When it comes to accessing treatments for rare diseases, the barriers standing in patients’ ways can be huge. In this week’s blog, Joanna Stenderup, a Pharmacy student at the University of Copenhagen, explores the main barrier people with Cystic Fibrosis face when accessing treatment: the financial barrier. This blog was originally written for BM Manifesto’s Student Voice Essay Competition 2017.

I remember doing an interview with a Danish patient suffering from cystic fibrosis (CF). She was telling me about a new drug she had been selected by her doctor to begin treatment with. The drug was still in a trial phase and therefore not part of the standard treatment in Denmark. I had just begun my second year as a pharmacy student, and I was fascinated by the improvement this patient told me about. She made a huge impact on me. Her lung function had increased. Her quality of life had improved. She had become more medical compliant. She suddenly experienced improvements in her everyday life compared to former medication. She was now able to spent time with her friends after school, instead of going home to take a nap. I thought that everyone suffering from CF should have a chance to be treated with this new drug.

I had more or less the same treatment for 3-4 years. I had reached a state where I thought; why should I take this medication,
if I don’t feel any improvements? That is why I got Orkambi – and I could definitely fell a huge difference. The new drug has
given me so much more energy. Increased quality of life!
” – Patient suffering from cystic fibrosis

In this essay, I consider the main barriers people with the rare disease CF, is facing when it comes to accessing treatment. The barrier I am focusing on is the financial barrier of newly developed and approved drugs on the market, especially for patients suffering from CF.

CF is the most common life-shortening inherited disease, which affects 1 in 3,500 people. It is caused by genetic mutations effecting the protein cystic fibrosis transmembrane conductance regulator (CFTR). This protein regulates the transport of sodium and chloride ions in the cell membranes. Different mutations can cause CF, but the most common one is the delta F508 mutation. Approximately 50% of the patients suffer from this particular mutation. CF patients has thick mucus in their lungs, pancreas and digestive system. This leads to chronic infections and blocking of the pancreas and digestive system, which effect the ability to produce insulin and digest food (1). Some persons with CF consume more than 50 tablets per day. They are more sensitive to infections, because of the chronic infection in their lungs. This means that they are often hospitalized and receiving even more medication such as antibiotics. They are not allow to have contact with other CF patients, due to risk of infection with different bacteria (2).

Most medication for CF only treats the symptoms of the disease. But recently two drugs was approved by the U.S. Food and Drug Administration (FDA). These two drugs acts as precision medicine, which mean they treat the cause of the disease and not just the symptoms (2).

After my interview, I dug deeper into the research of these particular drugs named Kalydeco and Orkambi. I learned that there are patients, whom these drugs may never be available to. Despite the fact that one of the drugs, approved by the FDA in 2015, have been licensed for use in some patients in the UK, the National Institute for Health and Care Excellence (NICE) rejected its use in England. This decision was based on lack of long-term evidence and cost-effectiveness. The drug was estimated to benefit almost 3000 patients suffering from CF (3).

As a pharmacy student, I am well aware of the complicated process of making a new drug available to patients. But I could not help thinking about the patients, who may never experience the potential benefits of this drug (4). I learned that the prices of these newly developed drugs are the center of a huge debate all over the world. I found several articles online stating that a barrier preventing patients from accessing treatment with this drug, was due to its expensive cost of £104,000 per patient for every year of treatment (3). In 2015, Arkansas Medicaid was sued by patients suffering from CF. They claimed the state denied using Kalydeco based on its high cost. The lawsuit ended with a settlement (5).

It is no secret that a medical company’s decision about choosing a new target can be market-driven. This is true for all other companies, whether they sell clothes, food, makeup or medicine. Large companies prefer to develop new drugs, which have huge potential markets, instead of new drugs, which is a need, but maybe only among populations and countries which are more or less unable to pay a high price. Furthermore, certain companies have a historical background for research in a specific medicinal area. The decision can also be driven by fashion, for example if several companies are doing research on the same target, due to increased literature or media coverage of a particular disease (6). The dilemma is difficult because the pharmaceutical company pays for the costs associated with development and clinical tests. It is unusual to find a person, who can afford these costs by themselves. It therefore relays on the government to decide if the patients are worth the treatment or not. But the government only has a certain amount of money – and there are more expenses than healthcare which should be covered. Since Kalydeco and Orkambi are some of the world’s most expensive drugs, it will be extremely costly to make them a part of the standard treatment in most countries (7).

Medics and researchers are trying to improve access to treatments for CF patients, by identifying patients who will benefit from the treatment with certainty (8). This is currently carried out by creating mini-organs with different types of CF mutations. The drugs are then tested in different organs, to clarify the individual effects (9). This is a really good idea, which could be implemented, not only when it comes to CF, but also a lot of different types of cancer and other diseases. The problem with expensive treatments are that they are sometimes “wasted” on patients, who do not benefit from it. But the doctors has to try it, because they do not know if it works before they have tried.

Another way to improve access to treatments for CF patients, would be if other pharmaceutical companies tried to develop a similar drug, or an entire new drug. Thereby creating competition and gradually lowering the price. This is already happening, but other companies are not allowed to developed and sell generic drugs before the patent has expired. Such drug patents can last 10-15 years, depending on which country is it issued by (10).

Since CF is a progressive incurable disease, the patients and their loved ones, hope for new drugs on the market. It must be difficult for these patients to be aware of the existence of two newly approved drugs, which may never be a part of their treatment. The life expectancy for CF patients is 42 years, so they do not have time to wait for a new drug, because the one existing might be too expensive. I cannot imagine what it would mean if the patient with CF I met no longer can be treated with Orkambi. It is difficult to talk about money when it comes to health and diseases, because who can justify if a person’s life and well-being is worth the expenses?

1. Flavell L. Cystic Fibrosis Overview2017. Available from: https://cysticfibrosisnewstoday.com/cystic-fibrosis-overview/.
2. Cystic Fibrosis Trust. What is cystic fibrosis? : Cystic Fibrosis Trust; 2017 [Available from: https://www.cysticfibrosis.org.uk/en/what is cystic fibrosis.
3. Gulland A. Cystic fibrosis drug is not cost effective, says NICE. BMJ. 2016;353:i3409.
4. U. S. Food & Drug Administration. Drug Trials Snapshots: ORKAMBI [WebContent]. Center for Drug Evaluation and Research; 2017 [Available from: https://www.fda.gov/Drugs/InformationOnDrugs/ucm455282.htm.
5. Walker J. Arkansas Reaches Settlement in Cystic Fibrosis Drug Suit2015. Available from: http://www.wsj.com/articles/arkansas-reaches-settlement-in-cystic-fibrosis-drug-suit-1423162197.
6. Heinrich M, Barnes J, Gibbons S, Williamson EM. Fundamentals of Pharmacognosy and Phytotheraphy 2ed. Great Britain: Elsevier Churchill Livingstone; 2012.
7. Werth B. A Tale of Two Drugs2013. Available from: https://www.technologyreview.com/s/520441/a-tale-of-two-drugs/.
8. Community Register [Internet]. EU Pharmaceutical Informations. Available from: https://ec.europa.eu/health/documents/community-register/.
9. Regalado A. Organoids Proposed to Screen Patients for High-Priced Drugs2017. Available from: https://www.technologyreview.com/s/608141/organoids-proposed-to-screen-patients-for-high-priced-drugs/.
10. Mehta SC, Mehta SS. Strategic options for brand-name prescription drugs when patents expire. Health Mark Q. 1997;14(3):107-14.