Subscribe to enjoy similar stories. Many diseases are the result of choice or circumstance: an unhealthy way of life, toxic living conditions or a chance encounter with a virus or bacterium. Others are predestined—because they are written in the genes.
Millions of people worldwide suffer from the most common genetic diseases, such as sickle-cell anaemia or thalassemia, two blood disorders. A long tail of rarer conditions each afflict an unfortunate few, who add up to many millions in total. Twenty years ago most patients had little hope of a treatment.
But exciting developments in genomic medicines are now promising to treat, and potentially cure, horrible and sometimes fatal genetic diseases. Gene-editing, for example, uses enzymes to snip a patient’s DNA precisely where needed and make a repair to harmful mutations. The technologies are mind-bending, but their cost, sadly, is bank-breaking.
At over $3m a time, several gene therapies count as the most expensive drugs ever approved. Even wealthy countries are struggling to absorb their costs. The Aspen Institute, a think-tank, reckons that by 2031 America may spend a combined $30bn a year on gene and cell therapies covering 550,000 people.
That would be roughly ten times the average cost per patient of America’s current prescription-drug bill. If they are to realise their potential, these medical innovations may therefore demand financial innovation, too. Pharma firms insist that the price is justified.
They correctly point out that creating new medicines is colossally expensive and fraught with risk. Once invented and approved, these treatments are also unusually fiddly to make. Gene therapies have been compared to “snowflakes", because each batch is unique.
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