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This Time, It’s Personal: New Cystic Fibrosis Drug (with $294,000 Price Tag)

Posted by Daniel Sharp on May 15th, 2012


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One rarely sees cystic fibrosis in the headlines. But a new experimental drug has recently boosted the disease into the pages of the New York Times, the Boston Herald, and Forbes. The relative flurry of coverage is based on both a legitimate and welcome medical discovery, and on speculative promises about personalized medicine.  

On January 31, the FDA announced its approval of the new cystic fibrosis (CF) drug, which is licensed to Vertex Pharmaceutical. The drug, Kalydeco (which goes by the generic name ivacaftor), gained quick approval  after it showed significant and sustained improvement in lung function in two 48-week clinical trials involving 213 CF patients. This is of course great news for people with CF.

Despite its promise, Kalydeco has a significant limitation. Cystic fibrosis is a genetic disorder, caused by mutation or damage in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene, which codes for a protein that regulates ion and water transport in the body.  The efficacy of medical treatments for CF is determined in part by the nature of the underlying mutation, which is not the same in all CF patients. Kalydeco is only effective in those who have the G551D mutation, only 4% (1,200 people) of the 30,000 individuals diagnosed with the disease in the US. The drug is not efficacious in CF patients with the F508 mutation that causes about half of CF cases.   

For reasons that remain largely unknown, Vertex made a conscious decision to go after the variant that affects 4% rather than 50% of CF patients.  However, new clinical evidence suggests that there might be a way for Kalydeco to overcome this limitation, and aid in the treatment of those with the F508 mutation as well. The results are far from definitive, but suggest that a combination of Kalydeco and another drug called VX-809 can have positive effects on lung function of patients with the more common variant of CF.

This is very good news for those afflicted by CF. Joe O’Donnell, a fundraiser for the Cystic Fibrosis Foundation, called the discovery a “game changer.”  

In the biotech and regulatory worlds, however, the discovery is being praised for another reason: its connection to personalized medicine.  FDA Commissioner Margaret A. Hamburg said that Kalydeco is “an excellent example of the promise of personalized medicine – targeted drugs that treat patients with a specific genetic makeup.” According to Stephen Spielberg, deputy commissioner for medical products at the FDA, the new Kalydeco and VX-809 combination treatment is “part of a revolution of how we will treat patients in the future, using increasingly targeted, personalized treatments.”

Though it is far too soon to tell whether personalized medicine will improve health care for many conditions or only a few, Kalydeco demonstrates that it has promise at least for some. But if Kalydeco is a model for the future of personalized medicine, there are some important questions to be raised. One big question mark concerns access.  A year’s worth of Kalydeco treatment is currently priced at $294,000. That’s an astronomically high price, one that reflects a general trend in medicines targeted to treat small subsets of patients.  

This trend has a certain logic: pharmaceutical companies want to make a large return on their investments, and since targeted medicines have small client bases, corporations charge patients more, often justifying this process by over-inflating the costs of R&D.

Of course, Vertex says they will work to ensure access, but there’s essentially no accountability mechanism in place to ensure this happens. In fact, there’s a wealth of data that suggests big pharma has notoriously limited access to essential medications in order to boost bottom lines.

The question of social justice looms in the background of an otherwise very hopeful story. As Karen Peterson-Iyer, a specialist in health care ethics, puts it:

In these circumstances, only the rich or the insured may be able to afford [personalized drugs]. From the standpoint of justice, one of the most disturbing possibilities raised by pharmacogenomics is that it will further entrench the already-deep socioeconomic divisions that characterize modern U.S. society.

The goal of personalized medicine – better treatment for patients – is certainly an admirable one, but we need to make sure that all patients can reap the benefits.

Previously on Biopolitical Times:

 





Posted in Biotech & Pharma, Media Coverage


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