Emerging market solutions for financing and reimbursement of gene therapies for sickle cell disease: Why do payment innovation?

Eric NormanFoCUS, Research Briefs

The FDA has approved two cell and gene therapies for sickle cell disease, marking a significant development for the largest population condition to date. ​ However, the high treatment costs, … Read More

Ask PAM: “How many?”

Eric NormanFoCUS

Individual Indication Workbook in actionHow many treatment-eligible people are in my population?The Pipeline Analysis and Modeling (PAM) in the NEWDIGS Paying for Cures Toolkit maintaints a unique, detailed, indication-by-indication analysis … Read More

Analyzing 340B and ASP interactions: Do Federal program rules disincentivize the use of VBCs, despite Medicaid Best Price reform?

Eric NormanFoCUS, Research Briefs

This paper describes the interactions among Medicaid Best Price rebate calculations and those for 340B Ceiling Prices and Average Sales Prices which can create disincentives for providers and therapy developers … Read More

Are Cell and Gene Therapy programs a better bet?

Eric NormanFoCUS, Research Briefs

Compared to traditional drug development programs, do durable cell and gene therapy (CGT) programs experience higher or lower clinical success rates? Estimating the Probability of Technical and Regulatory Success (PTRS) … Read More