Regulatory Framework:

Orphan drug legislation exhibits remarkable diversity on a global scale. In the United States, the Orphan Drug Act was enacted in 1983, while the Japanese archipelago implemented its Orphan Drug Regulations in 1993. For its part, Australia introduced its Orphan Drug Policy in 1998, and the European Union consolidated its position through Regulation (EC) No 141/2000, enacted in 2000.

Regulatory Entities Involved:

Each jurisdiction designates specific entities for the supervision and approval of these essential drugs: in the United States, the FDA and the OOPD; in Japan, the MHLW and the OPSR; in Australia, the TGA; and in the European Union, the EMEA together with the COMP.

Prevalence Criteria:

The criteria for classifying a condition as orphan-worthy vary considerably: in the United States, a threshold of 7.5 per 10,000 individuals is established; in Japan, 4; in Australia, 1.1; and in the European Union, 5. These thresholds determine the scope of the populations that can benefit from these policies.

Market Exclusivity and Tax Incentives:

The granted market exclusivity ranges from 7 years in the United States to 10 years in the European Union, with Australia offering a duration comparable to that of other drugs, of only 5 years. Only the United States and Japan provide tax incentives for clinical trials, and research support varies significantly between different regions.

Accelerated Authorization Procedures and Technical Support:

All jurisdictions examined facilitate accelerated marketing procedures and provide some level of technical assistance for the preparation of application dossiers. However, the possibility of reconsidering applications for orphan drug designation and the degree of technical assistance offered vary between them.

Conclusion:

While there is an international consensus on the need to promote the development and accessibility of orphan drugs, the specific policies and incentives differ substantially from one region to another, reflecting the different health priorities and regulatory capabilities.

Abbreviations:

* FDA: Food and Drug Administration

* OOPD: Office of Orphan Products and Development

* MHLW: Ministry of Health, Labour and Welfare

* TGA: Therapeutic Good Administration

* EMEA: European Agency for the Evaluation of Medicinal Products

* COMP: Committee for Orphan Medicinal Products

* NIH: National Health Institute

Sources:

#FDA #Orphanet

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