Approximately 80% of rare diseases have a genetic etiology. Gene therapies hold the potential to transform the treatment of rare diseases and may provide a functional cure for some. However, these innovative new treatments must come with equally innovative programs to optimize access in a global healthcare market that is still grappling with how to fairly assess their value and absorb their higher upfront costs. Nonetheless, the pipeline for gene therapies is burgeoning, as large pharmaceutical companies (e.g., Novartis, Johnson & Johnson) take an increasing stake in this arena. Here, we explore the clinical and commercial potential of gene therapies in key therapeutic areas (i.e., neurology, ophthalmology, and hematology).
United States, EU5, Japan
Approximately 30 country-specific interviews with thought-leading gene therapy experts. Supported by survey data collected for this and other DRG research.
Diagnosed prevalent and/or incident patient populations eligible for gene therapies in covered indications.
Ten-year, annualized, drug-level sales and patient share of key gene therapies through 2029.
Pipeline gene therapy coverage for select indications across therapy areas, including hematology, ophthalmology, and neurology.
Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.