Alan Maloney
  • Drug Development for Patients
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  1. Glossary
  • Welcome
  • 1  Introduction
  • 2  My Motivation For This Book
  • 3  Why Patients Outcomes Must Come First
  • 4  A Brief History Of Drug Development; The Good, The Bad And The Ugly
  • 5  Dose-Response Trials; A Brief History And Overview Of Current Practices
  • 6  The Science; Why We Must Care About Dose, Pharmacokinetics, Pharmacodynamics And Utility
  • 7  Introduction To IIV In PK And Its Consequences To D-E-R Trial Design
  • 8  Introduction To IIV In PD And D-E-R Analysis As Evidence For Regulators
  • 9  Personalised Dosing; Patients Are Different
  • 10  Where Does Precision Medicine And Personalised Medicine Fit In?
  • 11  Dose-Response Modelling; Why We Need Integrated Analyses Across All Doses/Trials
  • 12  Introducing The Most Important Dose-Response Model
  • 13  Population and Individual Dose-Exposure-Responses, Therapeutic Windows and Maximum Tolerated Doses
  • 14  What Should Be The Role For Regulators?
  • 15  The Two Regulatory Approval Pathways; “Approval P” and “Approval I”
  • 16  The Two Development Strategies; “Strategy P” and “Strategy I”
  • 17  Changing How We Pay For Drugs; Value/Outcome-Based Pricing And Subscription-Based Pricing
  • 18  What Product Should Drug Companies Sell? (can be skipped)
  • 19  Adaptive Randomisation In Population D-E-R Trials; Why We Should Learn As We Go
  • 20  The Half-Time Summary; What Have We Learnt, And What Solutions Are Outstanding?
  • 21  Technical Sections To Write
  • 22  Conclusions
  • 23  Acknowledgements
  • Glossary
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Glossary

ADME Absorption, Distribution, Metabolism and Excretion
ANCOVA Analysis of Covariance
ANOVA Analysis of Variance
bid twice daily (bis in die in Latin)
CPT Clinical Pharmacology and Therapeutics (journal)
CTS Clinical Trial Simulation
D-R Dose-Response
D-E-R Dose-Exposure-Response
FIM (with respect to trials) First In Man
FIM (with respect to statistics) Fisher Information Matrix
IIV Inter-Individual Variability
INR International Normalised Ratio
MAD Multiple Ascending Dose (trial)
MBMA Model-Based Meta-Analysis
MCP-MOD Multiple Comparison Procedure - Modelling
MoA Mechanism of Action
MTD Maximum Tolerated Dose
N Sample size (=number of trial participants)
NHST Null Hypothesis Significance Testing
NSCLC Non-Small Cell Lung Cancers
PD Pharmacodynamic
PK Pharmacokinetic
PPD Price Per Dose
qd once daily (quaque die in Latin)
RCT Randomised Controlled Trial
RWE Real World Evidence
SAD Single Ascending Dose (trial)
SSE Stochastic Simulation and Estimation
UX User Experience
23  Acknowledgements
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Copyright Alan Maloney, May 2023
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