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- A data-manipulation scandal has rocked the Swiss drug giant Novartis and its new rare-disease treatment Zolgensma.
- A clearer picture is now emerging with a new 59-page letter Novartis’ AveXis unit sent to the Food and Drug Administration.
- AveXis learned in March of an allegation that two senior executives led data manipulation at the company, according to the letter. The company said it initially led a secret investigation to make sure evidence would be preserved “without interference.”
- “Moreover, the investigation was significantly drawn out due to the implicated senior executives’ lack of cooperation and categorical denial of the allegations, which continues to this day,” the letter said.
- A representative for Brian Kaspar said Kaspar had cooperated with the company investigation and denied any wrongdoing. Allan Kaspar did not immediately return Business Insider’s request for comment.
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Scandal has rocked the Swiss drug giant Novartis since the US Food and Drug Administration revealed last month that the company submitted manipulated data on a key drug to the regulator.
Novartis later blamed Brian and Allan Kaspar, brothers who were top execs at its AveXis unit, for the problems. Now more allegations about the Kaspars are emerging in a 59-page letter from Novartis’s AveXis to the FDA, showing a clearer picture of how events unfolded earlier this year.
The letter was part of a response to the FDA after an FDA inspection found potential violations at AveXis’ San Diego laboratory. It includes the allegation that the two execs led the data manipulation in question and that their noncooperation and denials delayed AveXis’ investigation into the matter.
“Such conduct is unacceptable, and the two AveXis senior executives have been terminated,” the letter said.
The manipulated data was used to secure approval for Zolgensma, a treatment for spinal muscular atrophy. The FDA has said Zolgensma should remain on the market.
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A representative for Brian Kaspar said he had cooperated with the company investigation and denied any wrongdoing, while Allan Kaspar did not immediately return Business Insider’s request for comment. The FDA has said it could impose civil or criminal penalties.
The manipulated data was part of a large packet submitted to the FDA and reviewed as part of the drug’s approval. The particular issue pertained to data from product testing done in mice, according to the letter and a press release from the FDA last month.
In addition to the conduct itself, the timeline has also come under scrutiny, since Novartis had knowledge of potential data-manipulation issues before Zolgensma was approved in May — and disclosed the problem the next month, in June.
Read more:Elizabeth Warren and Bernie Sanders just slammed the Swiss drug giant Novartis over a new controversy swirling around the world’s most expensive drug
Here’s what else we just learned from the letter:
The story begins in mid-March, with mice.
Back then, an AveXis employee alleged that two senior execs at the company had led manipulation of data on a specific analysis done in mice, according to the letter.
The employee alleged the execs “altered or instructed others to alter a small amount of raw data” used in the analysis, the letter said.
The letter also reveals more specifics about the data in question.
According to the letter, the problematic data was mostly related to “the recorded date of death for some of the mice.”
Because the issue related to a small amount of product-testing data, the FDA has said the treatment, Zolgensma, should remain on the market.
AveXis then launched an internal investigation in March.
The investigation was led by top human-resources, quality, and legal officials, the letter said.
But when they contacted the employee who made the allegations, the person wasn’t available until about 12 days later, according to the letter. Novartis got looped in around then, in late March.
In April, AveXis brought in the heavy hitters: an external law firm hired to conduct what was initially a secret investigation.
In the beginning, the two execs weren’t told about the internal investigation because they “were still fixtures at AveXis with the potential to influence and impede the investigation,” and in the interest of preserving evidence, the letter said.
Later, AveXis said the two execs held up the process because of their “lack of cooperation and categorical denial” of the allegations.
Because of that, the company needed “significant resources” to go over thousands of hand-written and electronic records around the life history of mice, comparing those with spreadsheet entries — an estimated 2,000-plus hours of work, AveXis said.
The two execs were put on administrative leave in early May and then fired in mid-August.
After this initial investigation, “the two senior AveXis executives alleged to have been responsible could not offer a credible explanation for revisions to and inconsistencies in the data,” the letter said.
AveXis outlines changes to FDA.
In the letter, which is dated late August but was released on Tuesday to the public, AveXis then outlined to the FDA a number of changes, including training on data integrity. The company said it planned to link itself more closely to Novartis’ existing systems for ensuring that data is accurate.
The company said it hired new compliance workers and that Novartis would monitor AveXis’ policies and procedures more closely.
AveXis said that root causes of the data manipulation issue included “a lack of cultural emphasis on data integrity at the site, a lack of adequate training and real-time data recording.”