Thursday, April 13, 2017

UC Irvine Stem Cell Blog: Troubling New Federal Law on Stem Cell Research

The stem cell blog at UC Irvine this week published a piece that warned that a new federal law aimed at speeding commercialization of stem cell therapies is worrisome and could lead to harm to patients and damage the entire field of research.

The April 10 article was written by Navied Akhtar, a Ph.D. candidate in the biomedical engineering department.

Akhtar's topic was the 21st Century Cures Act. He wrote that the law, signed by President Obama and which had a broad range of supporters,
"...utilitizes evidence from clinical experience to 'help to support the approval of a new indication for a drug approved under [accelerated approval]' and 'to help to support or satisfy post approval study requirements.' The only requirements written in for accelerated approval past a 'reasonable likelihood' that there will be clinical benefit, is that one or both of two requirements are met: (1) That the manufacturer conducts studies after accelerated approval to verify the predicted effect on mortality or other clinical benefit and (2) That the manufacturer submits copies of marketing materials for the drug during the preapproval period." 
Akhtar said that the law 
"will allow for what is effectively anecdotal evidence to be used as actual evidence to support the requirement for accelerated approval. Furthermore, the verbiage surrounding the term 'surrogate endpoint' is loose at best. The idea of a surrogate endpoint is to produce a clinically relevant point in which to be able to measure the efficacy and safety of a drug. In the Cures Act, the definition of what constitutes a surrogate endpoint is left rather open-ended. It reads as such: "The term ‘surrogate endpoint’ means a marker, such as a laboratory measurement, radiographic image, physical sign, or other measure, that is not itself a direct measurement of clinical benefit, and is known to predict clinical benefit and could be used to support traditional approval of a drug or biological product; or is reasonably likely to predict clinical benefit and could be used to support the accelerated approval of a drug or biological product.' It is worrisome to write that this endpoint can be a marker that is not a direct measurement of clinical benefit, but is known to predict clinical benefit. This leaves open a large workaround for accelerated approval of drugs that may have no business being approved."
Akhtar goes on to cite cases of drugs that ultimately proved harmful even after clearing the usual approval process. 

In the case of novel stem cell therapies, he argued, serious mishaps or death because of loose federal standards could endanger progress in the entire field. 
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