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Why Pay $150,000 Year for a Lung Drug if it Does Not Work?

The Apha 1 group comprises about 10% of COPDers but they are the group who has made great organizational strides in bringing public awareness to COPD (Chronic Obstructive Pulmonary Disease)
They are politically astute and I like most of the folks I’ve met in the organization.

That said… the idea of paying $150,000. a year per person for a drug that, at best, does nothing for the patient is OUTRAGEOUS!

There is little to no research being done for regular COPDers…if this study is accurate, why can’t we use that wasted $150,000. Per patient for RESEARCH?

Roll the drug manufactures out of the profit at any cost bed and use the money where it will do the most good for the most people.
Why not?!

I am including the following verbatim for obvious reasons.

Wasted drug dollars? NO!
Research dollars for the COPD majority benefit? YES!

“Pricey lung disease drugs have no benefit: study

URL of this page: (*this news item will not be available after 10/04/2010)

Tuesday, July 6, 2010
By Kate Kelland
LONDON (Reuters) – Recommendations for expensive treatments made for a genetic disorder called alpha-1 antitrypsin deficiency should be withdrawn because the drugs have no benefit, scientists said on Wednesday.

The disorder causes chronic lung disease and researchers who reviewed data from two trials on 140 patients with it found no evidence that alpha-1 antitrypsin medicines — made by various drugmakers including Talecris, Kamada, CSL and Baxter — do any good.

Based on this evidence, the researchers said the treatment, which costs up to $150,000 a year in the United States, should not be recommended by doctors and advocacy groups.

“The drug has not shown any clinical benefit, is extremely costly and has important adverse effects,” said lead researcher Peter Gotzsche of the Nordic Cochrane Center at Rigshospitalet in Copenhagen, Denmark.

“In view of the lack of evidence and high cost of treatment, treating alpha-1 antitrypsin deficiency by replacement therapy cannot be recommended.”

According to the team, whose work was published in The Cochrane Library journal, recommendations by the American Thoracic Society and European Respiratory Society that promote alpha-1 antitrypsin replacement are “misguided”.

“Both societies recommend augmentation therapy for patients with breathing problems related to alfa-1 antitrypsin deficiency. In our opinion, these recommendations are not reasonable,” said Gotzsche.

Alpha-1 antitrypsin deficiency affects less than one in 1,600 people. Those who inherit the disorder have low levels of the protein alpha-1 antitrypsin, also called alpha-1 proteinase inhibitor, which protects the tissue of the lungs from destruction by the body’s own white blood cells.

At a relatively young age, this can result in symptoms of emphysema, including shortness of breath and wheezing.

The aim of alpha-1 antitrypsin replacement therapy is to give the patient back the protective protein they are missing. This should limit damage to lungs and, ultimately, prevent early death. The protein is usually extracted from blood donated by healthy volunteers.

The researchers reviewed data from two trials involving a total of 140 people with the disorder, all of whom were at a high genetic risk of developing chronic lung disease.

In one trial, patients were given intravenous alpha-1 antitrypsin or a placebo every four weeks for three years and in the other, the treatment or a placebo was given weekly for a minimum of two years.

There was no difference between treatment and control groups in terms of exacerbations of lung disease, or quality of life, the researchers found. Combining the results from the trials, Gotzsche’s team also found no evidence of a clinically important effect on lung function.

“Indeed the results suggested modest harm, or at best no effect,” they wrote in their study. They added that while the treatment might cause a reduction in the deterioration of lung appearance on CT scan, it was “not clear whether this is a clinically meaningful difference.”

More later… Sharon O’Hara

4 thoughts on “Why Pay $150,000 Year for a Lung Drug if it Does Not Work?

  1. John Morton
    Submitted on 2010/07/11 at 6:17pm

    I feel your passion for wanting to find a valid treatment for hereditary COPD but your ire is misdirected as are the conclusions you are basing your blog post upon.

    IV Alpha-1 augmentation therapy IS effective and has been so for years. The argument in the UK as in many other countries is that since it is an expensive orphan drug used to treat a relatively small number of needy citizens, the cost benefit analysis appears to favor simply refusing treatments and saving the state the money.

    First… a little about the study you are referring to:
    “Danish researcher Asger Dirksen, MD, originally listed as a co-author of the Gøtzsche review, had his name removed before publication.

    Dirksen, who was the lead author of both augmentation studies cited by Gøtzsche, said today:

    “After seeing the first draft I realized that our points of view were so far apart that collaboration with Peter Gøtzsche and his wife (Helle Krogh Johansen) would not be possible.””

    The Gøtzsche publication relies on combining the results of these two small randomized, placebo-controlled trials (each enrolling less than 100 study subjects), and is not based on the raw data from these studies. The review neglects the fact that the two studies had a significant number of individuals who enrolled in both studies and hence should not be included twice in combined analysis as this biases the data, said the Foundation statement.

