A Move to Cut Drug Prices Has Patients With Rare Diseases Worried

Published

2 years ago

September 1, 2023

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For people with cystic fibrosis, like Sabrina Walker, Trikafta has been a life-changer.

This story also ran on CBS News. It can be republished for free.

Before she started taking the drug, she would wind up in the hospital for weeks at a time until antibiotics could eliminate the infections in her lungs. Every day, she would wear a vest that shook her body to loosen the mucus buildup.

One particularly bad flare-up, known as a pulmonary exacerbation, had her coughing up blood in 2019, so she was put on the newly approved breakthrough medication.

Within a month, her lung function increased by 20%, she said, and her health improved. Before she started taking Trakafta, she could count on three to four hospitalizations a year. Over the four years on the medication, she has been hospitalized only once.

I was spending hours a day doing airway clearance and breathing treatments, and that has been significantly reduced, said the 37-year-old Erie, Colorado, mother. Ive gained hours back in my day.

Now she runs and hikes in the thin Colorado air and works a full-time job. Other patients have seen similar gains with the drug therapy, allowing many to resume regular lives and even take themselves off waiting lists for a lung transplant. Yet Walker and scores of other Colorado patients with cystic fibrosis are worried they could lose access to that transformative medication.

A state board charged with addressing the affordability of the most expensive prescription drugs has chosen Trikafta among its first five drugs to review, and it could move to cut the medications average in-state annual price of approximately $200,000, accounting for both insurers contributions and patients out-of-pocket costs. Drugmakers, including Trikafta’s maker, Vertex Pharmaceuticals, have said payment limits could hurt innovation and limit access, stoking panic among patients that the drug might no longer be sold in Colorado.

Two of the drugs chosen by the state board, the rheumatoid arthritis treatment Enbrel and the psoriasis medication Stelara, also appear on the initial list of 10 drugs for which Medicare will negotiate prices. Any federally negotiated price reductions wont go into effect until 2026, and its unclear how that effort will affect the Colorado boards work in the interim. Sabrina Walker, who has cystic fibrosis, has seen her lung health improve since taking Trikafta. Patients worry potential payment limits on the medication set by Colorados drug affordability board could cause the drugmaker to stop selling Trikafta in the state.(Adam Walker)

The Colorado boards choice of drugs to review elucidates one of the thorniest questions the board must wrangle with: Would lowering the price tag for rare-disease medications lead manufacturers to pull out of the state or limit their availability? State officials contend that the high cost of prescription drugs puts them out of reach for some patients, while patients worry that theyll lose access to a life-changing therapy and that fewer dollars will be available to develop breakthrough medications. And with affordability boards in other states poised to undergo similar exercises, what happens in Colorado could have implications nationwide.

It just puts Trikafta as a whole at risk, Walker said. It would start here, but it could create a ripple effect.

Cystic fibrosis is a genetic condition that causes the body to produce thick, sticky mucus that clogs the lungs and digestive system, leading to lung damage, infections, and malnutrition. It is a progressive disease that results in irreversible lung damage and a median age of death of 34 years. There is no cure.

The rare disease affects fewer than 40,000 people in the U.S., including about 700 in Colorado. That means research and development costs are spread across a smaller number of patients than for more common conditions, such as the millions of people with heart disease or cancer.

Officials from Vertex Pharmaceuticals declined a request for an interview. But company spokesperson Sarah DSouza emailed a statement saying that the price of this medicine reflects its value to patients, the small number of people living with CF, the billions of dollars Vertex has invested to date to develop the first medicines to treat the underlying cause of CF, and the billions more we are investing in CF and other serious diseases.

Setting an upper payment limit, the company said, could hinder access to drugs like Trikafta and curtail investment in scientific innovation and drug discovery.

State officials counter that Vertex and other drugmakers are resorting to fear-mongering to protect their profits.

Colorado Insurance Commissioner Michael Conway said that whenever the state talks about saving people money on health care, the affected entity be it a hospital, insurance company, or drug manufacturer cries foul and claims there will be an access problem.

This is just, from my vantage point, the pharmaceutical industry trying to scare people, he said.

