Safe PCI for Women: View from the Pharmaceutical Industry Mark B. Effron, MD, FACC, FAHA, FCCP Medical Fellow Cardiovascular Medical Affairs Eli Lilly and Company

SAFE-PCI for Women: View from Pharma Sponsored research vs Independent research – Sponsored Sponsor has a role in design of study Sponsor may create and maintain database Sponsor will have a copy of the database Sponsor is typically involved in review, interpretation and disclosure of information – Independent Sponsor has no role in design of study Sponsor has no direct access to database Sponsor is not involved in review, interpretation and disclosure of information

SAFE-PCI for Women: View from Pharma What are the advantages to Pharma from a “hybrid” study like SAFE-PCI for Women – Ability to quickly access a large number of patients – Data collection that is detailed and well established – Ability to “piggyback” onto established data collection system – Decrease cost of the study

SAFE-PCI for Women: View from Pharma What are the disadvantages to Pharma from a “hybrid” study like SAFE-PCI for Women – No monitoring of data collection – Source documentation not available – Input of data frequently by people not part of the “hybrid” study – Inability to audit data by sponsor – No control of the database

SAFE-PCI for Women: View from Pharma Is there the ability to use a “hybrid” study like SAFE-PCI for Women for new drug registration? – Data not under the control of the sponsor May affect the ability to quickly respond to inquiries from the FDA – May not be able to adequately design the study to put in enough controls to adequately test hypothesis – Inability to audit or monitor data input However, the FDA will likely audit and may uncover discrepancies not known to the sponsor Audit by NCDR Data Quality Program has shown high average participant average raw accuracy of data abstraction in the NCDR registries (Messenger et al, JACC 2012;60:1484–8)

SAFE-PCI for Women: View from Pharma “Hybrid” studies like SAFE-PCI for Women may be a good vehicle for post registration comparative effectiveness studies – Can provide large number of patients in relatively short time – Less expensive than designing a new study – Faster to implement than a new study – Databases such as those in the NCDR have more granularity in the data than payer database analyses – Payers (especially CMS) may (will?) be requiring comparative effectiveness studies in the future

TRANSLATE ACS Study Design

Data Collection Site CathPCI data elements being used in TRANSLATE ACS TRANSLATE ACS Database Non-NCDR data elements ACC – NCDR (CathPCI) Database NCDR data elements collected as usual by the site

SAFE-PCI for Women: View from Pharma “Hybrid” studies like SAFE-PCI for Women may also be a good vehicle for post-marketing requirements and commitments – Can provide large number of patients in relatively short time – Less expensive than designing new trial – Faster to implement than a new study – Potential for “real time” data – Data more granular and accurate than typical safety surveillance