Drugs

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Outcomes, Access, and Cost Issues Involving PCSK9 Inhibitors to Lower LDL-Cholesterol

Current Opinion

Abstract

The clinical importance and benefit of the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors appears well established for the high-risk cardiovascular (CV) patient. This applies especially to the statin-intolerant patient or the patient who does not attain an appropriate low-density lipoprotein cholesterol (LDL-C) target. Therefore, the barriers to appropriate clinical use of the PCSK9 inhibitors involve cost and not the documented CV benefit or LDL-C lowering. Multiple roadblocks affect many high-risk CV patients in arranging approval of a PCSK9 inhibitor. Overcoming these roadblocks may require legal pressures, some increased regulation, and facilitation by competitive forces.

Notes

Acknowledgements

The author thanks Colleen McMullen, MA, MBA, for her excellent editorial critique.

Compliance with Ethical Standards

Conflict of interest

The author confirms that he has no conflict of interest involving any pharmaceutical or medical device company or any other possible conflict.

References

  1. 1.
    Whayne TF Jr. Is there an ideal low-density lipoprotein cholesterol level? Confusion regarding lipid guidelines, low-density lipoprotein cholesterol targets, and medical management. Int J Angiol. 2017;26(2):73–7.CrossRefPubMedGoogle Scholar
  2. 2.
    Whayne TF. Low-density lipoprotein cholesterol (LDL-C): how low? Curr Vasc Pharmacol. 2017;15:374–9.CrossRefPubMedGoogle Scholar
  3. 3.
    Klose G. Introduction of PCSK9 inhibitors : new perspectives in treatment and practical implementation. Herz. 2016;41(4):307–12.CrossRefPubMedGoogle Scholar
  4. 4.
    McDonagh M, Peterson K, Holzhammer B, Fazio S. A systematic review of PCSK9 inhibitors alirocumab and evolocumab. J Manag Care Spec Pharm. 2016;22(6):641–53.CrossRefPubMedGoogle Scholar
  5. 5.
    Bays H, Gaudet D, Weiss R, Ruiz JL, Watts GF, Gouni-Berthold I, et al. Alirocumab as add-on to atorvastatin versus other lipid treatment strategies: ODYSSEY OPTIONS I randomized trial. J Clin Endocrinol Metab. 2015;100(8):3140–8.CrossRefPubMedPubMedCentralGoogle Scholar
  6. 6.
    Ray KK, Ginsberg HN, Davidson MH, Pordy R, Bessac L, Minini P, et al. Reductions in atherogenic lipids and major cardiovascular events: a pooled analysis of 10 ODYSSEY trials comparing alirocumab with control. Circulation. 2016;134(24):1931–43.CrossRefPubMedPubMedCentralGoogle Scholar
  7. 7.
    Sabatine MS, Giugliano RP, Keech AC, Honarpour N, Wiviott SD, Murphy SA, et al. Evolocumab and clinical outcomes in patients with cardiovascular disease. N Engl J Med. 2017;376(18):1713–22.CrossRefPubMedGoogle Scholar
  8. 8.
    FDA approves Amgen’s Repatha (evolocumab) to prevent heart attack and stroke. 2017. http://www.amgen.com/media/news-releases/2017/12/fda-approves-amgens-repatha-evolocumab-to-prevent-heart-attack-and-stroke/. Accessed 18 Dec 2017.
  9. 9.
    Bekkering GE, Kleijnen J. Procedures and methods of benefit assessments for medicines in Germany. Eur J Health Econ. 2008;9(Suppl 1):5–29.CrossRefPubMedGoogle Scholar
  10. 10.
    Kreis J, Busse R. From evidence assessments to coverage decisions?: the case example of glinides in Germany. Health Policy. 2012;104(1):27–31.CrossRefPubMedGoogle Scholar
  11. 11.
    Ioannides-Demos LL, Ibrahim JE, McNeil JJ. Reference-based pricing schemes: effect on pharmaceutical expenditure, resource utilisation and health outcomes. Pharmacoeconomics. 2002;20(9):577–91.CrossRefPubMedGoogle Scholar
  12. 12.
    Breckenridge A. Development and delivery of clinical pharmacology in regulatory agencies. Br J Clin Pharmacol. 2012;73(6):866–9.CrossRefPubMedPubMedCentralGoogle Scholar
  13. 13.
    Kumar B, Baldi A. The challenge of counterfeit drugs: a comprehensive review on prevalence, detection and preventive measures. Curr Drug Saf. 2016;11(2):112–20.CrossRefPubMedGoogle Scholar
  14. 14.
    Kuehn BM. Coverage and cardioprotective benefits of PCSK9 take center stage at the American College of Cardiology meeting. Circulation. 2017;135(25):2562–3.CrossRefPubMedGoogle Scholar
  15. 15.
    Caffrey M. Rival PCSK9 makers cut prices to get approval from UK health service. AJMC.com Managed Markets Network. 2016. http://www.ajmc.com/newsroom/rival-pcsk9-makers-cut-prices-to-get-approval-from-uk-health-service. Accessed 3 July 2017.
  16. 16.
    Elgin B, Langreth R. How big pharma uses charity programs to cover for drug price hikes. Bloomberg Business Week. 2016. https://www.bloomberg.com/news/articles/2016-05-19/the-real-reason-big-pharma-wants-to-help-pay-for-your-prescription. Accessed 3 Jul 2017.
  17. 17.
    Nunan M, Duke T. Effectiveness of pharmacy interventions in improving availability of essential medicines at the primary healthcare level. Trop Med Int Health. 2011;16(5):647–58.CrossRefPubMedGoogle Scholar