Colorados Prescription Drug Affordability Board has been working for more than a year to sort through 604 drugs eligible for review, with 17 data points for each, to create a prioritized list. In the end, they decided to focus this year only on drugs that had no brand-name competition or generic alternatives that could lower costs. Email Sign-Up

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Besides Trikafta, Enbrel, and Stelara, the board will review the affordability of the antiretroviral medication Genvoya, used to treat HIV, and another psoriasis treatment, Cosentyx.

Of those five, Trikafta had the highest average annual costs but the lowest five-year increase in price and the fewest patients taking it.

The boards review of the five drugs will happen over its next three to four meetings this year and early next year, allowing all stakeholders including patients, pharmacies, suppliers, and manufacturers to provide feedback on whether the drugs are indeed unaffordable and what a reasonable price should be. Any cost limits wouldnt take effect until next year at the earliest.

The board looked at what patients were paying out-of-pocket for their medicines, using a database that captures all the insurance claims in the state. But that data did not account for patient assistance programs, through which manufacturers reimburse patients for out-of-pocket costs. Such programs boost manufacturer sales of drugs because insurance covers most of the cost, and patients otherwise might not be able to afford them.

Through the first half of the year, Vertex reported profits of $1.6 billion, with 89% of its revenue coming from Trikafta (marketed as Kaftrio in Europe). At the beginning of the year, Vertex decreased copay assistance for people with cystic fibrosis, in what the company said was a response to insurers’ limiting patients’ ability to apply copay assistance to their deductibles.

Lila Cummings, director of the Colorado board, said its staff could not find any entity that collects data on patient assistance programs, so those figures were not available to the board. Once they begin reviewing the individual medications, board members will dig into what extra financial help patients are getting. Cummings also said the board is hoping manufacturers will convey in good faith what might prompt them to leave the Colorado market.

When Trikafta came up second on the Colorado boards prioritized list of drugs eligible for review, patients and advocacy groups flooded the board with pleas to leave pricing for the medication and other drugs for rare diseases untouched.

People are scared, Walker said. If you look at all the drugs out there, it’s one that has been so transformational that I think it will go down in history for how positively its impacted our population as a whole.

According to the Cystic Fibrosis Foundation, lung exacerbations dropped 65% and lung transplants dropped 80% after the drugs approval. More patients have been able to work, attendschool, or start a family. Clinicians have reported a baby boom among patients who take Trikafta.

A study published this year showed that two-thirds of people with cystic fibrosis struggled with finances, experiencing debt, food insecurity, or trouble paying for household or health expenses. The survey was conducted in 2019, before the FDA approval of Trikafta.

Years ago, the Cystic Fibrosis Foundation invested in Aurora Biosciences, later acquired by Vertex Pharmaceuticals, to promote development of cystic fibrosis therapies. The foundation completed the sale of its royalty rights in 2020.

Mary Dwight, chief policy and advocacy officer for the Cystic Fibrosis Foundation, said the board should ensure its review of Trikafta accounts for the overall value this drug has for someone with CF, including the impact on an individuals long-term health and well-being.

There is no guarantee that the Colorado board will take action on Trikafta. State officials have stressed that board members are solely focused on improving access and wouldnt jeopardize the availability of the medication.

We have a history of being able to save people money on health care that doesnt lead to access problems, Conway said. Were not talking about these companies losing money at all; were talking about making it more affordable so that more Coloradans can get access to the pharmaceutical needs that they have.

But Walker remains unconvinced.

They had so much testimony on their call and they still selected Trikafta, she said. Everyone was just saying how important this drug is, and it didnt matter. It still got pushed through.

Markian Hawryluk: MarkianH@kff.org, @MarkianHawryluk Related Topics Health Care Costs States Colorado Drug Costs Prescription Drugs Contact Us Submit a Story Tip

Business

Whitehall on alert for collapse of Gupta’s steel empire

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3 hours ago

August 16, 2025

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Whitehall on alert for collapse of Gupta's steel empire

The metals tycoon Sanjeev Gupta is this weekend plotting a controversial deal to salvage his remaining UK steel operations and avert their collapse into compulsory liquidation – a move that would put close to 1,500 jobs at risk.

Sky News has learnt that Mr Gupta is in talks about a so-called connected pre-pack administration of Liberty Steel’s Speciality Steel UK (SSUK) arm, which would involve the assets being sold – potentially to parties linked to him – after shedding hundreds of millions of pounds of tax and other liabilities to creditors.

