RiociguatAdempas
Withdrawn CardiovascularAuthority Required 💬 consumer voice
Treatment of inoperable chronic thromboembolic pulmonary hypertension (CTEPH) or persistent CTEPH subsequent to pulmonary endarterectomy (PEA) in adult patients with WHO functional class II-IV symptoms.
4
Submissions
3 resub
2014–16
On the record
$15k–75k
ICER range
1 sourced ICER · 2016
ICER stated
Cost basis
Decisions on record
- Meeting Mar 2023 Withdrawn Pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension no PSD
- Meeting Nov 2021 Recommended Chronic thromboembolic pulmonary hypertension (CTEPH) no PSD
- Meeting Mar 2016 Recommended Chronic thromboembolic pulmonary hypertension (CTEPH)
- Meeting Jul 2015 Not recommended For pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH)
- Meeting Nov 2014 Not recommended 500 microgram tablet, 42 and 84 1 mg tablet, 42 and 84 1.5 mg tablet, 42 and 84 2 mg tablet, 42 and 84 Adempas® Bayer Australia Ltd New Listing (Major submission) Chronic thromboembolic pulmonary hypertension Section 100 Authority Required listing for the treatment of chronic thromboembolic pulmonar
- Meeting Mar 2014 Recommended Adempas® Bayer Australia Ltd Pulmonary Arterial Hypertension (PAH) To request Section 100 Highly Specialised Drugs (HSD) program Authority Required listing for the treatment of pulmonary arterial hypertension (PAH) in patients who meet certain criteria.
Access path
- TGA registered · Adempas
TGA label narrower than the PBS population
- Mar 2014Recommended · restricted
vs bosentan and sildenafil (mixed comparator)
- Nov 2014Not recommended
Comparator changed: bosentan and sildenafil (mixed comparator) → placebo
- Jul 2015Not recommended
Cost-effectiveness of riociguat for CTEPH had not been established against the appropriate comparator; PBAC did not…
- ↻ resubmittedMar 2016Recommended · restricted
- PBS listing · Authority Required
RecommendedDeferredNot recommended
From the public summary
PBAC outcome
7.1 The PBAC recommended the listing of riociguat, on the basis that it should be available only under special arrangements under Section 100 (Highly Specialised Drugs Programme) for the treatment of patients with inoperable CTEPH or persistent CTEPH subsequent to pulmonary endarterectomy under certain conditions (seePSD · Mar 2016
Economic analysis
6.19 The re-submission presented a modelled cost utility analysis. The economic model differed from the model presented in the July 2015 re-submission in that survival was modelled on the basis of overall survival rather than being dependent upon changes in WHO functional class, and the costs and outcomes associated with the treatment of patients with inoperable CTEPH and those with persistent CTEPH after surgery were evaluated as separate cohorts, rather …PSD · Mar 2016
Outcomes Cost/improvement of ≥1 WHO FC, Cost/LYG, Cost/QALY (trial based EQ-5D utilities) A partitioned survival model comprising four health states: WHO FC health states (II, III, IV) and death: patients enter the model in either WHO FC II, III or IV according to the observed baseline distribution of patients in the CHEST-1 trial.PSD · Mar 2016
Clinical claim
6.8 Details of the trials presented in the re-submission are unchanged from the July 2015 re-submission, with the exception of the addition of two studies published in 2015, and are summarised in the following table. 8PSD · Mar 2016
Ghofrani HA, D'Armini AM, Grimminger F, Hoeper MM, Jansa P, Kim NH, Mayer E, New England Journal Simonneau G, Wilkins MR, Fritsch A, Neuser D, Weimann G and Wang C. of Medicine. 2013; Riociguat for the treatment of chronic thromboembolic pulmonary hypertension. 369(4):319-29 Ghofrani HA, Hoeper MM, Halank M, Meyer FJ, Stahler G, Behr J,, Ewert R, American Journal of Weinmann G, Neuser D, and Grimminger F.PSD · Mar 2016
Consumer comments
6.2 The PBAC noted and welcomed the input from an individual (1) via the Consumer Comments facility on the PBS website. The comment indicated that the individual would like to have increased mobility, fewer side effects, better quality of life and a decrease in progression of the disease.PSD · Mar 2016
Financial management – risk sharing
6.25 The ESC noted the Sponsor’s willingness to enter into a risk-share arrangement with the Commonwealth to provide further certainty regarding the financial impact of listing riociguat for the treatment of CTEPH.PSD · Nov 2014
For more detail on PBAC’s view, see section 7 “PBAC outcome”PSD · Nov 2014
Cost-effectiveness
ICER range varies by patient cohort: inoperable patients $15,000–$45,000; persistent patients $45,000–$75,000. Corrected base case ICER $45,000–$75,000.
The submission does not provide any economic analysis of riociguat compared with placebo in patients with CTEPH. PSD · 2014
ICER uncertain ×3Economic model disputed ×2Immature survival dataCost-effectiveness accepted
Decision context
PopulationAdult patients with inoperable CTEPH or persistent CTEPH following PEA with WHO functional class II, III, or IV symptoms, with documented PVR >300 dyn·sec·cm⁻⁵ and PAPmean >25 mmHg or right ventricular dysfunction on echocardiography.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2016 | Recommended · restricted | placebo | $15k–75k | RCT · 6MWD, WHO functional class |
| Jul 2015 | Not recommended | placebo | — | RCT · 6MWD |
| Nov 2014 | Not recommended | placebo | — | RCT · 6MWD |
| Mar 2014 | Recommended · restricted | bosentan and sildenafil (mixed comparator) | — | RCT · 6MWD |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| PATENT-1 | Ph 3 | 445 | 6 Minutes Walking Distance (6MWD) - Change From Baseline to Week 12 | completed |
| CHEST-1 | Ph 3 | 262 | 6 Minutes Walking Distance (6MWD) - Change From Baseline to Week 16 | completed |
| CHEST-2 | Ph 3 | 237 | Number of Participants With Treatment-emergent Adverse Events (TEAE) | completed |
Consumer voice
An individual consumer expressed a desire for increased mobility, fewer side effects, better quality of life, and decreased disease progression.
the individual would like to have increased mobility, fewer side effects, better quality of life and a decrease in progression of the disease Consumer comments · PSD
quality of lifeside effectsmobilitydisease progression
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricted to inoperable or persistent-post-PEA CTEPH only; TGA label includes both PAH and CTEPH.