nach oben

Journal of Public Health

Erschienen in:

01.06.2014 | Original Article

Pharmacoeconomics and clinical trials in multiple sclerosis: baseline data from the European Union

verfasst von: Roger Lee Mendoza

Erschienen in: Journal of Public Health | Ausgabe 3/2014

Einloggen, um Zugang zu erhalten

Abstract

Aim

One of the contemporary debates between science and economics is concerned with why, and to what extent, financial cost and economic efficiency considerations affect scientific inquiry into possible solutions to human illnesses. This study inquires into clinical drug trials for multiple sclerosis (MS) in the European Union to determine the validity of a prevailing claim, advanced in the medical community, that the clinical efficacy of these drugs is undermined by cost and efficiency considerations.

Subjects and methods

The first part of the study is devoted to a review of pharmacoeconomic literature (2000–2012) on clinical drug testing for MS, particularly in the member countries of the European Union. The second part presents survey results of discontinued clinical trials that we gathered from European Union registries.

Results

The study finds that cost and efficiency considerations exist in a limited number of trials in the European Union. Specifically, about 1/5 of MS drugs do not reach the pharmaceutical markets even if there may be some supporting efficacy data, or negative safety data is absent, during testing/experimentation. Their direct and controlling impact on decisions to withdraw suspend or prematurely terminate (as well as initiate) clinical testing and efficacy data collection for MS does not appear to be consistent. However, these could extend prevailing theoretical debates about the proper scope of clinical drug efficacy in MS.

Conclusion

The study suggests that pharmacoeconomic approaches to clinical efficacy help broaden and enhance our understanding and choices of disease management options for MS sufferers, their families and health care providers in the European Union and elsewhere. Finally, there is some empirical evidence to indicate an inverse relationship between transaction costs and drug marketability: The higher the transaction costs of clinical testing, the lower is the drug's potential for funding and eventual marketing

Baumhackl U, Kappos L, Radue EW, Freitag P, Guseo A, Daumer M, Mertin J (2005) A randomized, double-blind, placebo controlled study of oral hydrolytic enzymes in relapsing multiple sclerosis. Mult Scler 11:166–168PubMedCrossRef

Bryant J, Clegg A, Milne R (2001) Systematic review of immunomodulatory drugs for the treatment of people with multiple sclerosis: is there good quality evidence on effectiveness and cost? J Neurol Neurosurg Psychiatry 70:574–579PubMedCentralPubMedCrossRef

CAMMS223 Trial Investigators, Coles AJ, Compston DA, Selmaj KW, Lake SL, Moran S, Margolin DH, Norris K, Tandon PK (2008) Alemtuzumab vs. interferon beta-1a in early multiple sclerosis. N Engl J Med 359:1786–1801PubMedCrossRef

Chilcott J, McCabe C, Tappenden P, O’Hagan A, Cooper NJ, Abrams K, Claxton K (2003) Modelling the cost effectiveness of interferon beta and glatiramer acetate in the management of multiple sclerosis. BMC 326:522–526CrossRef

Coase R (1937) The nature of the firm. Economica 4:386–405CrossRef

Couzin J (2005) Drug discovery: magnificent obsession. Science 307:1712–1715PubMedCrossRef

Curtiss FR (2007) Pharmacoeconomic modeling of drug therapies for multiple sclerosis: are we building houses on sand? J Manag Care Pharm 13:245–261

Dahlman CJ (1979) The problem of externality. J Law Econ 21:141–162CrossRef

European Union Clinical Trials Register, https://www.clinicaltrialsregister.eu/ctr-search/search?query=multiple+sclerosis. Accessed February 24, 2012

Fuller GN, Bone I (2001) Disease modifying treatment in multiple sclerosis. J Neurol Neurosurg Psychiatry 71:i20–ii21

Hauser S (1994) Multiple sclerosis and other demyelinating diseases. In: Isselbacher KJ, Martin JB, Fauci AS (eds) Harrison’s principles of internal medicine. McGraw-Hill, New York, pp 2287–2295

Holmoy T, Celius EG (2008) Cost-effectiveness of natalizumab in multiple sclerosis. Expert Rev Pharm Outcomes Res 8:11–21CrossRef

