OBJECTIVES: Health technology assessment (HTA) assessment was introduced within the decision-making reimbursement processes of new molecules in Bulgaria from December 2015. HTA process might have started in Bulgaria if no negative HTA opinion was issued in Germany, France and UK. Two orphan molecules Pirfenidone and Nintedanib - indicated for treatment of Idiopathic Pulmonary Fibrosis (IPF) have been HTA assessed and reimbursed in all four countries.

METHODS: Review of the HTA recommendations of Pirfenidone and Nintedanib in Bulgaria -NCPHA, United Kingdom - NICE, France- HAS, and Germany- IQWIG, where clinical evidence, comparators and pharmaco-economic parameters were evaluated and reimbursement status in the selected countries was analyzed.

RESULTS: In all 4 countries (100%) Pirfenidone and Nintedanib have positive HTA assessment. In 90% of the countries the products are 100% reimbursed and only in France reimbursement is 30%. In 2 countries (UK and Germany) treatment with Pirfenidone is compared with best supportive care only. In UK and Bulgaria for Nintedanib the best supportive care and Pirfenidone have been used as relevant comparators. The added benefit has been evaluated based on the same clinical studies in all countries. Only in Germany according to the local legislation, an added benefit of orphan drugs was deemed as proven by the granted EU marketing authorization.

CONCLUSIONS: The results of the review would contribute to assess the need of clinical review within the HTA process for orphan drugs and will assess the possible benefits in terms of fast patients' access to treatment of rare diseases, where the HTA assessment of the first product helps the evaluation of the second one.