Begbies Traynor, the accountancy firm, is understood to be working on efforts to progress the pre-pack deal.

This weekend, Whitehall sources said that government officials had stepped up planning for the collapse of SSUK if an already-deferred winding-up petition scheduled to be heard next Wednesday is approved.

If that were to happen, SSUK would be likely to enter compulsory liquidation within days, with a special manager appointed by the Official Receiver to run the operations.

Mr Gupta’s UK business operates steel plants at Sheffield and Rotherham in South Yorkshire, with a combined workforce of more than 1,400 people.

SSUK is Britain’s third-largest steel producer.

Sources close to Mr Gupta could yet secure a further adjournment of the winding-up petition to buy him additional breathing space from creditors.

In May, a hearing was adjourned after lawyers acting for SSUK said talks had been taking place with “a third-party purchaser”.

Their identity has not been publicly disclosed, and it has been unclear in recent weeks if any such discussions were continuing.

A connected pre-pack risks stiff opposition from Liberty Steel’s creditors, which include HM Revenue and Customs.

UBS, the investment bank which rescued Credit Suisse, a major backer of the collapsed finance firm Greensill Capital – which itself had a multibillion dollar exposure to Liberty Steel’s parent, GFG Alliance – is also a creditor of the company.

Grant Thornton, the accountancy firm handling Greensill’s administration, is also watching the legal proceedings with interest.

The Serious Fraud Office launched a probe into GFG – which stands for Gupta Family Group – in 2022.

On Saturday, a Liberty Steel spokesperson said: “Discussions are ongoing to finalise options for SSUK.

“We remain committed to identifying a solution that preserves electric arc furnace steelmaking in the UK-a critical national capability supporting strategic supply chains.

“We continue to work towards an outcome that best serves the interests of creditors, employees, and the broader community.”

Last month, The Guardian reported that Jonathan Reynolds, the business secretary, was monitoring events at Liberty Steel’s SSUK arm, and had not ruled out stepping in to provide support to the company.

Such a move is still thought to be an option, although it is not said to be imminent.

The Department for Business and Trade has been contacted for comment.

It has previously said: “We continue to closely monitor developments around Liberty Steel, including any public hearings, which are a matter for the company.

“It is for Liberty to manage commercial decisions on the future of its companies, and we hope it succeeds with its plans to continue on a sustainable basis.”

Wednesday’s winding-up petition was filed by Harsco Metals Group, a supplier of materials and labour to SSUK, and is said to be supported by other trade creditors.

Mr Reynolds has already orchestrated the rescue of British Steel, the Scunthorpe-based steelmaker, after failing to reach a government aid deal with Jingye Group, the company’s Chinese owner.

Jingye had been preparing to permanently close Scunthorpe’s remaining blast furnaces, prompting Mr Reynolds to step in and seize control of the company in April.

The government has yet to make a decision to formally nationalise British Steel, although that is anticipated in the autumn.

Tata Steel, the owner of Britain’s biggest steelworks at Port Talbot, has agreed a £500m government grant to build an electric arc furnace capable of manufacturing greener steel.

Other parts of Mr Gupta’s empire have been showing signs of financial stress for years.

The Financial Times reported in May that he was preparing to call in administrators to oversee the insolvency of Liberty Commodities.

Separately, HMRC filed a winding-up petition against Liberty Pipes, another subsidiary, earlier this month, The Guardian reported.

Mr Gupta is said to have explored whether he could persuade the government to step in and support SSUK using the legislation enacted to take control of British Steel’s operations.

Whitehall insiders told Sky News in May that Mr Gupta’s overtures had been rebuffed.

He had previously sought government aid during the pandemic but that plea was also rejected by ministers.

SSUK, which also operates from a site in Bolton, Lancashire, makes highly engineered steel products for use in sectors such as aerospace, automotive and oil and gas.

The company said earlier this year that it had invested nearly £200m in the last five years into the UK steel industry, but had faced “significant challenges due to soaring energy costs and an over-reliance on cheap imports, negatively impacting the performance of all UK steel companies”.

World

Trump-Putin summit: No deal reached to end war in Ukraine

Published

4 hours ago

August 16, 2025

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Trump-Putin summit: No deal reached to end war in Ukraine

No deal has been reached to end the war in Ukraine – but Donald Trump has said there are “many points” he and Vladimir Putin agreed on during their highly anticipated summit.

