You need a miracle to pay for this miracle drug

 You need a miracle to pay for this miracle drug:  Vertex Pharmaceuticals Monopoly over Cystic Fibrosis Medications.

By Stephanie Ross

I grew up in a house where it was normal for my mom and brother to spend days on end in doctors offices.  Where it was normal for us to have a closet full of medications. Where it was normal for my brother to be too sick to go to school.  Where it was normal that there was a chance that my brother wouldn't live past the age of 20. The reason for this to all seem normal to me and the reason that my brother spent most of his childhood in and out of hospital rooms is because he was and is still suffering from Cystic Fibrosis. 

(all of the memes in this article are sourced from the Cystic Fibrosis Facebook group)

What is CF

Cystic Fibrosis (CF) is a genetic disorder that damages the lungs and digestive system. In individuals with CF, they have mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene which leads to the production of a faulty protein (CFF).  This faulty protein results in a thick, sticky and salty mucus.  This can cause a variety of issues in the body, with a wide range of severity.  This mucus can clog up the lungs, making it difficult to breathe, and it can also trap bacteria which can lead to infections, inflammation, respiratory failure, and other complications (CFF). This buildup of mucus can also prevent the release of digestive enzymes in the pancreas, resulting in malnutrition and poor growth.  And in the liver, this mucus can block the bile duct, resulting in liver disease. Symptoms and severity can vary greatly between patients. But some of the most common symptoms are salty skin, chronic coughing, frequent lung infection, frequent sinus infections and more.

     With all of the health complications that come with Cystic Fibrosis, that also comes with an added burden of paying for these doctors appointments and medications. For reference, the average Cystic Fibrosis patient takes about 30/40 pills a day (Cromwell). Plus add on the hospital visits, doctors appointments and procedures and it can become very expensive if you're having a rough year.  During my brother's toughest year, our out of pocket expenses got up to $25,000.  According to Gool, Annual health care cost for CF patients is US $15,571. For those with mild, moderate and severe symptoms, the averages are $10,151, $25,647 and  $33,691 respectively (Gool). 

Because Cystic Fibrosis was always well known to me, it was a surprise to find out that this is a rare disease. Only about 40,000 people in the United States suffer from CF, and there are an estimated 105,000 people who have been diagnosed with CF across 94 countries (Cromwell).  

    When my brother was first born, his life expectancy was only about 20 years old.  But since then great improvements have been made on treatments and care, and the life expectancy has increased by almost 30 years. And with a new medication called Trikafta, people are predicting the life expectancy to go up even more.

What is Vertex

This increase in life expectancy has a lot to do with the American biopharmaceutical company, Vertex. Vertex discovers, develops and commercializes transformative medications, many of which are for Cystic Fibrosis families. The Cystic Fibrosis foundation helps raise money for a lot of this research, and it has paid off.  Vertex has come out with four transformative medications for the CF community. These CFTR modulators help the CFTR protein function properly. So, these products allow cystic fibrosis patients to feel like a normal human. CF patients have a variety of different mutations, and different mutations cause different problems in CF patients. Vertex commercializes four different CFTR modulators, each targeting specific mutations. These modulators are called: Trikafta (which is a combination of elexacaftor/tezacaftor/ivacaftor), Symdeko (which is a combination of tezacaftor/ivacaftor), Orkambi (which is a combination of lumacaftor/ivacaftor) and Kalydeco (which is ivacaftor).  These modulators cater to different parts of the CF population. For example, Kalydeco caters to about 6% of the CF population.  And more importantly, their 2019 medication called Trikafta treats about a whopping 90% of the CF population or roughly 27,000 people in the United States (Myshko).  As geneticist and physician Francis Collins at the US National Human Genome Research Institute states,  “the development of Trikafta has been one of the most phenomenal and outstanding achievements of biomedical research in the last 30 years,” (Merali). It was so impressive that the three main collaborators on the project won a 3 million dollar Breakthrough prizes — a prestigious science award. 

