Development of drugs for rare diseases is extremely small patient population. This also resulted in extremely high price of rare disease therapies. How to measure the cost-effective therapy for rare diseases is high or low is placed in front of the world pharmaceutical administration is an important issue, and orphan drug prices with
pharmaceutical companies is the focus of the Governing Body Game. Recently, the British NICE price management department in considering whether to grant Alexion developed orphan drug for the treatment of infants Kanuma lysosomal acid lipase deficiency (LAL deficiency). However, NICE managers think we need more data to make definitive conclusions.
Previously published a draft NICE believes the therapy of up to 491,992 pounds ($ 704,630) price is too high, is not suitable to be generalized to all patients. However, Alexion is still plenty of time to persuade NICE to change his mind. Lack of lysosomal acid lipase disease more common in patients with a baby due to lack of lysosomal acid lipase, patient cells will accumulate excess fatty acids. Survival of sick babies often less than 12 months.
Alexion said, unfortunately NICE failed to see the seriousness of this disease. However, the company's requirements for how to deal with NICE also have experienced. December 2014, the company finally persuaded NICE approval of another rare disease treatment with atypical hemolytic-uremic syndrome drug Soliris for launch in the UK market. In this case, Alexion's Soliris company did not provide data to the R & D funding NICE. However, a year later, another drug company Strensiq not so good luck. Such a medicament for the treatment of rare hypophosphatasia each course cost more than $ 500,000, eventually NICE labeled low price tag and turned away. Today, Alexion carry Kanuma knock the door once again the British market, but also come up with their sincerity.
