PirfenidoneARX-PIRFENIDONE
Treatment of idiopathic pulmonary fibrosis (IPF). This submission was for new tablet forms (267 mg and 801 mg) as formulation changes to the previously listed pirfenidone capsule.
Decisions on record
- Meeting Mar 2018 Recommended Idiopathic pulmonary fibrosis
- Meeting Dec 2016 Recommended Idiopathic pulmonary fibrosis (IPF) no PSD
- Meeting Nov 2016 Deferred Idiopathic pulmonary fibrosis (IPF)
- Meeting Mar 2016 Not recommended Idiopathic Pulmonary Fibrosis
- Meeting Nov 2015 Not recommended Idiopathic pulmonary fibrosis (IPF)
Access path
- TGA registered · ARX-PIRFENIDONE
TGA label narrower than the PBS population
- Nov 2015Recommended · restricted
vs best supportive care (BSC)
- Mar 2016Not recommended
Comparator changed: best supportive care (BSC) → best supportive care
- Nov 2016Deferred
- Mar 2018Recommended · restricted
Comparator changed: best supportive care → pirfenidone 267 mg capsule
- PBS listing · Authority Required
From the public summary
6.1 The PBAC recommended the Authority Required listing of pirfenidone, as a 267 mg tablet form and as a 801 mg tablet, for continuing treatment only, for the treatment of idiopathic pulmonary fibrosis.PSD · Mar 2018
6.2 The PBAC noted that the recommended starting dose of pirfenidone is 1 x 267 mg capsule 3 times a day (tds) for 1 week, followed by 2 x 267 mg capsule tds for 1 week, and subsequently the maintenance dosage of 3 x 267 mg capsules tds to achieve 2,403 mg/day (Esbriet Product Information February 2016). The 801 mg tablet is to replace the requirement to take 3 x 267 mg capsules tds in the maintenance phase.PSD · Mar 2018
6.22 The current resubmission presented an updated economic model to evaluate the cost-effectiveness of pirfenidone versus BSC. The changes made since the March 2015 minor resubmission consisted of: Correcting an error in the previous model’s calculation of treatment costs.PSD · Nov 2016
Specifically, when the price reduction following market entry was switched off, instead of reverting back to the ''''''% rebate offered on the ex-manufacturer price, the rebate was incorrectly removed altogether. Consequently after 5 years, the full price of pirfenidone was applied rather than taking into account the '''''''% rebate. Unit costs were also updated to reflect current prices.PSD · Nov 2016
6.18 As in the November 2015 and March 2016 submissions, the current resubmission described pirfenidone as having superior efficacy and an acceptable safety profile compared with placebo. In November 2015, the PBAC considered that pirfenidone is 15PSD · Nov 2016
6.19 The current resubmission did not make an explicit clinical claim against nintedanib, but did state that the results of the indirect comparisons suggest that treatment with pirfenidone is non-inferior to nintedanib in FVC, all-cause mortality and IPF-related mortality, and that pirfenidone and nintedanib have a similar overall safety profile.PSD · Nov 2016
6.2 The PBAC noted and welcomed the input from individuals (50), health care professionals (5) and organisations (2) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with pirfenidone including the ability to slow disease progression and improve quality of life.PSD · Nov 2016
6.3 The PBAC noted the advice received from the Lung Foundation Australia and the Australian Pulmonary Fibrosis Consortium that treatment with pirfenidone may slow disease progression and improve quality of life. The PBAC specifically noted the advice that the only access to relatively affordable medication for IPF (i.e. nintedanib or pirfenidone) is through importation. The advice noted that alternative treatment 8PSD · Nov 2016
Cost-effectiveness
Minor submission with cost-minimisation analysis; no new ICER calculated as submission involved formulation change only.
The PBAC considered that the survival demonstrated in the historical data was acceptably similar to that of the registries. PBAC · 2016
Decision context
PopulationPatients with idiopathic pulmonary fibrosis meeting specified clinical criteria including FVC ≥50% predicted, FEV1/FVC >0.7, and DLCO ≥30% corrected for haemoglobin, diagnosed through a multidisciplinary team.
Risk sharingThe new presentations would join the current Risk Sharing Arrangement for pirfenidone and nintedanib.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2018 | Recommended · restricted | pirfenidone 267 mg capsule | — | Cost-minimisation · Cost-minimisation |
| Nov 2016 | Deferred | best supportive care | $45k–75k | RCT · OS |
| Mar 2016 | Not recommended | best supportive care | $45k–75k | RCT · FVC |
| Nov 2015 | Recommended · restricted | best supportive care (BSC) | — | RCT · Change in FVC%Pred |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| ASCEND | Ph 3 | 555 | Change in Percent Predicted Forced Vital Capacity (%FVC) From Baseline to Week 52 | completed |
Consumer voice
Consumers and health professionals reported that pirfenidone slows disease progression and improves quality of life for IPF patients. They highlighted the unmet need for PBS-subsidised treatment, financial burden of non-subsidised pirfenidone, and risks of obtaining cheaper versions online with uncertain safety and efficacy.
The comments described a range of benefits of treatment with pirfenidone including the ability to slow disease progression and improve quality of life. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to IPF patients meeting specific pulmonary function criteria (FVC ≥50%, FEV1/FVC >0.7, DLCO ≥30%) and MDT diagnosis; TGA label has no such restrictions.