Cost of Vertex in the US

But this life transforming medication comes at a high cost.  Trikafta can cost up to $326,239 per year (Guo).  Although most insurance companies bear the brunt of this cost, the out of pocket costs can still add up. Originally, out of pocket expenses were about 180$ a year, which is doable.  However, this 180 a year increased to a whopping $43,600 in 2023 for some (Nolen). The dramatic change is due to a decision by the drug’s manufacturer, Vertex Pharmaceuticals, to slash the amount of financial assistance that it offers to patients this year. They aimed to reduce its annual copay assistance for its cystic fibrosis treatments from approximately 100,000 dollars a year to 20,000 a year (Silverman).  These prices do not consider any rebates or discounts that Vertex might provide to pharmacy benefit managers for favorable placement on health plan formularies. But also because Vertex dominates the market, it doesn't face the same rebate pressure as competitors in other spaces. This reduction in copay assistance adds a new level of financial uncertainty for patients, as they also need to factor in other costs of medications like nebulizers and regular care into their insurance costs. While Vertex didn’t give an exact reason for this decrease in copayments, it can be speculated that they are hoping that insurance companies will be forced to start paying more.

Luckily the Cystic Fibrosis Foundation is very impactful in the Cystic Fibrosis Community, and provides many resources for Cystic Fibrosis patients to both learn more about the changes to insurance and also how to help cover the rising costs. 

The worst part about the high cost of the medication is that there are no generic brand options due to Vertex’s monopoly over the industry.  Additionally, an analysis led by researchers in Britain found that a year’s supply of the drug could be manufactured at an estimated cost of just $5,700 (Guo). While Vertex claims that they are upping the price to cover research and development costs, the company is still expected to profit billions of dollars in the upcoming years. If the pharmaceutical company wanted to, they could significantly decrease the cost of this life altering product, while still retaining a profit.

Vertex Internationally

While many may hear this information and start blaming the United States healthcare system, like I did at first.  This is not to say that countries with free healthcare systems are not struggling with these high prices. One of the earlier miracle drugs that vertex came out with is called Orkamni, which became available in the United States in 2015.  However, this medication only became available in places like the United Kingdom in 2019. The reason for the delay in availability in these countries was because Vertex asked the UK government for £105,000 per patient per year for Orkambi, which is more than three times higher than the NICE maximum guideline price of £30,000 per quality-life adjusted year (Nawrat). So the United Kingdom and Vertex were in a stalemate over this medication for 4 years. During this stalemate regarding prices for Orkambi 7,880 packs of drugs expired, forcing them to be thrown out without having even been used (Guo). Each pack covers 28 days, which would mean the destroyed packs amounted to the equivalent of more than 600 years’ supply – or six years for 100 patients. 

     In a similar dispute, Trikafta was approved in the US in 2019, while it was only approved in Canada in 2022. Other countries like India and Africa are still waiting for this life altering medication. While 3 years may not seem like a life changing amount of time, for Cystic Fibrosis Patients everyday makes a difference. As Sanja Stanojevic and others state in their research on the impact of delayed access to Trikafta, “the expected benefits of therapy are cumulative, therefore delayed access to elexacaftor/tezacaftor/ivacaftor will result in preventable health care utilization and deaths'' (Stanojevic). They find that earlier introduction of Trikafta “could reduce deaths by 15% and improve the median age of survival by 9.2 years” (Standojevic). The reason for this is that the health of Cystic Fibrosis patients declines rapidly each year, and these medications stop you where you are at.  So if you imagine a CF patient's health as a downward exponential line on a graph, waiting a year or two could significantly impact how bad your lung and liver health is. 

    By refusing to compromise, Vertex and the UK governments both wasted thousands of doses that could've been used on patients to change their lives, and they also allowed preventable health care utilization and deaths. To this day, there are many patients who still cannot receive these miracle medications because their country cannot afford its high cost.

