High hopes, higher costs seen for cystic fibrosis

Posted June 11th, 2015


The first treatment to address one of the underlying causes of cystic fibrosis (CF) was approved in 2012. The cost was high – around $300,000 per year – but the financial impact was relatively small, since Kalydeco™ (generic name ivacaftor) targets a specific gene mutation found in fewer than four percent of people with CF.1

Now a new combination drug is moving rapidly toward approval for CF: Orkambi.™ 2 Orkambi is a compound that combines the existing drug Kalydeco, plus a new experimental drug called lumacaftor. Orkambi can be used to fight the gene mutation found in nearly 50 percent of people with CF.1

Industry observers expect the new drug to be priced similarly to Kalydeco, which means potentially large new costs for employers.1, 2 In addition, we should note that Orkambi will likely be taken in addition to other standard care therapies (which are already costly), including bronchodilators, Pulmozyme®, inhaled and oral antibiotics and others.3

The FDA has committed to making a final decision on Orkambi by early July, although that action could also come sooner.


CF is a rare genetic disease that causes mucus to become thick and build up in the lungs and other organs. The build-up hinders breathing and makes it easy for bacteria to grow, which leads to repeated, life-threatening lung infections.4 Some patients end up needing lung transplants due to the damage caused by these repeated infections.

CF can also block small passages in the pancreas which prevents pancreatic enzymes from reaching the small intestine. Without these enzymes, the intestines can't fully absorb nutrients, which means that CF patients can also suffer from malnutrition.4

An estimated 30,000 children and adults in the United States have CF.4

As a result of these difficulties, an infant born with CF in years past was not expected to live more than a few months.5 Thanks to many advances in treatment and care, 90 percent of babies born today are surviving their first year of life, with many living into adulthood, well into their late 30s.5

From survival to quality of life

Until quite recently, the underlying causes of CF were untreatable. The available care could be as primitive as repeatedly clapping the patient on the chest and back in order to loosen the mucus building up in their lungs.3 As a result, most people with CF faced many challenges, including hospitalization to fight infections. At times even ordinary activities were hard to perform.6

This began to change with the introduction of the first genetically targeted therapy (Kalydeco) in 2012. For the first time we had an intervention that corrected for the genetic mutation and allowed the cells to properly regulate the transfer of critical chemicals in and out of the lungs and other organs.6 The resulting chemical balance allows the mucus to function normally, which allows patients to breathe and process food properly, all while being protected from infection. The proposed combination therapy contained in Orkambi is expected to work along similar lines.2, 6

These new CF drugs represent a significant advance in pharmaceutical design. Traditionally, many drugs were designed to find an overactive or otherwise offending target and stop it. For example, Lipitor reduces the production of “bad” LDL cholesterol.6

Because CF is caused by underperformance at the cellular level – certain chemical channels don’t function as well as they should – treating the underlying cause means helping them perform better, instead of blocking them. Therefore, Kalydeco and Orkambi are called “potentiator” and “corrector” drugs, because they actually encourage the body to function as it was intended to do.6

Despite their new method of action, neither Kalydeco nor Orkambi are considered to be cures, because their effects are only temporary.6  This is important, because it means that CF patients who take them must take them indefinitely. Nevertheless, some CF patients who have used the new genetically targeted drugs have reported dramatic improvements in their quality of life.

People who had previously found it impossible to exercise now say that they can walk and even run when using Kalydeco and Orkambi. 2, 6 One patient was able to go on an overseas mountain backpacking trip, a trip he says he could not have attempted previously.7

New combination drug expands treatments

While Kalydeco has shown encouraging results, it is effective for a very small percentage of CF patients. Only around four percent of CF patients have the exact genetic form of the disease that can be treated by Kalydeco alone.

