Intended for healthcare professionals


Orphan drugs and the NHS

BMJ 2005; 331 doi: (Published 10 November 2005) Cite this as: BMJ 2005;331:1144

Consider whom drug regulation is designed to protect

  1. Tom Marshall (, senior lecturer
  1. University of Birmingham, Birmingham B15 2TT

    EDITOR—Orphan drugs pose several dilemmas for policymakers, not all of which are addressed by discussing cost effectiveness.1

    To be reimbursed by a publicly funded healthcare system a drug needs to be effective, safe in animal testing, effective in humans, and a cost effective use of public resources.

    Orphan drugs will not generate sufficient sales to recoup the cost of jumping these …

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