Cholsäure (1) – Orphacol®

• Clinical trials
  • No controlled study is available to answer the question regarding the extent of the additional benefit; instead, the data presented in the European Public Assessment Report (EPAR) (from published case reports/case series and narrative reviews), as well as an additional literature search carried out by the G-BA.

Treatment of congenital disorders of primary bile acid synthesis due to 3β-hydroxy-Δ-C27-steroid oxidoreductase deficiency or Δ-3-oxosteroid 5β-reductase deficiency with cholic acid in infants, children and adolescents aged between 1 month and 18 years, and in adults

• The G-BA classifies the extent of the additional benefit of cholic acid as non-quantifiable on the basis of the criteria set out in Section 5(7) of the AM-NutzenV, taking into account the severity of the condition and the therapeutic objective in the treatment of the condition.
• There is an additional benefit, but it is non-quantifiable because the scientific evidence does not permit this.
• In the present case and for this indication, the limited evidence base is a key factor in the decision-making process, meaning that a valid and meaningful assessment of the results to quantify the additional benefit is not possible.
• Consequently, a quantitative comparative assessment of the extent of the effect and a quantification of the additional benefit into one of the categories ‘minor’, ‘considerable’ or ‘major’ is not possible on the basis of the data provided.
• mortality
  • The limited scientific evidence currently available does not permit a quantification of the extent of the additional benefit of cholic acid from the perspective of mortality.
• morbidity
  • Valid data on morbidity endpoints such as liver cirrhosis, failure to thrive and symptoms such as pruritus, rickets and coagulation disorders are not available due to incomplete data collection.
  • Taken together, the available results do not allow any conclusion to be drawn regarding the extent of the additional benefit in terms of morbidity.
• Health-related quality of life
  • No data on quality of life are available; therefore, no conclusion can be drawn regarding the extent of the additional benefit in terms of quality of life.
• Side effects
  • Valid data are not available due to incomplete recording.
  • Overall, based on the available results, no conclusion can be drawn regarding the extent of the additional benefit in terms of side effects.
• Overall assessment
  • In its overall assessment of the available results, the G-BA arrives at the following evaluation of the extent of the additional benefit: there is an additional benefit, but it is non-quantifiable because the available scientific evidence does not currently permit a quantifiable statement on the extent of the additional benefit for patient-relevant endpoints.

Courtesy translation only, please refer to the German original.