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).
QUESTIONS ANSWERED
GEOGRAPHIES
United States, EU5, Japan
PRIMARY RESEARCH
Approximately 30 country-specific interviews with thought-leading gene therapy experts. Supported by survey data collected for this and other DRG research.
EPIDEMIOLOGY
Diagnosed prevalent and/or incident patient populations eligible for gene therapies in covered indications.
FORECAST
Ten-year, annualized, drug-level sales and patient share of key gene therapies through 2029.
EMERGING THERAPIES
Pipeline gene therapy coverage for select indications across therapy areas, including hematology, ophthalmology, and neurology.
PRODUCT DESCRIPTION
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.