Advertisement

The use of medicines in exceptional circumstances in Spain: adding perspective to early access

  • A. Frutos Pérez-Surio
  • J. R. Lozano Ortíz
Current Opinion
  • 5 Downloads

Abstract

The use of medicines in exceptional circumstances refers to the use of non-authorized medicines or those used outside the authorized conditions, and comprises three different situations: (1) the use of medication in research or for compassionate use; (2) the use of foreign medicines (medicines unauthorized in one country but authorized in others); and (3) off-label use (prescription of drugs for conditions other than that for which they have been officially approved). In Spain, we intend to regulate the period between authorization and pricing through the use of therapeutic positioning reports containing standard criteria. In our opinion, the key to managing this period could be restrictive access based on the therapeutic positioning reports, prespecified agreements on acquisition cost, and restrictive time framework (for use or for decision making, e.g. orphan medicines).

References

  1. 1.
    Antoñanzas F, Terkola R, Overton PM, et al. Defining and measuring the affordability of new medicines: a systematic review. Pharmacoeconomics. 2017;35(8):777–91.CrossRefGoogle Scholar
  2. 2.
    Hatswell AJ, Baio G, Berlin JA, et al. Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999–2014. BMJ Open. 2016;6(6):e011666.CrossRefGoogle Scholar
  3. 3.
    Pace J, Ghinea N, Kerridge I, et al. Demands for access to new therapies: are there alternatives to accelerated access? BMJ. 2017;359:j4494.CrossRefGoogle Scholar
  4. 4.
    Alexander SP, Kelly E, Marrion N, et al. The concise guide to PHARMACOLOGY 2015/16: overview. Br J Pharmacol. 2015;172(24):5729–43.CrossRefGoogle Scholar
  5. 5.
    Wallerstedt SM, Henriksson M. Balancing early access with uncertainties in evidence for drugs authorized by prospective case series: systematic review of reimbursement decisions. Br J Clin Pharmacol. 2018;84(6):1146–55.CrossRefGoogle Scholar
  6. 6.
    Antoñanzas F, Rodríguez-Ibeas R, Juárez-Castelló CA. Personalized medicine and pay for performance: should pharmaceutical firms be fully penalized when treatment fails? Pharmacoeconomics. 2018;36(7):733–43.CrossRefGoogle Scholar
  7. 7.
    Eddy DM, Hollingworth W, Caro JJ, et al. Model transparency and validation: a report of the ISPOR-SMDM Modeling Good Research Practices Task Force-7. Med Decis Mak. 2012;32:733–43.CrossRefGoogle Scholar
  8. 8.
    Battisti WP, Wager E, Baltzer L, et al. Good publication practice for communicating company-sponsored medical research: GPP3. Ann Intern Med. 2015;163(6):461–4.CrossRefGoogle Scholar
  9. 9.
    Sampson CJ, Wrightson T. Model registration: a call to action. Pharmacoecon Open. 2017;1(2):73–7.CrossRefGoogle Scholar
  10. 10.
    Carswell CI, Paladino JA. Meandering down the road to transparency. Pharmacoeconomics. 2008;26(3):179–80.CrossRefGoogle Scholar
  11. 11.
    International Society for Pharmacoeconomics and Outcomes Research. ISPOR roadmaps. Available at: https://tools.ispor.org/htaroadmaps/. Accessed 15 May 2018.
  12. 12.
    National Institute for Health and Care Excellence. What we do. 2018. Available at: https://www.nice.org.uk/about/what-we-do. Accessed 15 May 2018.
  13. 13.
    French National Agency for Medicines and Health Products Safety (ANSM). Notice to applicants for marketing for temporary authorisation for use (ATU). 2015. Available at: http://ansm.sante.fr/var/ansm_site/storage/original/application/cadfbcf9594614d59c8915670853a28b.pdf. Accessed 15 May 2018.
  14. 14.
    The Federal Joint Committee. About us. Available at: http://www.english.g-ba.de. Accessed 15 May 2018.
  15. 15.
    Institute for Quality and Efficiency in Health Care. Responsibilities and objectives of IQWiG. Available at: https://www.iqwig.de/en/about-us/responsibilities-and-objectivesof-iqwig.2946.html. Accessed 15 May 2018.
  16. 16.
    German Institute for Medicinal Documentation and Information. HTA information system. Available at: https://www.dimdi.de/dynamic/en/further-services/health-technology-assessment/. Accessed 15 May 2018.
  17. 17.
    Care Institute of the Netherlands. Organisation. Available at: https://www.zorginstituutnederland.nl/over-ons/organisatie. Accessed 15 May 2018.
  18. 18.
    Agency for Health Technology Assessment. About us. Available at: http://www.aotm.gov.pl/www/. Accessed 15 May 2018.
  19. 19.
    Swedish Agency for Health Technology Assessment and Assessment of Social Services. Home page. Available at: http://www.sbu.se/en/. Accessed 15 May 2018.
  20. 20.
    European Medicines Agency. PRIME: priority medicines (2017). Available at: https://www.ema.europa.eu/human-regulatory/research-development/prime-priority-medicines. Accessed 15 May 2018.
  21. 21.
    European Medicines Agency: PRIME: a two-year overview (2018). Available at: https://www.ema.europa.eu/documents/report/prime-two-year-overview_en.pdf. Accessed 15 May 2018.
  22. 22.
    UK Government. Accelerated access review: final report (2016). Available at: https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/565072/AAR_final.pdf. Accessed 15 May 2018.

Copyright information

© Springer Nature Switzerland AG 2018

Authors and Affiliations

  1. 1.Department of Hospital PharmacyUniversity Clinical Hospital Lozano BlesaZaragozaSpain
  2. 2.Department of Microbiology, Preventive Medicine and Public HealthUniversity of ZaragozaZaragozaSpain
  3. 3.Department of Hospital PharmacyHospital Real Nuestra Señora de GraciaZaragozaSpain

Personalised recommendations