The problem
In 2015, Vertex Pharmaceuticals gained regulatory approval for its breakthrough cystic fibrosis drug, ORKAMBI®.
In 2018, some 5,000 English patients with this debilitating and life-threatening condition were still denied access.
Why?
Well, Vertex, it had spent millions developing ORKAMBI® and bringing it to market. Initial data showed it had the potential to transform patient lives, and they were asking NHS England for what they believed was a fair price.
Understandable.
NHS England, on the other hand, with a limited budget, were being asked to assess ORKAMBI®’s benefits based on only a few years of clinical data and, unable to take the risk of wasting money, they asked for a discount, a big discount.
Understandable.
The result? Three years of painful price negotiations and delay. For Vertex, three years of missed peak annual sales. For patients, huge frustration, and three years of unnecessary suffering.
That's just one example of a huge industry problem.
Some twenty years ago, the Human Genome Project completed heralding the genomics revolution, and now mankind is beginning to reap its benefits.
A tsunami of Advanced Therapy Medicinal Products (ATMPs); cell therapies and gene therapies, as well as other breakthrough medicines (many transformative and potentially curative), is on the horizon. However, many of these medicines are being approved for use based on emergent and extrapolated data.
The health and economic benefits of these medicines are potentially huge but, if they are to be made available to patients, we must resolve the problem, the problem of pricing.