PralatrexateFolotyn
Recommended OncologyAuthority RequiredSecond-line line 💬 consumer voice
Treatment of relapsed or refractory peripheral T-cell lymphoma (PTCL) in adult patients who have progressed after at least one prior therapy.
4
Submissions
3 resub
2015–17
On the record
$15k–75k
ICER range
3 sourced ICERs · 2016–2017
ICER stated
Cost basis
Decisions on record
- Meeting Nov 2017 Recommended Peripheral T-Cell Lymphoma
- Meeting Jul 2017 Not recommended FOLOTYN is indicated for the treatment of adult patients with peripheral T-cell lymphoma (nodal, extranodal, and leukaemic/disseminated ) who have progressed after at least one prior therapy.
- Meeting Mar 2016 Not recommended Patients with peripheral T-cell lymphoma who have progressed after at least one prior therapy
- Meeting Nov 2015 Not recommended Peripheral T-cell lymphoma
Access path
- TGA registered · Folotyn
TGA label equal than the PBS population
- Nov 2015Not recommended
single-arm study design without randomisation subject to considerable bias, uncertain overall survival benefit (matched…
- Mar 2016Not recommended
insufficient evidence of incremental clinical benefit against currently available treatments, concerns regarding a high…
- Jul 2017Not recommended
Comparator changed: basket of treatments (DHAP, brentuximab, gemcitabine containing…
- ↻ resubmittedNov 2017Recommended · restricted
ICER dropped $75,000 → $45,000 (-40%)
- PBS listing · Authority Required
RecommendedDeferredNot recommended
From the public summary
PBAC outcome
6.1 The PBAC recommended the S100 Efficient Funding of Chemotherapy Program Authority Required listing for pralatrexate for treatment of relapsed or refractory peripheral T-cell lymphoma (PTCL), on the basis that the substantially reduced price for pralatrexate adequately addressed the uncertainty with the cost-effectiveness estimates, noting that pralatrexate will fulfil a clinical need.PSD · Nov 2017
6.2 The PBAC did not consider use of pralatrexate outside the proposed restriction was likely and hence considered that a risk share agreement would not be required.PSD · Nov 2017
Economic analysis
5.8 In the July 2017 resubmission, a stepped cost-utility analysis was presented with an incremental cost-effectiveness ratio (ICER) of $45,000/QALY - $75,000/QALY gained.PSD · Nov 2017
A summary of the outstanding issues with the economic model from July 2017 and the sponsor’s response to the issues in this minor resubmission is shown in Table 1.PSD · Nov 2017
Clinical claim
6.29. The clinical claim presented in this resubmission was that pralatrexate was superior in effectiveness and had comparable or non-inferior safety compared to the basket of comparator treatments. This was unchanged from the November 2015 submission. The March 2016 minor resubmission did not explicitly state a clinical claim. 6.30.PSD · Jul 2017
The ESC considered that the small size of the Australian cohort also meant that differences in the distribution of key confounders between the local and OS historical cohorts were difficult to determine. The treatments received by patients in the combined historical control cohort were not representative of the nominated basket of comparator treatments.PSD · Jul 2017
Consumer comments
5.2 The PBAC noted and welcomed the input from individuals (1) via the Consumer Comments facility on the PBS website. The comment highlighted the tolerability and effectiveness of pralatrexate in a patient with PTCL. 2PSD · Nov 2017
Financial management – risk sharing
6.60. The resubmission requested a Special Pricing Arrangement such that the published ex-manufacturer price per 20 mg vial of pralatrexate was no less than $'''''''''''. The resubmission proposed a risk share arrangement to alleviate any concerns the PBAC might have regarding eligible patient numbers.PSD · Jul 2017
Cost-effectiveness
With the reduced price proposed for pralatrexate in this minor resubmission and the removal of brentuximab costs from the comparator arm, the revised base case ICER was $15,000/QALY - $45,000/QALY gained. PSD · 2017
ICER uncertain ×2Economic model disputed ×2Immature survival dataSurrogate endpointNo head-to-head trialIndirect comparisonCost-effectiveness accepted ×2
Decision context
PopulationAdults with relapsed or refractory peripheral T-cell lymphoma who have progressed after at least one prior therapy
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2017 | Recommended · restricted | basket of comparators (chemotherapy regimens) | $15k–45k | Single-arm · OS |
| Jul 2017 | Not recommended | Basket of comparator treatments (DHAP, brentuximab, gemcitabine containing regimens, methotrexate, romidepsin, ESHAP, IC | $45k–75k | Single-arm · OS |
| Mar 2016 | Not recommended | basket of treatments (DHAP, brentuximab, gemcitabine containing regimens, methotrexate, romidepsin, ESHAP, ICE) | $45k–75k | Single-arm · OS |
| Nov 2015 | Not recommended | basket of treatments including DHAP, brentuximab, gemcitabine-containing regimens, methotrexate, romidepsin, ESHAP, and | — | Single-arm · OS |
Consumer voice
One individual provided consumer input highlighting the tolerability and effectiveness of pralatrexate in their experience with peripheral T-cell lymphoma (PTCL).
tolerabilityeffectiveness
Similar precedents
Regulatory · TGA
Label equal than PBS population — Both describe treatment of relapsed/refractory PTCL in adults after ≥1 prior therapy; terminology differs but population is identical.