At €1 million a pop, expectations are high.  [[wysiwyg_imageupload:134:]]
But European regulators have given the initial thumbs up to Glybera (alipogene tiparvovec), the western world’s first gene therapy, paving the way for approval by the European Commission.

The treatment is for a rare type of pancreatic disorder in which patients have damaged copies of a gene which is essential for breaking down the fat content in foods.  Affecting one person in a million, the disease causes a build up of lipids in the blood stream, leading to abdominal pain and potentially fatal inflammation of the pancreas.

The decision marks an important advance for gene therapy, a field which has been hotly debated for many years due to shortcomings of previous investigational therapies.  Approval for Glybera, which was previously rejected on three occasions, will make it the first gene therapy to be available outside a clinical trial in Europe or the US.  China approved a gene therapy for head and neck cancer in 2004.

The aim of gene therapy is simple: to replace faulty genes with healthy copies.  But the reality has not been so straightforward.  Many early treatments were abandoned because they failed to deliver healthy genes to the right cells in sufficient numbers, and some very early trials were halted after patients died when therapies switched on the wrong genes.  Glybera works by using an inactivated virus to transport healthy genes to a patient's muscle cells. Once in place, the gene helps the cells to manufacture the enzyme needed to break fat down.

Made by a Dutch biotechnology company, Uniteq, Glybera will be the world’s most expensive medicine.  The company has defended the price on the basis of high development costs coupled with the fact that the therapy will be limited to just a few hundred patients in Europe.  Uniteq has also emphasised the longevity of the therapeutic benefit, as well as the potential cost savings on reduced hospital admissions and treatment of complications for patients.

What does this development mean for us? 

Gene therapy is a new and exciting field that’s likely to attract increasing interest in the coming years across many different disease areas, and therefore an important field for healthcare communicators to be aware of.  The good news is that there is likely to be a keen appetite among both scientific and consumer media for such novel and cutting edge technology. 

Clear education using simple messaging, compelling visuals and engaging animations will be needed for the media to help them understand the challenges and complexities of the field, and enable them to appropriately contextualise every step forward.  It’s also essential that expectations are not inappropriately raised among potential patients and their loved ones.  Progress is slow and gene therapy is currently prohibitively expensive to be considered as a mainstream treatment, and is likely to remain so for at least the next decade.  Continued austerity in Europe means the availability of treatment is likely to be restricted to relatively few patients who meet specific clinical criteria.  Finally careful messaging will be required to explain and contextualise the value story for gene therapies to encourage acceptance of the eye-watering price tag.