    “The article also discards an important endpoint of both studies, the evaluation of loss of lung tissue as judged by CT scans, as being of no clinical interest. In fact, CT scans are the most direct method for evaluating the extent and progression of emphysema – the primary lung disease suffered by those with Alpha-1 – and now accepted as the best predictor of mortality in this disease,” said Robert A. Sandhaus, MD, PhD, Foundation Clinical Director. “In fact, the meta-analysis by Gøtzsche did show a benefit of augmentation therapy in reducing the loss of lung tissue as measured by chest CT.”

    Sandhaus said the review also “overlooks the effect of study dropouts related to worsening lung function in the placebo groups, which unbalances the differences in treatment groups.
    Finally, while arguing that augmentation therapy is too costly, the authors ignore the several publications that have actually studied the cost effectiveness of augmentation therapy.”

    Added Sandhaus: “In selecting only these two small studies on which to base their sweeping recommendation, Gøtzsche and his co-author have ignored the wealth of other data in the medical literature regarding the effectiveness of augmentation therapy in Alpha-1.” Several large observational studies have concluded that augmentation therapy slows the progression of lung disease and the largest of these studies, including over 1,100 individuals with Alpha-1, has also shown longer survival among those on augmentation therapy, Sandhaus said.

    “While large randomized, controlled, blinded studies remain the gold standard in the evaluation of therapeutics, performing such studies in a population of patients with a rare genetic condition presents almost insurmountable obstacles,” said Sandhaus. “In these situations, scientists, clinicians, and patients must rely on the best evidence available, and the preponderance of scientific data support the use of augmentation therapy in Alpha-1 as recommended by experts representing the leading respiratory societies in both the USA and Europe.”

    The Foundation statement said that virtually everyone in the Alpha-1 community agrees that additional studies are needed to help evaluate the effectiveness of augmentation therapy, but Gøtzsche and his co-author end their article with the conclusion that “further studies with surrogate markers cannot be recommended, if the aim is to elucidate whether or not augmentation therapy with alpha-1 antitrypsin has a relevant clinical effect.”

    The Foundation’s Medical and Scientific Advisory Committee notes that not every patient with
    Alpha-1 requires this therapy, and along with several recognized experts in the international scientific community, both in the USA and Europe, is currently preparing a formal response to the Gøtzsche article.

    Kamada shares dropped 4% yesterday on a turnover of NIS 49 million, over six times the share’s average daily turnover, a week after receiving U.S. Food and Drug Administration approval for its flagship drug, Glassia. At one point during the trading day the share had fallen by 10%. The collapse was a response to research published by Copenhagen’s Nordic Cochrane Center that found that drugs for treating a rare genetic disorder called alpha-1 antitrypsin deficiency, which can cost up to $150,000 a year in the United States, have no clinical benefit.

    One has to wonder what the real motivations are behind the timing of the release of this report based on Bad Science (BS).

    I don’t know you Sharon and pray that you or any of your loved ones will ever be told they have it have alpha-1 antitrypsin deficiency related COPD. Your posting reflects a true passion for doing something to help those with this condition through valid research for treatments and eventually a cure. I would invite you or your readers to visit or their fellow organization to find out more about the lives of many of us who ARE on weekly infusions – including myself.

    I was diagnosed in 1999 after having lost 50% of my lung function between 1994 and 1999. After being diagnosed, I started IV therapy and have not lost any documented function since.

    The audacity of Cochoran Library to allow something so flawed to be published and then mindlessly reported by Reuters/Kate Kelland and then your blog, makes the ignorance about the condition and the treatments irresponsible at best… inexcusable at worst.

    It’s always good to do your homework before reported what someone wants you to believe is the news.

  2. Thank you for your response…it is ALWAYS good to educate the public.

    I’m curious to know why the plain jane, majority Mutt and Jeff variety COPDer has NO RESEARCH and no new drugs to combat loosing our lung function… for any amount of money …whether they work or not…we don’t have any.

    How can that be?

  3. Author : DC Young …

    This study is fundamentally flawed in stating that augmentation therapy for Alpha-1’s is not functional. Any Alpha that follows this recommendation has not studied the issue. Augmentation therapy will not “restore” lung function. But it will – under most situations stop or retard the rapid deterioration to lungs. If the researchers want to verify that, they should just get with those who augment and have kept their lung function stable for 5 – 10 – 15 years compared to when it was fading rapidly every year before the therapy was started.

    The study does a disservice to all those who are campaigning for a cure to Alpha-1 Antitrypsin Deficiency. It needs to be retracted.

    DC Young – a grateful Alpha – who Augments and knows it helps.

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