OBJECTIVES: Health technology assessment (HTA) as multidisciplinary process of evaluation of the social, economic, organizational and ethical issues of a health technology is used within the decision-making processes concerning reimbursement of new molecules. It is also applied for orphan drugs in Germany, France, UK and Bulgaria. 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%) marketing authorization (MA) holder applied for HTA assessment. The added benefit has been evaluated based on the same clinical studies in all countries and same comparators have been used. Only in Germany according to the local legislation, an added benefit of orphan drugs was deemed as proven by the granted EU MA. Patients have access to the treatment 1 to 5 years after granting the MA by European Commission.

CONCLUSIONS: The results of the review show that patient access to treatment options depends on the MAH despite products’ Orphan designation. Also, the results of the review may help to reassess the need of clinical review within the HTA process for orphan drugs in each EU country. This is in line with the proposals for new EU HTA regulation – no more duplication of work for health authorities and industries, and fast patients' access to treatments for rare diseases.