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

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

How can self-insured employers prepare for the portfolio impact of highcost gene therapies coming to market?

Eric NormanFoCUS, Research Briefs

Self-insured employers (SIEs) currently rely on traditional stop-loss insurance to protect against unexpected high-cost claims, including those for cell and gene therapies. Given that current approvals are in rare diseases, … Read More

Updated projection of US durable cell and gene therapies product-indication approvals based on December 2019 development pipeline

Eric NormanFoCUS, Research Briefs

Ongoing pipeline modeling by the MIT NEWDIGS FoCUS team projects 10X growth from July 2020 levels, but with large uncertainties. Download FoCUS Research Brief 2020F207v51Share the research

Tracking the CAR-T revolution: Analysis of clinical trials of CAR-T and TCR therapies for the treatment of cancer

Eric NormanFoCUS, Whitepapers

Chimeric antigen receptor and T-cell receptor (CAR-T/TCR) cellular immunotherapies have shown remarkable success in the treatment of some refractory B-cell malignancies, with potential to provide durable clinical response for other … Read More

Pediatric Gene Therapy Launches

Eric NormanFoCUS, Research Briefs

Gene therapy cures for hereditary genetic conditions are likely to reach the United States market in the coming decades. Several disease areas with potential cures are orphan conditions, which affect … Read More