  18. 18.
    Mekonnen AB, McLachlan AJ, Brien JA. Pharmacy-led medication reconciliation programmes at hospital transitions: a systematic review and meta-analysis. J Clin Pharm Ther. 2016;41(2):128–44.CrossRefPubMedGoogle Scholar
  19. 19.
    Alliance for Patient Access. 2017. https://www.guidestar.org/profile/20-5130312. 13 Oct 2017.
  20. 20.
    Hillman BJ, Frank RA, Rodriguez GM, I Medical, Technology Alliance Workshop p. New pathways to medicare coverage for innovative PET radiopharmaceuticals: report of a medical imaging & technology alliance (MITA) workshop. J Nucl Med. 2012;53(2):336–42.CrossRefPubMedGoogle Scholar
  21. 21.
    Hillman BJ, Frank RA, Rodriguez GM, Medical I, Technology Alliance Workshop P. New pathways to medicare coverage for innovative PET radiopharmaceuticals: report of a Medical Imaging & Technology Alliance (MITA) workshop. J Am Coll Radiol. 2012;9(2):108–14.CrossRefPubMedGoogle Scholar
  22. 22.
    Lobban TC, Camm AJ. Patient associations as stakeholders: a valuable partner for facilitating access to therapy. Europace. 2011;13(Suppl 2):ii21–4.PubMedGoogle Scholar
  23. 23.
    McCormick D, Sayah A, Lokko H, Woolhandler S, Nardin R. Access to care after Massachusetts’ health care reform: a safety net hospital patient survey. J Gen Intern Med. 2012;27(11):1548–54.CrossRefPubMedPubMedCentralGoogle Scholar
  24. 24.
    Fay M, Malley J. HSA growth and the associated opportunities/challenges presented by pharmacy benefits. Benefits Q. 2014;30(3):8–15.PubMedGoogle Scholar
  25. 25.
    Dong X, Fetterolf D. Specialty pharmacy: an emerging area of interest for medical management. Dis Manag. 2005;8(2):73–85.CrossRefPubMedGoogle Scholar
  26. 26.
    Patterson CJ. Best practices in specialty pharmacy management. J Manag Care Pharm. 2013;19(1):42–8.PubMedGoogle Scholar
  27. 27.
    Kesselheim AS, Tan YT, Darrow JJ, Avorn J. Existing FDA pathways have potential to ensure early access to, and appropriate use of, specialty drugs. Health Aff (Millwood). 2014;33(10):1770–8.CrossRefPubMedGoogle Scholar
  28. 28.
    Penington R, Stubbings JA. Evaluation of specialty drug price trends using data from retrospective pharmacy sales transactions. J Manag Care Spec Pharm. 2016;22(9):1010–7.CrossRefPubMedGoogle Scholar
  29. 29.
    Lotvin AM, Shrank WH, Singh SC, Falit BP, Brennan TA. Specialty medications: traditional and novel tools can address rising spending on these costly drugs. Health Aff (Millwood). 2014;33(10):1736–44.CrossRefPubMedGoogle Scholar
  30. 30.
    Kirschenbaum BE. Specialty pharmacies and other restricted drug distribution systems: financial and safety considerations for patients and health-system pharmacists. Am J Health Syst Pharm. 2009;66(24 Suppl 7):S13–20.CrossRefPubMedGoogle Scholar
  31. 31.
    Patel BN, Audet PR. A review of approaches for the management of specialty pharmaceuticals in the United States. Pharmacoeconomics. 2014;32(11):1105–14.CrossRefPubMedPubMedCentralGoogle Scholar
  32. 32.
    Gleason PP, Alexander GC, Starner CI, Ritter ST, Van Houten HK, Gunderson BW, et al. Health plan utilization and costs of specialty drugs within 4 chronic conditions. J Manag Care Pharm. 2013;19(7):542–8.PubMedGoogle Scholar
  33. 33.
    Starner CI, Alexander GC, Bowen K, Qiu Y, Wickersham PJ, Gleason PP. Specialty drug coupons lower out-of-pocket costs and may improve adherence at the risk of increasing premiums. Health Aff (Millwood). 2014;33(10):1761–9.CrossRefPubMedGoogle Scholar
  34. 34.
    Baldini CG, Culley EJ. Estimated cost savings associated with the transfer of office-administered specialty pharmaceuticals to a specialty pharmacy provider in a medical injectable drug program. J Manag Care Pharm. 2011;17(1):51–9.PubMedGoogle Scholar
  35. 35.
    Tu HT, Samuel DR. Limited options to manage specialty drug spending. Res Brief. 2012;22:1–13.Google Scholar
  36. 36.
    Fitzgerald K, White S, Borodovsky A, Bettencourt BR, Strahs A, Clausen V, et al. A highly durable RNAi therapeutic inhibitor of PCSK9. N Engl J Med. 2017;376(1):41–51.CrossRefPubMedGoogle Scholar
  37. 37.
    Gomes VS, Amador TA. Studies published in indexed journals on lawsuits for medicines in Brazil: a systematic review. Cad Saude Publica. 2015;31(3):451–62.CrossRefPubMedGoogle Scholar
  38. 38.
    Pettignano R, Caley SB, McLaren S. The health law partnership: adding a lawyer to the health care team reduces system costs and improves provider satisfaction. J Public Health Manag Pract. 2012;18(4):E1–3.CrossRefPubMedGoogle Scholar

Copyright information

© Springer International Publishing AG, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Gill Heart and Vascular InstituteUniversity of KentuckyLexingtonUSA

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