Glybera, the first gene therapy drug that has been approved by European regulators, is set to launch in Germany in early 2015 with a 1.1 million euro (U.S. $1.4 million) price tag, far surpassing the $440,000 annual price tag of Alexion’s Soliris, the previous most expensive drug. Gene therapy treats diseases caused by a mutation in a gene using a modified virus to insert a corrected copy of the gene into a person’s cells. Because gene therapy provides a working copy of a gene, it offers the possibility of a long-term, possibly permanent, cure; in the case of Glybera, benefits have been noted for up to six years so far.
Glybera is a treatment for lipoprotein lipase deficiency (LPLD), a rare genetic disorder affecting about 1-2 persons per million people (or about 150-200 people in Europe) that causes fat to build-up in the blood leading to cardiovascular disease, diabetes, and life-threatening, recurrent bouts of pancreatitis. The only prior treatment option was severe dietary restrictions and symptomatic treatment of pancreatitis. Glybera, developed by Dutch biotech firm UniQure and to be marketed by its partner Italian firm Chiesi in the European Union, has been given orphan drug status in the European Union and the United States.
The pricing of Glybera, which may only need to be given over one treatment course, was designed to match the annual cost of treating other lysosomal-storage diseases similar to LPLD with enzyme replacement therapies. Yet, the high price of Glybera, along with the prices for other drugs for rare diseases and for some new cancer therapies, is raising concerns that these drug costs are not sustainable in the long term. However, as is the case with Glybera, patients often have no other treatments and receive significant improvements in health and in quality of life from new therapeutics. Healthcare costs and benefits cannot be easily analyzed when the focus is only on the pricing of individual drugs or even on costs within individual healthcare sectors. Other options such as improving benefit-risk assessments, assessing more cost-effective means of delivering clinical care, and even exploring new financing models are critical to explore.