That’s why anticipation is high for the new Orkambi combination therapy. It is aimed at patients with two copies (homozygous) of the F508del mutation.8 The pie chart below shows that an estimated 50 percent of all CF patients belong to this category. Of these, it is projected that approximately 90 percent will request the new drug.1


Cost is a major concern. Kalydeco is among the most expensive of all prescription drugs, costing upwards of $300,000 per year.1 Looking at the graph above, we see that the approximate life expectancy a person with CF is age 40. If someone was consistently treated with Kalydeco for 30 years they would accumulate $9 million in drug costs alone (in simple, extrapolated current dollars).1

The new combination drug is expected to be priced similarly to Kalydeco.1, 2

As noted at the beginning, we can probably expect that Kalydeco and Orkambi will be used together with existing standard therapies, some of which are also expensive. To some extent these combined drug costs may be offset if the new genetically targeted drugs are successful at preventing major medical expenses, like lung transplants. CF is the third most common reason for lung transplantation, which can cost nearly $600,000, plus additional medication and medical care in subsequent years.1, 9 About 900 people receive lung transplants due to CF each year in the US.10

Employers seeking to understand their potential cost exposure to these new drugs would need to evaluate their CF population according to which genetic mutation each person suffers from.

Strategies to manage

OptumRx will continue to monitor the CF space. As always, we have prepared a robust total approach to cost management that combines a proactive drug management strategy together with clinical management and adherence programs designed to promote the use of clinically appropriate medications and support each member for better outcomes.

  1. Formulary Journal. New cystic fibrosis drugs may significantly increase pharmacy benefit costs. April 15, 2015. Accessed at: http://formularyjournal.modernmedicine.com/formulary-journal/news/new-cystic-fibrosis-drugs-may-significantly-increase-pharmacy-benefit-costs?page=full on 04.28.2015.
  2. Boston Globe. Vertex wins key approval for cystic fibrosis treatment. May 12, 2015. Accessed at: http://www.bostonglobe.com/business/2015/05/12/fda-panel-meets-today-rule-vertex-treatment/mxu38yBwiSUh9aTWKbItmL/story.html on 05.14.2015.
  3. Cystic Fibrosis Foundation. Therapies for Cystic Fibrosis. Feb. 14, 2012. Accessed at: http://www.cff.org/treatments/Therapies/ on 05.27.2015.
  4. National Institutes of Health. What Is Cystic Fibrosis? Dec. 26, 2013. Accessed at: http://www.nhlbi.nih.gov/health/health-topics/topics/cf on 04.29.2015.
  5. Medical Daily. Cystic Fibrosis Patients Living To Adulthood to Increase 75% By 2025; Health System Must Adapt To Treat Influx of Adult Patients. Accessed at: http://www.medicaldaily.com/cystic-fibrosis-patients-living-adulthood-increase-75-2025-health-system-must-adapt-326148 on 04.28.2015.
  6. Scientific American. New Vertex drug combination for cystic fibrosis represents a triumph of drug discovery research. June 28, 2014. Accessed at: http://blogs.scientificamerican.com/the-curious-wavefunction/2014/06/28/new-vertex-drug-combination-for-cystic-fibrosis-represents-a-triumph-of-drug-discovery-research/ on 04.28.2015.
  7. Pittsburgh Business Times Kalydeco transforms Pitt grad student’s life. Mar 15, 2013. Accessed at: http://www.bizjournals.com/pittsburgh/print-edition/2013/03/15/kalydeco-transforms-pitt-grad-life.html?page=all on 04.29.2015.
  8. The Street. What to Expect From Vertex Pharma's Next Cystic Fibrosis Drug Results. Feb. 23, 2015. Accessed at: http://www.thestreet.com/story/13054375/1/what-to-expect-from-vertex-pharma-next-cystic-fibrosis-drug-results.html on 04.28.2015.
  9. Transplant Proceedings. Lung transplantation in patients with cystic fibrosis. April, 2013. Accessed at: http://www.ncbi.nlm.nih.gov/pubmed/23622646 on 04.30.2015.
  10. Cystic Fibrosis Foundation. Lung Transplants. Accessed at: http://www.cff.org/Mobile/livingwithCF/lungtransplants/ on 04.30.2015.