The explosion in real-world data (RWD) from digital platforms is creating new insights into the understanding of rare diseases and patient experiences. The value and validity of RWD generated from registries, patient records, wearables and apps is increasingly recognized by pharma and stakeholders. Pharma’s challenge now is less about data collection for their RWD programs, but more about how to effectively process and analyze data from the many varied data sources to create significant insights that inform and support patients, physicians, payers and regulators.
To explore the opportunities and challenges of harnessing the power of RWD for orphan drugs we interviewed rare disease and data collection/analysis experts. In Real World Data for Orphan Drugs: Meeting the Data Analysis Challenge they examine the current trends in RWD generation/analysis and the technologies, practice and strategies that are generating new critical insights.