Teduglutide
Type III (chronic) short bowel syndrome with intestinal failure in paediatric patients aged 2 years and above who are dependent on parenteral support.
Decisions on record
- Meeting Mar 2021 Recommended Short bowel syndrome; paediatric patients
- Meeting Mar 2019 Recommended Short Bowel Syndrome (SBS)
- Meeting Jul 2018 Not recommended The treatment of adult patients with short bowel syndrome who are dependent on parenteral support. Patients should be stable on their parenteral support regimen for at least 4 weeks prior to initiating teduglutide.
- Meeting Nov 2017 Not recommended Short Bowel Syndrome (SBS)
Access path
- Nov 2017Not recommended
vs standard care (best supportive care focusing on…
- Jul 2018Not recommended
Uncertain long-term benefit; inappropriate restriction criteria (12-month parenteral support dependency may not…
- ↻ resubmittedMar 2019Recommended · restricted
Comparator changed: standard care (best supportive care) → standard care (best supportive…
- Mar 2021Recommended · restricted
- PBS listing · Authority Required
From the public summary
5.1 The PBAC recommended amending the Section 100 (Highly Specialised Drugs Program) Authority required listing of teduglutide for the treatment of paediatric patients with Type III (chronic) short bowel syndrome (SBS) with intestinal failure (SBS- IF) to include specific response criteria for paediatric patients.PSD · Mar 2021
5.2 The PBAC recalled it considered that the restriction should not specify any age criteria given teduglutide has been studied in paediatric patients and was shown to be well tolerated (paragraph 7.6, teduglutide PSD, March 2019).PSD · Mar 2021
6.33 The resubmission presented a modelled economic evaluation assessing the cost effectiveness of teduglutide plus standard care compared to standard care alone, in patients with short bowel syndrome associated with Type III intestinal failure.PSD · Mar 2019
6.34 A comparison of the economic models presented in the current resubmission and the July 2018 resubmission is presented in Table 9. 19PSD · Mar 2019
6.29 The resubmission described teduglutide plus standard care as superior in terms of effectiveness compared to standard of care alone. The PBAC previously considered that the claim of superior comparative effectiveness of teduglutide over standard care was reasonable, based on the statistically significant difference in the primary efficacy outcome of the proportion of patients who achieved at least a 20% reduction in 18PSD · Mar 2019
6.30 The PBAC previously considered that the non-inferior safety claim was not adequately justified (Paragraph 6.37, July 2018 teduglutide PSD). The resubmission described teduglutide plus standard care as inferior in term of safety compared to standard of care alone.PSD · Mar 2019
4.2 The PBAC noted and welcomed the input from Parenteral Nutrition Down Under (PNDU) via the Consumer Comments facility on the PBS website. The comment described the significant effects on quality of life for children with SBS-IF.PSD · Mar 2021
4.12 No changes are proposed to any aspect of the current RSA for teduglutide. For more detail on PBAC’s view, see section 5 PBAC outcome.PSD · Mar 2021
Cost-effectiveness
This is a minor submission requesting amendments to existing Authority Required listing; no new economic evaluation was undertaken.
Decision context
PopulationPaediatric patients (aged 2 years and above) with Type III chronic short bowel syndrome with intestinal failure who are dependent on parenteral support and have been on a stable parenteral support regimen for at least 3 days per week in the previous 4 weeks.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2021 | Recommended · restricted | — | — | RCT · Parenteral support reduction |
| Mar 2019 | Recommended · restricted | standard care (best supportive care focusing on optimisation of remnant intestinal function) | — | RCT · Other |
| Jul 2018 | Not recommended | standard care (best supportive care) | — | RCT · Reduction in parenteral support volume (≥20% reduction from baseline); days off parenteral support; number of patients weaned off parenteral support |
| Nov 2017 | Not recommended | standard care (best supportive care focusing on optimisation of remnant intestinal function through parenteral support, | — | RCT · Other |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| STEPS | Ph 3 | 86 | Responder | completed |
Consumer voice
Parenteral Nutrition Down Under (PNDU) highlighted the significant quality of life impacts for children with short bowel syndrome-intestinal failure, noting that reductions in parenteral support volume directly reduce treatment hours and improve quality of life, and advocating for this reduction to be reflected in PBS restrictions.
The comment described the significant effects on quality of life for children with SBS-IF. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to paediatric patients only and requires Type III chronic SBS classification; TGA label includes both adult and paediatric patients.