Following the meeting in Alaska, which lasted more than two-and-a-half hours, the two leaders gave a short media conference giving little detail about what had been discussed, and without taking questions.

Mr Trump described the meeting as “very productive” and said there were “many points that we agreed on… I would say a couple of big ones”.

Trump-Putin summit – latest updates

But there are a few left, he added. “Some are not that significant. One is probably the most significant, but we have a very good chance of getting there…

“We haven’t quite got there, we’ve made some headway. There’s no deal until there’s a deal.”

Mr Putin described the negotiations as “thorough and constructive” and said Russia was “seriously interested in putting an end” to the war in Ukraine. He also warned Europe not to “torpedo nascent progress”.

After much build-up to the summit – with the US president threatening “severe” consequences for Russia should it not go well – it was ultimately not clear whether the talks had produced meaningful steps towards a ceasefire in what has been the deadliest conflict in Europe in 80 years.

Mr Trump said he intended to speak with Ukrainian President Volodymyr Zelenskyy and other European leaders, who were excluded from the discussions, to brief them.

Despite not reaching any major breakthrough, the US leader ended his remarks with a thank you, and said he would probably see Mr Putin again “very soon”.

When the Russian president suggested that “next time” would be Moscow, he responded by saying he might face criticism, but “I could see it possibly happening”.

The red carpet treatment

The news conference came after a grand arrival at the Elmendorf-Richardson military base in Anchorage, where the US president stepped down from Air Force One and later greeted his Russian counterpart with a handshake and smiles on a red carpet.

Mr Putin even travelled alongside Mr Trump in the presidential limousine, nicknamed “The Beast”.

It was the kind of reception typically reserved for close US allies, belying the bloodshed and the suffering in the war.

Before the talks, the two presidents ignored frantically-shouted questions from journalists – and Mr Putin appeared to frown when asked by one reporter if he would stop “killing civilians” in Ukraine, putting his hand to his ear as though to indicate he could not hear.

Our US correspondent Martha Kelner, on the ground in Alaska, said he was shouting “let’s go” – apparently in reference to getting the reporters out of the room.

A ’10/10′ meeting

During his first day back in the White House in January, Mr Trump had pledged confidently to bring about an end to the war in Ukraine.

But seven months later, after infamously berating Mr Zelenskyy during a meeting at the Oval Office in February, and then stanching the flow of some US military assistance to Kyiv, he still does not appear to have brought a pause to the conflict.

In an interview with Fox News before leaving Alaska, Mr Trump described the meeting with Mr Putin as “warm” and gave it a “10/10”, but declined to give details about what they discussed.

He also insisted that the onus going forward could be on Mr Zelenskyy “to get it done”, but said there would also be some involvement from European nations.

What happens next?

Mr Trump is expected to speak to Mr Zelenskyy, Sir Keir Starmer and European leaders about the talks.

A meeting of ambassadors from European countries has been scheduled for 8.30am UK time, EU presidency sources have told Sky News.

European heads of state and Mr Trump are also likely to have a virtual meeting later in the day.

Despite the US president’s efforts to bring about a ceasefire, Russian attacks on Ukraine have only intensified in the past few months.

On 9 July, Russia carried out its largest aerial attack on Ukraine since the start of the war, launching more than 740 drones and missiles.

Furthermore, Mr Zelenskyy has said Russia is preparing for new offensives.

Ahead of the summit, one of the key commanders of Ukraine’s drone forces told Sky News in a rare interview that there would be no let-up in its own long-range drone attacks on Russia until Moscow agrees to peace.

World

What we expected from the Trump-Putin summit – and what actually happened

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4 hours ago

August 16, 2025

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What we expected from the Trump-Putin summit - and what actually happened

A warm handshake, big smiles, and a red carpet – this was the welcome for Vladimir Putin as he touched down on US soil for critical negotiations on the war in Ukraine.

There had been much build-up to the summit in Anchorage, Alaska, not least from Donald Trump himself – with the US president having threatened “severe” consequences for Russia should it not go well.

But more than two-and-a-half hours of talks resulted in just a brief news conference with little detail given away – and ultimately, no talk of a ceasefire and no deal on Ukraine reached yet.

