Decitabine + CedazuridineINQOVI 35/100
Treatment of myelodysplastic syndrome (MDS) and chronic myelomonocytic leukaemia (CMML) in high-risk patients.
Decisions on record
- Meeting Jul 2021 Recommended High-risk myelodysplastic syndromes and chronic myelomonocytic leukaemia
- Meeting Mar 2021 Not recommended Intermediate-2/high-risk MDS and CMML
Access path
- TGA registered · INQOVI 35/100
TGA label narrower than the PBS population
- Mar 2021Not recommended
vs azacitidine
From the public summary
The PBAC did not recommend the listing of decitabine+cedazuridine for the treatment of patients with myelodysplastic syndrome (MDS) classified as intermediate-2 or high- risk according to the International Prognostic Scoring System (IPSS) and patients with chronic myelomonocytic leukaemia (CMML) due to the claims of non-inferior effectiveness and safety compared with azacitidine being uncertain.PSD · Mar 2021
The PBAC noted the comments received from consumers and organisations, and the sponsor’s hearing, which highlighted the advantages, in particular for patients living in rural and remote areas, of an oral treatment over the current standard of care, azacitidine, which is administered subcutaneously or intravenously and requires attendance at an outpatient clinic for seven days each month.PSD · Mar 2021
The submission presented a cost-minimisation analysis based on the indirect comparison versus azacitidine and the pharmacokinetic evidence comparing 20PSD · Mar 2021
The submission estimated equi-effective doses based on the dosing regimen proposed for decitabine+cedazuridine and the dosing recommended for azacitidine in its PI (see Table 12).PSD · Mar 2021
The submission described decitabine+cedazuridine as non-inferior in terms of effectiveness compared with azacitidine and non-inferior in terms of safety compared to azacitidine.PSD · Mar 2021
The ESC considered while the pharmacokinetic evidence has adequately demonstrated that decitabine+cedazuridine is bioequivalent to IV decitabine, the claim of non-inferiority of IV decitabine and azacitidine was not strongly supported by the evidence, for the following reasons: 19PSD · Mar 2021
PBAC noted and welcomed the input from health care professionals (4) and organisations (2) via the Consumer Comments facility on the PBS website. The comments from health care professionals noted that current treatment with azacitidine requires patients to attend outpatient clinics over several days each month.PSD · Mar 2021
The Leukaemia foundation and Rare Cancers Australia strongly supported listing decitabine+cedazuridine on the PBS, noting that blood cancers are associated with debilitating effects on quality of life and the availability of an oral agent which reduces the need to travel for treatment, would be valuable for these patients.PSD · Mar 2021
Cost-effectiveness
Cost-minimisation analysis; no ICER calculated. Economic evaluation presented but not detailed in the public summary.
The PBAC considered that the claim of non-inferior comparative effectiveness was uncertain given the issues raised in paragraph 6.35. PBAC · 2021
Decision context
PopulationAdult patients with myelodysplastic syndrome classified as intermediate-2 or high-risk according to IPSS with ≤30% marrow blasts, and chronic myelomonocytic leukaemia patients with 10% to 29% marrow blasts without myeloproliferative disorder.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2021 | Not recommended | azacitidine | — | RCT · Surrogate |
Consumer voice
Health care professionals and consumer organisations noted that an oral hypomethylating agent would reduce treatment burden by decreasing the need for frequent outpatient clinic attendance and travel, particularly benefiting patients in remote areas and reducing clinic burden.
current treatment with azacitidine requires patients to attend outpatient clinics over several days each month Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to high-risk MDS (intermediate-2 or high-risk IPSS) and CMML with specific blast percentages; TGA label includes all adult MDS and CMML.