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Regulatory Focus™ > News Articles > EMA, TGA Launch New 'Collaboration' to Accelerate Access for Orphan Medicines

EMA, TGA Launch New 'Collaboration' to Accelerate Access for Orphan Medicines

Posted 07 April 2014 | By Alexander Gaffney, RAC

EU and Australian regulators have announced that they will begin sharing information about orphan medicines in an attempt to accelerate approval for new drugs intended for rare diseases.


Orphan medicines are those intended for diseases or conditions which affect a small number of individuals-so-called "rare diseases"-and are thus less likely to have a ready market to incentivize investment in new therapies.

The definition of "rare disease" differs throughout the world. In the US, the Orphan Drug Act defines the term as any disease affecting fewer than 200,000 individuals. In the EU, the term means fewer than five in 10,000 people in the EU, or about 250,000 people. In Australia, the Therapeutic Goods Regulations of 1990 define "rare disease" as any disease or condition affecting fewer than 2,000 individuals in Australia at any time.

Drugs intended to treat rare diseases are often granted "orphan drug" status by local regulators, enabling them to receive expedited review by regulators, increased access to scientific advice, and often reduced regulatory filing fees and other tax incentives. All these incentives are intended to make it easier to develop and obtain approval for orphan drugs in the hopes that more companies will develop products.

New Collaboration

Now two regulators have announced the creation of a new policy intended to further cut down approval times for some products.

On 4 April 2014 the European Medicines Agency (EMA) and Australia's TGA announced a new agreement that will see both regulators sharing full assessment reports-scientific evaluations of medicines-related to marketing authorizations of orphan medicines.

Explained EMA in a statement: "If the same marketing-authorisation application (MAA) is received in parallel by EMA and TGA, the two regulators have the possibility of scientific exchange to facilitate the evaluation of the medicine. Both regulators will still reach their own conclusions about the suitability of each medicine to be authorised in their respective markets."

The hope, the regulators said, was that rare disease patients would benefit from accelerated access to new medicines. Also implied in the statement is that some medicines will benefit from accelerated approvals, though neither EMA nor TGA provided any estimate regarding how many companies submit applications to the regulators in parallel.

EMA Statement


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