It is notoriously difficult to reach an individual with a rare disease in the middle of an Eastern European country when their diagnosing physician is in Berlin, their treating physician is 300 miles away and their local pharmacy with no experience in specialist medications is 10 miles away. Robert Donnell, Head of Business Development at Durbin, one of the world’s largest specialist distributors of pharmaceuticals, discusses how you supply drugs to that patient with unmet medical needs.
As patents expire and generics are developed, it is widely acknowledged that the blockbuster (products achieving peak sales of £1 billion or more) mass market model is considerably less profitable than it has been in the past.1 At the same time, regulatory authorities around the world have created incentives for the pharmaceutical industry to develop treatments for rare conditions, often known as orphan diseases.2 These two factors have led to changes in the pharmaceutical industry’s approach to research and development, as well as the subsequent commercialisation of orphan disease drugs.
An orphan disease is a condition that affects fewer than 200,000 people nationwide. There are between 6000–8000 orphan diseases recognised worldwide, of which only 500 have established treatment options.3 Orphan diseases include familiar conditions such as cystic fibrosis and Tourette’s syndrome, as well as less familiar ones such as Hamburger disease.4
Many orphan diseases have as few as a couple of hundred patients around the world and, in extreme cases, it can be even less, such as RPI deficiency wherein only one person has been diagnosed during a 17-year period.5
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