Pirfenidon (1) – Esbriet®

Adults for the treatment of mild to moderate idiopathic pulmonary fibrosis (IPF)

• The G-BA notes that the results of the two Phase III trials, PIPF-004 and PIPF-006, which were decisive for marketing authorisation, are inconsistent.
• In summary, based on the marketing authorisation and the desired and undesired effects observed in the aforementioned studies, as well as taking into account the comments received, the oral hearing and the severity of the disease, the G-BA arrives at the following assessment of the extent of the additional benefit: there is an additional benefit, but it is non-quantifiable because the available scientific data do not currently permit this.
• Morbidity – Change in forced vital capacity (FVC) as a percentage of the target value after 72 weeks compared with baseline
  • For the primary endpoint ‘change in forced vital capacity (FVC)’ as a percentage of the target value after 72 weeks compared with baseline, a statistically significant effect of pirfenidone was observed in Study-004; however, this effect was not statistically significant in Study-006.
  • It is questionable to what extent the primary endpoint ‘forced vital capacity’ is suitable for drawing valid conclusions regarding patient-relevant endpoints.
• Morbidity – Change in the 6-minute walk distance after 72 weeks compared with baseline
  • The results for the secondary endpoints of the studies, in terms of the ‘change in 6-minute walk distance at 72 weeks compared with baseline’, were significant in Study PIPF-006, but not in Study PIPF-004.
• Morbidity – Progression-free survival (PFS)
  • With regard to the endpoint ‘progression-free survival (PFS)’, the results were significant in study PIPF-004, but not in study PIPF-006.
  • However, the meta-analysis of the pooled data from both studies showed a statistically significant effect.
• Health-related quality of life
  • Although data are available for the patient-relevant endpoint ‘health-related quality of life’, they do not show any significant improvement.
  • These data are of particular importance in a palliative care setting for assessing the patient-relevant additional benefit.

Courtesy translation only, please refer to the German original.