Related Articles or Presentations

  • ISPOR conference, retrospective observational study to capture economic burden evidence in a rare disease AbstractPoster
  • EphMRA, “Precedent and consequence research to undergird orphan reimbursement strategy – A multi-layered, non-traditional approach” Presentation
  • Japanese Respiratory Society, treatment patterns for a rare lung disease in EU5, Japan and US Abstract
  • European Respiratory Society, prevalence and incidence of a rare lung disease in Europe Abstract
  • European Respiratory Society, treatment patterns for rare disease in EU5, Japan and US Abstract
  • European Respiratory Journal, treatment patterns for a rare lung infection in Europe, US and Japan, and article on prevalence in Europe, co-written with international team of medical experts,  Abstract 1 & 2
  • KIT 2014 (Congress for Infectious Diseases and Tropical Medicine), co-author, rare lung infection prevalence in Germany Abstract
  • Annals of the American Thoracic Society, US study of rare lung infection Abstract  
  • American Thoracic Society (ATS) International Conference, study of rare lung infection Abstract
  • ​Metastatic Melanoma Treatment (PDF)

Rare diseases, orphan drugs


Our Methodology Helps Size the Market 
and Boost Investor Confidence

Orphan drugs and rare diseases are part of our research focus – We have studied various rare diseases, including blood disorders and cancers, NTMPD (pulmonary disease), and connective tissue disorders, and we have studied orphan drug treatments for some of these conditions. The graduate thesis by one of our staff explored the impact of federal orphan drug legislation. The scope of our orphan drug/rare disease research has included the US, Europe, and Asia.

Our methodology is well-suited to orphan drugs and rare diseases. What matters is a representative sample of target physicians and patients whose experiences can be generalized to the target universe, even if that universe is small. Ours is the only methodology that can do that. With patient records and treating physician research, you also get the advantage of capturing not only physicians perceptions but facts from the actual patient charts.


Our methodology is more accurate than any other in correctly sizing the market for an orphan drug. We often find that a clients estimation of the market for an orphan drug may be too low because foundational claims and other secondary data:

                •   are missing ICD codes for the disease

                •   or attribute the target disease to the wrong code.


Our patient records/treating physician methodology identifies and quantifies these errors. Our triangulation process allows you to optimize your use of costly claims / secondary data and increase your ROI for these data.

The accuracy of our work promotes confidence in our clients by investors, the FDA, and internal stakeholders.

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