Kendrick M, Johnson KI (2000) Long-term treatment of multiple sclerosis with interferon may be cost effective. PharmacoEconomics 18:45–53PubMedCrossRef

Kleinschnitz C, Meuth SG, Wiendl H (2008) The trials and errors in MS therapy. Int MS J 15:79–90PubMed

MacLean HJ, Freedman MS (2001) Immunologic therapy for relapsing-remitting multiple sclerosis. Curr Neurol Neurosci Rep 1:277–285PubMedCrossRef

Maggon K (2010) Daclizumab (Biogen, Abbott) Review: multiple sclerosis. http://knol.google.com/k/daclizumab-biogen-abbott-review-multiple-sclerosis#Clinical_trials. Accessed July 11, 2011

National Institute of Neurological Disorders and Stroke (NINDS) Multiple sclerosis information page. http://www.ninds.nih.gov/disorders/multiple_sclerosis/multiple_sclerosis.htm. Accessed May 13, 2007

O’Connor PS, Goodman AD, Kappos L, Lublin FD, Miller DH, Polman CH, Rudick RA, Forrestal F, Jurgensen S, Panzara MA, Sandrock AW (2009) Return of disease activity after cessation of natalizumab. Mult Scler 15:S240

Pugliatti M, Rosati G, Carton H, Riise T, Drulovic J, Vecsei L, Milanov I (2006) The epidemiology of multiple sclerosis in Europe. Eur J Neurol 13:700–722PubMedCrossRef

Ryan M (2008) Multiple sclerosis. In: Dipiro JT, Talbert RL, Yee GC, Matzke GR, Wells BG, Posey LM (eds) Pharmacotherapy a pathophysiologic approach. McGraw Hill, New York, pp 431–441

Sorensen PS, Mellgren SI, Svenningsson A, Elovaara I, Frederiksen JL, Beiske AG, Myhr KM, Søgaard LV, Olsen IC, Sandberg-Wollheim M (2009) NORdic trial of oral Methylprednisolone as add-on therapy to Interferon beta-1a for treatment of relapsing-remitting multiple sclerosis (NORMIMS study): a randomised, placebo-controlled trial. Lancet Neurol 8:519–529PubMedCrossRef

Tan IL, Lycklama à Nijeholt GJ, Polman CH, Adèr HJ, Barkhof F (2000) Linomide in the treatment of multiple sclerosis: MRI results from prematurely terminated phase-III trials. Mult Scler 6:99–104PubMedCrossRef

Weinshenker BG, Noseworthy JH (2001) Multiple sclerosis: study design, sample sizes and pitfalls. In: Guiloff RJ (ed) Clinical trials in neurology. Springer, London, pp 371–386CrossRef

Wiendl H, Hohlfeld R (2002) Therapeutic approaches in multiple sclerosis: lessons from failed and interrupted treatment trials. BioDrugs 16:183–200PubMedCrossRef

Wiendl H, Neuhaus O, Kappos L, Hohlfeld R (2000) Multiple sclerosis: current review of failed and discontinued clinical trials of drug treatment. Nervenarzt 71:597–610PubMedCrossRef

Titel: Pharmacoeconomics and clinical trials in multiple sclerosis: baseline data from the European Union
verfasst von: Roger Lee Mendoza
Publikationsdatum: 01.06.2014
Verlag: Springer Berlin Heidelberg
Erschienen in: Journal of Public Health / Ausgabe 3/2014
Print ISSN: 2198-1833
Elektronische ISSN: 1613-2238
DOI: https://doi.org/10.1007/s10389-013-0561-z

Springer Medizin

Abstract

Aim

Subjects and methods

Results

Conclusion

Bitte loggen Sie sich ein, um Zugang zu diesem Inhalt zu erhalten

Weitere Artikel der Ausgabe 3/2014

Impact of gender and age on risk factor distribution and health perception: evaluation in a prospective population with heart disease

Public hospital accounting reform: the case of Austria introduction of mandatory cash flow statements for hospitals

Regional differences in the incidence of inpatients with pressure ulcers in Germany

Injuries to 15–19-year olds in road traffic crashes: a cross sectional analysis of police crash data

Access to and perceptions towards water, sanitation and hygiene: a case of HIV/AIDS affected and non-affected households in rural Uganda

Dropping out of school and chronic disease in the United States