Investigation won 'miracle' drug for this little girl

Hey there,

Earlier this month, a team of researchers announced the first therapy that seems to successfully treat Huntington’s disease. It felt like a great step forward, not for one country or one scientist, but for humanity as a whole. 

But it may take a while for the world to benefit. Drug distribution is a thorny issue, and one that exposes many of the inequalities in healthcare across the globe.

Some drugs are simply unavailable in countries that can’t afford them. Other nations are hampered by a lack of infrastructure to keep medicines the right temperature, or transport them to where they’re needed. Opting for cheaper versions can mean buying drugs that don’t work as they should.

The health team at my newsroom has exposed brands of children’s cancer drugs that were contaminated and ineffective (which we spoke about in this Spark issue here). They also found snake antivenoms being marketed in areas of sub-Saharan Africa where they’d be unlikely to work, because they weren’t made using the venom of local snakes. (Snakebites kill more than 500,000 people a year).

Investigate Europe is another punchy and powerful investigative newsroom that has also been looking into drugs, specifically how they’re priced in the EU. The team there found cystic fibrosis patients across the continent were being priced out of access to life-saving medications.

I spoke to Max Peigné, one of Investigate Europe’s reporters, about this recent investigation. Alongside Eurydice Bersim, Max revealed that ‘miracle’ cystic fibrosis drugs cost more for poorer EU countries than richer ones. In Czechia, for example, it cost roughly twice as much to treat one patient for a year as it did in France.

Cystic fibrosis is a rare inherited condition that causes breathing and digestive problems. Thick mucus will blog your airways, causing breathing problems and recurrent infections. There’ll also be blockages in your digestive system, preventing your body from absorbing important nutrients.

More than a decade ago, however, a revolutionary set of drugs were developed that can significantly improve lung function. Even the NHS called it a “miracle”.

In the EU, new, innovative drugs need to be approved by the European Medicines Agency. But after that centralised regulation, it’s a matter of cloak and daggers: drug makers negotiate secret prices with each country individually.

This means that some cancer drugs, for example, may be available in Germany, which has strong buying power and leverage to negotiate, but not in smaller countries. Even nations like Ireland, Max told me, may struggle to secure drugs that keep people alive.

Both drug companies and governments aren’t transparent about how much each country pays. European healthcare systems, much like the NHS, report their spending and patient numbers, but the figures they publish don’t include rebates – the confidential discounts companies give back.

But Max realised he could dig into the drug companies’ accounts, which do include rebates, since those show the companies’ real revenue. He then compared those figures with the number of patients receiving the drugs to get a more accurate picture of what countries were really paying. With something like cancer drugs, this is difficult because it’s a crowded market, with many treatments made by dozens of companies. But with cystic fibrosis it’s simpler: just one company produces the drugs, Vertex Pharmaceuticals, and there are only three or four treatments on the market.

The disparity Max uncovered was striking. Countries like France have had access to all of Vertex’s drugs for the past decade, but in Lithuania the drugs are so expensive the country can only afford to treat up to 48 patients.

“Countries like Germany and France can afford to pay and also have the negotiating power to secure substantial rebates,” Max explained. “With strong domestic pharmaceutical industries, they have little incentive to push for price transparency or to negotiate collectively with poorer countries. Doing so could undermine their own deals.”

The impact of this is heart-wrenching. Max spoke with Milda, a three-year-old Lithuanian girl with cystic fibrosis, and her mum, Urtė Gylienė. Milda has coughed and struggled to gain weight all her life. While other children play in the park, Milda’s daily routine involves two hours of physiotherapy, pills and, when things take a turn for the worse, hospital visits.

Milda wasn’t one of the lucky 48 patients Lithuania could afford to treat with Vertex’s game-changing medicines Kaftrio and Kalydeco.

“There are so many kids that can't wait a day anymore,” Urtė said. “I’m really angry because Vertex makes a drug that’s not available.” Cystic fibrosis affects about 54,000 people in Europe, and more than half of related deaths in 2022 were of those aged between 18 to 39.

Vertex declined to comment on its negotiations with individual countries, but told Investigate Europe: “The price of our medicines is based on their innovation and the value they bring to the CF community, caregivers and healthcare systems.” The company disputed some of Investigate Europe’s calculations on the prices healthcare systems paid for the drugs.

After Investigate Europe published, the Lithuanian government announced it had renegotiated its deal with Vertex. While it did not disclose the exact terms, it confirmed that far more people would now have access to the treatment, with no restrictions on who would qualify.

Max believes the team’s reporting played a role in this shift, and they received encouraging feedback from several sources supporting that view. It’s clear that journalism does make a difference.

Milda now has access to the life-changing drugs she so desperately needs. When Max and Eurydice returned to interview her, they wrote: “Judging by the sparks of joy in Milda’s eyes, one would think the four-year-old has just received a new toy. Instead, the Lithuanian girl is finally holding the medicines that help her breathe, ridding her small lungs of the mucus clogging them day and night.”

“Carbon credit schemes to offset a company’s emissions can be bought anywhere in the world. They may be out of sight, but they should not avoid scrutiny.

When we investigated the environmental claims of companies buying carbon credits, our reporting took us all the way to Uruguay (not in person, sadly). There, Fin Johnston discovered that British American Tobacco (BAT) had invested in a project where the supposed environmental benefits would have happened anyway. The scheme produced junk credits – it was seemingly a money-making exercise, not a planet-saving one.

After we published, BAT was hit with a $5m class action lawsuit in the US. BAT had marketed Vuse as the “world’s first carbon-neutral vape brand.” Some of the vapers who bought into that claim are now suing BAT and its subsidiary for misleading them. Our evidence is enabling others to use the power of the law to push for accountability.

If the Vuse plaintiffs win, it would send a powerful message, not just to BAT but to every company using carbon credits, that the environmental benefits must be real. You cannot buy your way out of responsibility without proving what’s genuine.

For now, the case hangs in the balance. A judge still has to decide whether it qualifies as a class action. That decision matters: if approved, many more Vuse users could step forward without filing individual complaints.

But the process is slow. There may be an out-of-court settlement. If not, and it goes to a jury trial, it will be nearly three years from the initial filing before it is heard.

We are being forced to be patient.”

These investigations don't just happen overnight – our team toils away for months to get to the bottom of the story, and we do it with the help of our brilliant community of Bureau Insiders. Please join us today:

A bit more on that Huntington’s gene therapy, to keep the good news coming. Patients treated in the trial so far appear to show slower disease progression, raising hope that this could be the first therapy that gives patients years more “good quality life”. 

You may also have seen on Friday that the police arrested Noel Clarke; last week’s Spark edition was about the libel suit the Guardian faced after reporting about Clarke’s sexual misconduct. You can catch up here if you missed it. 

And that’s all! Thanks for reading and have a lovely week,