Vertex Offers England a Portfolio Approach for Access to Orkambi and Future CF Therapies

Vertex Pharmaceuticals is offering the England arm of Britain’s National Health Service a package deal on the pricing of its current and future cystic fibrosis therapies.

What the company describes as “a bold new portfolio approach” to pricing comes in the wake of the National Health Service balking at covering the approved therapy Orkambi (lumacaftor/ivacaftor) over cost concerns. The U.S. price of Orkambi (lumacaftor/ivacaftor) is $272,000 a year, Forbes magazine reported in late 2017.

Britain’s National Health Service is covering another approved Vertex therapy, Kalydeco (ivacaftor), whose U.S. cost Forbes pegged in late 2017 at $311,000 a year.

The Cystic Fibrosis Trust, the U.K.’s biggest CF patient advocacy group, hailed Vertex’s portfolio proposal as a step in the right direction.

The company said the aim of its portfolio approach, details of which have yet to surface, is to facilitate patient access to both current and future treatments.

It said the approach should give eligible patients fair and equal access to its therapies while providing the National Health Service with value and budget certainty.

“We share the CF community’s sense of urgency on access to new Vertex medicines, and we are committed to working with the NHS [National Health Service] to find a sustainable funding solution,” Vertex announced.

The company’s offer is similar to one that Ireland adopted. It covers Orkambi, Kalydeco and any future Vertex therapies that regulators approve.

Vertex is conducting Phase 3 trials of triple-combo therapies that include its CFTR correctors VX-659 and VX-445.

And the U.S. Food and Drug Administration recently approved Symdeko (tezacaftor/ivacaftor and ivacaftor) as a treatment for children with cystic fibrosis who are 12 or older. The authorization applies to children with an F508del mutation in their CFTR gene.

“At Vertex, we are developing medicines to treat 90 percent of CF patients,” the company said. The therapies will address the underlying cause of the disease, it said.

Although Vertex has offered a portfolio proposal only to England’s arm of the National Health Service, it is also talking with officials in Scotland, Wales, and Northern Ireland.

“After over a year of waiting, this proposal from Vertex is an important step in gaining access to Orkambi for people with CF in the U.K.” David Ramsden, the chief executive of the Cystic Fibrosis Trust, said in a press release.

“We also urge the government to consider this offer seriously and to recognize its significance not just for Orkambi, but for other new, innovative treatments in the CF development pipeline,” he said. “We will keep pushing to see progress across the whole of the U.K., and will not stop fighting until all who are eligible have access as soon as possible.”

The trust is asking patients to help make Vertex’s proposal a reality by sharing their story of living with cystic fibrosis. A parliamentary roundtable on the medicines is scheduled for Feb. 28 and a debate March 19.

“We call on everyone affected by CF to help us ensure these discussions are robust, and ask those willing to help to continue to tell their stories” to members of Parliament and other officials, Ramsden said. This will help “ensure that U.K. governments take responsibility for making progress in negotiations” over access to the treatments, he said.

To learn more about participating in the campaign, please visit Your voice: Orkambi and new medicines for cystic fibrosis.