A clinician’s perspective: The reimbursement of a medicine impacts the selected treatment

The basic reimbursement granted for medicinal substance teduglutide was a significant step forward for children with short bowel syndrome, their parents and the healthcare personnel treating them. How could it not be: it is a case of a patient group with a risk of death, who need lifetime intravenous nutrition and fluidification without the medication.

According to HUS Deputy Chief at Paediatric Gastroenterology, Laura Merras-Salmio, 2–5 children with short bowel syndrome are annually treated at HUS.

Short bowel syndrome causes a risk of death

Without medicine, most of the patients have major problems and need intravenous nutrition and fluidification throughout their life. This is why most children are forced to have a central vascular catheter in their neck veins.

– Intestine transplant causes costs of hundreds of thousands of euros and the disease continues afterwards. That is, you cannot tell the patient and their parents that let’s do this and you will then walk home. Mortality is also high in intestine transplant, tells Merras-Salmio.

Objective is to live at home

The objective is that children with short bowel syndrome can live at home. That is why the parents are taught to treat the children: in 1-7 nights a week, they provide a 10-14-hour infusion for the child. According to Merras-Salmio, most parents want to treat their child and learn the infusion.

– Our starting point is that a child has the right to live in their home. This is of course related to the coping of the parents, because treating the child every night is tough. This can cause, for example, absences from work, states Merras-Salmio.

Teduglutide injected once a day has brought a great relief for the life of the families with short bowel syndrome.

– Now we finally have an effective medicine for a patient group with a risk of death.

Teduglutide has provided promising treatment results

Teduglutide increases villus height and blood circulation in the intestine, which improves the absorption of nutrients and liquids. Treatment results have been promising, as Merras-Salmio’s two examples demonstrate.

1. Patient A was first discharged from hospital at the age of 8 months. They had 20 per cent of the small intestine left. At the age of one year, they were started with teduglutide medication. Infusions could be stopped at the age of 2 and teduglutide at the age of 3. They no longer need any medicine.
2. Patient B received infusion seven nights a week at the age of 7. He had 15 per cent of the small intestine left. At second grade, he started to receive teduglutide and finally infusions were decreased to three nights a week.

– I never forget his expression when he was told that he can be free from the tubes for one night, Merras-Salmio tells.

The quality of life of the whole family improves

Teduglutide is an expensive medicine, and the families could not afford to use it without the society’s support. According to Merras-Salmio, its use is, however, rational in a patient group with high costs of treatment throughout their life without medication and whose quality of life is improved with the medication, including the well-being of the whole family.

– Of course, tax money needs to be reasonably spent. Everyone should not get everything, and one should consider what is the right price tag. It feels strange and unfortunate that in this patient group, the contribution of the parents is not included in the calculation. It is not considered significant, states Merras-Salmio.

She thinks one should also consider whether the quality-of-life studies are sufficient to guide decision-making.

– Children’s quality of life is a curious matter. These children do not consider having a particularly poor quality of life. It is a sign of resilience. It is their life, and they cannot compare it with anything else.

The reimbursability criteria of teduglutide are strict.

– The medicine needs to be paused once a year to see if the patient still needs it. When the medicine is suddenly stopped, the patients can end up in a poor condition. The reimbursability process needs to be more human in the future, states Merras-Salmio.

HUS Deputy Chief at Paediatric Gastroenterology, Laura Merras-Salmio, talked about a clinician’s perspective to the reimbursement of medicine at Medaffcon’s customer evening organised at Espoo Museum of Modern Art Emma on 14 Sep 2022.