11/28/23, 2:38 PM Vertex pressured to expand access to cystic fibrosis treatments https://www.statnews.com/pharmalot/2023/11/28/vertex-cystic-fibrosis-medicines/ 1/5 Skip to Main Content Cyber Week: Get 50% off 12 months of STAT+. Reporting from the frontiers of health and medicine Reports E-Books Don't miss out Subscribe to STAT+ today, for the best life sciences journalism in the industry Learn more Subscribe Now To access exclusive content, subscribe to STAT+ View Latest View the latest STAT+ stories 1 Vertex pushes a donation program to widen access to cystic fibrosis treatments, but advocates say it’s not enough By Ed Silverman 2 3 Nov. 28, 2023 11/28/23, 2:38 PM Vertex pressured to expand access to cystic fibrosis treatments https://www.statnews.com/pharmalot/2023/11/28/vertex-cystic-fibrosis-medicines/ 2/5 V Bill Sikes/AP ertex Pharmaceuticals is being accused by a group of advocates and physicians of refusing to take several steps that would widen access to its pricey cystic fibrosis medicines in predominantly poor countries. In a recent Zoom call, company executives were urged to drop patent claims and implement a global pricing model reflecting R&D and production costs, which could make the treatments more affordable. Instead, the Vertex team maintained the company will use a nascent donation program to address the concerns, according to people who attended the Oct. 12 session. The outcome frustrated a coalition pushing the company to alter its stance toward countries where cash-strapped governments are unable to meet pricing demands. The advocates and physicians also complained that, while Vertex maintained its donation program is a workable solution, the executives refused to disclose details about the extent of the initiative and how many people are benefiting. “They have determined that low- and lower-middle income countries are not commercially viable. They are continuing to enforce patents and fight efforts to overturn patents in those countries despite having no intention to supply [medicines] there,” said Diarmaid McDonald, who heads Just Treatment, a U.K. advocacy group that has worked with patients and their families to access Vertex medicines. “They have said their access solution in those countries will be a donation program. There is nothing in the public realm about this. They want to have total control over their proposed solution to global inequality of access, but without any transparency... It’s never worked as a solution for any other drug and it seems almost impossible to see how it might work for cystic fibrosis.” A Vertex spokeswoman declined to comment about the patent strategy or provide specifics about the donation program. Instead, she pointed us to previous responses to questions about widening access in low-income 11/28/23, 2:38 PM Vertex pressured to expand access to cystic fibrosis treatments https://www.statnews.com/pharmalot/2023/11/28/vertex-cystic-fibrosis-medicines/ 3/5 countries. Earlier this year, for instance, the company said it was looking at a “variety of different approaches,” although only the donation program was mentioned. The call was held amid an escalating number of efforts to widen access to the cystic fibrosis medicines sold by Vertex. One of its medications, in particular, is effective in treating roughly 90% of those who suffer from the disease, which severely damages the lungs and greatly limits life expectancy. The drug — known as Trikafta in the U.S. and Kaftrio elsewhere — has a U.S. list price of about $310,000. Vertex has battled with several countries over the cost of its medicines, notably the U.K., where the company clashed with a government agency over cost-effectiveness. A four-year deal recently lapsed and the same U.K. agency earlier this month issued new draft guidance 7 again questioning the cost-effectiveness of three cystic fibrosis medicines sold by the company. It is worth noting, though, that preliminary recommendations can sometimes change. Earlier this year, for instance, a Brazilian government advisory committee determined Trikafta was not cost-effective and initially recommended that the health ministry not provide reimbursement. But after a consultation period, the committee changed gear and recommended 8 reimbursement. However, price recently became an issue in Poland, which is considered a high-income country by the World Bank. The health ministry earlier this month announced 9 that reimbursement talks with Vertex were not successful and the company pulled its application. As a result, children six years and older will no longer be eligible for two Vertex drugs under a subsidized government access program. As of July 2023, Vertex estimated that more than 65,000 people with cystic fibrosis worldwide are currently on at least one of its medicines. The treatments are approved and available in more than 60 countries, although these are mostly wealthy nations and are not available in low- and lower-middle-income countries, except where Vertex says it is establishing its donation program. Through the first nine months of this year, cystic fibrosis medicines yielded $7.35 billion in revenue on a worldwide basis, an 11% increase, according to recent Vertex regulatory filings. Of that, $4.5 billion was generated in the U.S. The biggest-selling medicine was Trikafta, which generated $6.6 billion in worldwide sales, a 17% gain. Meanwhile, cystic fibrosis patients and their families have argued that prices often mean the medicines are kept out of reach in many countries. At different times, they have urged the company to provide its drugs at affordable prices, offer compassionate use where the drugs are not yet available, or issue licenses to generic makers in countries where the company does not plan to supply treatments any time soon. In February, patients filed patent challenges 10 asking South Africa and Ukraine to issue compulsory licenses, which allow a government to sidestep patents so lower-cost generics can be made and distributed. A different tactic is being pursued in India, where patients asked the government to revoke Vertex patents, and plan to petition for compulsory licensing if the patents are not revoked. In June, an academic gave a talk on international treatment disparities at the Cystic Fibrosis Conference in Vienna, and highlighted ways to address patents, generics, and reimbursement strategies. The presentation 11 prompted Jane Davies, a professor at the Imperial College London and president-elect of the European Cystic Fibrosis Society, to openly question Vertex executives on hand about access issues. 