Module 3 Presentations

14/05/2024

The Importance of CMC  Even if you have the best drug in the world it could (will?) fail from: o Market supply constrained by manufacturing problems (DS and/or DP) o Lack of stable RT formulation (>18 months shelf-life preferred by wholesalers) o Wrong dosage form (not liked by patients) o Competition from similar but cheaper products  CMC needs to look at the long-term not only getting product to market but keeping it there

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Reimbursement Issues  Drug budget under increasing pressure

o Formularies prohibit availability of all drugs in the same class o ‘Value for Money’ (NICE etc) assessments prevent or limit use of perceived expensive new drugs or ‘me too’ products  Orkambi (ivacaftor + lumacaftor) for F508del Cystic

Fibrosis treatment (~ 4,000 UK patients) o US price £207,000 ($272,000) per patient year

o Initial UK price £105,000 per patient year rejected by NHS o Confidential commercial deal struck to allow NHS use (could be as low as £20,000 per patient year to match generic from Argentina)

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