Additional file 2 of The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis

Additional file 2. Framework for fostering the development, review and approval of medicines for rare and serious life-threatening conditions in the US and in the EU. Definitions: MA = Marketing Authorization, SMEs = small & medium-sized enterprises. At the time of marketing authorization LUM/IVA was withdrawn from the Community Register of designated Orphan Medicinal Products of the EU upon request of the sponsor [25]. In the EU, the designation to accelerated assessment - which shortens the review time from 210 to 150 days - was granted to IVA and LUM/IVA, while the EMA did not agree to the applicant’s request for TEZ/IVA being considered not of major public health interest [69]. The triple combination ELX/TEZ/IVA was initially reviewed under EMA’s accelerated assessment program, but since the applicant requested a 3-month clock stop during assessment - ultimately reduced to 2 months - the conditions for accelerated assessment could no longer be met [35].