For Two Years, Australia Failed People With Cystic Fibrosis; It Can’t Happen Again

Governments around the world have withheld access to life-changing medications for people with cystic fibrosis (CF) for years in the name of being hawkish on drug pricing. These countries allow bureaucrats to dictate which drugs are “worth it” but do so based on demonstrably flawed calculations of value. Patient communities must hold them accountable; their bad math and delay tactics cost lives and health.

Trikafta, a drug that corrects the underlying cause of CF, was approved in the US at the end of 2019. The drug is nothing short of a miracle. It slows the condition that has historically suffocated patients with thick sticky mucus in our airways by the time we enter our early thirties.

I live with CF and write this from a position of bias. My conflict is that I am alive today, I am married today, and I am a father today because I take Trikafta, and it shakes me to my core to think that others with my condition remain condemned to severe disease by bad math.

Before I started on the drug, I relied on overnight tube feedings. I required hospital-based interventions many times per year. I took dozens of prescription medications that merely mitigated my worsening respiratory symptoms. Those days feel like ancient history.

Despite the benefits thousands of Americans experience from the drug, Trikafta wasn’t approved in the United Kingdom until August 2020 or made widely available in Canada until the Fall of 2021.

In those countries, the response has also been mind-blowing. In Canada, a 61-year-old woman’s improvement in pulmonary function gave her “a second lease on life.” In the UK, an actor says he was able to land his “dream role” after the drug cut his daily treatment routine from six hours to 20 minutes.

These gains were possible only after extensive patient advocacy campaigns to persuade governments to allow access to Trikafta. These campaigns continue in Australia, which we can only hope will soon grant approval.

Outside the US, countries evaluate novel drugs’ cost-effectiveness through Health Technology Assessments (HTAs), which consider how well a drug works and calculate whether it’s “worth it.” With a price tag over $300,000 per year, Trikafta is unaffordable to nearly all patients out-of-pocket. It would be unaffordable even if it cost a tenth of this price. That’s why we have insurance. Commercial or government-subsidized health coverage is supposed to make expensive treatments affordable to the few who need them at any one time. When places like Australia, Canada, and the UK say these medicines aren’t “worth it,” they do so because their calculations say that the cost exceeds the value to patients and society.

Where Australia’s arbiter of drug value, the Pharmaceutical Benefits Advisory Committee, and others have gone wrong, is precisely this cost-effectiveness math.  Among their errors, they ignore that Trikafta’s price is only temporarily high. Trikafta will go generic and will continue to save lives and avert hospitalization costs in perpetuity at a much lower price. This is demonstrably true of so many drug classes: statins, antibiotics, anti-depressants, cancer treatments. All were expensive at one time and now they are inexpensive generics, still saving lives.

HTA bodies claim they are looking out for society’s interests, but they only consider the here and now. The savings from a new medicine someday going generic is an input left out of 95% of published cost effectiveness analyses.

In 20 years, people will still be born with CF, but when they start on the generic version of Trikafta, which will likely remain a core CF treatment, they will not suffer from the same CF I have suffered from.

Missed school and work due to CF exacerbations will be a thing of the past, and so will much of the need for solid organ transplants. In that way, Trikafta benefits more than just patients, but also the communities around us.

Leading health economists have written about the importance of accounting for these benefits, referring to caregiver spillover, productivity, insurance value, genericization, and option value, but you almost never find them in the cost-effectiveness equations of HTA bodies. These critical omissions imply bureaucrats want to underestimate the value of medicines like Trikafta so their client governments can deny coverage.

The irony is crippling. Patients, who have advocated and fundraised for decades hoping to see a drug like Trikafta, are trapped in a web of flawed equations. Worse, some patients have been misled by the flawed cost-effectiveness math into believing that Australia was right to deny coverage of Trikafta based on price.

I wish they could see the ongoing research that I’ve seen (and will soon be published), showing that when accounting for just some of the missing variables, Trikafta is more than 10-fold more cost-effective than conventional HTA has claimed. In other words, Trikafta offers far more societal value than is reflected in its price.

If Australians finally gain access to Trikafta, they deserve to take a victory lap. For them, it will represent the end of a hard-won advocacy campaign. But we still owe it to them to question the several years’ delay they have suffered in the name of bad math. That cannot be forgotten. The math flaws made by leading HTA bodies need to be put in the spotlight and then reformed or they will continue to cause harm. These errors are so clear that any politician or bureaucrat who leans on such math to deny patients a necessary medicine is essentially telling us our lives have little worth. If math central to healthcare coverage is to be a battleground, then patients need to understand it well enough to fight for our values, or we will be rounded down.

Gunnar Esiason, an MPH Candidate at Dartmouth College, lives with cystic fibrosis and serves on the board of directors at No Patient Left Behind (NPLB), a non-profit organization dedicated to creating and maintaining patient access to affordable drugs by holding both insurance and drug companies accountable for their policies and practices. Like many people with CF, Gunnar has benefited from the advocacy of his family’s non-profit organization, the Boomer Esiason Foundation, among whose many supporters is Vertex Pharmaceuticals, the manufacturer of Trikafta.