Teduglutid (2) – Revestive®

Patients aged between one and 17 years inclusive

• Non-quantifiable.
• There is additional benefit, but it is non-quantifiable because the scientific evidence currently available does not permit a quantifiable statement on the extent of the additional benefit for patient-relevant endpoints.
• Due to the significant methodological limitations of the study and the overall limited evidence base, the G-BA classifies the extent of the additional benefit of teduglutide in the group of patients aged one to under 18 years as ‘not quantifiable’ on the basis of the criteria in Section 5(7) of the AM-NutzenV, taking into account the severity of the disease and the therapeutic objective in the treatment of the disease, as non-quantifiable.
• mortality
  • No deaths occurred in the study. Consequently, no conclusion can be drawn regarding the extent of the additional benefit in terms of mortality.
• morbidity
  • One of the endpoints of the study was the reduction in the volume of parenteral nutrition. In the responder analysis, a response – defined as a 10% or 20% reduction in the volume of weekly parenteral nutrition at week 12 compared with baseline – was achieved only in the teduglutide group, specifically in 8 patients when the volumes prescribed by the investigator were analysed.
  • Analysis of the diary data revealed 10 (10% reduction in parenteral nutrition) and 7 (20% reduction) responders, respectively.
  • The relative and absolute change in volume per week was more pronounced in the teduglutide group in both cases: There was a median reduction of 25.4 per cent and 1.3 litres, respectively, with teduglutide, compared with 0 per cent with placebo, when analysing the volumes prescribed by the investigator.
  • Analysis of the diary data showed a reduction of 29.3 per cent and 2.0 litres per week, respectively, with teduglutide, compared with an increase of 9.1 per cent and 0.5 litres per week, respectively, with placebo.
  • A clear assessment of the clinical relevance of the reduction in parenteral nutrition volumes cannot be derived from the available data.
  • Furthermore, the proportion of patients who were able to reduce their parenteral nutrition by 1, 2 or 3 days per week after 12 weeks was recorded. Based on the volumes prescribed by the doctor, 4 patients in the teduglutide group were able to achieve this, whereas none in the control group did.
  • According to diary data, there was a reduction of a median of 3 hours per day on teduglutide versus 0 hours per day in the control group.
  • Complete discontinuation of parenteral nutrition was achieved by 3 patients in the teduglutide group, based on the volumes prescribed by the investigator, but not in the control group.
  • Based on diary data and the volumes prescribed by the investigator, one patient out of 15 in the teduglutide group achieved complete discontinuation of parenteral nutrition, compared with no patients in the control group.
  • It is not possible to quantify the additional benefit on the basis of the available data on morbidity due to the methodological limitations of the study described above.
• Health-related quality of life
  • Health-related quality of life was not assessed as a separate endpoint. No conclusions can be drawn regarding the extent of the additional benefit in terms of quality of life.
• Side effects
  • Adverse events (AEs) occurred in all patients in both study arms.
  • At least one serious AE was reported in 53% of patients in the intervention group and 60% in the control group.
  • In no case was treatment discontinued due to AEs.
  • Given the small study size and insufficient comparability of the treatment groups, it is not appropriate to analyse individual AEs in order to determine the extent of any additional benefit.
  • Overall, no conclusion can be drawn regarding the extent of the additional benefit with respect to side effects.
• Overall assessment
  • In summary, it is not possible to quantify the additional benefit of teduglutide on the basis of the mortality, morbidity and adverse event data.

Courtesy translation only, please refer to the German original.