Orphan drugs are those used to treat rare diseases. They are also called "orphans". It seems that if the disease is rare, then it is difficult to meet it.
A paradox is actually at work. Despite the rarity, in the aggregate there are a lot of patients with such diagnoses. For example, in European countries there are 30 million of them, that is, approximately every 15th person suffers from an orphan disease. Special medicines are intended for their treatment.
Orphan drugs registration
These drugs usually follow the same production and registration path as conventional medicines. But they are given some benefits that encourage manufacturers to invest in their developments.
Government intervention to support production comes in a variety of ways: tax breaks and incentives, simplified access to the domestic market, development subsidies, extended market exclusivity.
Who assigns status
Orphan drug status assignedmedicines by the Committee for Orphan Medicinal Products, European Medicines Agency. Then approved by the European Commission. Only after this is the registration certificate issued.
Requirements
In order for an application for orphan status to be approved, it must meet the following requirements:
- Should be used to diagnose, treat, or prevent a life-threatening or chronic disease.
- The disease should affect no more than 5 people out of 10,000 in the European Union or it should be unlikely that sales of a pharmaceutical product will cover the costs needed to develop it.
- There is no appropriate method for diagnosing, treating or preventing the disease or, if it is created, the drug in question should help patients with such an ailment.
At the level of the law
In Russia at the end of March 2010, the bill "On the Circulation of Medicines" was approved, which provides for state regulation of prices for drugs that are considered essential and vital. At the same time, there was no mention of the so-called orphan drugs in the final version. Such an amendment disappeared from the law after the first proofreading by the State Duma.
Because of this, several dozen charitable foundations unitedin an address to the President of Russia. They asked to legalize the circulation of orphan drugs and simplify the regime for their import and registration in the country.
As a result, an official statement was published on the website of the Ministry of He alth. Thus, the procedure for importing such drugs has been simplified. Now it is no more than 5 days.
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, stand in line at the offices to get permission.
Orphan diseases
Examples of orphan diseases are:
- Mucopolysaccharidosis. Hereditary disease in severe form, which concerns metabolic disorders. In Russia, on average, about 15 people are born with this disease every year.
- Hemophilia. A hereditary pathology in which a deficiency of a blood clotting factor is diagnosed, which slows down the process. 8,000 patients have been registered in Russia.
- Chronic mucous candidiasis. The causative agent is a fungus of the genus Candida. Usually occurs in children under 18.
In addition, this list includes mucormycosis, zygomycosis, thymoma, malignant soft tissue sarcoma, cystic fibrosis, hyperprolactinemia, Rett syndrome, gastric ulcerative colitis, Majid syndrome, aniridia. These are not the only orphan pathologies - their list is much longer.
List of popular drugs
The most famous orphan drugs for diseases are:
- "Soliris". The active ingredient is eculizumab. The drug is an immunosuppressant. Indications for use - atypical hemolytic-uremic syndrome, as well as paroxysmal nocturnal hemoglobinuria. Contraindications include Neisseria meningitides bacteria carrier, active infection, lack of vaccination, severe liver and kidney pathology.
- "Enplate". The main active ingredient is romiplostim. The drug is prescribed for idiopathic thrombocytopenic purpura.
- "Elapraza". The main component is idursulfase. The medicine is prescribed for mucopolysaccharidosis type 2.
- "Trucklear". The main ingredient in the drug is bosentan. It is used for pulmonary hypertension.
- "Enbrel". The drug contains etanercept. It is prescribed for juvenile arthritis with a systemic onset.
- "Let's see." The active ingredient is galsulfase. The tool is used for mucopolysaccharidosis of the fourth type.
- "Actemra". Contains tocilizumab. It is prescribed for juvenile arthritis with a systemic onset.
- "Revoleyd". The active ingredient is eltrombopag. The tool is used for idiopathic thrombocytopenic purpura.
- "Aldazurim". The main substance is laronidase. It is prescribed for mucopolysaccharidosis type 1.
- "Ilaris". The active ingredient is canakinumab. The remedy is used for juvenile arthritis with a systemic onset.
- "Privigen". HisThe active ingredient is human immunoglobulin. The remedy is prescribed for idiopathic thrombocytopenic purpura.
- "Replagal". The active substance is agalsidase alfa. It is prescribed for Fabry disease.
- "Exjade". The active ingredient is deferasirox. The remedy is used for unspecified aplastic anemia.
- "Orfadin". The active substance is nitisinone. It is prescribed for tyrosinemia.
- "Adempas". Contains riociguat. It is prescribed for pulmonary hypertension.
- "Opsumite". Contains macitentan. It is used for pulmonary hypertension.
- "Revation". Contains sildenafil. Prescribed for pulmonary hypertension.
- "Myozyme". The main component is alglucosidase alfa. The drug is used for Pompe disease.
- "Veil". The main active ingredient is miglustat. It is prescribed for sphingolipidoses.
- "Ventavis". Contains iloprost. Prescribed for pulmonary hypertension.
- "Fabrazim". Contains agalsidase beta. It is prescribed for sphingolipidoses.
- "Volibris". The active compound is ambrisentan. Prescribed for pulmonary hypertension.
- "Ilomedin". The active ingredient is iloprost. The drug is used for pulmonary hypertension.
This list of orphan drugs is considered the most popular among those prescribed for diseases.
Contraindications
Drugs are forbidden to take whenincreased 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 permission of the doctor.
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, incentives are provided to companies. Registration is carried out in the same way as for other pharmaceuticals, but with additional confirmation from the relevant commission.
As for patients, it must be remembered that only a doctor should prescribe a medicine. Self-acquisition of drugs is not recommended, as it may not bring the desired result, but only aggravate the course of the disease.
