Click here to read: Rare disease in America

After the United States enacted the "Orphan Drugs Act" in 1983, EMA formally implemented orphan drug laws and regulations in April 2000 to encourage drug manufacturers to research, develop and market orphan drugs and also to formulate orphan drug regulations in European countries Provide a key legal framework.

According to some information released by the European Medicines Agency (EMA), there are about 30 million people in Europe suffering from rare diseases, the incidence of most diseases less than one hundred thousandth.

According to a document published by the EMA (Orphan Medicines Figures 2000-2016), pharmaceutical companies submitted 2,714 orphan drug applications during 2000-2016, while the Orphan Drug Products Council (COMP) gave affirmative opinions on 1,827 of these applications. 24 applications received a negative opinion. The European Commission finally found 1805 orphan eligibility claims and 684 applications were withdrawn during the assessment period.

See Table 1 for details.

Table 1 Number of orphan medicines identified during 2000-2016

According to the data released by the EMA, among the 1805 orphans prescribed for these orphans, the top three therapeutic areas are: Antineoplastic Drugs 36%; Muscle, Skeletal and Neurological Drugs 12%; Gastrointestinal and Metabolic Drugs 11 %, See Figure 1 for details.

Figure 1 Distribution of therapeutic areas for COMP affirmed orphan medicine during 2000-2016

In addition, the data shows that a total of 128 orphan medicines were obtained from the EU within 2000-2016 for market approval, as shown in Figure 2.

Figure 2 2000 - 2016 orphan drug market

Let us take a look at the basic situation and indications of 2017 orphan drug approved by the EU:

In 2017, EMA approved a total of 13 orphan medicines, of which five (Spinraza, BRINEURA, Bavencio, Rydapt and Xermelo, respectively) have been approved by the FDA in advance (see Table 2 for details)

Table II 2017 orphan drugs approved by the EMA