With the Orphan Drug Act, the companies are able to get tax benefits as these drugs address diseases with small pool of patients and with very few other treatment options available. The average cost per patient in year 2016 for an orphan drug was $140,443 versus $27,756 for a non-orphan drug. For example, a drug that treats ultra rare CLN2 disease, Brineura by BioMarin is priced at $702,000. Another example is Ravicti by Horizon Pharma which is used in treatment of urea cycle disorders and is priced at $793,632.
According to World Preview report, orphan drug sales are projected to represent 32% of pharmaceutical growth through 2022. It is expected to add $95 billion more to the sales by 2022.
This greater interest of companies can be attributed to two main reasons –
- drug companies can address a greater unmet need and
- they can win development incentives for advancing medicines to treat rare diseases.
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