About Deerfield

Launched in 1994, Deerfield Management Company is an investment firm dedicated to advancing healthcare through information, investment, and philanthropy—all toward the end goal of cures for disease, improved quality of life, and reduced cost of care.

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Supporting companies across the healthcare ecosystem with flexible funding models…

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Delivering market research to the Deerfield team, its portfolio companies and other partners.

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A New York City-based not-for-profit devoted to advancing innovative health care initiatives.

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Portfolio Companies

Deerfield generally maintains a combined portfolio of more than 150 private and public investments across the life science, medical device, diagnostic, digital health and health service industries at all stages of evolution from start-up to mature company.

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Research Collaborations

Deerfield partners with leading academic research centers, providing critical funding and expertise to further sustain and accelerate the commercialization of discoveries toward meaningful societal impact by advancing cures for disease.

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Strategic Partners

As a strategic partner, Deerfield offers capital, scientific expertise, business operating support, and unique access to innovation.

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Deerfield Foundation

The Deerfield Foundation is a New York City-based not-for-profit organization whose mission is to improve health, accelerate innovation and promote human equity.

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Cure Campus

Cure is a 12-story innovations campus in New York City that intends to bring together innovators from academia, government, industry, and the not-for-profit sectors to advance human health and accelerate the fight against disease.

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Cure Programming

Cure has a series of expert lectures intended to advance thought in healthcare, management, innovation, policy, and other relevant subjects. This fosters growth and education for those at Cure and its guests.

Events at the Cure

Calculating the prevalence of urea cycle disorders in the EU5 using an incidence-survival model


Gene therapies for rare diseases have ignited industry-wide debates concerning patient access, pricing and efficacy. Urea cycle disorder (UCD), a genetic rare disease, is a target for enzyme replacement therapies, yet UCD epidemiology data are scarce. The objective of this study is to estimate the prevalence of UCD in the 0–17 year-old population in five major European markets (EU5) (France, Germany, Italy, Spain, UK).


We designed an incidence-survival model utilizing birth incidence, annual life-expectancy and country-specific newborn data. Birth incidence estimates for UCD were obtained from the Urea Cycle Disorders Consortium (UCDC). We conducted a literature review to determine the annual life expectancy of UCD. Finally, we reviewed available EU5 newborn census data to obtain the number of newborns in each country from 2001 to 2012. We extrapolated the trend from 2001-2012 to forecast the number of newborns through 2024. These data were entered into the incidence-survival model to calculate the prevalence of UCD from 2015-2024.


We estimated a prevalence of 1.71 per 100,000 population in the 0–17 year age groups resulting in 1,027 and 1,033 UCD cases in the EU5 in 2015 and 2024, respectively. The prevalence was 1.89 per 100,000 in the 0-4 year-old age group, 1.63 per 100,000 in the 10-14 year-old age group, and 1.57 per 100,000 in 15-17 year-old age group.


To our knowledge, this study provides the first prevalence estimate of UCD in the EU5 markets. With 1,027 estimated cases in 2015, it is expected that UCD treatments will be expensive given the small size of the patient population. Further, these estimates can be used to approximate the total cost burden of UCD and the resulting cost-effectiveness of UCD treatments