Here is what was expected from the meeting – based on information from the White House, Mr Trump and the Kremlin beforehand – and what happened on the night.

One-on-one turned into three-on-three

It was thought this would be a one-on-one meeting between Mr Trump and Mr Putin.

Instead, the US president was joined by US secretary of state Marco Rubio and special envoy Steve Witkoff, while the Russian leader was supported by his foreign affairs advisor Yuri Ushakov and Russian foreign minister Sergei Lavrov.

The change seemed to indicate the White House was perhaps taking a more guarded approach than during a 2018 meeting in Helsinki, where Mr Trump and Mr Putin met privately with interpreters. The US leader then shocked the world by siding with the Russian leader over US intelligence officials on whether Russia meddled in the 2016 presidential campaign.

Rolling out the red carpet

Mr Putin was given the kind of reception typically reserved for close US allies, belying the bloodshed and the suffering in the war he started.

The two men greeted each other with a handshake and a smiling Mr Trump even applauded the Russian president as he approached him on the red carpet.

Our international affairs editor Dominic Waghorn, in Kyiv, gauged the Ukrainian reaction to the arrival – and said people were furious at the welcome extended by the Trump team.

Images of US soldiers on their knees, unfurling the red carpet at the steps of the Russian leader’s plane, went viral, he said, with social media “lit up with fury, anger, and disgust”.

He added: “There are different ways of welcoming a world leader to this type of event, and Trump has gone all out to give a huge welcome to Putin, which is sticking in the craw of Ukrainians.”

Any questions?

Plenty. But no one was really given a chance to ask.

Ahead of the talks, cameras were allowed inside for just a minute – and while this was enough time for a few journalists to shout some questions, these were ignored by the two leaders.

“President Putin, will you stop killing civilians?” one shouted. In response, Mr Putin put his hand up to his ear as if he could not hear.

In their brief media conference after the talks, Mr Putin spoke for almost nine minutes, while Trump took just three-and-a-half to say what he wanted to say.

The two men then did not stay to answer questions from reporters.

Before the event, the Kremlin said it could last between six and seven hours, but the whole visit lasted about four-and-a-half hours.

‘Severe consequences’

Ever since his inauguration in January, Mr Trump had been threatening serious consequences for Russia should a deal on Ukraine not be reached soon. Just two days after the ceremony, he took to social media to declare there could be “high levels of taxes, tariffs and sanctions” and called for an end to the “ridiculous” war.

In February, he held what he described as a “productive” call with the Russian leader, and about two weeks later he infamously berated Ukrainian President Volodymyr Zelenskyy during a visit to the Oval Office – this one taking place in front of the world’s media.

In July, he started to set deadlines for an end to the war – first giving Mr Putin 50 days and later reducing this to “10 or 12 days”, before announcing the summit last week.

Yesterday, Mr Trump insisted his Russian counterpart was “not going to mess around with me”.

However, while both men insisted the talks were “productive”, it is not clear what agreements have been reached, and whether Ukraine is any closer to finding peace. The word ceasefire was not mentioned by either leader. Instead, they praised each other, with Mr Trump describing Mr Putin’s remarks as “very profound” – and there was no mention of sanctions.

A meeting with Mr Zelenskyy?

It was expected that after the talks, Mr Trump could set the table for the next meeting with the Ukrainian president.

While he said he would call Mr Zelenskyy, he made no public commitment to a meeting during the media conference.

In an interview with Fox News after the summit, he said Russia and Ukraine would set a date to discuss next steps and a potential ceasefire deal, but did not provide further details on specifics or timings.

“They’re going to set up a meeting now, between President Zelenskyy and President Putin and myself, I guess,” Mr Trump said. He also said that European nations “have to get involved a little bit” but it is “really up to President Zelenskyy to get it done”.

Putin brought his own limo – but travelled in The Beast instead

After shaking hands on the red carpet, the two leaders made their way towards their waiting vehicles.

But despite Mr Putin arriving with his “Aurus” limousine, and it being spotted on the tarmac near the planes, he got into the American presidential limousine, known as “The Beast”, to travel to the meeting location.

The Russian president was seen with a wide smile on his face, while Mr Trump appeared to be waving to the crowds.