Conclusion

All of this information is not to say that Vertex is a villain.  This medication has saved and altered so many lives.  Given the rarity of Cystic Fibrosis, Vertex could have opted for broader research benefiting a larger demographic. Instead, they chose to make a monumental impact on the lives of this relatively small population. Below, you'll find an image of my brother during his college graduation. When he entered this world, the prospects of him reaching this milestone seemed improbable. Yet, with these groundbreaking medications, he now has the prospect of a long, healthy, and joyful life. The transformative impact of Trikafta became evident within the initial months — my brother expressed, "I finally felt like a normal person." 

However, the accessibility of these medicines demands attention. While I acknowledge the inherent expenses in healthcare, the inability or refusal to pay for medication should not condemn people to unnecessary suffering. The reduction in copay assistance by Vertex seems like a gamble with insurance companies, assuming they'll absorb the cost difference at the expense of their customers' well-being. One potential solution could involve a gradual decrease in Vertex's copay assistance, allowing patients more time to prepare and giving insurance companies a chance to increase the amount they are giving their consumer. Additionally, reflecting on the four-year stalemate in the UK regarding Trikafta, a compromise should have been made earlier. Wasting almost 8,000 boxes of medication, each capable of changing lives, raises questions about the practicality of discarding them. If the production cost of this medication is indeed high, they should have been more motivated to sell them. One would think that getting something for them is better than throwing it out. 

         In essence, Vertex's contributions are commendable, yet there's an undeniable need for a more balanced approach to ensure the accessibility of these life-changing medications. 

Citations

Cromwell EA, Ostrenga JS, Todd JV, Elbert A, Brown AW, Faro A, Goss CH, Marshall BC.

Cystic fibrosis prevalence in the United States and participation in the Cystic Fibrosis Foundation Patient Registry in 2020. J Cyst Fibros. 2023 May;22(3):436-442. doi: 10.1016/j.jcf.2023.02.009. Epub 2023 Mar 13. PMID: 36922288.

CFF, About Cystic Fibrosis. Cystic Fibrosis Foundation

https://www.cff.org/intro-cf/about-cystic-fibrosis

Gool K, Norman R, Delatycki MB, Hall J, Massie J. Understanding the costs of care for

cystic fibrosis: an analysis by age and health state. Value Health. 2013 Mar-Apr;16(2):345-55. doi: 10.1016/j.jval.2012.12.003. PMID: 23538187.

Guo J, Wang J, Zhang J, Fortunak J, Hill A. Current prices versus minimum costs of production

for CFTR modulators. J Cyst Fibros. 2022 Sep;21(5):866-872. doi: 10.1016/j.jcf.2022.04.007. Epub 2022 Apr 16. PMID: 35440408.

Myshko, Denise. “Cystic Fibrosis Medication Adds Significantly to Healthcare Costs.”

Formulary Watch, Formulary Watch, 8 July 2022, www.formularywatch.com/view/cystic-fibrosis-medication-adds-significantly-to-healthcare-costs. 

Nolen, Stephanie, and Rebecca Robbins. “The Drug Is a ‘miracle’ but These Families Can’t Get

It.” The New York Times, The New York Times, 7 Feb. 2023, www.nytimes.com/2023/02/07/health/cystic-fibrosis-drug-trikafta.html. 

Nawrat, Allie. “NHS England and Vertex Break Stalemate over Price of CF Drugs.”

Pharmaceutical Technology, 24 Oct. 2019, www.pharmaceutical-technology.com/features/nhs-england-vertex-cf/?cf-view. 

Silverman, Ed. “‘Caught in the Middle’: A Battle between Vertex and Insurers Is Leaving Cystic

Fibrosis Patients with Crushing Drug Costs.” STAT, 31 July 2023, www.statnews.com/pharmalot/2023/02/20/cystic-fibrosis-drug-costs-copays-vertex/. 

Stanojevic, Sanja, et.al , Projecting the impact of delayed access to

elexacaftor/tezacaftor/ivacaftor for people with Cystic Fibrosis, Journal of Cystic Fibrosis, Volume 20, Issue 2, 2021, Pages 243-249, ISSN 1569-1993, https://doi.org/10.1016/j.jcf.2020.07.017.