Gene Therapy is Finally Here, But Who Will Foot the Bill

Biopharmaceutical companies are struggling with this next challenge as treating rare disorders through the technique of gene therapy may be an economical impossibility for many.


Human protein-coding genes number from 20,000 up to 25,000.

If just one of these genes gets altered or a code gets missing, it can be fatal to an individual.

In fact, approximately 30 per cent of infant mortality at birth in developed countries are caused by genetic disease. Almost 50 per cent of all miscarriages worldwide are due to chromosomally defective fetus.

Furthermore, according to the World Health Organization, over 10,000 human diseases are linked to single gene mutation alone. Among these monogenic diseases are thalassaemia, sickle cell anemia, haemophilia, Fragile-X syndrome, cystic fibrosis, and Huntington’s disease.

The other two major types of genetic disorders are chromosomal and complex disorder, where there’s mutation in two or more genes.

Genetic disease is not also simply inherited, our environment is another factor that can trigger mutation. Cancer, diabetes, and heart disease are classified as multifactorial inheritance genetic disorders.

Considering all these, one would expect that the world will be welcoming the revolutionary gene therapy with wide-open arms.

Yet, UniQure’s Glybera has been recently withdrawn from the European market in spite of its promising one-time cure for lipoprotein lipase deficiency (LPLD).

LPLD is a rare genetic disorder characterized by the body’s lack of lipase, which is an enzyme that breaks down triglycerides from the blood. The deficiency results to recurrent abdominal pain, fat deposits in the skin (xanthomata), and repeated attacks of acute pancreatitis. LPLD is known to affect one person in a million. However, UniQure’s Glybera costs as much as $1 million per patient. Since the drug’s introduction in 2012, only one patient has been subscribed to the treatment.

Another genetic drug that offers one-time cure for Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) is GlaxoSmithKline’s Strimvelis. ADA-SCID is an inherited genetic condition characterized by a damaged immune system. People with SCID are prone to persistent and recurring infections since they absolutely have no immune protection from microbes. Symptoms begin to appear in a baby’s first 6 months of life, and afflicted infants hardly reach two years of age without treatment.

GlaxoSmithKline’s Strimvelis can cure the genetic disease and save precious lifes. But the $700,000 drug had only a couple of sales in 2016 and another two expected this year. With this disappointing development, GSK might simply sell its rare diseases unit.

Data shows that the prices of the current gene therapy in the market are too hard if not impossible for most families to reach, especially since it has to be a one-time payment. And health care systems which only pay on monthly basis are not of much help to pharmaceuticals, which have made such enormous investments to formulate genetic cures.

Is there real hope?

Many drug companies still think so.

Pfizer, Sanofi, and Shire are now also making the revolutionary pursuits. And GSK has not completely given up as it strives to use its gene therapy platform in the development of cure for more common genetic illnesses.

Yes, at the moment, the whole picture may appear dim. But, by creating new business models, the leading companies in the biopharmaceutical industry if they are really serious about doing something in relation to rampant increases in drug prices, can start by creating a business model which is first based on humanism and then their respective bottom lines.

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