Rare disease drugs and cost

The attempt to make them cheaper


Allison Moore, Founder Hereditary Neurotherapy Foundation, has CMT
Allison Moore, Founder Hereditary Neurotherapy Foundation, has CMT

Pharnext is a French drug maker developing a treatment for a ‘close cousin’ disease of HSP, namely Charcot-Marie-Tooth (CMT). CMT is an inherited muscular atrophy condition resulting in debilitating muscle weakness similar to HSP. The company is determined to keep any drug treatment it produces affordably priced.


If Pharnext succeeds in its plans, it will charge $20,000 to $60,000 for annual treatments — a hefty sum, but considerably less than the kinds of drug prices that have triggered allegations of price-gouging among patients and consumer advocates. It could also show other drug makers that it’s possible to drastically reduce research-and-development costs to produce breakthrough treatments, which in turn would allow them to charge less.


Pharnext has “been a game-changer,” said Allison Moore, who has Charcot-Marie-Tooth, or CMT, and is the founder of the Hereditary Neurotherapy Foundation. “I do think that they will deliver on the pricing,” she said. “If the pricing can be low, that can also be a trend for these other rare diseases.”


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SOURCE: http://www.statnews.com/2016/01/20/pharnext-rare-disease-charcot-marie-tooth-disorder/


This biotech company is trying to make a rare disease drug cheaper. Will it work?


By Dylan Scott, January 20, 2016



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