The regulatory and Health Technology processes in Europe and drug market access. The case of cystic fibrosis

The regulatory and Health Technology processes in Europe and drug market access. The case of cystic fibrosis

Authors

  • Mayra Latorre Martinez Universidad Nacional de Educación a Distancia (UNED)
  • Pedro Serrano Servicio de Evaluación, Servicio Canario de Salud Red de Investigación en Servicios Sanitarios y Enfermedades Crónicas (REDISSEC)
  • Antonio Sarria Santamera Red de Investigación en Servicios Sanitarios y Enfermedades Crónicas (REDISSEC) Agencia de Evaluación de Tecnologías Sanitarias, Instituto de Salud Carlos III Universidad de Alcalá

DOI:

https://doi.org/10.7175/fe.v16i4.1207

Keywords:

Cystic Fibrosis, Agencies/organization & administration, Health Technology Assessment

Abstract

In order to reach the European market, a new drug needs to receive a positive evaluation regarding its quality, safety and efficacy by regulatory health authorities and also obtain a positive HTA appraisal regarding its cost-effectiveness by HTA bodies. Regulators and HTA bodies are collaborating in several projects at European level in order to harmonize the scientific requirements of both evaluations to the maximum extent possible. The comparison of the regulatory evaluation performed by EMA for Kalydeco and the HTA appraisals issued by several EU bodies exemplifies the dilemma between scientific evidence and local economic considerations and the difficulties in the achievement of harmonization and therefore equity in the access to drugs.

References

EU Pharmaceutical informations. Eudralex Volume 1. Pharmaceutical Legislation Medicinal Products for Human Use. Available at: http://ec.europa.eu/health/documents/eudralex/vol-1/index_en.htm (last accessed October 2015)

European Network of Health Technology Assessment (EunetHTA). Available at: http://www.eunethta.eu/ (last accessed October 2015)

European Network of Health Technology Assessment (EunetHTA). HTA Core Model. Available at: http://www.eunethta.eu/hta-core-model (last accessed October 2015)

Berntgen M, Gourvil A, Pavlovic M, et al. Improving the Contribution of Regulatory Assessment Reports to Health Technology Assessments—A Collaboration between the European Medicines Agency and the European network for Health Technology Assessment. Value Health 2014; 17: 634-41; http://dx.doi.org/10.1016/j.jval.2014.04.006

Directive 2011/24/EU of the European Parliament and of the Council of 9 March 2011 on the application of patients’ rights in cross-border healthcare. Available at: http://eur-lex.europa.eu/homepage.html (last accessed October 2015)

European Commission. Register of designated orphan medicinal products. Available at: http://ec.europa.eu/health/documents/community-register/html/alforphreg.htm (last accessed October 2015)

European Medicines Agency. European public assessment report (EPAR) for Kalydeco. Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/epar_search.jsp&mid=WC0b01ac058001d124 (last accessed October 2015)

Cohen D, Raftery J. Paying twice: question over high cost of cystic fibrosis drug developed with charitable funding. BMJ 2014; 348: 1445.

Kalydeco (ivacaftor). European Public Assessment Report. Available at: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Summary_for_the_public/human/002494/WC500130744.pdf (last accessed October 2015)

Scottish Medicines Consortium (SMC): Ivacaftor 150mg film-coated tablets (Kalydeco). SMC Nº (827/12). Available at: https://www.scottishmedicines.org.uk/files/advice/ivacaftor_Kalydeco_FINAL_December_2012_amended_11_01_13_for_website.pdf (last accessed October 2015)

NHS Commission Board. Clinical Commissioning Policy: Ivacaftor for cystic fibrosis. Available at: http://www.england.nhs.uk/wp-content/uploads/2013/04/a01-p-b.pdf (last accessed October 2015)

Agencia Española de Medicamentos y Productos Sanitarios. Informe de Posicionamiento Terapéutico de Ivacaftor (Kalydeco). Available at: http://www.aemps.gob.es/medicamentosUsoHumano/informesPublicos/docs/IPT-ivakaftor-kalydeco-aprobado-GCPT.pdf (last accessed October 2015)

Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWIG)- Ivacaftor (neues Anwendungsgebiet) – Bewertung gemäß. Available at: https://www.iqwig.de/download/G14-09_Ivacaftor-neues-Anwendungsgebiet_Bewertung-35a-Abs1-Satz10-SGB-V.pdf (last accessed October 2015)

National Centre for Pharmacoeconomics (NCPE). Ireland. Cost-effectiveness of Ivacaftor (Kalydeco) for the treatment of cystic fibrosis in patients aged 6 years an older who have the G551D mutation. Available at: http://www.ncpe.ie/wp-content/uploads/2012/08/Ivacaftor-Summary.pdf (last accessed October 2015)

Haute Autorité Santé. Transparency Committee. Kalydeco 150 mg film-coated tablets. Available from: http://www.has-sante.fr/portail/upload/docs/application/pdf/2013-05/kalydeco_ct_12474.pdf (last accessed October 2015)

The Canadian Drug Expert Committee. Final CDEC Recommendation. Ivacaftor. Available from: http://www.cysticfibrosis.ca/wp-content/uploads/2013/09/CADTHKalydeco_March-25-13.pdf (last accessed October 2015)

Australian Government. Department of health. Public Summary Document. Ivacaftor. PBAC Meeting. Available at : http://www.pbs.gov.au/industry/listing/elements/pbac-meetings/psd/2014-03/ivacaftor-psd-03-2014.pdf (last accessed October 2015)

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Published

2015-12-23

How to Cite

Latorre Martinez, M., Serrano, P., & Sarria Santamera, A. (2015). The regulatory and Health Technology processes in Europe and drug market access. The case of cystic fibrosis. Farmeconomia. Health Economics and Therapeutic Pathways, 16(4), 103–110. https://doi.org/10.7175/fe.v16i4.1207

Issue

Vol. 16 No. 4 (2015)

Section

Original research

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