For a while now, members of NORD’s leadership have been interested in finding ways to better understand the economic costs of not being able to manage rare diseases effectively and safely. In other words, we often have little idea of the total annual costs of looking after, and caring for, someone with a specific rare disorder – especially when this may include extended periods in hospitals, multiple visits to emergency departments, and other high-cost services.

At the most simplistic level, if we really did have some idea about the annual costs of caring for a patient with rare disease X, then it would help us to understand a “reasonable” price to pay for a new form of treatment that was able to eliminate many of the current, existing costs. As one example, just keeping a patient out of an intensive care unit can save thousands of dollars a day here in the USA.

Last week, we heard that an organization called Pro Bono Economics (PBE) is potentially interested in being able to explore ways to address this problem. PBE is a not-for-profit organization based in the UK that has conducted studies in conjunction with an organization known as FindACure. It works with specialized economists who volunteer their time and expertise to address a whole variety of different economic issues for other non-profits.

One of the things that PBE needs if it is to proceed with an initiative on the economics of rare diseases is examples of case studies in which other charitable organizations have been able to conduct cost analyses of living with specific rare disorders. At present we know of two rare diseases for which such analyses have been done (alkaptonuria in the United Kingdom and muscular dystrophy in Australia). However, we would encourage readers who know of other, analogous cost analyses to please let NORD and Pro Bono Economics know about such analyses:

Information about such cost analyses should be e-mailed to orphan@rarediseases.org with “Blog” as the subject. We don’t need all the details immediately – just that such an analysis has been done for a specific rare disorder and who to get in touch with about this.

NORD is keen to find ways to support PBE in exploring this potential initiative – in addition to other US-specific initiatives designed to address the current costs of care for rare disorders. Our advocacy mission includes the desire to develop “A social, political, and financial culture of innovation that supports both the basic and translational research necessary to create diagnostic tests and therapies for all rare disorders.” Understanding the economics of current levels of care for those living with rare disorders is a critical component of addressing that mission.

Thanks in advance for any help that you can offer to NORD and to PBE in identifying this type of economic case study.