Orphan drugs are those that are used to treat rare diseases. They are also called "orphan". It seems that if the disease is rare, then it is difficult to meet it.
In fact, the paradox is in effect. Despite the rarity, there are a lot of patients with such diagnoses in the aggregate. For example, in European countries there are 30 million of them, that is, approximately every 15th person suffers from orphan disease. For their treatment, special medications are intended.
Registration of orphan drugs
Such drugs usually follow the same path of production and registration as conventional drugs. But they are provided with some benefits that encourage manufacturers to invest specifically in their development.
Government intervention to support production is carried out in various ways: tax incentives and incentives, a simplified procedure for admitting to the domestic market, subsidizing developments, increasing the period of exclusivity in the market.
Who assigns status
Orphan drug status is assigned to drugs by the Committee for Orphan Medicinal Products, European Medicines Agency. Then approved by the European Commission. Only after that a registration certificate is issued.
Requirements
In order for the application to be granted orphan status to be approved, it is necessary that it meets the following requirements:
- It should be used for the diagnosis, therapy or prevention of a disease that threatens a person’s life or has a chronic form.
- The disease should not occur in more than 5 people out of 10,000 in the countries of the European Union or it should be unlikely that the sale of a pharmaceutical product will cover the costs necessary for its development.
- There is no appropriate way to diagnose, treat or prevent a disease, or if it is created, the drug in question should help patients with such an ailment.
Law level
In Russia, at the end of March 2010, the draft law “On the Circulation of Medicines” was approved, which provides for state regulation of prices for drugs that are considered to be the most important and vital. However, in the final version there was no mention of the so-called orphan drugs. Such an amendment disappeared from the law after the first deduction of the State Duma.
Because of this, several dozen charitable foundations united in an appeal to the president of Russia. They asked to legalize the circulation of orphan funds and to simplify the regime of their import and registration in the country.
As a result, an official statement was published on the website of the Ministry of Health. Thus, the procedure for importing such drugs was simplified. Now it is no more than 5 days.
An import permit for personal use is issued in electronic format with a digital signature. Thanks to this, patients from remote regions will not have to visit the capital, queue for offices to get permission.
Orphan diseases
Examples of orphan diseases are:
- Mucopolysaccharidosis. Severe hereditary disease, which relates to metabolic disorders. In Russia, an average of approximately 15 people are born with such an ailment every year.
- Hemophilia. Hereditary pathology, in which a coagulation factor deficiency is diagnosed, due to which the process slows down. In Russia, 8 thousand patients are registered.
- Chronic mucous candidiasis. The causative agent is a fungus of the genus Candida. Usually appears in children under 18 years of age.
In addition, this list includes mucormycosis, zygomycosis, thymoma, malignant sarcoma of soft tissues, cystic fibrosis, hyperprolactinemia, Rett syndrome, ulcerative colitis of the stomach, Majid syndrome, aniridia. These are not the only orphan pathologies - their list is much larger.
The list of popular drugs
The most famous orphan drugs for diseases are:
- "Solaris". The active ingredient is eculizumab. The tool is an immunosuppressant. Indications for use - atypical hemolytic-uremic syndrome, as well as paroxysmal nocturnal hemoglobinuria. Contraindications include the carrier of Neisseria meningitides, active infection, the absence of vaccination, severe liver and kidney pathologies.
- "Enplate". The main active ingredient is romiplostim. The medicine is prescribed for idiopathic thrombocytopenic purpura.
- "Elapraz". The main component is idursulfase. The medicine is prescribed for mucopolysaccharidosis of the second type.
- Traklir. The main substance in the drug is bosentan. It is used for pulmonary hypertension.
- Enbrel. In the composition of the drug, etanercept is present. It is prescribed for juvenile arthritis with a systemic onset.
- "Eyed." The active ingredient is halsulfase. The tool is used for mucopolysaccharidosis of the fourth type.
- "Actemra". Contains tocilizumab. It is prescribed for juvenile arthritis with a systemic onset.
- Revolade. The active ingredient is Eltrombopag. The drug is used for idiopathic thrombocytopenic purpura.
- Aldazurim. The main substance is laronidase. It is prescribed for mucopolysaccharidosis of the first type.
- "Hilaris." The active ingredient is canakinumab. The tool is used for adolescent arthritis with a systemic onset.
- "Attached." Its active component is human immunoglobulin. The drug is prescribed for idiopathic thrombocytopenic purpura.
- "Repay". The active substance is agalsidase alpha. It is prescribed for Fabry disease.
- Exidjad. The active ingredient is Deferasirox. The tool is used for unspecified aplastic anemia.
- Orfadin. The active substance is nitisinone. It is prescribed for tyrosinemia.
- Adempas. Contains riotsiguat. It is prescribed for pulmonary hypertension.
- "Opsamite." Contains Macitentan. It is used for pulmonary hypertension.
- "Revolution." The composition contains sildenafil. It is prescribed for pulmonary hypertension.
- Mayozaym. The main component is alglucosidase alpha. The drug is used for Pompe disease.
- "Curtain". The main active ingredient is miglustat. It is prescribed for sphingolipidosis.
- Ventavis. Contains iloprost. Assign with pulmonary hypertension.
- "Fabrazim." Contains agalsidase beta. It is prescribed for sphingolipidosis.
- Volibris. The active compound is ambrizentan. It is prescribed for pulmonary hypertension.
- "Ilomedin." The active ingredient is iloprost. The medicine is used for pulmonary hypertension.
This list of orphan drugs is considered the most popular among those prescribed for diseases.
Contraindications
Drugs are prohibited for use with increased susceptibility to the active compound of the drug, as well as in severe forms of liver and kidney pathologies. Children are given only after the doctor’s permission.
Conclusion
Thus, the list of orphan drugs is a list of drugs that are prescribed for diseases from the group of the same name. Such pathologies are considered rare.
For the development of orphan drugs, benefits are provided to companies. Registration is carried out in the same way as with other pharmaceuticals, but with additional confirmation from the relevant commission.
As for patients, it must be remembered that the doctor should prescribe the medicine only. Self-medication is not recommended, as it may not bring the desired result, but only exacerbate the course of the disease.