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.