W hen do clinicians usually extrapolate in their practice? Daniel - PowerPoint PPT Presentation

W hen do clinicians usually extrapolate in their practice? Daniel Brasseur CHMP Christoph Male PDCO

Frequent approaches in the Clinical setting Extrapolation across • Patients • Products • Pathologies

Patients Sam e m edicine than in adults used ‘dow n the age’ 1 / dow n/ up the age class 3

Patients ‘dow n the age’ How Far can w e go ? 5

Morphology? Function?

Patients ‘Up the age’ Cystic fibrosis Congenital disorders (orphan conditions) … 7

Patients ‘Up and dow n again…’ Cystic fibrosis Congenital disorders (orphan conditions) … 8

Frequent approaches in the Clinical setting Extrapolation across • Patients • Products • Pathologies

Products Different m edicines belonging to a class Drug/ pro-drug • ( [ des] loratadine...) Bio-equivalence • ( Release form ulation, conversion factor) Therapeutic equivalence • ( anti-TNF in MS, Blood factor substitution, ERT) ( vaccines, based on sero-conversion factors) ( one statine to another, m onitored by LDL-Chol) ) Me-too, is not equi-potency • ( for E [ long term effect statines] &/ or S...) 10

Frequent approaches in the Clinical setting Extrapolation across • Patients • Products • Pathologies

Sam e products used for different diseases pain / fever • leukaem ia/ lym phom a • arthritis… • HTA [ prim ary,secondary] / heart failure • Allergy/ exercise induced asthm a • Heart failure stage I ,I I ,I I I ,I V • Cancer stage, relapse... • Pathologies 12

Pathologies Sam e product but used in different diseases epsy FOCAL GENERALISED IDIOPATHIC 10 years Reading epilepsy Adolescent Acquired epileptic absence Aphasia epilepsy 2 years JME Epilepsy with CT spikes BMEI Childhood Epilepsy 1 month Absence with GTCS BIFS epilepsy on BIS awakening BFNS BNS Doose syndrome WEST EI EE Symptomatic or cryptogenic focal EME epilepsies IDIOPATHIC MPSI Lennox Gastaut syndrome E. partialis continua Dravet syndrome NON E. With myoclonic Epilepsy absences with CSWS Sylvie N'Guyen 13

How do Clinicians behave? I nterlinking • Mechanism of action of the disease ( pathogenesis/ pathw ays) • Mechanism of action of the drug ( target, receptors) • Clinical condition/ presentation ( individual sign & sym ptom s)