Funding plea for early-onset emphysema therapies


By Michael Woodhead

22 Feb 2018

Patients with Alpha 1 Antitrypsin Deficiency (AATD), which causes early onset emphysema, will have to wait at least a year for any decision on funding for TGA-listed augmentation treatments, the government says.

A patient petition to fast track funding for alpha I proteinase inhibitor products such as Zemaira and Prolastin-C has a received a response from Federal minister for health Greg Hunt, who said decisions on funding had to go through the usual assessment process for blood-based products overseen by the National Blood Authority.

In a response tabled in parliament, the minister said the augmentation products for AATD were derived from human plasma and therefore would require evaluation by the Jurisdictional Blood Committee, which would then provide advice to the Council of Australian Governments (COAG) Health Council for a decision.

“Based on the timing for consideration of previous submissions, the timeframe for the evaluation and approval processes may take approximately 12 months,” he said.

AATD affects about one in 3000 people and may cause emphysema onset before the age of 40, as well as liver problems.

The two augmentation products have been  listed on the Australian Register of Therapeutic Goods (ARTG) for the treatment of AATD since February 2017 and November 2016), respectively

Patients would usually gain subsidised access to such products through hospitals under the national blood arrangements; with funding shared between the Commonwealth and the states and territories.

Mr Hunt said the National Blood Authority had received applications for funding of alpha-I -proteinase inhibitor products, which would make its decision based on efficacy and cost.

“I would like to note the potential for registered products to be supplied to individuals outside of the national arrangements. It may be possible for medical practitioners to prescribe products for medical conditions that are not funded under the national blood arrangements through a public hospital via the local hospital therapeutics committee,” said Mr Hunt.

“These local arrangements vary between hospitals and jurisdictions. Those with AATD can approach their doctors or their relevant Government regarding funding through such an arrangement,” he said.

A 2012 review recommended that AATD be treated with measures similar to COPD, and suggested that augmentation therapy with intravenous alpha-1 antitrypsin should be considered for nonsmokers who have an FEV1 of 25%–80% of predicted.

However it noted that because of the relatively rarity of the condition there was only limited clinical trial evidence showing that augmentation therapy  – which may cost up to $100,000 a year – slowed the progression of lung disease in AATD.

Some respiratory professional groups recommend that all patients with COPD be screened for AATD, while others recommend more selective testing of people whose symptoms appear at a young age.

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