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Availability of CFTR modulators in countries of Eastern Europe: The reality in 2022

Published:August 23, 2022DOI:https://doi.org/10.1016/j.jcf.2022.08.014
      The 3rd V4 (i.e., Visegrad) international cystic fibrosis (CF) conference, held in November 2021 in Krakow, provided the opportunity to compare the availability of CFTR modulator (CFTRm) therapies in several Eastern European countries. Our interest in collecting the numbers of patients on CFTRm who live in this geographical region was primarily driven by concerns about the overall standards of CF care in less resourced countries, which if not accurate, have a substantial negative impact on CF health [
      • Bell S.C.
      • Mall M.A.
      • Gutierrez H.
      • Macek M.
      • Madge S.
      • Davies J.C.
      • et al.
      The future of cystic fibrosis care: a global perspective.
      ]. Past surveys [
      • Madge S.
      • Bell S.C.
      • Burgel P.R.
      • De Rijcke K.
      • Blasi F.
      • Elborn J.S.
      • et al.
      Limitations to providing adult cystic fibrosis care in Europe: results of a care center survey.
      ,
      • Walicka-Serzysko K.
      • Peckova M.
      • Noordhoek J.J.
      • Sands D.
      • Drevinek P.
      Insights into the cystic fibrosis care in Eastern Europe: results of survey.
      ] implied that the region of Eastern Europe struggled to achieve standards of CF care as recommended in the European CF Society consensus documents [
      • Castellani C.
      • Duff A.J.A.
      • Bell S.C.
      • Heijerman H.G.M.
      • Munck A.
      • Ratjen F.
      • et al.
      ECFS best practice guidelines: the 2018 revision.
      ,
      • Conway S.
      • Balfour-Lynn I.M.
      • De Rijcke K.
      • Drevinek P.
      • Foweraker J.
      • Havermans T.
      • et al.
      European cystic fibrosis society standards of care: framework for the cystic fibrosis center.
      ]. Findings such as a lack of centralized CF care in some countries, the absence of complete and functional multidisciplinary teams, CF centers not having been recognized by national health care authorities or restricted financial resources for CF care [
      • Walicka-Serzysko K.
      • Peckova M.
      • Noordhoek J.J.
      • Sands D.
      • Drevinek P.
      Insights into the cystic fibrosis care in Eastern Europe: results of survey.
      ] indicate that access to costly CFTRm drugs can be very limited there. This concern was further underlined by the Waiting to Access Innovative Therapies (W.A.I.T.) indicators, which showed that the time from marketing authorization of orphan medicinal products to their actual availability (i.e., reimbursement) for the period of 2017–2020 takes, on average, 636 days for European countries, but this interval is often considerably longer for countries of Eastern Europe (e.g., Bulgaria 787 days, Poland 993 days) [

      EFPIA Patients W.A.I.T. Indicator 2021 Survey 2022. Available from: https://www.efpia.eu/media/636821/efpia-patients-wait-indicator-final.pdf [accessed April 2022].

      ].
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