Legal Routes for Researching an Orphan Product
The research and market admission of orphan drugs — medicines designed for rare diseases — are regulated in Georgia by the general norms of the Law on Drugs and Pharmaceutical Activities. For a product calculated for small populations this means that the organiser chooses the most suitable of three possible routes: conducting a clinical trial in Georgia, recognising a product already registered abroad, or the full national registration procedure. Articles 5¹, 11⁷ and 11¹¹ of the law govern precisely these three routes, and knowing them accurately saves both time and resources.
One general principle also governs the choice of research: in Georgia, preclinical and clinical research into pharmacological agents is conducted in accordance with the standards and guidelines approved by the Minister, and the rights of patients and healthy volunteers participating in the research are protected by the legislation of Georgia. For an orphan product these standards carry particular weight, since the circle of participants is narrow and every datum carries decisive significance.
The Condition for Researching an Advanced Therapy Product
Medicines for rare diseases are often advanced therapy products. The law sets a separate condition for this case: a clinical trial of a pharmacological agent intended as an advanced therapy medicinal product is permissible if the agent is manufactured under a standard of good manufacturing practice recognised by the Government of Georgia, at an enterprise holding the authorisation to manufacture biological medicinal products.
In practical terms this means that before the trial begins the sponsor must verify the enterprise's authorisation to manufacture biological medicinal products and confirm that the manufacturing standard applied is on the list recognised by the Government. In addition, the sponsor — the person, company, institution or organisation responsible for the initiation, management or funding of the research — must, before the start of the trial, secure insurance of the research risk for its duration, reimbursing or compensating the participants' treatment costs.
Recognition of a Product Registered Abroad
When an orphan product is already registered in another country or by an international regulatory body, its admission to the Georgian market may use the recognition regime of state registration established by Article 11⁷ of the law. This regime applies to a product admitted by such a body to the relevant market; the Government establishes the list of recognised bodies, and the interested person may be any person. Advanced therapy medicinal products may be admitted to the Georgian market precisely under the recognition regime.
The advantage of this route is speed: the Agency carries out the administrative expertise of the homologous identification documentation and places the product information in the official registry within seven working days. The documentation includes an authorised Georgian translation of the instruction, the product's form, dosage and labelling sample, the period of admission and unique number, the certificate, and the document confirming manufacture under a good manufacturing practice standard. The period of admission under this regime follows the corresponding foreign market's period, but may not exceed five years.
The Full National Registration Procedure
If the orphan product is registered for Georgia under the national regime, the full procedure established by Article 11¹¹ of the law applies. The interested person — the product's manufacturer or the holder of the trading licence — submits to the Agency an application with accompanying documents. The registration dossier consists of administrative and scientific-technical parts; the administrative part is submitted in Georgian, and the scientific-technical part in Georgian, Russian or English.
The Agency checks the compliance of the submitted documents no later than fourteen days — this is the administrative expertise. After a positive conclusion the documents undergo scientific-technical expertise in order to establish the product's standardisation, quality, safety and therapeutic efficacy. To eliminate defects the interested person is additionally granted a period of up to two months. The decision on registration or refusal in first-time registration is taken within a period of three months, and the document confirming the right of admission to the market is issued within ten days of the issuance of the administrative act.
A product registered under the national regime may circulate on the territory of Georgia for five years from its registration, and after expiry — until its shelf life runs out. For re-registration the documents must be submitted no later than two months before expiry; otherwise registration proceeds under the first-time registration regime. These periods must be factored in advance when planning the supply of an orphan product.
Frequently Asked Questions
On what condition is a clinical trial of an advanced therapy product conducted?
The trial is permissible if the pharmacological agent is manufactured under a good manufacturing practice standard recognised by the Government, at an enterprise holding the authorisation to manufacture biological medicinal products, and if the sponsor has secured insurance for the duration of the research.
How long does registration under the recognition regime take?
The Agency examines the homologous identification documentation and places the information in the official registry within seven working days. The admission period under this regime does not exceed five years.
What periods does the national registration procedure have?
The administrative expertise is carried out no later than fourteen days; up to two months are granted to eliminate defects; the decision in first-time registration is taken within three months; and the confirming document is issued within ten days.
What happens after the registration period expires?
The product may circulate for five years from registration; after expiry, the sale of the product already in circulation is possible until its shelf life runs out. Documents for re-registration must be submitted no later than two months before expiry.
How We Help on Legal.ge
Orphan drug trials and market admission have several alternative routes, and the right choice depends on the product's status and the urgency of supply. On Legal.ge we help verify the conditions for research, compare the recognition and national regimes and prepare the documentation. Contact us for a consultation — we will analyse your circumstances and point to the shortest legal route.