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Research & Reports

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Rare Diseases and Orphan Drugs | Landscape & Forecast | Disease Landscape & Forecast - Gene Therapies for Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs | Landscape & Forecast | Disease Landscape & Forecast - Gene Therapies for Rare Diseases and Orphan Drugs

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Coming Soon – October 2019


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 treatment options 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, with large pharmaceutical companies (e.g., Novartis, Johnson & Johnson) taking an increasing stake in this arena. Here we will explore the clinical and commercial potential of gene therapies in key therapeutic areas (e.g., neurology, ophthalmology, hematology).


  • What are the sizes of the eligible populations for gene therapies in key indications across the G7?
  • What are the key advantages and disadvantages of marketed gene therapies, and where do clinicians want to see improvements?
  • As potential single-administration cures, how will the treatment-eligible population change over time in indications served by a gene therapy?
  • Who are the key decision makers for gene therapy purchase and administration?
  • What are the key drivers and barriers for the adoption of gene therapies?
  • What type of clinical trial data will be necessary to support adoption and reimbursement of gene therapies?
  • What is the clinical and commercial potential for emerging gene therapies in key therapy areas (e.g., ophthalmology, neurology)?


United States, EU5, Japan


Approximately 20 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


10-year, annualized, drug-level sales and patient share of key gene therapies through 2028


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, indepth
pipeline assessments, and drug forecasts supported by detailed primary
and secondary research.

Table of contents

  • Disease Landscape & Forecast - Gene Therapies for Rare Diseases and Orphan Drugs
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  • Pub Date: February 2019
  • Author(s): Claudia Dall'Osso
  • Claudia is a Senior Business Insights analyst on the Infectious, Niche, and Rare Diseases team at Decision Resources Group, specializing on niche and rare indications. Before joining DRG, she held a management and strategy consultant position at Precision Medicine Group, where she worked for clients in the biopharmaceutical, medical device and diagnostic industries. Claudia completed her Master’s in Management at Harvard University; she also holds a in medical genetics from Brescia University in Italy and a BS/MS degree in medical biotechnology from University of Milano in Italy.

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