A Global Perspective on Orphan Medicines and Rare Diseases

By Danélia Botes

February 29, 2024


Rare diseases, albeit individually affecting a small number of people, collectively impact millions globally. Addressing these illnesses necessitates the creation of ‘orphan medications’, a process fraught with particular hurdles. This article offers a global perspective on orphan medicines, focusing on the efforts of the European Medicines Agency (EMA), US Food and Drug Administration (FDA), and Japan’s Ministry of Health, Labour and Welfare.

Understanding Orphan Medicines and Their Importance

When it comes to diagnosing, preventing, and treating uncommon diseases, orphan medications are exceptionally important. Due to the fact that there are over 26 million people living with a rare disease in the European Union alone, there is a considerable need for these treatments. However, their development is often hindered by limited and fragmented medical expertise and research, coupled with a lack of profitability for pharmaceutical companies.

Overcoming Development Challenges: The Role of Regulatory Agencies

In order to overcome these challenges, regulatory bodies play an extremely important role. The EMA, for instance, has a specific program that supports orphan medicines’ development by offering incentives such as free scientific advice, fee reductions, and ten years of market exclusivity. The FDA, on the other hand, provides tax credits, exemption from user fees, and potential market exclusivity for seven years after approval. Japan’s Ministry of Health, Labour and Welfare offers a range of incentives, including subsidy payments, guidance, and consultation, priority review, and preferential tax treatment.

The Impact of Orphan Medicines on Rare Diseases

Since 2000, the EMA has granted over 2,700 orphan designations, with more than 230 orphan medicines approved to treat rare diseases. Similarly, the FDA and Japan’s Ministry of Health, Labour and Welfare have implemented incentive programs that have led to the development and approval of several orphan medicines, significantly impacting the lives of millions of patients.

The Future of Orphan Medicines

The European Union currently reviews the regulation on orphan medications. This ongoing work promises a brighter future for patients dealing with rare diseases. Regulatory authorities worldwide, along with breakthroughs in life sciences and artificial intelligence, contribute to this positive outlook. 


The development of orphan medicines is a global effort, requiring the collaboration of regulatory agencies, pharmaceutical companies, and healthcare professionals. Despite the challenges, significant progress has been made, providing hope for millions of patients living with rare diseases worldwide.

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