Research Sizes Global Markets, Measures Burden of Illness, Builds Evidence Package for Pre-Launch Rare Lung Disease Treatment

•Situation: A client developing a treatment for pulmonary nontuberculous mycobacterial (PNTM) disease, a rare lung infection, sought to size the market and understand the treatment landscape in
                          the US, Canada, Europe and Japan.

•The client also sought to build an evidence package to persuade regulators and
payors of the need for and value of its prospective orphan drug, via a subsequent
representative, 4-nation burden of illness (BOI) study of refractory patients and a
strong presence in medical literature and at medical/health outcomes conferences.

•Methods: Our 7-nation market sizing study employed a representative treating physician/patient chart audit methodology, bolstered in Europe and Japan with a Delphi consensus method among epidemiologists to establish prevalence. It included >1,500 physicians providing >2,300 patient cases.

•Results: We captured detailed information about current treatment practices, patient characteristics and disease states, and other medications used as therapy, helping the client to see where its product might fill gaps. We estimated there were about 40% (20,000) more patients with this rare disease in the United States than the client previously had thought, important to an orphan product. The client’s news release about this finding was reported in several investment publications. Our studies included two NIH epidemiologists and several key medical experts in the disease area.

•We validated our US market size estimates with 2 models, one using survey-corrected claims data and the other, Medicare data.

•To date, more than a dozen publications or presentations have resulted from our research, which Clarity co-authored with several international medical and epidemiologic experts in the target illness, including 2 from NIH, including publications related to the completion of our subsequent burden of illness study. See a sampling of these publications and presentations at our website: https://www.claritypharma.com/publications.html