Lacking access to an effective-yet-costly cystic fibrosis (CF) therapy from Vertex Pharmaceuticals, patients and activists in four countries are attempting to take matters into their own hands.
Monday, a coalition of CF patients and their families, helmed by Vertex Save Us and U.K.-based patient advocacy group Just Treatment, petitioned governments in South Africa, Brazil, India and Ukraine to either revoke or suspend Vertex patents surrounding the company’s Trikafta.
Also known as Kaftrio, the Vertex medicine is a combination of elexacaftor, ivacaftor and tezecaftor. It works by helping defective cystic fibrosis transmembrane conductance regulator (CFTR) proteins work more effectively.
But the CF modulator is out of reach for patients across “all low- and middle-income countries,” as well as some high-income countries, Diarmaid McDonald, director of Just Treatment, said in an email.
Even in countries where the drug is available, like the U.S., Trikafta’s cost before discounts is prohibitively expensive, clocking in at roughly $326,000 per patient per year, according to McDonald.
The initiative’s goal is to “secure affordable generic access to these extremely expensive medicines,” McDonald explained.
The Boston-based company, for its part, aims to “provide our medicines to as many CF patients around the world as possible,” a Vertex spokesperson said over email.
Vertex has inked formal reimbursement agreements in more than 40 countries outside the U.S., and its CF medicines have been used by patients in more than 50 countries, the spokesperson added. “We continue to work actively to expand access including in lower-income countries, recognizing the complexities and access challenges in these markets.”
The activist coalition’s strategy mainly hinges on compulsory licenses, a World Trade Organization provision that allows governments to permit someone else to produce a patented product or process without the consent of the patent owner.
That’s the route the patients and activists are taking in South Africa, Ukraine and Brazil, where petitions were submitted Monday. If granted, those governmental bids could secure access to more affordable generics of Trikafta.
The group is taking a slightly different approach in India, where it’s asked the government to revoke Vertex’s patents on CFTR modulator drugs.
Vertex, for its part, believes “intellectual property rights are critical to encourage and protect innovation," the company's spokesperson said.
Beyond this latest transcontinental campaign, Vertex is no stranger to pricing criticism.
Notably, the company butted heads with the United Kingdom for four years over the cost of its CF drug Orkambi, which combines ivacaftor and lumacaftor. That pricing spat ultimately saw Vertex's top brass summoned to participate in a parliamentary hearing focused on pricing negotiations.
Off-and-on negotiations between Vertex and U.K. health officials over Orkambi pricing lasted so long that the drugmaker had to destroy almost 8,000 packs of the medicine that expired in 2018.
Last year, meanwhile, the Journal of Cystic Fibrosis issued a report suggesting just 12% of some 162,000 people with CF worldwide were receiving Vertex’s Trikafta. The researchers called CFTR modulators like Trikafta an “unparalleled opportunity to increase quality and length of life for almost all CF patients,” but cautioned that “the medicines are so expensive they are essentially unavailable unless reimbursed by the government or health system authorities.”
https://www.fiercepharma.com/pharma/vertexs-trikafta-pricing-under-pressure-again-patients-and-activists-press-4-governments
