Australia Looking to Reform Orphan Drug Regulations

The Therapeutic Goods Administration is considering raising the threshold for classifying rare diseases, as well as the number of patients in specific subsets of a disease, to broaden the scope of drugs that qualify for orphan status.

The proposed changes, outlined in a recent discussion paper, are needed to better align the agency’s orphan drug definitions and incentives with a world of evolving targeted treatments that are helping people live longer, the TGA says.

Currently, the upper limit for classifying rare diseases under the orphan drug program is 2,000 patients. However, Australia’s population has increased by nearly 4 million since those regulations were drafted in the late 1990s, and the change risks cutting the number of patients eligible to be treated with orphan drugs.

That’s because increasingly targeted treatments for diseases including hemophilia A are extending patients’ lives, the TGA explains. The number of patients with factor VIII deficiency hemophilia A alone has grown by 161 in the last six years, the agency says.

In light of that, it may be appropriate to classify specific stages or subsets of patients with hemophilia A, for example, as rare diseases needing orphan drug treatment, the TGA suggests. This would also reflect the fact that it has become increasingly common for orphan drugs to treat more narrow indications.

The agency also notes that its proportion threshold for rare diseases — 0.88 in 10,000 — may no longer be accurate and might be better expressed as a percentage of the population or as a specific prevalence of a disease per 10,000 people, which is what the EU uses — i.e., five in 10,000.

Rethinking Incentives

The agency is also rethinking the incentives it gives to sponsors of orphan drugs. Since the 1990s, sponsors have had their evaluation and registration fees waived — an amount the TGA says is about US $173,000 per sponsor for new chemical entities, $67,000 for a major indication change and $103,000 for an expanded indication.

While preserving these waivers remains an option, so is waiving the initial registration fee, but sponsors would still be charged for changing or expanding a drug’s indication. Charging 50 percent of the fees levied for non-orphan drugs is another option, as is waiving fees only for specific patient demographics, such as children, the TGA says.

Discontinuing fee waivers may make little difference, however, as that cost is minimal compared with what it costs to bring a drug to market, and sponsors recoup much of their R&D expenses through drug pricing, the agency adds.

Charging for subsequent indications might also have the unintended consequence of delaying orphan drugs’ time to market, as sponsors may attempt to submit a single application for multiple indications, requiring more time to gather the supporting data.

Comments are due to by June 15. Read the consultation document at — Lena Freund

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