Should the FDA approve Vertex’s new cystic fibrosis drug?

The scene at the FDA hearing was familiar. All the  advisory committees have seen it play out again and again. Yesterday, it was the Pulmonary-Allergy Drug committee: The pharma doc with the convincing statement. The weeping patients. The drug likely to cost a couple thousand a month offering a slim  benefit over an existing drugs. But it works, and it’s safe.

From the Globe:VertexLogoSOP

“I think this is a much-needed advance for patients with cystic fibrosis,” said committee member Dr. Michelle S. Harkins, associate professor of medicine at the University of New Mexico Albuquerque, one of the majority voting to recommend approval.

The lone dissenter in the 12-1 vote recommending approval of the drug, Dr. Yanling Yu, the president and cofounder of Washington Advocates for Patient Safety in Seattle, said she was not convinced the data generated by the Vertex testing supported the approval of Orkambi.

“I really understand the patients critically need a new drug, but sometimes a new drug does not provide [the needed effectiveness],” she said.

From The New York Times story:

An issue for the advisory committee was that Orkambi had what the F.D.A. said was only modest effectiveness, improving lung function by only about 3 percentage points relative to placebo.

Some family members or advocates, some of them crying, pleaded with the committee to endorse the drug.

Some patients who took the drug in clinical trials said it had made a huge difference in their lives, reducing their coughing, allowing them to exercise better, helping them gain weight or reducing how often they ended up in the hospital…

Michael Yee of RBC Capital Markets, for instance, expects the price will be $225,000 to $250,000 a year.

The vote is advisory. The FDA staff will make the final call.

More here:

The financial stakes for Vertex.

FDA briefing for meeting.

Vertex briefing for the meeting

2 Boston studies: Breast cancer costs and medical records benefits

After pushing mammograms for years, breast cancer advocates have had a hard time pulling back. Still the evidence keep piling up that message about surviving breast cancer is more complicated than early detection.  What this study by Boston researchers doesn’t measure: the scores of women who say — If there’s a chance it’s cancer, I want to know.  From the AP, in the back pages of the Globe A section; buried in the web site.

Sharpening a medical debate about the costs and benefits of cancer screening, a new report estimates that the United States spends $4 billion a year on unnecessary medical costs due to mammograms that generate false alarms, and on treatment of certain breast tumors unlikely to cause problems.

On a lighter note, another study centers on the benefits of giving patients their own records. From the NYTimes.

ONC_PSA_BannerAd_300x250_Red_Button_1Some of the most advanced medical centers are starting to make medical information more available to patients. Brigham and Women’s, where Mr.  (Steven) Keating had his surgery, is part of the Partners HealthCare Group, which now has 500,000 patients with web access to some of the information in their health records including conditions, medications and test results.

Other medical groups are beginning to allow patients online access to the notes taken by physicians about them, in an initiative called OpenNotes. In a yearlong evaluation project at medical groups in three states, more than two-thirds of the patients reported having a better understanding of their health and medical conditions, adopting healthier habits and taking their medications as prescribed more regularly.

Here’s the Globe’s take.

Some patient advocates have been pushing for this for years, and having a little fun with it.

What is wrong with medical research? Boston health writers raise important questions

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Do we need evidence-based evidence? How effective is our medical research system? What are we getting for the $30 billion that goes into university and hospital labs? Here are a couple of stories from local writers with some discouraging answers to those questions.

Carolyn Johnson’s piece on poor quality of medical research was tucked under the Globe’s Sunday editorial urging Elizabeth Warren to run for president. If only it could get as much attention:

Evidence of a quiet crisis in science is mounting. A growing chorus of researchers worry that far too many findings in the top research journals can’t be replicated. “There’s a whole groundswell of awareness that a lot of biomedical research is not as strongly predictive as you think it would be,” said Dr. Kevin Staley, an epilepsy researcher at Massachusetts General Hospital. “People eventually become aware because there’s a wake of silence after a false positive result,” he added. The same is true in every field of science, from neuroscience to stem cells.

Ideally, science builds on and corrects itself. In practice, the incentives facing scientists can hamper the process. It’s more exciting and advantageous to publish a new therapeutic approach for a disease than to revisit a past discovery. Yet unless researchers point out the limitations of one another’s work, the scientific literature can end up cluttered with results that are partially or, in some cases, not at all true.

Recently, researchers and the US government alike have sought to assess how much research is irreproducible — and why — and are looking for systematic ways to retest experiments that make headlines but yield no further progress.

Her story follows a piece by another local writer on how stem cell research has not yet delivered on its promises. Karen Weintraub’s piece was in the New York Times in September.     

Stem cells broke into the public consciousness in the early 1990s, alluring for their potential to help the body beat back diseases of degeneration like Alzheimer’s, and to grow new parts to treat conditions like spinal cord injuries.

Progress has been slow. The Michael J. Fox Foundation for Parkinson’s Research, an early supporter of stem cell research, pulled much of its financial backing two years ago, saying that it preferred to invest in research that was closer to providing immediate help for Parkinson’s disease patients.

But researchers have been slowly learning how to best use stem cells, what types to use and how to deliver them to the body — findings that are not singularly transformational, but progressive and pragmatic.

Finally, don’t miss this week’s Health Wonk Review, a digest of health policy blog posts.

Boston Globe reports: Biolab & ebola

Two recent Boston Globe items of note:

Local hospitals prep for ebola

emergencyIn response to the West African outbreak of the deadly Ebola virus, some Boston hospitals are instructing clinical staff to ask patients as soon as they arrive about their travel histories, and reminding doctors and nurses of the symptoms.

But hospital officials say they would be ready to quickly identify the illness and prevent its spread if an infected patient showed up, using protocols and equipment already in place.

