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International agreement on treatments for rare diseases

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Patients with rare diseases may get quicker access to advanced treatments under an agreement struck between the Australian medicine watchdog and its European counterpart.

The Therapeutic Goods Administration and the European Medicines Agency have agreed to share their assessments of so-called orphan drugs, which are medicines developed specifically to treat a rare medical condition.

Developing and testing treatments for conditions that may only affect a small fraction of the population present special challenges, including the lack of a large market to offset the cost of research and development, and the fact that there may not be sufficient people with the condition to meet the typical quota required for a scientifically robust clinical trial.

The agreement between the TGA and the EMA is seen as a way to help lower the barriers to orphan drugs while still ensuring they meet safety and efficacy standards.

The TGA said that where a manufacturer simultaneously seeks marketing authorisation in both Australia and Europe, there is an opportunity for “scientific exchange” that could facilitate the evaluation of the medicine concerned.

But, indicating the limits to cooperation, “both regulators will still reach their own conclusions about the suitability of each medicine to be authorised in their respective markets,” the TGA added.

Nonetheless, the regulator said, “the agreement will reinforce collaboration and work-sharing between the two regulatory authorities, and will contribute to accelerating access to new medicines for patients with rare diseases in Europe and Australia”.

It said such collaboration was particularly important “in view of the small number of patients worldwide, and the need for the limited number of studies performed to benefit patients, regardless of where they live”.

In addition to the agreement on orphan medicines, the two regulators have also reached a mutual recognition agreement on good manufacturing practice for medicines.

Adrian Rollins

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