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. 2012 Feb;109(7):117-23.
doi: 10.3238/arztebl.2012.0117. Epub 2012 Feb 17.

New drugs: evidence relating to their therapeutic value after introduction to the market

Mariam Ujeyl  1 , Claudia Schlegel, Siegbert Walter, Ursula Gundert-Remy
Affiliations

New drugs: evidence relating to their therapeutic value after introduction to the market

Mariam Ujeyl et al. Dtsch Arztebl Int. 2012 Feb.

Abstract

Background: Drug approval is based on three criteria: quality, efficacy, and safety. We investigated the types of study design and statistical methods employed to demonstrate safety and efficacy of proprietary medicinal products (PMPs) that were approved for use in the European Union through the centralized procedure.

Methods: We retrospectively analyzed the European Public Assessment Reports of PMPs that the European Medicinal Agency approved, either initially or for extended indications, in 2009 and 2010.

Results: Data were analyzed for 39 PMPs: 64% of these were new active substances, and 36% were approved for extended indications. 46% of the PMPs had been studied in an active-control trial. In only 28%, superiority of the new PMPs compared to active control had been tested. 46% of the approvals included testing of a patient-relevant primary endpoint. The median size of population used to demonstrate safety was 1700 persons.

Conclusion: The centralized procedure does not require comparative information from active-control trials. Accordingly, as our descriptive analysis revealed, this information is often not available at the time of market introduction. Pivotal studies only rarely clearly demonstrate an added therapeutic value of a new PMP compared to existing alternatives.

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References

    1. Europäische Arzneimittel-Agentur. Zulassung und Überwachung von Arzneimitteln - Europäische Arzneimittel-Agentur. http://europa.eu/legislation_summaries/internal_market/single_market_for.... Last accessed on 1 June 2011.
    1. EMA. Status of EMEA scientific guidelines and European pharmacopoeia monographs and chapters in the regulatory framework applicable to medicinal products. www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/.... EMEA/42371/2008; London, 11. September 2008 - corr. Last accessed on 1 June 2011.
    1. EMA. European public assessment reports. www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/general/general_con... Last accessed on 1 June 2011.
    1. Garattini S, Bertele V. Mossialos E, Mrazek M, Walley T, editors. The role of the EMEA in regulating pharmaceutical products. Regulating pharmaceuticals in Europe: striving for efficiency, equity and quality. Open University Press; Maidenhead: European Observatory on Health Systems and Policies Series. 2004
    1. Hart D. Risk-benefit evaluation of medicinal products. An element of health technology assessment. Bundesgesundheitsbl - Gesundheitsforsch - Gesundheitsschutz. 2005;48:204–214. - PubMed

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