The European Medicines Agency (EMA) and Australia’s Therapeutic Goods Administration (TGA) have announced that they are to share the full assessment reports related to marketing authorisations (MA) for orphan drugs.
If the same MA application is received in parallel by the EMA and TGA, the two regulators will be able to use scientific exchange to facilitate evaluation of the medicines, according to EMA executive director Professor Guido Rasi and Professor John Skerritt, national manager of the TGA, who announced the collaboration at a meeting held at the EMA in London.
However, both the regulatory agencies will still reach their own conclusions about the suitability of each medicine to be authorised in their respective markets.
The agreement will reinforce collaboration and work-sharing between the EMA and TGA, and will contribute to accelerating access to new medicines for patients with rare diseases in Europe and Australia, the agencies say. They also point out that global collaboration on orphan medicines and rare diseases is particularly important in view of the small number of patients affected worldwide and the need for the limited number of studies performed to benefit patients regardless of where they live.
Under the framework of greater international work-sharing, the EMA and TGA are also collaborating and exchanging information in a number of other areas, including: through a mutual recognition agreement on Good Manufacturing Practice (GMP) for medicines; their work to rationalise international GMP inspections of manufacturers of active substances; and as part of multilateral relationships involving other regulatory partners.
Since 2008, the EMA and the US Food and Drug Administration (FDA) have been working together to encourage applications for orphan designation to be submitted to the two agencies in parallel. In 2012, this programme was extended to Japan’s Ministry of Health, labour and Welfare (MHLW) and Pharmaceutical and Medical Devices Agency (PMDA).
- Rare diseases are defined as conditions which affect no more than five in every 10,000 people. Around 70 million people around the world are estimated to be suffering from a rare disease, including more than 1.2 million in Australia (or 8%-9% of the population), and about 30 million across the European Union (EU).