Orphan Drugs in Surgery

Introduction. Rare diseases, also called orphan diseases, are life-threatening or chronically debilitating conditions of different origin. Majority of them are genetic disorders, others being rare cancers, congenital malformations, autoimmune, toxic and infectious diseases. Rare conditions may also be related to surgery, e.g. acute sensorineural hearing loss after surgery induced acoustic trauma, scarring post glaucoma filtration surgery, and short bowel syndrome following intestinal surgery. Besides, surgery as a specific area for orphan drugs, has not been studied yet in Latvia.

Aim of the study. This study aims to determine orphan drugs associated with surgery (used pre-, during or post-surgery) and their availability and access in Latvia.

Materials and methods. European register of designated orphan medicinal products and EMA approved Summary of Product Characteristics were analyzed, to find orphan drugs with approved labeled indications related to surgery. Drug availability and access in Latvia were determined, by using data available from State Agency of Medicines of Latvia and National Health Service. A literature review was performed to compare Latvian situation in field of orphan medicines with other European countries.

Results. 15 orphan drugs were identified, 8 of them (53.3%) indicated for different kinds of tumors. 6 drugs (40%) are available in Latvia, including one drug (6.7%) included in the reimbursement list.

Conclusions. Oncology is the biggest therapeutic area of orphan drugs. Majority of orphan drugs are not available in Latvia, moreover those drugs that are available are often not accessible.