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Fee crisis hits orphan drugs
Posted 24 September 2018
Australian patients' access to orphan drugs could be cut off by an "indirect and possibly unforeseen effect" of changes recently introduced by the TGA, says the director of orphan drug specialist Emerge Health.
Under the new rules, drugs are orphan designated for six months initially and then can apply for one six-month extension. They no longer retain orphan designation for an open-ended period
Emerge Health and Illuminate Health director Peter Davey told Pharma in Focus the change has resulted in the reimposition of PBS cost recovery fees on orphans.
"Historically, orphan drug designation has meant no government submission and evaluation fees in Australia. Both TGA and PBAC submission and evaluation fees used to be waived," he said.
The rationale was that the fees would discourage the filing and supply of orphan treatments to patients suffering from rare diseases (historically conditions effecting less than 2,000 people). Drugs which were orphan designated kept that status, it didn't lapse and neither did the fee waiver.
But with orphan designations now truncated, fees can quickly come into play, meaning orphan drug suppliers face cost recovery of as much as $240,000 for TGA and $135,000 per major PBAC submission, then another $135,000 for each subsequent major PBAC submission and so on, Davey said.
"No changes have been made to the PBAC fees process and this is the cause of the problem," he said. "Twelve months is simply too short a period for most companies to submit to the TGA and then go through the PBAC process."
He said Emerge Health had recently been denied a fee waiver on an orphan drug for which the orphan designation had lapsed four days before PBAC submission.
"This means that most orphan drugs will not be exempt from PBAC lodgement fees under the current system. For a number of suppliers of orphan drugs these fees will be prohibitive, and the treatments will not be made available in Australia. As a result, some patients will be denied access to new treatments for rare diseases," Davey said.
Nick Lush
Posted 24 September 2018
Australian patients' access to orphan drugs could be cut off by an "indirect and possibly unforeseen effect" of changes recently introduced by the TGA, says the director of orphan drug specialist Emerge Health.
Under the new rules, drugs are orphan designated for six months initially and then can apply for one six-month extension. They no longer retain orphan designation for an open-ended period
Emerge Health and Illuminate Health director Peter Davey told Pharma in Focus the change has resulted in the reimposition of PBS cost recovery fees on orphans.
"Historically, orphan drug designation has meant no government submission and evaluation fees in Australia. Both TGA and PBAC submission and evaluation fees used to be waived," he said.
The rationale was that the fees would discourage the filing and supply of orphan treatments to patients suffering from rare diseases (historically conditions effecting less than 2,000 people). Drugs which were orphan designated kept that status, it didn't lapse and neither did the fee waiver.
But with orphan designations now truncated, fees can quickly come into play, meaning orphan drug suppliers face cost recovery of as much as $240,000 for TGA and $135,000 per major PBAC submission, then another $135,000 for each subsequent major PBAC submission and so on, Davey said.
"No changes have been made to the PBAC fees process and this is the cause of the problem," he said. "Twelve months is simply too short a period for most companies to submit to the TGA and then go through the PBAC process."
He said Emerge Health had recently been denied a fee waiver on an orphan drug for which the orphan designation had lapsed four days before PBAC submission.
"This means that most orphan drugs will not be exempt from PBAC lodgement fees under the current system. For a number of suppliers of orphan drugs these fees will be prohibitive, and the treatments will not be made available in Australia. As a result, some patients will be denied access to new treatments for rare diseases," Davey said.
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