Column : Local biolab sits mostly empty as the CDC copes with its own lab safety crisis.

Over a decade of community protests, Boston University has beaten back lawsuits aimed at closing the lab and won City Council backing. Final approvals are still pending from the Boston Public Health Commission and the federal Centers for Disease Control and Prevention.

Its critics, though, have been given fresh ammunition: The CDC confessed earlier this month to sloppy handling of anthrax and avian flu at its laboratories elsewhere, exposing dozens of employees to the deadly bacteria. The mistake was the kind that proponents of the Albany Street lab had called nearly impossible.

“The CDC example is a wake-up call, if you needed a wake-up call,” said David Ozonoff, a Boston University professor and the longtime dean of its department of environmental health who opposes the lab.

In today’s Globe: Stem cells, retractions, cystic fibrosis and lung transplants.

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Two items of note, including one from obit writer Bryan Marquard on a patient who survived Mass General’s first living-donor double-lung transplant  which was “so new that no one could venture odds for long-term survival.Mr. Bean was 20 that July day as he lay on the operating table, helping advance science as much as he hoped to extend his life. He was 38 when he died in Mass. General on April 14, several months after his body began to reject the transplanted lungs and complications set in.”

 

And from Carolyn Y. Johnson’s always solid but hard-to- find science blog. Read it inside the Monday business section. Good luck finding it online.  Here’s some help:

Retracted stem cell papers get public, private scrutiny

Something would turn out to be wrong with both papers. Where these two tales diverge is how these problems have been handled.

In the weeks since the paper describing the acid bath technique was published in the journal Nature, it has been thoroughly — and publicly — picked apart. Several of its Japanese authors have held press conferences. The president of the RIKEN research institution in Tokyo, where many of the authors work, has apologized to the scientific community, prefacing his public remarks with a deep bow. RIKEN has released detailed reports and been specific about what portions of the paper it was investigating and what was found. It publicly accused a young scientist named Haruko Obokata of fraud, a finding she is appealing.

The incident has sparked a national discussion about the state of science in Japan and the need to ensure high standards in order not to lose the world’s trust.

In contrast, the 2012 paper was withdrawn in April without fanfare: a barebones retraction notice posted by the journal Circulation stated an institutional review had found that the paper contains unspecified “compromised” data. No details were provided about what was wrong with the data.

HIV, gone after marrow transplant, returns to Boston’s “Berlin” patients

Update: Health News Review used this story as an example of overuse of the term “cure.” HNR also pointed out how the “cure” got a lot of coverage and the news that it wasn’t a cure did not.

The Globe’s story is behind the paywall. Here is the nut.

Or here

Boston researchers are reporting the return of the HIV virus in two patients who had become virus-free after undergoing bone marrow transplants, dashing hopes of a possible cure that had generated widespread excitement

The story reports that doctors detected the return of the virus in one patient in August. The second patient chose to continue in the study, but in November, doctors found traces of HIV and he went back on his medication

Some background here from Nature Boston. 

More on the meeting where this was reported here.

As pointed out above in HNR, the overuse of the term “cure” often leads to disappointment? Here’s what the Google search looks like:

ssniOr here.

Genetics and autism: One study, one story

Two Boston-linked stories today on the genetics of Autism.

ss sciFrom the Scientist Last year a team of Australian scientists claimed to have developed a genetic test that predicts risk for autism spectrum disorder (ASD) with “72 percent accuracy.”y night at a Boston fundraiser in support of his research into the functioning of brain synapses in autism

The Scientists reports that they said the test  “may provide a tool for screening at birth or during infancy to provide an index of at-risk status.”

But a new study, led by Benjamin Neale from Massachusetts General Hospital, suggests that those claims were overblown. Neale’s team replicated the Australian group’s research in a larger sample, and found that the proposed panel of markers did not accurately predict ASDs.

“The claims in the original manuscript were quite bold. If they were true, it really would have been quite a major advance for the field, with serious ramifications for patients and other risk populations,” said Neale. “I think it’s important to ensure that this kind of work is of the highest quality.”

More here from SciBlogger Emily Willingham. 

And, this from WBUR

BOSTON — For Timmy and Stuart Supple, a pool is one of the best places to be. That’s where their mother thought the boys, who are 8 and 10 years old and severely autistic, would be the most calm and least stressed for a very important introduction.

“We, we, we go see the doctor?” 10-year-old Stuart asked his mother.

His mother, Kate Supple, tells him the man standing in front of him by the pool is the doctor. Dr. Thomas Sudhof has never met the boys, but he wants to see their autism unchecked.

Sudhof isn’t a pediatrician or one of the myriad of therapists trying to get into their world and bring them out. The Stanford University neuroscientist — who this year shared the Nobel Prize in medicine for his decades of study into how brain cells communicate — has been studying Tommy and Stuart’s genes, specifically an alteration in one gene, for five years. The Supples hosted Sudhof Wednesda

From the Scientist Last year a team of Australian scientists claimed to have developed a genetic test that predicts risk for autism spectrum disorder (ASD) with “72 percent accuracy.”y night at a Boston fundraiser in support of his research into the functioning of brain synapses in autism

The Scientists reports that they said the test  “may provide a tool for screening at birth or during infancy to provide an index of at-risk status.”

But a new study, led by Benjamin Neale from Massachusetts General Hospital, suggests that those claims were overblown. Neale’s team replicated the Australian group’s research in a larger sample, and found that the proposed panel of markers did not accurately predict ASDs.

“The claims in the original manuscript were quite bold. If they were true, it really would have been quite a major advance for the field, with serious ramifications for patients and other risk populations,” said Neale. “I think it’s important to ensure that this kind of work is of the highest quality.”

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