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Regulatory Focus™ > News Articles > TGA to Move Forward With Orphan Drug Revamp

TGA to Move Forward With Orphan Drug Revamp

Posted 18 April 2017 | By Michael Mezher 

TGA to Move Forward With Orphan Drug Revamp

Australia's pharmaceutical industry is "broadly supportive" of proposed changes to the country's orphan drug program, the Therapeutic Goods Administration (TGA) says.

On Tuesday, TGA released feedback it received in response to a 2016 consultation on the proposed changes, which include raising the population threshold for rare diseases, taking the seriousness of the condition into account when making designations and requiring sponsors to demonstrate significant benefit over existing treatment options.

Now that the public consultation has concluded, TGA says it plans to introduce new regulations to implement the proposed changes, though it notes that it will provide industry with a transition plan before implementing any reforms.

However, not all drugmakers are backing the proposed changes, and one in particular—that orphan designations would automatically lapse after a set amount of time if not submitted for approval—drew nearly universal disapproval.


Most of the 39 responses to the proposal TGA received were from the pharmaceutical industry, including industry associations Medicines Australia and the Generic and Biosimilar Medicines Association as well as major drugmakers AbbVie, Alexion, Amgen, Bayer, BioMarin, GlaxoSmithKline (GSK), Johnson & Johnson, Pfizer, Novartis, Sanofi and Shire.

The consultation calls to revamp TGA's designation criteria for orphan drugs to be more in line with the criteria used by the European Medicines Agency (EMA).

Overwhelmingly, industry supported part of TGA's first proposal, which would raise the threshold for disease prevalence nearly six-fold, from 2,000 patients in Australia to 5 in 10,000 patients, or roughly 12,000 patients.

However, TGA's other proposals, which include a requirement that the disease be life threatening or seriously debilitating and have no approved alternatives or offer a significant benefit over existing treatments, drew a mixed reaction from industry.

Some companies, such as Amgen, GSK, Novartis and Sanofi say they support the requirement, while others such as Johnson & Johnson and Bayer raised questions about how TGA would define a disease's seriousness.

Drugmakers also supported TGA's proposal to continue to waive submission fees for orphan drugs, though some respondents including AbbVie, Johnson & Johnson, GSK and Novartis called for the agency to consider providing companies with additional incentives, such as longer periods of marketing exclusivity or tax incentives for approved orphan products.

While TGA appears to be moving forward with most of the proposals from the consultation, including the provision that orphan designations will lapse after six months, the agency says it will allow sponsors to renew the designation for an additional six months in certain circumstances.


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