April 12

Nobody Should Volunteer for Clinical Trials As Long As Research Data Is Secret

In their compelling Op Ed article in The New York Times, Drug Data Shouldn’t Be Secret, (below) Peter Doshi and Tom Jefferson, take their case advocating for clinical trial data disclosure to the wide public. They describe how pharmaceutical companies like Roche, manufacturer of the drug, Tamiflu, get away with generating false claims about the safety and effectiveness of their products when published in medical journals whose peer-review process is supposed to ensure scientific honesty. They do so, by concealing the data from their trials, which they alone control–thereby undermining the integrity of the peer-review process and the credibility of the published reports.

Despite evidence exposing corrupted peer-review process, company-generated assertions in medical journals about safety and efficacy of drugs, vaccines, and medical devices, journal reports are presumed to be science-based. Indeed, those largely unsubstantiated assertions in journals form the basis for medical practice guidelines and public health policies which may–and often have–proven harmful to our health and also to our fiscal policies.

But as the authors correctly note, by law, companies are required to disclose to the FDA all clinical trial data pertaining to drugs for which they seek a license to market. But the FDA has never required drug or medical device manufacturers to share their data with independent researchers or academics.  In fact, the FDA treats the company data as secret and proprietary. In effect, the FDA collaborates in the concealment of vital drug safety information.

FDA’s assessment of the data submitted by Roche shows that Tamiflu is of minimal benefit–no more than aspirin. In adults, the label states, "there was a 1.3 day reduction in the median time to improvement in influenza-infected subjects receiving TAMIFLU compared to subjects receiving placebo."  In geriatric patients there was a  1 day reduction, and in children 1.5 days compared to placebo. The  label states that "Tamiflu has not been shown to prevent serious bacterial infections" (for instance, pneumonia). 

So why, did the US government stockpile this drug paying more than $1.5 billion at taxpayer expense?  The public interest–both health and fiscal–is ill served when government agencies, lacking access to the scientific data, make public health policy decisions based on the false assertions in medical journal reports because they put their trust in the integrity of the peer- review process.

Officials of the US Centers for Disease Control, the Dept. of Health and Human Services, and the World Health Organization disregarded FDA’s Tamiflu efficacy assessment. They issued pronouncements–without supporting data–declaring that the drug was effective in reducing the duration of hospitalizations, reducing serious complications (such as pneumonia), cutting transmission of the virus.  Roche didn’t need sales reps to convince doctors to use their drug–authoritative public health agencies were Tamiflu’s best emissaries.

Company controlled clinical trial data has been accessible during legal litigation in the US many years after the drugs have been marketed and have caused harm.  Invariably, the data has contradicted safety and efficaacy assertions in journal reports, which explains why manufacturers routinely conceal the data.
 
Although the authors focus on their experience in trying to obtain Tamiflu data from Roche, they make the case that public trust in public health policies can only be won if the scientific basis informing those policies–namely, the data documenting the risks and benefits–are made public and open to independent analysis.
Human beings who volunteer to serve as research subjects have been led to believe that their sacrifice–undertaking risk and discomfort–will serve a humanitarian purpose. If sponsors of research are not compelled by law to disclose the data from those trials–the integrity of the research is thrown into doubt. Absent the humanitarian raison d’etre for enrolling in a clinical trial, no human being should be put at any–even minimal risk–without adequate compensation as a laborer and the protection of Workmen’s Compensation insurance.

See also, Peter Doshi and Tom Jefferson’s article in PLos Medicine, 2012:
     http://www.plosmedicine.org/article/info%3Adoi%2F10.1371%2Fjournal.pmed.1001201   doshi jefferson bmj

 Vera Sharav

http://www.nytimes.com/2012/04/11/opinion/drug-data-shouldnt-be-secret.html
THE NEW YORK TIMES

Drug Data Shouldn’t Be Secret
April 10, 2012

By PETER DOSHI and TOM JEFFERSON

In the fall of 2009, at the height of fears over swine flu, our research group discovered that a majority of clinical trial data for the anti-influenza drug Tamiflu — data that proved, according to its manufacturer, that the drug reduced the risk of hospitalization, serious complications and transmission — were missing, unpublished and inaccessible to the research community. From what we could tell from the limited clinical data that had been published in medical journals, the country’s most widely used and heavily stockpiled influenza drug appeared no more effective than aspirin.

