Reason for request

New indication Availability of a new form

Key points

Favourable opinion for reimbursement in the treatment of Clostridioides difficile infections (CDI) in paediatric patients (for more details, see MA).

What therapeutic improvement?

Therapeutic improvement in management.

Role in the care pathway?

The two most commonly used antibiotics in the treatment of C. difficile infections (CDI) are metronidazole and vancomycin, by the oral route. These two products are effective in most CDI cases but almost a third of initially recovered patients relapse.

Role of the medicinal product in the care pathway

Fidaxomicin is a first-line therapy in the treatment of C. difficile infections due to its comparable efficacy and safety profile to that of vancomycin in terms of recovery from the infection, but with a lower risk of relapse and a greater convenience of use than vancomycin.

Special recommendations

The Committee reiterates that there is a risk of misuse, with potential use of fidaxomicin in cases of antibiotic-associated diarrhoea without established C. difficile infection (CDI). However, not all cases of antibiotic-associated diarrhoea are linked to C. difficile and may be due to poor colonic fermentation as a result of a reduction in the dominant flora, for example. Since this is generally a short-term situation, it will be difficult for clinicians to differentiate between the efficacy of fidaxomicin in this indication and that of no treatment.

Hence treatment should only be considered in the event of CDI with the presence of toxin A and/or B in the stools. It should be noted that the presence of toxins in the stools before the age of 3 years only has a diagnostic value in the event of intestinal blockage.


Clinical Benefit

Substantial

The Committee deems that the clinical benefit of DIFICLIR (fidaxomicin) is substantial in the MA indications.


Clinical Added Value

moderate
  • CAV in paediatric patients (for all forms)

Considering:

  • available data in the paediatric population suggesting an efficacy profile for fidaxomicin comparable to that of vancomycin in terms of clinical response at the end of treatment (77.6% versus5%, a difference adjusted by 7.5% (CI95%: [-7.4; 23.9]);
  • a suggested advantage in terms of reduction in the rate of recurrence of difficile-associated diarrhoea compared to vancomycin (11.8% versus 29.0%);
  • a size effect of the same magnitude as that described in adults with a comparable safety profile;
  • a simplification of use compared to vancomycin with the availability of an appropriate paediatric formulation;
  • the absence of robust data in the most severe forms (ileus, toxic megacolon, septic shock) and/or with multiple recurrent infections (more than one previous episode of difficile infection);

the Committee considers that, in the same way as in adults, DIFICLIR (fidaxomicin) provides a moderate clinical added value (CAV III) in the treatment of Clostridioides difficile infections in paediatric patients.

 

  • CAV in adults (for the oral suspension form)

This medicinal product is a range supplement that does not provide any clinical added value (CAV V) compared to the 200 mg film-coated tablet form already approved. This form may be used in adult patients with difficulties swallowing tablets.

 

no clinical added value

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