Formulary Request

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Clinician’s request

Imagine that you are one of the pharmacists making formulary decisions at a medical center. A new hematologist has moved to your medical center. She is one of the nation’s best known experts on paroxysmal nocturnal hemoglobinuria (PNH).  She has been treating quite a few patients with eculizumab.   Eculizumab is FDA-approved for treatment of hemolysis in PNH, but not all of the hematologist’s eculizumab-treated PNH patients require treatment for hemolysis.  Some require treatment because of significant thrombotic complications.  She asks that eculizumab be made available at your institution for PNH patients with either hemolysis or thrombosis.

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Pharmacy’s Response

The pharmacy doesn’t have much experience with eculizumab which is one of the most expensive drugs in the world.  Yearly costs for treatment of PNH are estimated at between $400,000 –  $700,000.  You have been asked to prepare a formulary monograph on the use of eculizumab in PNH with thrombosis.

You decide to start by searching PubMed for journal articles that describe the results of phase II-IV trials and any other randomized controlled trials.  To perform a thorough PubMed search, you need to understand the differences between the two major sets of journal article records that are included in PubMed.