Implementation Brief: Understanding access issues for new therapies

Eric NormanFoCUS, Research Briefs

Our first implementation brief outlines several major barriers to patient access for novel cell and gene therapies. It also describes how providers, payers, and developers may overcome these barriers, with … Read More

Managing the Challenges of Paying for Gene Therapy: Strategies for Market Action and Policy Reform

Eric NormanFoCUS, Publications, Whitepapers

Gene therapies delivered through a single administration have revolutionized treatment possibilities for many patients living with serious or fatal conditions such as spinal muscular atrophy, hemophilia, and sickle cell disease. … Read More

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