'Clear statistical reservations' surround Bial's fatal clinical trial

The Royal Statistical Society has questioned the design of the trial of Bial's candidate painkiller BIA-102474-101 in which one person died and others were hospitalised last month.

The Royal Statistical Society (RSS) has issued a statement calling for more information to be disclosed about the circumstances surrounding the fatal Bial/Biotrial clinical trial. According to the Society, there are “clear statistical reservations about the trial's study-design.”

In its statement the RSS, “urges that the French investigatory teams include independent statistician-members.”

Apparently, the trial did not follow previously made recommendations, which were issued following the hospitalization of six healthy men in 2007 during a different clinical trial (TGN1412).

Professor Stephen Senn, who contributed to the publishing of these initial recommendations (Statistical Issues in First-in-Man Studies) commented on the recent developments:

“Our thoughts are with the families of all the men in the affected cohort. Key recommendations in the RSS report cover proper risk assessment, sharing that assessment with all parties and the sharing of information across studies to improve the assessment of risk. We hope that, as the inquiry proceeds, these matters will be properly and expertly addressed not only by physicians but also by statisticians.”

The French National Agency for Medicines and Health Products Safety (ANSM) has published Bial laboratory's clinical trial BIA-102474-101 protocol (after it was leaked by a French investigative journalist.)

However, Bial has not agreed to publish two other documents, the Investigational Product Dossier, and the Investigator’s Brochure, which would provide more pharmaceutical information as well as information on the animal studies conducted.

In its statement, the RSS has also called for the publication of these documents. In their defense, Bial is citing trade secrets protection.

Additionally, the RSS also stated that the escalated dose-levels that were tested in the trial’s single-administered-dose (SAD) phase need to be disclosed, “together with the number of volunteer cohorts on whom each dose-level was tested. We note that back-tracking to re-test a dose-level was allowed as well as escalation.”

The RSS also called for the publication of the regiment selected for ten days of administration to the first cohort of volunteers in the trial’s multiple-administered-dose (MAD) phase.

The British Pharmacological Society has also issued a statement calling for improved early access to data from catastrophic clinical trials:

As experts in the field of drug discovery and development, drug safety assessment and early clinical trials, pharmacologists can contribute to diagnosing the causes of – and preventing future instances of – serious adverse reactions.

We recommend release of the study design and protocol, the full Investigation Medicinal Product Dossier (IMPD) and the batch release data for such studies, at the earliest possible stage. This level of transparency is critical to maximise patient safety in the future and should outweigh considerations of commercial confidentiality."