Bringing gene therapies to market

Doug Danison, vice-president, head of access, value and evidence strategy at bluebird bio, a gene therapy company, looks at the obstacles, opportunities and required change in thinking needed to ensure patients have access to innovative personalised medicines.

Rare diseases are more common than you might think. The World Health Organization (WHO) gives the rough estimate that about one in fifteen people worldwide could be affected by a rare or orphan disease. This equates to 400 million people worldwide, with 30 million in Europe and 25 million in the United States. These 400 million are affected by one of the between 5,000–8,000 rare diseases, some of which we know more about than others and most of them with a genetic basis. Many of the diseases in this group have a deep impact on the lives of patients and their families, and very few have any form of approved treatment available.

As our understanding of these diseases has grown in parallel with developments in genome sequencing, so has our understanding of how to potentially treat them using personalised gene therapies.

Treatments such as those recently launched to treat cancer are ground-breaking in themselves, but represent only the very tip of the iceberg. With many personalised therapies in the pipeline for a wide variety of different conditions, the next few years are set to be busy for the regulators charged with assessing these novel treatments. We are potentially on the cusp of gene therapy becoming more widely available to patients.

• Read the full article in pharmaphorum's Deep Dive digital magazine here