EARLY ACTIVATION OF VALUE DEMONSTRATED BY THE COLLECTION OF REAL WORLD DATA IN PRE-APPROVAL ACCESS SETTINGS
Author(s)
Eslami N1, Zaidi Q2, Sadana R3, Garfield S4, Richardson S5
1Ernst & Young, Malden, MA, USA, 2Ernst & Young, Iselin, NJ, USA, 3Ernst & Young, Haryana, India, 4Ernst & Young, Wayland, MA, USA, 5Ernst & Young, New York, NY, USA
OBJECTIVES: Ubiquity of data is driving regulators and payers to consider RWD in evaluating drug value, compelling companies to look at ways to collect data that supports their product value story before launch. This abstract focuses on RWD collected in a pre-approval access (PAA) setting, and assesses how companies currently collect and leverage this data, and its impacts on regulator and payer decision-making. A data collection framework is also discussed considering ethical and legal concerns facing data collection in this setting. METHODS: An assessment was conducted in US, UK and France to understand the value of collecting data in PAA settings. Publically available information on EAPs, specifically SPRs and NPPs, was collected, with an overview of guidelines and examples, while primary research focused on benefits, risks, and considerations. RESULTS: US, UK, & France have established programs to support PAA collection; regulators and payers want to use this data to aid decision-making. Manufacturers are leveraging data for approval or publication, e.g. Vistogard by Wellstat Therapeutics and Tecentriq by Genentech. Primary research highlighted benefits, including enriching stakeholder relationships and learning more about RW use of a drug and its population. Risks were ensuring data privacy/security, and the weight of negative data on launch. A framework was developed to guide setup and data collection in PAA programs, including mitigating risks. CONCLUSIONS: There is growing interest in collecting RWD in PAA settings. The data will educate stakeholders around the use of a drug beyond CT populations, especially within rare/orphan diseases or oncology where additional subgroups may be identified. Markets are looking to use this data as part of their approval and pricing analyses, resulting in a push to develop robust data collection practices. It is recommended manufacturers implement a framework to ensure equitability and data quality, aiding payers and regulators as they look to leverage RWE in decision-making.
Conference/Value in Health Info
2019-05, ISPOR 2019, New Orleans, LA, USA
Value in Health, Volume 22, Issue S1 (2019 May)
Code
PRO43
Topic
Health Policy & Regulatory, Patient-Centered Research, Real World Data & Information Systems
Topic Subcategory
Approval & Labeling, Data Protection, Integrity, & Quality Assurance, Patient-reported Outcomes & Quality of Life Outcomes, Reimbursement & Access Policy
Disease
Drugs, Oncology, Rare and Orphan Diseases