Out-of-pocket healthcare expenditures of Turkish households living with rare diseases.

Introduction: This study aims to determine the out-of-pocket health expenditures of households in Turkey where individuals with rare diseases are residing. Methods: The research population consisted registered members of associations who are members of the Rare Diseases Network. In addition to the general analysis including all participants, expenditures based on characteristics of disease holders were also calculated. Results: A total of 439 participants were included in the analysis. We determined that special nutrition was the highest expenditure group and emergency departments were the lowest expenditure group. When all the participants were evaluated, the average cost of rare diseases was found to be L22,743 (€2,877). A significant relationship was found between income status and out-of-pocket health expenditures (p = 0.012). Discussion: Policy makers should consider inclusion of special nutritional products and medical/non-medical devices used in treatment of rare diseases within the scope of reimbursement and the development of orphan drug legislation as the first actions to be taken.

Cost-Effectiveness Analysis of the Triclip™ Transcatheter Tricuspid Valve Repair System in Patients with Tricuspid Regurgitation.

BACKGROUND: Tricuspid regurgitation is a condition that affects 1.6 million patients in the United States and is independently associated with morbidity and mortality. The TriClip™ procedure repairs the tricuspid valve without the need for open-heart surgery. The aim of this study is to evaluate the cost-effectiveness of TriClip™ treatment in patients with advanced tricuspid regurgitation from the Turkish reimbursement agency perspective. METHODS: Within the scope of this study, the general literature was searched in order to reach data on tricuspid regurgitation. The utilization of health care services used in the expert panel was re-calculated with the current reimbursement costs to determine the cost of heart failure in Turkey. In this study, Markov analysis, Tornado analysis, cost-effectiveness analysis, and partitioned survival analysis have been performed to determine whether TriClip™ is an effective treatment method compared to medication treatment. RESULTS: In according to calculations, 5-year survival rate was found as 49.91% for medication treatment and 57.64% for TriClip™ treatment. According to the analysis performed, the cost of medication treatment was calculated as €3879.72 and TriClip™ Transcatheter Tricuspid Valve Repair System treatment as €25 661.15 for a 60-month period in patients with tricuspid regurgitation and New York Heart Association III-IV. In the calculation, it was found that TriClip™ treatment gave patients an average of 1.64 life years and it was found to be cost-effective compared to medication treatment. CONCLUSIONS: Considering the positive effect of TriClip™ treatment on patients with tricuspid regurgitation in terms of mortality and regression of the heart failure stage, as recommended in the guidelines, widespread of its use has great importance.

Analysis of patient access to orphan drugs in Turkey.

BACKGROUND: Rare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases. This study aims to analyze the accessibility, reimbursement status, licensed status, and Anatomical Therapeutic Chemical (ATC) codes of drugs that the European Medicines Agency (EMA) in Turkey considers to be "orphan" pharmaceuticals. METHODS: The drugs included in this analysis were obtained from the list of orphan drugs published by the EMA. Orphan drugs' accessibility and licensing status in Turkey were obtained from the Health Implementation Communiqué published by the Social Security Institution (SGK) and the List of Abroad Active Substance and List of Licensed Products published by the Turkey Pharmaceuticals and Medical Devices Agency (TITCK). Descriptive analysis was applied to determine the accessibility status of orphan drugs identified by the EMA in Turkey. RESULTS: Based on the EMA, 105 pharmaceuticals were approved with "orphan drug" status except for drugs that have lost orphan drug status, decommissioned in the European Union and withdrawn from the European Community Register by January 2020. Of the 105 rare drugs on the EMA list, 34 were inaccessible in Turkey. Of the 71 available drugs, 23 (32%) were licensed and 48 (68%) were unlicensed in Turkey. 17 (74%) of licensed products and 17 (35%) of unlicensed products were covered by reimbursement. When orphan drugs' ATC codes were examined, the most common ATC group was found to be "L-Antineoplastic and Immunomodulatory" agents. CONCLUSION: An orphan drug incentive policy is very important to ensure early access to the drugs used to treat rare diseases. Considering the capacity and prices for orphan drugs in Turkey, it can be said that many patients with rare diseases have difficulty in their treatment. It is obvious that such a policy must prepare for the regulation of orphan drugs in Turkey.