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Getting Orkambi on the NHS is a bittersweet victory, but the fight goes on

AFTER a long and fraught four years, an agreement between Pharma company Vertex and the NHS has finally resulted in Orkambi and Symkevi, the “game-changing” cystic fibrosis drugs, being made available on the NHS. 

Vertex were originally asking £100,000 per patient, per year for Orkambi, which the NHS couldn’t afford, resulting in a protracted price negotiation that above anything else illustrates a system which requires fundamental change.

This conflict between private companies and their need to give returns to their shareholders operating in the medical arena where much more is at stake was highlighted by Just Treatment patient leader Emily Birchall who said: “This is absolutely brilliant news for patients and for all the families who have fought so hard to access Orkambi and other medicines for cystic fibrosis over the last four years.

“However, four years is an unacceptable wait for people who so desperately need treatment,” Birchall said: “In this time, hundreds have died and many more have suffered irreversible lung damage and disease progression. So, while we’re celebrating this news today, this last four years is proof again that the system doesn't work — we need to make sure that access to medicine is never again down to the whim of a private company.”

The Morning Star spoke to two families in January, when talks at that point in time had broken down between Vertex and the NHS. These parents described the gruelling regimes their children had to undergo each and every day and their fragile health. They knew Orkambi could help in 50 per cent of cases — where those with cystic fibrosis who had two copies of the F508 mutation — slowing down the deterioration in lung function by up to 42 per cent. Yet, even though the European Medicines Agency had approved Orkambi, and Nice had described it as “important and effective,” it was still only accessible in “compassionate cases” where lung function was already below 40 per cent.

One of these parents described what Orkambi would mean for her and her son. She said: “It is a lifeline, a light of hope…It gives us the ability to stop the clock on cystic fibrosis. It could mean his lung function and liver won’t deteriorate further, that he could gain and sustain weight without the need for supplements.”

Families affected were critical of Vertex who put profits before patients, but also how the NHS had handled the negotiations. This was echoed at the time by Rachel Gaskell MP who criticised the system devised by Nice as unethical arguing that the decision made about Orkambi was both “economically and clinically illiterate.”

Tabitha Ha, an advocacy manager at STOPAIDS, said: “This announcement is welcome but long overdue. The agreement between the NHS and Vertex hides the final price of these medicines. If Health Secretary Matt Hancock genuinely wants to push for affordable medicines in Britain and across the world, then we need to know the prices of medicines and the true costs of research and development, including the contributions of British taxpayers to research. Keeping this information in a black box only works to serve the profit-driven interests of Big Pharma. Britain has promised to improve transparency of medicines prices, but we are waiting to find out if, when and how this will happen.”

Global Justice Now’s Radhika Patel says: “Government inaction is at the heart of this four-year-long wait for patients. Issuing a Crown Use Licence, a legal tool at their disposal to access fairly priced medicine, should have been the default in order to get cheaper generic versions that patients desperately needed. Corporate monopolies over medicines like Orkambi should never have come before the needs of the patients.

“This has been just one case of many around the world: Bedaquiline has been recommended by the World Health Organisation as a breakthrough medicine to treat multidrug resistant tuberculosis. Yet because of its hefty price tag only 2 per cent of patients who could benefit from it (many of whom live in the Global South) have been treated with it. The pharmaceutical system needs urgent reform to ensure patients everywhere get the medicines they need.

“The power and greed of big pharmaceutical companies repeatedly comes above patient access. We are facing a crisis in high-priced medicines here and around the world.’

Just Treatment  can be found at

STOPAIDS can be found at 

Global Justice Now can be found at and the letter urging the UK government to support the transparency resolution can be found at

Ruth F Hunt is an author and freelance journalist


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