Orphan drugs

The term orphan drug or orphan drug (from English orphan , "the orphan ", from Greek  ορφανός ) was first used in 1983 for drugs that are used to treat rare diseases . A more precise term is "drugs for rare diseases" (English orphan pharmaceutical drug or orphan medicinal product ). These drugs are not of interest to the pharmaceutical industry because of the sometimes tiny market and their low sales during the statutory patent protection period - with high development costs at the same time .

In 1983 the United States passed the Orphan Drug Act , which aims to promote and develop such drugs.

In January 2000 the EU put the Orphan Medicines Regulation into force. The Committee on medicines for rare diseases (Committee for Orphan Medicinal Products, COMP ) of the European Medicines Agency (EMA) adopted at its regular intervals held meetings regularly recommendations for granting the status of "drug for a rare disease."

The criteria for classification as a rare disease are regulated differently in different locations:

• EU: fewer than 5 patients per 10,000 population
• USA: fewer than 200,000 patients per year, or 7.5 per 10,000 population
• Japan: fewer than 50,000 patients per year, or 4 per 10,000 population
• Australia: fewer than 2,000 patients per year, or around 1 in 10,000 people

Orphan medicines in Europe

Legal act

Some of the key points of the EU regulation for designation as orphan medicinal products are:

• the economic and epidemiological criteria (there must be no satisfactory method so far, or if such a method exists, the product must promise considerable benefit)
• Formation of an Orphan Medicines Committee in the European Medicines Agency (EMA)
• Automatic access to the centralized procedure ( authorization )
• Market exclusivity for the approved indication for a period of ten years for the person who has approved the orphan drug

Thus, if a pharmaceutical manufacturer of orphan drug status for a supplement issued, it means ten years for the company exclusive rights from market approval of the new drug and the reduction of fees .

Community register

The assignment of orphan drug status is done by the EU Commission on the recommendation of the Committee for Orphan Medicinal Products of the European Medicines Agency . A medicinal product with orphan drug status is entered as such in the EU community register.

Approval and Marketing

Drugs for orphan diseases are promoted through a simplified approval process. The entry in the community register does not yet mean approval of the medicinal product in the indication . This must be granted independently in the centralized procedure . Medicines with orphan status authorized in the EU can be called up in the database on Orphanet , for example, and are also published in a booklet (“List of Medicines for Rare Diseases in Europe”).

Additional benefit

The law for the reorganization of the drug market (AMNOG) passed in Germany in November 2010 requires, among other things, that the manufacturer must submit evidence of an additional benefit for all new drugs. The extent of the additional benefit of a drug determines its reimbursement amount. Since approval as a drug for a rare disease (orphan drug) is only granted if the disease is rare and there is no adequate therapy for this, there is no need for an orphan drug to provide - repeated - evidence of an additional benefit.

The Federal Ministry of Health (BMG) considers this relief to be appropriate only for medicinal products that have a low turnover due to their approval for rare diseases. If, on the other hand, the pharmaceutical entrepreneur achieves sales of more than 50 million euros with the orphan drug, he must provide evidence of the additional benefit. Manufacturer associations are critical of this regulation and advocate the additional benefit of orphan drugs in the AMNOG process without considering a sales threshold. In contrast, the National Association of Statutory Health Insurance Funds wants to see the special statutory status of orphan drugs in the “early benefit assessment” revised.

See also

• Compassionate Use
• Off-label use

Web links

• Register of designated Orphan Medicinal Products (alphabetical)
• Orphan Drug List of the Association of Researching Pharmaceutical Manufacturers (continuously updated)

Individual evidence

1. ↑ Regulation (EC) No. 141/2000 of the European Parliament and of the Council of December 16, 1999 on medicinal products for orphan diseases. Retrieved November 9, 2019 . 
2. ^ Public Health - European Commission. Retrieved November 9, 2019 . 
3. ↑ Directory of medicines for rare diseases in Europe (PDF)
4. ↑ See. § 35a paragraph 1, sentence 10 SGB V .
5. ↑ BPI information brochure on orphan drugs and key political demands from January 1, 2015 , accessed on February 28, 2019.
6. ↑ vfa- / vfa bio position paper Orphan Drugs, as of December 2018 , accessed on February 28, 2019.
7. ^ Orphan Drugs - Cash box against special status , Doctors newspaper, January 22, 2016.