TY - JOUR

T1 - Innovation at the Intersection of Clinical Trials and Real-World Data Science to Advance Patient Care

AU - Swift, Brandon

AU - Jain, Lokesh

AU - White, Craig

AU - Chandrasekaran, Vasu

AU - Bhandari, Aman

AU - Hughes, Dyfrig

AU - Jadhav, Pravin

PY - 2018/9

Y1 - 2018/9

N2 - While efficacy and safety data collected from randomized clinical trials are theevidentiary standard for determining market authorization, this alone may no longer be sufficient to address the needs of key stakeholders (regulators, providers and payers) and guarantee long-term success of pharmaceutical products. There is a heightened interest from stakeholders on understanding the use of real-world evidence (RWE) to substantiate benefit-risk assessment and support the value of a new drug. This review provides an overview of real-world data (RWD) and related advances in the regulatory framework,and discusses their impact on clinical research and development. A framework for linking drug development decisions with the value proposition of the drug, utilizing pharmacokinetic-pharmacodynamic-pharmacoeconomic (PK-PD-PE) models, is introduced. The summary presented here is based on the presentations and discussion at the symposium entitled Innovation at the Intersection of Clinical Trials and Real-World Data to Advance Patient Care at the American Society for Clinical Pharmacology and Therapeutics (ASCPT) 2017 annual conference.

AB - While efficacy and safety data collected from randomized clinical trials are theevidentiary standard for determining market authorization, this alone may no longer be sufficient to address the needs of key stakeholders (regulators, providers and payers) and guarantee long-term success of pharmaceutical products. There is a heightened interest from stakeholders on understanding the use of real-world evidence (RWE) to substantiate benefit-risk assessment and support the value of a new drug. This review provides an overview of real-world data (RWD) and related advances in the regulatory framework,and discusses their impact on clinical research and development. A framework for linking drug development decisions with the value proposition of the drug, utilizing pharmacokinetic-pharmacodynamic-pharmacoeconomic (PK-PD-PE) models, is introduced. The summary presented here is based on the presentations and discussion at the symposium entitled Innovation at the Intersection of Clinical Trials and Real-World Data to Advance Patient Care at the American Society for Clinical Pharmacology and Therapeutics (ASCPT) 2017 annual conference.

U2 - 10.1111/cts.12559

DO - 10.1111/cts.12559

M3 - Article

VL - 11

SP - 450

EP - 460

JO - Clinical and Translational Science

JF - Clinical and Translational Science

SN - 1752-8054

IS - 5

ER -