When a drugmaker applies for FDA approval of its product, it is responsible for providing the data to the agency. The FDA then reviews the data to reach its decision about whether to grant or withhold approval of the drug and what possible conditions to require. The process demands that pharmaceutical manufacturers provide accurate and truthful data to the FDA. Otherwise, the agency may be put in a position where it approves a drug that is either dangerous or ineffective. Recently, the agency learned that large drugmaker Novartis manipulated data that it provided to the FDA in connection with the approval of one of its drugs. As a result of the violation, which Novartis self-reported, the company may face some time of regulatory or disciplinary action.
The FDA recently approved Novartis’ blockbuster drug called Zolgensma. This drug is intended to treat spinal muscle atrophy through the use of gene therapy. This disease affects infants under the age of two, and it is usually fatal. Zolgensma made headlines because the cost of the drug was $2.1 million per patient. This makes Zolgensma the most expensive pharmaceutical product in the entire world. The drug is manufactured by AveXis, which was bought by Novartis in April 2018. However, not all was well with the drug. On June 28, the manufacturer of the drug informed the FDA that there was data manipulation that impacted a small amount of what it reported to the agency regarding the preclinical phase of the testing. This involved a small amount of testing of the drug on animals.
Notwithstanding the data manipulation, the FDA expressed the opinion that the approval should not be overturned and the drug should remain on the market. The agency reassured the public that the data manipulation issue only affected a small part of the testing and the regulator continues to believe that the drug is safe and effective. The FDA continues to believe that the data from the human clinical trials supports its decision to keep the drug available. The testing involved the manufacturing process as opposed to the efficacy of the drug. According to Novartis, the part of the manufacturing process that was implicated is no longer in use.
What troubled the FDA is that the drug’s manufacturer learned of the data manipulation in the time after the application was submitted, but before the FDA granted its approval of the drug. The manufacturer should have reported the issue to the agency at the time it learned of it, but did not do so. It was only after the product was approved by the FDA that the manufacturer alerted the agency of the data manipulation. Novartis has claimed that it launched an internal investigation immediately after it learned of the problem and informed the FDA once it learned the results of the investigation. By the time that Novartis informed the FDA, the drug had already been approved for five weeks.
In the wake of the revelation, the FDA conducted an inspection of the manufacturing process. While the drug will remain on the market for now, the FDA may take some kind of action regarding the manufacturing. As it processes the data from the inspection, the FDA will determine what action, if any, needs to be taken. Novartis continues to maintain that the drug is entirely safe and the data manipulation was minimal and does not have any impact on the current commercial usage of the drug.
In the meantime, the manufacturer of the drug may find itself in trouble with the FDA. The regulatory system of drug approvals requires that manufacturers submit honest and accurate data. If not, patients are at risk of taking dangerous and ineffective drugs. According to the agency’s press release, the agency is considering further action against the drugmaker. While the drug will not be pulled from the market, the matter of the data manipulation places AveXis at risk to receive a significant civil or even criminal penalty. There are penalties in federal criminal law for false statements that are made to the government and even entire corporations can be indicted.
The entire FDA approval process depends on the submission of truthful and accurate data, and the agency takes this breach seriously. This data manipulation caused Novartis’ stock price to fall four percent in the immediate aftermath of this revelation.
Unfortunately, Novartis is far from the only drugmaker to engage in this practice. Data manipulation is often done by large multinational corporations who seek FDA approval for their products. In 2018, there were 30 data integrity violations in connection with the FDA approval process. Data integrity is a common issue and companies may sometimes feel that they have an incentive to submit falsified data. In essence, they are taking a chance that either they will not get caught or that the penalty will be less severe than the benefits. The FDA has recently charged one company with data manipulation and sent warning letters to others. It remains to be seen how hard the FDA will come down on Novartis, but given the high profile of the drug involved, the agency’s hand may be forced into taking significant legal action against the company.
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