Petition to FDA Seeks to Modify Trial Regulations Following Deaths

Posted 18 May 2017 By Zachary Brennan

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A petition submitted to the US Food and Drug Administration (FDA) in 2016 has been updated recently, as it seeks to reform FDA requirements for investigational new drug (IND) applications, investigational device exemptions (IDEs) and new drug applications (NDAs).

In a letter submitted in April, the Center for Responsible Science noted 19 treatment-related deaths in clinical trials from July 2016 through April 2017. Among those were five clinical trial participants who died in a phase II trial for Juno Therapeutics' chimeric antigen receptor T-cell (CAR-T) therapy due to severe neurotoxicity.

Michael Vokhgelt, a computer programmer from Ohio and a father of one of the participants in the cancer trial who died, said in a submitted statement: "I was shocked and dismayed that Juno did not disclose Max's death to the public and that they continued the trial without investigating what killed my son. On June 4, 2016, just a week after my son's death, I was shocked again to discover that Juno issued a press release saying that the ROCKET trial data showed 'encouraging' and 'impressive' results…It wasn't until two more trial volunteers died and FDA issued a clinical hold that on July 7, 2016, Juno announced the deaths."

The petition also notes the three who died, not due to disease progression, in a Kite Pharma clinical trial that is currently ongoing for the treatment of refractory, aggressive non-Hodgkin Lymphoma (NHL). In addition, the petition pointed to deaths from a trial conducted in France that led the European Medicines Agency (EMA) to begin reviewing first-in-human trials.

The Center for Responsible Science added: "In the case of the Juno ROCKET trial, transparency from FDA outlining the rationale for lifting the clinical hold in July after three deaths could have helped inform trial participants and others about why FDA believed it was safe to proceed … We argue that everything that can be done to protect clinical trial volunteers must be done. Rather than resume a trial without knowing the actual cause of the deadly toxicity, sponsors should have made the drug available so that it could be tested in a human-relevant platform."

In an interim response sent in March 2016, FDA said it "has been unable to reach a decision on your petition because it raises issues requiring further review and analysis by agency officials. This interim response is provided in accordance with FDA regulations on citizen's petitions (21 CFR 10.30(e)(2)). We will respond to your petition as soon as we have reached a decision on your request."

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Categories: Biologics and biotechnology, Drugs, Clinical, Postmarket surveillance, News, US, FDA

Tags: citizen petition, FDA petition, clinical trial regulations

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