PERTH, Australia – Australia's Therapeutic Goods Administration (TGA) will start accepting priority review applications, and it has expanded its orphan drug designation criteria, thanks to new legislation passed last month that overhauls the country's drug regulations.

The new pathway is expected to bring Australia in line with other global regulators and will shave three months off the drug registration process. The target time frame is 150 working days compared to 190 days for a standard review. (See BioWorld Today, Dec. 7, 2016.)

Effective July 1, the TGA will open its priority review pathway for prescription medicines, at which point it will accept notifications from sponsors of their intent to lodge a priority review designation application.

"Notifications at least one month prior to lodgement are strongly encouraged," TGA spokeswoman Kay McNiece told BioWorld Asia. She said the agency will start to accept lodgement of priority review designation applications by Aug. 1.

If a priority review application is approved, the TGA will publish the details of the eligible designation decision on its website.

The new priority pathway ensures certain drugs that provide a therapeutic advance compared to existing therapies are given priority evaluation, but they will still be subject to a full evaluation of safety, quality and effectiveness.

To be eligible for the new pathway, a new active ingredient or fixed combination would not have previously been listed on Australian Register of Therapeutic Goods (ARTG).

A new medicine may be eligible for the priority review designation if all three of the following eligibility criteria are met:

• The priority indication treats, prevents or diagnoses a life-threatening or seriously debilitating condition.

• There are no comparable treatments approved on the ARTG or there is substantial evidence that the therapy provides a significant improvement in efficacy or safety compared to the existing therapy.

• There is substantial evidence demonstrating that the therapy provides a major therapeutic advance.

Sponsors must also include an assessment of the magnitude of the improvement in safety or efficacy. Supporting evidence demonstrating significant improvement in safety or efficacy should be based on clinical trial data, the guidance stated.

Sponsors may only submit a specific novel medicine for a specific indication. Companies wanting to submit applications for more than one indication must submit separate applications for each indication.

If an application also has a supporting companion diagnostic, sponsors need to list the name of the manufacturer and diagnostic as well as ensure that an application for the diagnostic has been lodged or is planned. Sponsors should consider diagnostic sensitivity and specificity, predictive values and likelihood ratios.

More orphans

The priority review designation and orphan pathway overlap considerably, and the agency is allowing joint or sequential applications for both pathways for eligible therapies under certain conditions.

The eligibility criteria for Australia's orphan drug program have been updated for the first time in 20 years. The guidance lays out the criteria for the standard orphan drug designation as well as a pathway for new dosage forms that would not be financially viable to bring to market in the absence of a TGA fee waiver.

The eligibility criteria for the orphan drug program is similar to the priority review program in that the condition must be serious and there are no drugs to treat the condition or the new treatment provides a significant benefit over approved therapies.

To meet the orphan drug prevalence threshold, the drug would treat a condition that affects fewer than five in 10,000 people in Australia. Sponsors should include statistics for life expectancy and morbidity for patients with the orphan disease, as well as the effect of the disease on patients' daily lives.

The new regulations, effective July 1, encompass the following changes:

• The current 100 percent fee waiver is retained as an incentive to develop orphan drugs.

• A more generous orphan disease prevalence threshold has been established, widening the eligibility criteria.

• The proposed orphan condition must be seriously debilitating or life threatening.

• Additional criteria aim to bring orphan products to market for conditions that lack current treatments or that provide significant benefit over existing therapies.

• The validity of the orphan drug designation would lapse after six months, and sponsors could apply for extensions in certain circumstances.

A transition period will remain in effect for existing orphan drug designations that have already been submitted, and those would lapse after 12 months rather than six months.

A drug would not be eligible for orphan designation if it has been rejected due to safety concerns by the TGA, the FDA, the EMA, Health Canada or the U.K.'s Medicines and Healthcare products Regulatory Agency.

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