Health Care Transparency in Need of Academic Vigilance

According to the Food and Drug Administration Amendments Act (FDAAA), controlled clinical trials of drugs and biologics that were initiated on or before September 27, 2007, are required to submit results of the trial within 12 months of trial completion date—failure to do so can result in fines that can including withholding grant funds for federal-funded studies.

However, an investigation by STAT into the ClinicalTrials.gov website identified about 9000 trials that had failed to abide by the requirements.

According to the report, defaulting institutes include Stanford University, Memorial Sloan Kettering Cancer Center (MSKCC), University of Pennsylvania, and the University of Pittsburgh. Importantly, these failures were not occasional—researchers were either late or failed to report their results 95% of the time.

While this issue has been floating around for a while, data curated by STAT has underscored the need for increased vigilance by academic institutions to adhere to reporting requirements.

Why do we need this transparency? Because that was the original intent of the National Institutes of Health website, ClinicalTrials.gov: rapid dissemination of trial data to the public that would prevent the pharmaceutical industry from shrouding important information on safety and efficacy.

Data transparency brings an important value proposition to the healthcare market, and examining what STAT has reported, the onus lies beyond the drug developers. Academic researchers actively collaborate with the pharmaceutical industry in developing a drug product.

Considering that treatment decisions are made based on historic research findings, withholding clinical data can have serious consequences.

According to STAT, MSKCC did not report adverse effects of an experimental drug, ganetespib, in breast and colon cancer patients, that included heat, liver, and blood disorders, bowel and colon obstructions, as well death of a patient. Turns out, the cancer center was keen to publish its results in a medical journal prior to making the knowledge public through the NIH website.

A spokesperson for MSKCC told STAT that the drug’s sponsor would be responsible for disseminating information regarding the drug to other investigators evaluating the drug.

Another example is of Avastin, which was found ineffective but responsible for serious side effects in patients with metastatic breast cancer. Failure to report these events by Hoosier Cancer Research Network in 2009 (when they identified these events) had doctors still debating using the drug in breast cancer patients, till the FDA revoked the drug’s approval for breast cancer.

While Hoosier published a paper in 2013, reporting the findings, they have not posted results on ClinicalTrials.gov, according to STAT.

Francis Collins, MD, PhD, who heads NIH, told STAT that by next year, NIH and FDA will have “a firmer basis for taking enforcement actions.” If an NIH grantee fails to meet reporting deadlines, their funding can be terminated. “Staff scientists will be required to follow the law and NIH policy, and there will be consequences…for failing to do so.”