AnakinraKineret
Not recommended Rare diseaseAuthority RequiredNot applicable line 💬 consumer voice
Amendment of PBS listing from Authority Required (STREAMLINED) to Authority Required for treatment of patients with moderate to severe cryopyrin associated periodic syndromes (CAPS).
3
Submissions
2 resub
2014–17
On the record
—
ICER range
Not modelled
Cost basis
risk sharing
Decisions on record
- Meeting Jul 2017 Not recommended Moderate to severe cryopyrin associated periodic syndromes
- Meeting Jul 2015 Recommended Cryopyrin-associated periodic syndromes
- Meeting Nov 2014 Recommended 100 mg/0.67 mL, 28 x 0.67 mL syringes Kineret® A.Menarini Australia Pty Ltd New listing (Major submission) Cryopyrin Associated Periodic Syndromes Section 100 Authority Required listing for the treatment of cryopyrin-associated periodic syndromes.
- Meeting Jul 2004 Recommended Used to treat no PSD
Access path
- TGA registered · Kineret
TGA label narrower than the PBS population
- Nov 2014Recommended · restricted
vs best supportive care
- Jul 2015Recommended · restricted
Evidence: Single-arm → Registry
- Jul 2017Not recommended
No clinical basis to change the level of authority; current restriction level considered appropriate; submission…
RecommendedDeferredNot recommended
From the public summary
PBAC outcome
5.1 The PBAC did not recommend amending the Authority Required (STREAMLINED) listing for anakinra to an Authority Required listing for the treatment of patients with moderate to severe cryopyrin associated periodic syndromes (CAPS).PSD · Jul 2017
5.2 The PBAC noted that the Sponsor presented utilisation data for anakinra in the first 21 months of listing, and claimed that the number of scripts used was higher than anticipated. Based on these utilisation data and the sponsor’s assumption of leakage to patients with other conditions the sponsor requested a change in restriction level to Authority Required. 2PSD · Jul 2017
Economic analysis
6.23 The submission presented one cost-effectiveness analysis (short-term) and two cost- utility analyses (one short-term and one long-term). This was consistent with the clinical claim that anakinra is superior in terms of effectiveness but inferior in terms of safety to best supportive care. The submission described the approach as a stepped economic evaluation.PSD · Nov 2014
Therefore, the Commentary presented the short term (Steps 1 and 2) and long term (Step 3) analyses separately, and retained the submission’s terminology of Steps 1, 2 & 3. The steps of the economic evaluation and model structures are summarised below.PSD · Nov 2014
Clinical claim
6.21 The submission described anakinra ± best supportive care as superior in terms of comparative effectiveness and inferior in terms of comparative safety over best supportive care.PSD · Nov 2014
6.22 The Commentary and ESC observed that the superior treatment effect of anakinra over best supportive care in terms of resolving common CAPS symptoms and inferior safety is supported by the evidence presented in the submission. The Commentary and ESC further observed that the evidence indicated that anakinra had a limited effect on some important complications of CAPS (e.g.PSD · Nov 2014
Consumer comments
6.2 The PBAC noted and welcomed the input from individuals (9), a health care professional (1) and organisations (2) via the Consumer Comments facility on the PBS website. The individual comments described some of the benefits of treatment with anakinra including improvement in symptoms and quality of life, and the need for treatment options in a rare disease such as CAPS.PSD · Nov 2014
6.3 Rare Voices of Australia outlined the supportive evidence and experience with anakinra for CAPS in Europe and the United States of America (USA). This organisation further commented that PBS listing would achieve greater equity in access for Australian patients and noted the difficulty in the current consumer comments process reaching audiences with a rare disease.PSD · Nov 2014
Cost-effectiveness
Minor submission with no economic evaluation; no ICER calculated.
Overall, the ESC advised that the Step 3 model is highly likely to be unreliable due to concerns with the transformation of DLQI scores to utility weights, the incomparability of the utility weights of the other health states, the length of the model duration, potential underestimates of the dose of anakinra used in practice and therefore the estimated treatment costs, the probability of developing complications and the costs of managing these complications. PBAC · 2014
ICER uncertainEconomic model disputedSurrogate endpoint
Decision context
PopulationAdult and paediatric patients with moderate to severe cryopyrin associated periodic syndromes (CAPS).
Risk sharingA Risk Sharing Arrangement was established at the time of the November 2014 PBAC recommendation to manage any risk of leakage into rheumatoid arthritis and mild forms of CAPS.
Why it was knocked back
- No clinical basis to change the level of authority; current restriction level considered appropriate; submission provided no evidence to support claim of use outside current restriction.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2017 | Not recommended | — | — | Cost-minimisation |
| Jul 2015 | Recommended · restricted | — | — | Registry |
| Nov 2014 | Recommended · restricted | best supportive care | — | Single-arm · Surrogate |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| Study 03-AR-0298 | Ph 1, PHASE2 | 43 | Diary Symptom Sum Score (DSSS) (Fever, Rash, Joint Pain, Vomiting, and Headaches) | terminated |
Consumer voice
No consumer comments were received for this item.
The PBAC noted that no consumer comments were received for this item. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to moderate to severe CAPS; TGA label includes all severity levels of CAPS.