Parents of a toddler in Europe with the most severe form of spinal muscular atrophy used crowdfunding campaigns to raise $2.7 million to access Zolgensma, a lifesaving treatment, according to Politico.
Zolgensma, made by Novartis, is the world's most expensive drug and is not yet authorized in Europe. It is designed to treat spinal muscular atrophy, a rare genetic disease that causes muscles to deteriorate and affects less than 10,000 people in the European Union. Most children die within a couple years of diagnosis.
Zolgensma was approved in the U.S. in May and is the closest thing to a cure for SMA.
Since it is not yet approved in Europe, and the government does not provide reimbursements for the drug, SMA patients have had to use crowdfunding campaigns to raise money to get the drug for their children.
Other parents in countries including Belgium, Slovenia, Greece, Hungary, Cyprus and Estonia have taken similar measures to access Zolgensma for their children, according to Politico.
Portugal, France and Germany have agreed to pay under provisional agreements allowing patients access to drugs that treat life-threatening diseases before they're approved in Europe, according to Politico. Other countries, such as Estonia, have refused to reimburse Zolgensma before it hits the market, arguing it's unaffordable.
Read the full article here.
More articles on pharmacy:
Novartis pays $9.7B to acquire Medicines Co.
Drug importation plan will be released 'soon,' Trump tweets
From data analytics to biosimilars — How 5 pharmacy leaders are tackling drug pricing