A recent report from technology market research company BCC predicted that the total global market for orphan drugs would rise from $82.6 billion in 2011 to $112 billion by 2015.
Until legislation such as the U.S. Orphan Drug Act of 1983 was passed, treatments for rare diseases were not profitable enough to be pursued by pharma companies, due to the small number of patients involved. Following this and other government measures such as tax breaks, the situation has changed significantly.
For the market access departments of pharma companies, this represents an enormous opportunity, and an enormous challenge at the same time. The relatively high cost of orphan drugs can make it extremely difficult to supply an economic argument - something payers now look for more than ever.
One lesson learned by companies seeking market adoption for orphan drugs is the importance of long-term data. Since the number of patients is relatively small, it’s essential to look at the details in each case.
As one Head of Health Economics working in the area of orphan drugs put it:
To secure funding we have to follow the patients, include them in a registry and look at their outcomes, to assess and confirm the value of the treatments.
As spending on orphan treatments grows by an average of 5.4% per year globally, it’s clear that health economics is increasingly important to the profitability of the rare diseases business unit.