Clinical trial design and management

Research output: Contribution to journalArticlepeer-review


The randomised, controlled trial (RTC) is considered the gold standard for the assessment of the effect of any therapeutic intervention. However, rare diseases carry several issues in terms of population size, life-threatening nature of the condition and lack of other available treatments, which basically render this approach not always feasible. Alternative methodological approaches are being developed to better suit the need of an orphan drug development.

Original languageEnglish
Pages (from-to)57-62
Number of pages6
JournalPharmaceuticals Policy and Law
Issue number1-2
Publication statusPublished - 2010


  • controlled trial
  • population size
  • Randomised
  • rare disorder

ASJC Scopus subject areas

  • Health Policy
  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)


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