Orphan drugs (medicines for rare diseases) are drugs that are developed for the diagnosis, prevention or treatment of rare diseases. These diseases are also known as orphan diseases. Rare diseases usually occur in a very small number of people, which means that developing drugs to treat them can be a challenge – both in terms of funding and research.

Main characteristics of orphan drugs:

• Rare diseases: Orphan drugs are intended for diseases that only occur in a limited number of patients. This number can vary from country to country and from region to region. For example, a disease is considered rare in the EU if no more than one person in 2,000 EU citizens is affected by it.
• Commercial aspect: Drugs for rare diseases are often not lucrative due to the limited number of potential patients, i.e. buyers. This is why there are few financial incentives (German translation: finanzielle Anreize) for pharmaceutical entrepreneurs to develop and manufacture them.
• Treatment gap: For many rare diseases, there are either no effective treatments (translation into German: Behandlungen) or the treatments have only limited success and are therefore unsatisfactory. Orphan drugs are intended to fill this niche.
• Development challenges: Due to the limited number of patients and the complexity of research in connection with rare diseases, the development of orphan drugs can require an immense amount of time and resources.
• Marketing authorization: In various countries, there are special programs and laws to support and accelerate the development of orphan drugs. They can provide for more flexible requirements for clinical trials and accelerated marketing authorization procedures.

Orphan drugs play an important role in improving the quality of life (German: Lebensqualität) of patients suffering from rare diseases and fill a gap in healthcare. In most cases, the development of drugs for rare diseases requires collaboration between pharmaceutical companies, research organizations and medical institutions.

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