CF Reading: Research, Drug Costs and the Loss of a Beautiful Life

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by Stephen Shannon |

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Being informed on the science, drug developments, and public policy of CF is an excellent way to engage with the CF community and connect with your healthcare team. If you become easily overwhelmed by the churning waves of internet news outlets, I would like to extend you a small life-saver with four of my top articles on the current events in CF (disclaimer: three are recent and one is an old standard we should all come to love.)

She Always Had Hope. She Needed a New lung.

I can’t help but to start with this article. To live with CF is to have a perspective not characteristic of one who is carefree but of someone who understands the richness that can be brought in life through suffering, pain and loss. I felt an intimate connection reading this story of a friend who retained hope in the midst of losing her life to CF.

In a short article published in 2007, in the New York Times, Annie Lehmann shares an experience of losing a dear friend to CF at a time when organ transplants were given to those who had waited the longest and not who were necessarily the most sick. The pain of loss shared in this story is fresh in my heart as last month I also lost my sister, Jennifer, to the disease. From my experience, I ached in reading when Annie last saw her friend in the hospital room. “I was afraid that she would sense the effort behind my smile. As I left, she gave me two thumbs up, and I kissed her blanketed leg and told her, ‘I love you.’” Annie shares a piece of her friends life as she was diagnosed late in life; and although her health began to decline she maintained a relentless optimism and hope as she managed life with her husband and kids.

I appreciate the perspective the article gives from someone who experiences CF through the loss of someone loved. In having CF I often forget that the pain goes beyond what I feel in myself, but is extended to those who love me and are helpless but to watch in a unique suffering. I found a beautiful connection with this story and I would urge you to take a moment to read it for yourself.

Cystic Fibrosis Discovery May Lead to New Treatment Strategy to Help Patients Breathe Easier.

The next article is an abrupt transition from the emotion of the first article to the objective and, some could argue, bland findings of a study from the University of California San Francisco (UCSF). The article, published on February 25, 2015 by UCSF, relates the findings of a recent collaborative study between UCSF, University College Dublin, and the Cleveland Clinic in Ohio,

The study helps to elucidate the mechanism underlying the thick mucous characteristic of CF. “Fahy and his team found that — contrary to previous belief — inflammation causes new molecular bonds to form within mucus, transforming it from liquid to an elastic sludge.” The findings led the team to begin developing a medicine to help disrupt the “disulfide bods in the mucin polymers.”

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Will a New Vertex Drug for Cystic Fibrosis Become a Budget Buster?

The article published by The Wall Street Journal on April 15, 2015 seems to give more concern than consolation. The writing helps to provide context to the financial burdens both to the healthcare system as well as to individuals as new medications continue to be released for rare diseases.

“In an analysis of insured patients, Prime Therapeutics estimates that it may cost as much as $4.5 billion annually for the nation’s health care system to pay for a combination medicine that includes Kalydeco – an existing and treatment sold by Vertex Pharmaceuticals – along with another Vertex compound.”

This article finds considerable relevance with the release of the new combination therapy by Vertex in July of this year. The article raises the question of what is fair compensation for a company who takes on the risky financial investment to develop medications for rare diseases. I believe it is fair for these companies to profit from their investment, because without their effort the ability for patients to take the medication would not be possible. However, I believe accessibility of the medication to patients is the first and primary objective when releasing these medications.

Science, industry economics, patients are boosting research on rare disease drugs _ and hope.

This article, published in US News on March 25, is related to the prior article in highlighting the changing direction of the pharmaceutical industry in investing in research and development in drugs for rare diseases.

“Last year, the Food and Drug Administration approved 17 medicines for rare diseases. More than 450 others are in development to treat a wide variety of ailments…in the 1970s, only a handful of rare-disease drugs got approved. Then the 1983 Orphan Drug Act helped rouse interest by providing expedited drug reviews, tax credits and other benefits for drug companies.”

There is a palpable stir in the CF community as the past few years have brought life changing medications into the homes of a percentage of CF patients. The CF Foundation, highlighted in the article, is at the forefront of the rapid progress in the treatment of CF with its ‘venture philanthropy’ model used to strategically invest in research projects in an effort to bring them quickly through clinical trials and into market.