DifelikefalinKORSUVA
Treatment of moderate to severe pruritus associated with chronic kidney disease in adult patients on haemodialysis who have inadequate response to best supportive care.
Decisions on record
- Meeting Nov 2023 Recommended Chronic kidney disease associated pruritus (moderate to severe, haemodialysis patients)
- Meeting Mar 2023 Not recommended Chronic kidney disease (CKD) associated pruritus in adult patients receiving haemodialysis
Access path
- TGA registered · KORSUVA
TGA label narrower than the PBS population
- Mar 2023Not recommended
vs best supportive care
- ↻ resubmittedNov 2023Recommended · restricted
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the Authority Required (Telephone/Online) listing of difelikefalin for the treatment of moderate to severe pruritus associated with chronic 41PSD · Nov 2023
7.2 The PBAC noted the consumer comments discussed the severe impact of the condition on quality of life and the current lack of effective treatment options. The PBAC noted that health care professionals described the effectiveness of difelikefalin in reducing CKD-associated pruritis symptoms observed in patients accessing it though special access programs.PSD · Nov 2023
The PBAC noted the reliability of the economic The resubmission provided additional justification to address evaluation for decision-making was contingent on PBAC’s concerns with the large utility decrements in the acceptance of the modelled utility decrements for previous submission based on an updated literature review pruritus. The PBAC noted concerns regarding the and comparisons with other sources of utilities.PSD · Nov 2023
PBAC meeting). The resubmission argued that the updated estimates have greater plausibility than the estimates previously considered by the PBAC, as the utility values were lower at baseline and yielded smaller utility decrements for pruritus.PSD · Nov 2023
6.50 The resubmission described difelikefalin as superior in terms of efficacy and inferior in terms of safety compared to best supportive care. These claims were unchanged from the previous submission and were primarily based on the same evidence previously considered by the PBAC. In March 2023, the PBAC considered that the claim of superior efficacy and inferior safety was reasonable (para 6.60 and 6.61, difelikefalin PSD, March 2023 PBAC meeting).PSD · Nov 2023
6.51 The PBAC noted the clinical claims and key clinical evidence supporting them were unchanged from the March 2023 submission and reaffirmed its view that the claims of superior comparative effectiveness and inferior comparative safety compared to best supportive care were reasonable.PSD · Nov 2023
6.2 The PBAC noted and welcomed the input from individuals (4), health care professionals (7) and organisations (1) via the Consumer Comments facility on the PBS website. The comments from individuals and health care professionals described the severe impacts of CKD-associated pruritis on quality of life and everyday functioning, including reduced sleep quality, impacts on mental health, decreased compliance with treatment and limited social function.PSD · Nov 2023
6.3 The PBAC also noted the input from Kidney Health Australia. The input supported that provided by individuals and health care professionals regarding the quality of life impacts of CKD-associated pruritis. The comments stated that there are limited effective therapeutic options available and noted those which may offer some benefit may be challenging to use in patients on dialysis and are associated with many adverse effects.PSD · Nov 2023
Cost-effectiveness
ICER values are redacted in the public summary document. The document indicates ranges ('$45,000 to < $55,000' and '$55,000 to < $75,000') but the specific numeric ICER is marked as redacted (shown as '$___1' and '$___2').
Decision context
PopulationAdult patients on haemodialysis (in-centre or at-home) with chronic kidney disease and moderate to severe pruritus (WI-NRS score > 4) who have inadequate response to best supportive care.
Risk sharingRisk sharing arrangement (RSA) proposed by the sponsor based on predicted financial estimates and a rebate percentage (redacted). PBAC advised that an RSA would be required to address uncertainty regarding use outside the proposed restriction.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2023 | Recommended · restricted | best supportive care | — | RCT · QoL |
| Mar 2023 | Not recommended | best supportive care | — | RCT · QoL |
Consumer voice
Consumers and healthcare professionals reported severe quality-of-life impacts from CKD-associated pruritis, including sleep disruption and mental health effects, with limited current treatment options. Kidney Health Australia supported these comments and noted difelikefalin's effectiveness and ease of administration alongside dialysis.
The comments from individuals and health care professionals described the severe impacts of CKD-associated pruritis on quality of life and everyday functioning, including reduced sleep quality, impacts on mental health, decreased compliance with treatment and limited social function. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to patients with inadequate response to best supportive care; TGA label has no such prior treatment requirement.