Limits of add-on trials

Antirheumatic drugs

Research output: Contribution to journalArticle

7 Citations (Scopus)

Abstract

Purpose: This paper assesses the design of clinical studies used in the process of regulatory approval, focusing on how add-on studies affect regulatory decisions. Methods: The sample case taken is that of the new agents for rheumatoid arthritis (RA) authorised by the European Medicine Agency (EMEA). The European Public Assessment Reports (EPARs) accompanying the marketing authorisations were the source of information on the studies presented in the registration dossiers. Results: The recently approved anti-RA agents are all indicated in combination with methotrexate (MTX) for treating adults with active RA who have responded inadequately to disease-modifier drugs (DMARDs). The add-on design was frequently used in registration studies. For infliximab, etanercept, adalimumab and rituximab, add-on trials contributed, together with parallel-group trials, to gaining the approval as combination therapy. Anakinra and abatacept were authorised on the basis of add-on trial results only. Conclusions: Add-on trials do not allow assessment of the intrinsic efficacy and safety of new agents and their value as alternatives to available treatments. The indications granted for the new anti-RA agents do not specify whether newer drugs can replace standard treatments in nonresponders, can do better in the overall patient population or can be used as first-line treatment.

Original languageEnglish
Pages (from-to)33-41
Number of pages9
JournalEuropean Journal of Clinical Pharmacology
Volume65
Issue number1
DOIs
Publication statusPublished - Jan 2009

Fingerprint

Antirheumatic Agents
Rheumatoid Arthritis
Interleukin 1 Receptor Antagonist Protein
Therapeutics
Marketing
Methotrexate
Pharmaceutical Preparations
Medicine
Safety
Population

Keywords

  • Abatacept
  • Adalimumab
  • Add-on studies
  • Anakinra
  • Disease-modifying antirheumatic drugs
  • Etanercept
  • European Medicine Agency
  • Infliximab
  • Marketing authorisation
  • Methotrexate
  • Registration trials
  • Rheumatoid arthritis
  • Rituximab

ASJC Scopus subject areas

  • Pharmacology (medical)
  • Pharmacology

Cite this

Limits of add-on trials : Antirheumatic drugs. / Ottolenghi, Laura; Bertele', Vittorio; Garattini, Silvio.

In: European Journal of Clinical Pharmacology, Vol. 65, No. 1, 01.2009, p. 33-41.

Research output: Contribution to journalArticle

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