Right to breathe takes precedence over company profits: Families of cystic fibrosis patients seek access to life-saving drug in face of company intransigence

“Not even the doctors believed it would be so miraculous,” says 22-year old Rafaella Pereira. In two days, a new cystic fibrosis medicine took her from being so ill she was bed-ridden and on oxygen, and deemed too ill to survive a needed lung transplant, to being able to leave the house without oxygen. Eventually, she was deemed healthy enough to no longer need a lung transplant. Unfortunately, getting access to this medicine in Brazil, where she lives, was not easy. She had to take legal action.

Now a group of health advocates, including Ms Pereira, are launching a campaign to make sure that no one else needs to go to such great lengths for life saving medicine. “It does not make sense that only I have access,” she said; and others should not “have to be as sick as I was” to access this medicine.

“Hundreds of thousands of patients are being put at risk due to abuse of monopoly and high prices,” said Diarmaid McDonald, founder and director of Just Treatment, which is working with the global cystic fibrosis patient coalition “Vertex Save Us” and several other advocacy groups on the campaign.

The medicine in question, made by company Vertex Pharmaceuticals, retails for the prohibitively high $300,000 per person per year in the US. It is a combination of three different molecules (elexacaftor/ivacaftor/tezacaftor) that help modulate a badly functioning protein that causes cystic fibrosis. Cystic fibrosis is a rare genetic condition; it usually begins in childhood and sufferers usually deal with progressive lung disease and risk of respiratory failure. 

Vertex has secured patent rights even in countries where it has no intention of selling its original product: notably, it has not submitted drug registration information in India, South Africa, or Ukraine. 

This creates a “really problematic situation,” noted Olga Gurgula, a Ukrainian lawyer teaching in the UK, at a press conference, adding it is an “abuse of the patent system” for a company to neither provide treatment nor allow others to do so.

The campaign seeks to push for lower-cost generic versions of the medicine, by pressuring Vertex to drop its patent claims in low- and middle-income countries as well as by lobbying governments to issue compulsory licences if Vertex prices remain high.

The campaign is focusing on four countries where access has been a particular challenge: Brazil, India, South Africa, and Ukraine. In South Africa and Brazil, compulsory licences are being sought. In India, a coalition of patients are seeking to revoke Vertex’s patents on grounds that Vertex has failed to work the patent (i.e., supply the medicine) in India, as well as for unjustifiably high prices. In Ukraine, either a compulsory licence or a waiver of all intellectual property (IP) on the medicines is being sought. Additionally, there is an effort to temporarily suspend IP rights during the war using the security exception provided for in the World Trade Organization’s Trade-Related Aspects of Intellectual Property Rights (TRIPS) Agreement. 

This is not the first time that Vertex has come under fire from health activists – or governments – for abusive pricing. In 2019, the company was the subject of a ‘Crown use’ compulsory licence in the UK after the company turned town a generous £500 million offer from the National Health Services for its Orkambi, an earlier cystic fibrosis treatment, which was retailing at £104,000 per patient per year.

“It is not for the pharma industry to decide who lives and who dies in order to maximise its profits,” said Brazilian attorney Susana van der Ploeg. We agree and wish their campaign success. 

Full details of the campaign can be found here.

A letter sent to Ambassador Katherine Tai, the US Trade Representative, about the campaign can be found here.

Right to breathe takes precedence over company profits: Families of cystic fibrosis patients seek access to life-saving drug in face of company intransigence

“Not even the doctors believed it would be so miraculous,” says 22-year old Rafaella Pereira. In two days, a new cystic fibrosis medicine took her from being so ill she was bed-ridden and on oxygen, and deemed too ill to survive a needed lung transplant, to being able to leave the house without oxygen. Eventually, she was deemed healthy enough to no longer need a lung transplant. Unfortunately, getting access to this medicine in Brazil, where she lives, was not easy. She had to take legal action.

Now a group of health advocates, including Ms Pereira, are launching a campaign to make sure that no one else needs to go to such great lengths for life saving medicine. “It does not make sense that only I have access,” she said; and others should not “have to be as sick as I was” to access this medicine.

“Hundreds of thousands of patients being put at risk due to abuse of monopoly and high prices,” said Diarmaid McDonald, founder and director of Just Treatment, which is working with the global cystic fibrosis patient coalition “Vertex Save Us” and several other advocacy groups on the campaign.

The medicine in question, made by company Vertex Pharmaceuticals, retails for the prohibitively high $300,000 per person per year in the US. It is a combination of three different molecules (elexacaftor/ivacaftor/tezacaftor) that help modulate a badly functioning protein that causes cystic fibrosis. Cystic fibrosis is a rare genetic condition; it usually begins in childhood and sufferers usually deal with progressive lung disease and risk of respiratory failure. 

Vertex has secured patent rights even in countries where it has no intention of selling its original product: notably, it has not submitted drug registration information in the Ukraine. 

This creates a “really problematic situation,” noted Olga Gurgula, a Ukrainian lawyer teaching in the UK, at a press conference, adding it is an “abuse of the patent system” for a company to neither provide treatment nor allow others to do so.

The campaign seeks to push for lower-cost generic versions of the medicine, by pressuring Vertex to drop its patent claims in low- and middle-income countries as well as by lobbying governments to issue compulsory licences if Vertex prices remain high.

The campaign is focusing on four countries where access has been a particular challenge: Brazil, India, South Africa, and Ukraine. In South Africa and Brazil, compulsory licences are being sought. In India, a coalition of patients are seeking to revoke Vertex’s patents on grounds that Vertex has failed to work the patent (i.e., supply the medicine) in India, as well as for unjustifiably high prices. In Ukraine, either a compulsory licence or a waiver of all intellectual property (IP) on the medicines is being sought. Additionally, there is an effort to temporarily suspend IP rights during the war using the security exception provided for in the World Trade Organization’s Trade-Related Aspects of Intellectual Property Rights (TRIPS) Agreement. 

This is not the first time that Vertex has come under fire from health activists – or governments – for abusive pricing. In 2019, the company was the subject of a ‘Crown use’ compulsory licence in the UK after the company turned town a generous £500 million offer from the National Health Services for its Orkambi, an earlier cystic fibrosis treatment, which was retailing at £104,000 per patient per year.

“It is not for the pharma industry to decide who lives and who dies in order to maximise its profits,” said Brazilian attorney Susana van der Ploeg. We agree and wish their campaign success. 

Full details of the campaign can be found here.