Talks with England drag on; Scotland nixes Vertex cystic fibrosis drugs routine use

As the battle between Vertex and England’s National Health Service (NHS) continues — the Scottish NHS has rejected two of the drugmaker’s cystic fibrosis medicines.

The US drugmaker has been locked in negotiation with NICE, which has refused to allow the drug in to England’s NHS until Vertex $VRTX offers it a discount on the treatment’s price tag that would compel the agency to look favorably upon its cost-effectiveness. On Monday, the Scottish NHS spurned two of the company’s medicines — Orkambi and Symkevi — for the same reason, to the dismay of thousands of patients.

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.
Campaigners in the United Kingdom have been coaxing the NHS to cover Vertex’s CF drugs but to no avail. To the horror of UK cystic fibrosis patients, a Vertex executive disclosed in March, 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.
The cystic fibrosis drugs made by Vertex 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.

The company is disappointed that Orkambi and Symkevi have not been recommended for routine use in Scotland, a Vertex spokesperson told Endpoints News, adding that since learning about the decision, Vertex is working with the Scotting government to allow “broad access” for all eligible patients — which the drugmaker hopes to settle in the coming weeks.

“Vertex will continue to provide access to our medicines via the PACS Tier 2 process — which allows clinicians to apply for access for individual patients based on clinical need — and was put into place prior to Vertex’s submission in Scotland. So far, about 65 patients have been approved to receive these medicines under this scheme,” the spokesperson added.

Apart from its ruling on Vertex’s medicines, Scotland’s cost-effectiveness watchdog — the Scottish Medicines Consortium — has made the decision to back Akcea Therapeutics’ drug for hereditary transthyretin-mediated amyloidosis, Tegsedi; AbbVie’s blood cancer drug, Venclyxto; Camurus’ treatment for opioid dependence, Buvidal, in a restricted capacity; and Almirall’s therapy for plaque psoriasis, Ilumetri, for limited use.

As negotiations with England labor on, Scotland rejects routine use of Vertex’s cystic fibrosis drugs