Precision therapies are often approved based on small trials, in specialized settings, and using biomarkers as endpoints. Thus, despite their potential high cost, the evidence of value is lacking which makes real-world evidence crucial. Join Schaeffer Center Director of Research Darius Lakdawalla in discussion with MIT NEWDIGS Director of Research Kay Larholt and Merck Center for Observational and Real-World Evidence Executive Director Megan O’Brien for a discussion about how we should measure and leverage real-world data to assess the value…

Mark Trusheim participated in the World Orphan Drug Congress USA 2021 on August 25–27.

Mark Trusheim participated in the ASCGT Policy Forum panel "The Status of Medicaid Coverage and Access for Gene and Cell Therapies" on September 22, and on September 28 he led the roundtable "Preparing for 2022 regulatory change and the performance-based contracting explosion" for the Reuters Cell and Gene Therapy USA meeting. On September 30 Mark spoke in the OHE Master Class Adaptive Pathways: Generating Evidence of Value.

Jane Barlow discussed FoCUS emerging market solutions research at the Huron Virtual Roundtable discussion "Unlocking the Full Potential of Outcomes Based Contracting Work for CGT: Collaborate or Compete?" on September 22.