Safe PCI for Women: View from the Pharmaceutical Industry

1. 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

2. 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

3. 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

4. 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

5. 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)

6. 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

7. TRANSLATE ACS Study Design

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

9. 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