MassBio President Knows Difficulty of Living with Cystic Fibrosis

According to the Boston Globe, the president of the Massachusetts Biotechnology Council has a personal stake in his continued advocacy for pharmaceutical development in his home state.

Robert Coughlin has been a public figure in Massachusetts for some time, having previously served in the state legislature from 2003 to 2007. Around the time of Robert’s campaign, his son Bobby was born.

Bobby is 16 now, and a sophomore in high school. He balances friends, basketball, and homework like any other person his age – on top of the daily doses of dozens of pills and hour-long therapy sessions.

Bobby has cystic fibrosis (CF), an inherited genetic disorder that makes the mucous produced by his body thick and viscous. It must be treated daily, or health complications could arise. Every day Bobby spends forty-five minutes using a nebulizer, which he follows up with just under a half hour of using a sound-emitter to loosen the mucous in his lungs. Though manageable on a daily basis, the outlook for most CF patients is serious – average life expectancy being somewhere around 37 years. A particularly lucky patient might live into their fifties. It can be a grim diagnosis, and despite medical advances is likely to affect over 30,000 Americans.

Massachusetts Biotechnology Council

Bobby’s medical challenges quickly inspired his father’s line of work. In 2007 Robert left politics to helm the nonprofit Massachusetts Biotechnology Council, or MassBio. His work there finds him lobbying for pharmaceutical developers, mostly. His political pedigree was welcome by the nonprofit – providing a more intimate understanding of the legalese that makes up a good part of any pharmaceutical undertaking. It was perhaps the experiences with his son, however, that ultimately convinced the board to pursue hiring Coughlin in 2007, former MassBio board of directors chairman Mark Leuchtenberger acknowledged. Leuchtenberger offered that someone personally affected by such a condition has a deeper understanding of the personal costs involved, and stated that it remains a “source of [Coughlin’s] energy.”

Encouraging Pharmaceutical Development

Cystic fibrosis is more manageable now than ever, though certain cases elude effective pharmaceutical treatment. In 2012 the FDA approved their first medication designed to treat CF patients – Kalydeco, from Vertex Pharmaceuticals Incorporated. However, there are hundreds upon hundreds of possible mutations that can lead to someone being born with cystic fibrosis – no single drug is enough to treat everyone. Over the next several years, Vertex had two more CF drugs approved by the FDA. By Vertex’s estimate, over half of America’s 30,000 known cystic fibrosis patients are eligible for at least one of their three drugs.

Bobby Coughlin, however, is not one of them. But thanks to his father’s work, that might be changing.

Vertex is in late clinical testing for a new drug they hope to market – and its expected to treat people with the same genetic mutation as Bobby. The Coughlins, and thousands of others, eagerly await further results to be announced, expected to come some time early this year.

Despite the excitement, the steep price of Vertex’s drugs has some heads spinning. A year’s supply of each of their three CF drugs averages out to just around $292,500. Many have decried the figure as price-gouging the desperate, though Robert Coughlin and the nonprofit he leads adamantly defend such sky-high figures. Robert claims that such prices are necessary to fund the next stages of drug development, a notoriously expensive undertaking.

That revenue, he argues, is an investment in drugs that are yet to come – like those that he hopes might treat his son.

Although the high prices of this kind of medication are sometimes covered by insurance, for some patients the financial strain continues to be too great. Are better incentives for pharmaceutical manufacturers required for development? Or do you think the high price tags are more about profit than research? Share your thoughts with Patient Worthy!

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