Rare Diseases and Orphan Drugs – Landscape & Forecast – Disease Landscape & Forecast (G7)

Approximately 80% of rare diseases have a genetic etiology. Gene therapies have the potential to transform the treatment of rare diseases and, in some cases, provide a functional cure. However, these innovative treatments must come with equally innovative programs to optimize access in a global healthcare market that is still grappling with how to fairly assess these therapies’ value and absorb their high up-front costs. Nonetheless, the pipeline for gene therapies is burgeoning, as large pharmaceutical companies (e.g., Janssen, Roche) assume increasing stakes in this arena. In this content, we explore the clinical and commercial potential of select gene therapies in rare disorders such as spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), cerebral adrenoleukodystrophy (CALD), beta thalassemia, and inherited retinal dystrophies such as retinitis pigmentosa.

Questions answered

• What are the sizes of the eligible populations for gene therapies in key indications in the G7?
• What are the main advantages and disadvantages of marketed gene therapies, and where do clinicians want to see improvements?
• With gene therapies being potential single-administration cures, how will the treatment-eligible population change over time in indications served by a gene therapy?
• What are the key drivers of and barriers to the adoption of gene therapies?
• What is the expected uptake of the first CRISPR-based gene editing therapy (Casgevy) approved for beta thalassemia?
• What is the commercial potential of emerging gene therapies in key indications (e.g., DMD, Leber hereditary optic neuropathy, CALD)?

Content highlights

Geography: United States, EU5, Japan

Primary research: More than 28 country-specific interviews with thought-leading ophthalmologists, neurologists, and hematologists, supported by survey data collected for this and other Clarivate research

Epidemiology: Diagnosed prevalent and/or incident patient populations eligible for gene therapies in the covered indications

Forecast: 10-year, annualized, drug-level sales and patient share of key gene therapies through 2033, segmented by geography and key indication

Drug treatments: Coverage of key current and emerging gene therapies in select indications across therapy areas

Product description

Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current and emerging therapies, and drug forecasts supported by detailed primary and secondary research.

Key features

Disease Landscape & Forecast will be updated throughout the year to provide timely insights and analyses as material indication-specific news and events unfold.