Vertex deal for Scotland — no deal for England
Cystic fibrosis (CF) drug maker Vertex Pharmaceuticals — which is still locked in negotiation with NHS England to endorse the use of its medicines — has successfully negotiated a deal with Scottish authorities.
A month ago, the Scottish Medicines Consortium spurned two of the company’s medicines — Orkambi and Symkevi — citing uncertainty over their long-term efficacy in relation to their cost.
Each drug carries a list price of more than £100,000 per patient per year. The UK has more than 10,400 cystic fibrosis patients – the largest CF population outside the US, according to the Cystic Fibrosis Foundation.
But on Thursday, Vertex announced a deal had been reached with Scotland. The price negotiated for the treatments is confidential, but as part of the five-year agreement Vertex will collect real-world evidence on the drugs that support any future submissions, the company said, adding that about 400 out of the 900 CF patients in Scotland carry the mutations addressable by Orkambi or Symkevi.
“We celebrate the news in Scotland today, but our campaign must continue to focus on Vertex and all parties in England, Wales and Northern Ireland. Those in need of the drugs have already waited too long and we must ensure that thousands more people are not subjected to a postcode lottery. Scotland’s success must now be replicated across the UK without further damaging delay,” Cystic Fibrosis Trust chief David Ramsden said in a statement.
The cystic fibrosis drugs made by Vertex $VRTX are the first treatments that address the underlying genetic causes of cystic fibrosis, which is characterized by a thick sticky mucus in the lungs, digestive system and other organs that reduces life expectancy. In its second-quarter results — published in late July — Vertex’s trifecta of CF medicines combined generated nearly $1 billion in sales, up 25% from the preceding quarter. The FDA is also reviewing the Boston biotech’s three-drug cocktail for CF, which is expected to treat 90% of CF patients.
Vertex has been haggling with UK’s cost-effectiveness watchdog NICE, which has refused to allow the drug into England’s NHS until Vertex offers it a discount on the treatment’s price tag that would compel the agency to look favorably upon its cost-effectiveness. Earlier this year, NHS England offered Vertex £500 million over five years and £l billion over the next 10 years to access the company’s medicines — but the US drugmaker rejected the offer. Negotiations are still ongoing.
To the horror of UK cystic fibrosis patients, a Vertex executive disclosed that last year close to 8,000 packs (each containing a 28-day supply) of the company’s treatment, Orkambi, were destroyed after crossing their expiry date. In a standoff with UK parliament in March, Vertex chief Jeff Leiden stood his ground, despite being chastised by a plethora of MPs for Vertex’s pricing strategy, business model and ethics.
Social image: Vertex