Orphan drugs and the NHS

BMJ 2005; 331 doi: https://doi.org/10.1136/bmj.331.7525.1144-b (Published 10 November 2005) Cite this as: BMJ 2005;331:1144

Consider whom drug regulation is designed to protect

  1. Tom Marshall (T.P.Marshall@bham.ac.uk), 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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