11/28/23, 2:38 PM Vertex pressured to expand access to cystic fibrosis treatments https://www.statnews.com/pharmalot/2023/11/28/vertex-cystic-fibrosis-medicines/ 4/5 The same presentation highlighted arguments that access in poor countries could be widened if pricing more closely reflected R&D and production costs. The academic, Jonathan Guo, a cystic fibrosis researcher and medical student at Imperial College London, has maintained the data on production costs are supported by real- world evidence of generics that are currently being produced. His talk occurred not long after an analysis 12 concluded the cost to produce Trikafta is less than $5,700. The researchers who conducted the analysis said that a generic version could be produced for a fraction of the list price, but that the current pricing model restricts access to the best available therapy and exacerbates inequalities in treating cystic fibrosis patients. And so the advocates hoped that, during the Oct. 12 call, Vertex could be persuaded to change its approach, but also obtain more information about the donation program. The company launched the program in 10 countries on four continents on a pilot basis last year with help from Direct Relief, a charity that works to expand access to medicine and health care services internationally. The session, however, yielded little progress, according to those who participated. For one, the Vertex team — which included Amit Sachdev, chief patient and external affairs officer, and Fosca De Iorio, vice president for international global medical affairs — declined to provide details about either the extent or progress of the donation program. For instance, it remains unclear which countries were chosen and why, how many patients are being treated and plans for expansion. Moreover, the Vertex executives maintained patents will be enforced in low- and lower-middle income countries, according to the participants. The executives added there are no plans to drop resistance to the patent challenge in South Africa or register its medicines there, but the company has since agreed to allow importation of unregistered medicines into the country, according to Gayle Pledger of the Right to Breathe Campaign. This means the treatments will only be available on an individual basis. Vertex did not comment. “The approach [taken by] Vertex is really too difficult for me to understand,” said Bulent Karadag, a pediatric pulmonologist at Marmara University in Istanbul, who was on the call. “The selection of certain countries or a subgroup of patients in others would only increase the inequity and the suffering of the parents” with children who have cystic fibrosis. By maintaining certain patents, Vertex preserves the exclusive rights to sell the medicines in those countries, even if it has not registered the treatments with authorities. Meanwhile, generic companies are generally precluded from making and distributing lower-cost versions of the medicines, which means patients must rely on their governments to reach coverage deals with Vertex. This strategy has caused friction among pharmaceutical companies, governments, and advocates elsewhere. A recent example involved Johnson & Johnson and its tuberculosis drug. The company agreed not to enforce 13 its patent rights only after losing a patent challenge in India, where many generic makers are based, and a subsequent high-profile pressure campaign that created negative publicity. “When I started caring for people with cystic fibrosis 26 years ago, I was able to hold the hands of the parents of a dying, seven-year-old little girl and know that cystic fibrosis was a desperately sad and ultimately life- shortening condition, but that everything possible had been done to extend their daughter’s life for as long as possible,” said Cathy Baird, who treats adults with cystic fibrosis at Netcare Milpark Hospital in Johannesburg. 11/28/23, 2:38 PM Vertex pressured to expand access to cystic fibrosis treatments https://www.statnews.com/pharmalot/2023/11/28/vertex-cystic-fibrosis-medicines/ 5/5 “How, today, do I hold the hands of the children of my cystic fibrosis patients and tell them that we have done everything possible to extend the life of their parent? Knowing that there is medication that can essentially turn a life-threatening condition into a chronic controllable condition, but that patients may not access it because of extreme patent abuse?” About the Author Ed Silverman 2 Pharmalot Columnist, Senior Writer Ed Silverman covers the pharmaceutical industry. ed.silverman@statnews.com 14 @Pharmalot 3 Exciting news! STAT has moved its comment section to our subscriber-only app, STAT+ Connect 21 . Subscribe 22 to STAT+ today to join the conversation or join us on Twitter 23 , Facebook 24 , LinkedIn 25 , and Threads 26 . Let's stay connected! 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