Three factors to consider when working with rare diseases
Roland Gerke, Immedica Pharma in Germany, Austria, and Switzerland has over 20 years of experience serving patients with rare diseases. Over time he has accumulated extensive experience and knowledge. Now, he shares three key factors to be successful in the business.
The search for the “needle in the haystack” requires extraordinary employees.
An orphan drug company aims to meet the medical needs of patients that are very few and can be difficult to find. Only a few hundred patients in the DACH region are affected by a rare disease, and only a handful of medical specialists exist who can diagnose and treat these patients. For orphan drug companies, this requires particularly skilled employees who can master the many different aspects and requirements of rare diseases with passion and experience.
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"Working with rare diseases is not an ordinary job, it is a vocation. So, how do you succeed to help as many people as possible?"
The diseases are rare, but the drugs should ways be available.
As an orphan drug company, you are usually the only provider of essential therapy. The patients – mainly suffering from congenital genetic disease – are therefore highly dependent on the reliability of the drug supply in every situation. For an orphan drug company, this means a high level of responsibility to avoid delivery delays and stock-outs, even in the challenging times of a pandemic.
Newborn screening – the first step to success.
Orphan diseases are always about very few largely underdiagnosed patients. It means they often suffer from consequential damages that could have been avoided if diagnosed in good time. These consequential damages are often irreversible. In the past, treating physicians, patient organizations, and orphan drug companies had to invest a lot of money and efforts into awareness campaigns and market research to find these patients. Advances in newborn screening will help diagnose and treat these patients more quickly in the future.