RARE Daily

CF Patient Advocates Launch Global Campaign Targeting Costs of Vertex’s Drugs

February 8, 2023

Rare Daily Staff

A group of cystic fibrosis patient advocates have launched a global campaign aimed at overriding patents held by Vertex Pharmaceuticals on life-saving but costly treatments for the disease in low- and middle-income countries.

The effort led by the grassroots CF community group Vertex Save Us and the patient-led organization Just Treatment, launched the campaign against Vertex, which they charge is putting profits ahead of the lives of patients. They note that World Trade Organization agreements and national laws are designed to ensure intellectual property rights do not threaten citizens’ right to life and they said mechanisms exist that break the company’s patent protections to allow for the production of lower cost generic forms of the medication.

“We had to fight for four years to win access to Vertex’s drugs in the UK. It was only when their monopoly, and the profits it generated, were threatened did we see a deal done,” said Gayle Pledger a CF parent, co-founder of Vertex Save Us, and part of the Just Treatment team. “Today is an important and unparalleled step to challenge their monopoly power in four countries on four continents on one day – but we are in talks with patients and families in lots of other countries. This campaign will keep growing until every CF patient, everywhere has access to these lifesaving medicines.”

Advocates filed papers in the South Africa High Court seeking a compulsory license to allow for the production of generic versions of Trikafta, Vertex’s triple combination therapy for cystic fibrosis. Advocates also filed petitions to revoke or suspend Vertex’s patents to the governments of India, Ukraine, and Brazil.

Cystic fibrosis (CF) is a rare, life-shortening genetic disease. It is caused by mutations in the CFTR gene that lead to a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein. The genetic condition causes a build-up of mucus on the lungs and other organs. Without treatment patients often die in infancy, and life expectancy is just 20.5 years in South Africa, while Indian patients typically die in their teenage years.

Vertex has pioneered a new class of medicines known as CFTR modulators. These have revolutionized treatment and significantly extended the life expectancy of patients in a number of high-income countries. But Vertex patents allow it to price the drugs without fear of competition. Just Treatment said Vertex prices these drugs for as much as $326,239 for Trikafta. The pricing has fueled a controversy in the United States and Europe and fostered global inequalities of access. The group said no low- or middle- income country has been able to agree to a deal to enable patients to access the medicine.

“Quite simply – if CF patients don’t get access to these drugs, they die. Vertex has priced these medicines so high even western healthcare systems are struggling to pay for them – so patients like me and tens of thousands more in poorer countries don’t stand a chance,” said Cheri Nel, a CF patient from South Africa who filed paper in South Africa to seek a compulsory license. “They are making tens of billions of dollars yet haven’t lifted a finger to help CF patients around the world. Worse – they have patented their medicines in countries they are not supplying, like South Africa, blocking patients’ access to more affordable generic versions of the drugs in order to protect their profits.”

A spokeswoman for Vertex told The New York Times that the company has started a product donation program in low-income countries but didn’t specify which lower-resourced countries have access to Trikafta.

“Our teams are working every day to expand access to even more patients around the world through a range of routes, including in low-middle-income countries and low-income countries where access barriers are high due to challenging economic conditions and limited health care infrastructure,” she told the newspaper.

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