in July 2018. The boy was not born with short bowel syndrome. He acquired it about a month after he was born, as his intestines were cut due to a sudden onset of necrotizing enterocolitis. Huh’s son is now receiving special calculation benefits.

He was born at 27 weeks gestation, weighing only 1 kg after his amniotic fluid suddenly ruptured, and he was in the NICU at Seoul National University Children's Hospital. He was only 270 grams heavier than when he was born. Due to necrotizing granulomatous inflammation in his tiny body, he had to have about 80 centimeters of his 90-centimeter small intestine removed. Still, fortunately, he was eligible for special calculation from the start.

“I know how valuable this benefit is because I narrowly missed the criteria (acquired short bowel syndrome within the first month of life),” Huh said.

For this reason, Huh emphasized that the system should be improved to ensure that all patients with SBS who need fluid therapy to survive can access the special calculation benefit.

The good news is that the reality of short bowel syndrome treatment is becoming more widely known thanks to the efforts of people with short bowel syndrome, medical professionals, medical societies, and the Korean Organization for Rare Diseases. The number of cases of children with acquired short bowel syndrome within the first year of life being unofficially recognized as congenital short bowel syndrome and receiving special benefits has recently increased, and the Korea Disease Control and Prevention Agency (KDCA) is promoting institutional improvements.

“Not all patients with short bowel syndrome need total parenteral nutrition (TPN) fluid therapy, and I hope that the policy will be detailed to benefit all patients who need it (whether congenital or acquired),” Huh said.

She added that it is also necessary to create a treatment environment where patients with short bowel syndrome who can live their daily lives while receiving fluid therapy outside of the hospital can receive stable treatment at their local hospital and live healthy daily lives. Currently, only one hospital in Korea has such a system: Seoul National University Hospital (SNUH).

Patients with short bowel syndrome who are treated with fluids 24/7 can try stopping the fluids for three hours a day if their dependence on fluids falls below 30 percent of their total nutritional supply, and if such time is increased, SBS patients who need to receive daily injections can be discharged from the hospital and try “Home TPN,” which allows them to attend kindergarten, school, or work. However, only Seoul National University Hospital is now equipped with the Home TPN system, so SBS patients from outside the Seoul metro region are hospitalized due to the limitations of their area of residence, even if Home TPN is available.

In addition, because SBS patients who have poor nutritional status must have TPN customized for them by checking even the trace nutrients in their bodies through monthly blood tests, hospital pharmacies manufacture TPN separately, and unlike TPN made by pharmaceutical companies, hospital-dispensed TPN has a shelf life of only 48 hours. Of course, once the patient's nutritional status is stabilized, they can switch to pharmaceutical TPN, but this is a long way off for patients who are trying home TPN for the first time, but the right to home TPN treatment for SBS patients outside the capital area is limited due to geological reasons.

“For home TPN, patients must visit Seoul National University Hospital every two days even after being discharged,” Huh said. A mother with a child with SBS whose home is in Yeoju, Gyeonggi Province, drives her child to SNUH for TPN every other day, rain or shine,” Huh said, pointing out that she is even lucky to be able to do home TPN.

Seoul National University Hospital is not the only hospital with a home TPN system. However, it is difficult for SBS patients to receive stable Home TPN services in other hospitals.

“Asan Medical Center also has a Home TPN system, but it limits the number of patients to seven, making it difficult for SBS patients to be included. That limit should be lifted,” Huh emphasized.

There is a reason other hospital, except for SNUH, the national central hospital, do not have a well-equipped home TPN system. Dispensing TPN for each SBS patient requires a heavy workload, and since there is no reimbursement for dispensing TPN, hospitals are unwilling to increase the number of home TPN systems at a loss.

To ensure that SBS patients can be treated reliably anywhere in Korea, regardless of location, an additional fee for dispensing TPN by hospital pharmacists should be created in line with the domestic reimbursement system, which is based on a fee-for-service system. There is a new drug for short bowel syndrome called Gattex (teduglutide), a GLP-2 analog with a short intestinal absorption rate licensed in Korea.

However, the Short Bowel Syndrome Patients’ Association says that creating a stable fluid therapy environment should be prioritized over reimbursing a new drug that costs hundreds of millions of won.

“Gattex does not guarantee that all SBS patients requiring fluid therapy can stop injecting fluids,” Huh said. “TPN is the most reliable way to treat short bowel syndrome, so creating a stable environment for TPN treatment should be the priority.”

She continued, “We also need to take care of patients with short bowel syndrome, who have difficulty reaching the average height and weight even with fluid therapy, have diarrhea dozens of times a day even when they go to kindergarten or elementary school and are unable to wean themselves off diapers because they have a strong aversion to food.”

Currently, there is no social consideration for the rare disease short bowel syndrome, which makes it easy for patients to fall behind in many ways. For this reason, some parents of children with short bowel syndrome even say that children diagnosed with SBS and other diseases are luckier than those with only SBS because the former group is eligible for diagnosis of disabilities.

“It's a sad reality,” Huh said, pointing out that a child with short bowel syndrome becomes eligible for special education because of a disability. However, children with only SBS are not categorized as such and must get homeschooled.

Moreover, SBS patients who receive intravenous fluids have to be hospitalized for two to three weeks at least once a year due to central line infections, no matter how well they manage their TPNs. So, the short bowel syndrome itself must be designated as a disability to prevent children from falling behind in education due to absenteeism.

“The number of days of education is different from the number of days of general education if you receive a disability designation,” Huh said. “We hope that children with short bowel syndrome will be designated as a disability to prevent them from falling behind in the educational environment.”