MethoxsalenUVADEX
Treatment of steroid dependent, steroid intolerant or steroid refractory chronic graft versus host disease (cGVHD) in patients following allogeneic haematopoietic stem cell transplantation, as part of treatment with integrated, closed system extracorporeal photopheresis (ECP).
Decisions on record
- Meeting Sep 2021 Recommended Steroid dependent, steroid intolerant or steroid refractory chronic graft versus host disease (cGVHD)
- Meeting Jul 2021 Deferred Steroid-dependent/intolerant/refractory chronic graft versus host disease
- Meeting May 2020 Recommended Erythrodermic cutaneous T-cell lymphoma
- Meeting Jul 2017 Deferred Erythrodermic cutaneous T-cell lymphoma
Access path
- TGA registered · UVADEX
TGA label narrower than the PBS population
- Jul 2017Deferred
vs basket of second-line treatments: interferon alfa-2b (32%)…
- May 2020Recommended · restricted
Comparator changed: basket of second-line treatments: interferon alfa-2b (32%)…
- Jul 2021Deferred
Comparator changed: methotrexate, interferon alfa, vorinostat, brentuximab vedotin →…
- Sep 2021Recommended
Comparator changed: current standard of care (continued use of steroids in combination…
- PBS listing · Authority Required
From the public summary
6.1 The PBAC deferred its consideration of methoxsalen for the treatment of patients with steroid dependent, steroid intolerant or steroid refractory chronic graft versus host disease (cGVHD), to await the outcome of the Medical Services Advisory Committee (MSAC) consideration on the funding of the co-dependent extracorporeal photopheresis (ECP), before making a final decision on PBS funding. Outcome: Deferred 8PSD · Sep 2021
4.01 METHOXSALEN, Solution for blood fraction 20 microgram per mL, 10 mL, Uvadex®, Terumo BCT Australia Pty LtdPSD · Sep 2021
5.3 The submission claimed superior comparative effectiveness and non-inferior safety of ECP plus methoxsalen compared to current standard of care alone for the treatment of treatment refractory cGVHD.PSD · Sep 2021
5.4 The comparative efficacy and economic analysis will be considered by MSAC.PSD · Sep 2021
Cost-effectiveness
The submission is co-dependent with an MBS-listed procedure (ECP). The PBAC notes that the medicine component is substantially smaller in overall scope and financial implications compared to the procedure component. The comparative efficacy and economic analysis was to be considered by MSAC, not PBAC. No ICER is stated in the PBAC PSD.
The PBAC was satisfied that ECP involving methoxsalen provides, for some patients, a significant reduction in toxicity over the nominated comparators (methotrexate, interferon alfa, vorinostat, and brentuximab vedotin). PBAC · 2020
Decision context
PopulationPatients with steroid dependent, steroid intolerant or steroid refractory chronic graft versus host disease (cGVHD) following allogeneic haematopoietic stem cell transplantation, receiving integrated closed system extracorporeal photopheresis (ECP).
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Sep 2021 | Recommended | current standard of care, which consists of the continued use of steroids from first-line treatment, in combination with | — | Single-arm · Response |
| Jul 2021 | Deferred | current standard of care (continued use of steroids in combination with mycophenolate mofetil or calcineurin inhibitors) | — | Other · Other |
| May 2020 | Recommended · restricted | methotrexate, interferon alfa, vorinostat, brentuximab vedotin | — | Single-arm · Safety and efficacy (skin score response) |
| Jul 2017 | Deferred | basket of second-line treatments: interferon alfa-2b (32%), methotrexate (46%), and alemtuzumab (22%) | — | Single-arm · Other |
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to second-line cGVHD only; TGA label includes CTCL and does not specify treatment line.