After we published this finding in the British Medical Journal at the end of that year, Tamiflu’s manufacturer, Roche, announced that it would release internal reports to back up its claims that the drug was effective in reducing the complications of influenza. Roche promised access to data from 10 clinical trials, 8 of which had not been published a decade after completion, representing more than 4,000 patients from every continent except Antarctica. Independent verification of the data seemed imminent. But more than two years later, and despite repeated requests, we have yet to receive even a single full trial report. Instead, the manufacturer released portions of the reports, most likely a very small percentage of the total pages. (One of us, Tom Jefferson, has been retained as an expert witness in a lawsuit relating to some of these issues.)

This is entirely within Roche’s rights. After all, regulators have never required drug or medical device manufacturers to share their data with independent researchers or academics. They are required to show the information only to the regulators themselves, who treat the data as secret.

Some may argue that, because the Food and Drug Administration approves drugs for the United States market based on these data, this is not a major cause for concern. But the actual use of drugs is often driven by assumptions about drug safety and effectiveness put forth by articles in peer-reviewed journals (sometimes written by doctors affiliated with the drug manufacturers) and clinical practice guidelines that can be entirely inconsistent with the F.D.A.’s assessments.

In the case of Tamiflu, some of these assumed properties led to stockpiling at great taxpayer expense — more than $1.5 billion. The F.D.A. approved Tamiflu for the treatment of influenza (on the basis that it could reduce the duration of flu symptoms by about a day); not for the prevention of transmission. But other agencies are far more enthusiastic about Tamiflu’s benefits. The Centers for Disease Control and Prevention has argued that it reduces the duration of hospitalizations and serious complications like pneumonia, citing Roche-authored papers. The Department of Health and Human Services, also citing Roche, assumed in its national influenza pandemic plan that Tamiflu would cut complications. And the World Health Organization’s pandemic planning assumed that the drug would cut transmission of the virus. But here’s the rub: none of these organizations have vetted the original trial data.

The only agency in the United States that seems to have independently reviewed the original trial data never made these claims. The F.D.A.’s conclusion — which it required Roche to print on Tamiflu’s product labeling — is that “Tamiflu has not been shown to prevent” complications like serious bacterial infections (for instance, pneumonia). It seems that federal agencies like the C.D.C. and H.H.S., instead of conducting an independent evaluation of Tamiflu, advocated stockpiling by referencing claims in journal publications written by the drug’s manufacturer, ignoring the F.D.A.’s assessment that those very claims were unproven.

Why would they do this? Unwarranted trust in the peer-review process of medical journals probably has something to do with it. So, too, does wishful thinking; lacking good alternatives, it’s tempting to hope that the drug we have works wonders. And it’s important to remember that correcting the statements of medical journals or public health agencies falls outside the F.D.A.’s jurisdiction — when it comes to drugs, the F.D.A. is responsible for regulating industry, not other government agencies.

But this is no way for supposedly evidence-based decision making to work, and the F.D.A. could do much more. As a result of new freedom of information policies in Europe, the Continent’s version of the F.D.A., the European Medicines Agency, has released 22,000 more pages of Roche’s Tamiflu trial reports. But even this represents an incomplete picture, as the most detailed portions of the reports are not in the European drug regulator’s files.

Nevertheless, the data point to a drug of minimal benefit. In accordance with the F.D.A.’s findings, it appears to shave a day off the duration of influenza symptoms, but we found no decrease in risk of hospitalization and no evidence that it could stop the spread of the virus. More worrisome, we found suggestive evidence that Tamiflu interfered with the body’s ability to produce antibodies against influenza — which could affect the body’s response to influenza vaccine and its ability to fight off future influenza infections. But to do a complete analysis, including evaluating Tamiflu’s potential harms, we need the remainder of the data — the full “clinical study report” — promised by Roche, but never delivered.

In response to our conclusions, which we published in January, the C.D.C. defended its stance by once again pointing to Roche’s analyses. This is not the way medical science should progress. Data secrecy is a disservice to those who volunteer their bodies for clinical trials, and is dangerous to those being asked to swallow approved medicines. Governments need to become better stewards of the scientific process. The European regulator’s announced intention to release clinical study reports after it finishes reviewing a manufacturer’s application is an important precedent. But the F.D.A. — guardian of arguably more trial data than any other entity in the world — appears stuck in the era of data secrecy.

We should not have to wait for patients to be hurt by the medications they take, as recently happened with the diabetes drug Avandia, before reviewing this wealth of data.

Peter Doshi is a postdoctoral fellow in comparative effectiveness research at Johns Hopkins University School of Medicine.
Tom Jefferson is an independent epidemiologist with the Cochrane Collaboration, an international nonprofit research organization.


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