15
Views
2
CrossRef citations to date
0
Altmetric
Review

Societal implications of the pharmacoeconomics of α1-antitrypsin deficiency

, &
Pages 243-249 | Published online: 09 Jan 2014

References

  • Perlmutter DH. Clinical manifestations of alpharantitrypsin deficiency. Pediatric Gastroenterology Part II 24(1), 27–43 (1995).
  • ••Good overview of clinical aspects ofAATdeficiency.
  • Seersholm N, Wencker M, Banik N et al Does alpha l-antitrypsin augmentation therapy slow the annual decline in FEV1 in patients with severe hereditary alphal-antitrypsin deficiency? Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (VVATL) alphal -AT study group. Eur. Respir. 10(10), 2260–2263 (1997).
  • •Addresses the impact of FEV1 values.
  • Dirksen A, Dijkman JH, Madsen F et al A randomized clinical trial of alpha(1)-antitrypsin augmentation therapy. Am.Respir. Crit. Care Med. 160,1468-1472 (1999).
  • Stockley RA. Alpha-1-antitrypsin deficiency: what next? Thorax 55 (7), 614–618 (2000).
  • Stoller JK. Augmentation therapy for severe alpha 1-antitrypsin deficiency: is the jury still out on a trial? Thorax 53(12), 1007–1009 (1998).
  • McElvaney NG, Stoller JK, Buist AS et al Baseline characteristics of enrollees in the National Heart, Lung and Blood Institute Registry of alpha 1-antitrypsin deficiency. Chest. 111 (2), 394–403 (1997).
  • NIH-Registry. Survival and FEV1 decline in individuals with severe deficiency of alphal-antitrypsin. The Alpha-l-Antitrypsin Deficiency Registry Study Group. Am. .1. Respir. Crit. Care Med. 158,49-59 (1998).
  • Rooney C, Taggert C, O'Neill SJ et al. Alpha 1-antitrypsin deficiency. Irish Med. .1. 92(1), 227–228 (1999).
  • Banasik J. Diagnosing alphal-antitrypsin deficiency. The Nurse Practitioner 26(1), 58–67 (2001).
  • Wall M, Moe E, Eisenberg J et al. Long- term follow-up of a cohort of children with alpha-l-antitrypsin deficiency. .1. Pediatrics 116(2), 248–251 (1990).
  • Hubbard RC, Crystal RG. Strategies for aerosol therapy of al-antitrypsin deficiency by the aerosol route. Lung 168 (Suppl.), 565–578 (1990).
  • Hay JVV, Robin ED. Cost-effectiveness of Al replacement therapy in treatment of congenital chronic obstructive pulmonary disease. Am.j Public Health 81, 427–433 (1991).
  • ••First published cost-effectiveness analysis of AAT deficiency replacement therapy.
  • Alkins SA, O'Malley P Should healthcare systems pay for replacement therapy in patients with Al deficiency? A critical review and cost-effectiveness analysis. Chest 117, 875–880 (2000).
  • ••Describes the policy debate regarding reimbursement for replacement therapy.
  • Mullins CD, Huang X, Merchant S, Stoller JK. The Direct Medical Costs of Alpha 1-Antitrypsin Deficiency. Chest. 119(3), 745–752 (2001).
  • ••First report of burden of illness of AATdeficiency.
  • Horowitz, ID. Alpha sub 1-proteinase inhibitor (human) shortage. Letter to the editor. Chest 110(6), 1624 (1996).
  • Lutes, R. Alpha subl-proteinase inhibitor (human) shortage. Letter to the editor. Chest 110(6), 1165 (1996).
  • Anonymous. Bayer Direct: New distribution program creates unanswered questions. Infusion 6, 16–21 (1999).
  • Alpha-1 Bulletin. 1(1), May (2001).
  • US Congress Office of Technology Assessment. Genetic Tests and Health Insurance: Results of a Survey. Office of Technology Assessment, Washington, DC, USA (1992).
  • Wulfsberg EA, Hoffman DE, Cohen MM. al-Antitrypsin deficiency: impact of genetic discovery on medicine and society. JAMA 271(3), 217–222 (1994).
  • •Discussion on the importance of genetic aspects of treatment.
  • US Department of Health and Human Services Healthcare Financing Administration publication no. HCFA - 02110 revised February 2001.
  • Stoller JK, Spray J, Smith P Demographics and impact of alpha 1-antitrypsin deficiency: results of a mail survey. Am. Rev Respir Dis. 147, A871 (1993)
  • US Gov. Printing Office. Federal Register 66(213), Friday, November 2 (2001).
  • US Gov. Printing Office. Federal Register 66(231), Friday, November 30 (2001).
  • US Gov. Printing Office. Federal Register 66(250), Monday, December 31(2001).
  • Personal Conversation with Miriam O'Day, Senior Director of Public Policy for Alpha - 1 Foundation, February 13 (2002).
  • Kropp J, Wencker M, Hotze A et al. Inhalation of [1231] al-protease inhibitor: toward a new therapeutic concept of al-protease inhibitor deficiency. J. Nuclear Med. 42(5), 744–751 (2001).
  • •• Describes potential for aerosol augmentation therapy.
  • Hubbard RC, McElvaney NG, Sellers SE et al. Recombinant DNA-produced a 1-antitrypsin administered by aerosol augments lower respiratory tract antineutrophil elastase defenses in individuals with al-antitrypsin deficiency. Clin. Invest. 84,1349-1354 (1989).
  • Hubbard RC, Brantly ML, Sellers SE et al. Antineutrophil-elastase defenses of the lower respiratory tract in al-antitrypsin deficiency directly augmented with an aerosol of al-antitrypsin. Ann. Intern. Med 111,202–212 (1989).
  • Hubbard RC, Casolaro MA, Mitchell M et al. Fate of aerosolized recombinant DNA-produced al-antitrypsin: use of the epithelial surface of the lower respiratory tract to administer proteins of therapeutic important. Proc. Nati Acad. Sc1 USA 86, 680–684 (1989).
  • Vogelmeier C, Kirlath I, Warrington S et al. The intrapulmonary half-life and safety of aerosolized al-protease inhibitor in normal volunteers. Am. J. Respir. Crit. Care Med. 155, 536–541 (1997).
  • Mason J, Drummond M. Biotechnology: a special case for health technology assessment? Health Policy 41, 73–81(1997).

Websites

  • Bureau of Labor Service. Consumer price index for all urban consumers (CPI-U): US city average, by medical category. Available at http://146.142.4.24/cgi-bin/surveymost?cu. (Accessed on March 18, 2002).
  • Statement to Healthcare Providers Regarding Augmentation Therapy during Reduced Availability of Prolastin®. Available at www.alphaone.org/news/ announcements/ statement_to_providers_re_prolastin.html (Accessed March 21, 2002).
  • Bayer Direct Frequently Asked Questions. Available at www.bayerdirect.com/ pro_faq.htm (Accessed February 28, 2002).
  • Prescription Drug Assistance Programs. Available at www.medicare.gov./ Prescription/Home.asp (Accessed February 28, 2002).
  • The Alpha-1 Foundation contacts all members of Congress urging them to take action opposing change in Alpha-1 Medicare benefits. Available at www.alphaone.org/publicpolicy/ regulatory%20issues/ opposing_statement.htm (Accessed March 21, 2002).
  • Our mission. Available at: www.alphal.org/ (accessed on February 20, 2002, last updated on February 18, 2002).
  • Advocacy the Alpha-1 Way A Vital Mission of the Alpha-1 Association. Available at www.alphal.org/advocacy/ advocacy_statement.htm (Accessed March 21,2002).

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.