I found this early 2014 article below quite relevant to NEU coming into 2015. Note I have added the following words and brackets: (or there is no existing therapy) - my apologies to the author.
Not all FDA approvals have equal meaning, Yale study says
NEW HAVEN Approval by the U.S. Food and Drug Administration may not mean the same thing for each drug and shouldn’t take the place of detailed discussions with your doctor, according to a Yale study.
The study by researchers at the Yale School of Medicine found that while many drugs went through several clinical trials for extended lengths of time, others did not.
Looking at 188 drugs over seven years, the researchers found that one-third went through a single trial. Others tested only for a limited result of the drug, such as whether it reduced the size of a tumor, and not a “clinical endpoint,” such as whether it extended life, for example, according to Dr. Joseph Ross, assistant professor of internal medicine at Yale School of Medicine and senior author of the study.
About 40 percent of drugs studied were those that patients would take “for the rest of their life,” such as for diabetes or asthma, Ross said. However, “less than half were studied in trials that lasted six months or longer.” So, long-term effects of a medicine would not necessarily be included in the FDA’s approval, he said.
The study, published in the Jan. 22/29 issue of the Journal of the American Medical Association, shows a wide variation in such trials. “I think it’s reasonable to have a somewhat flexible standard of approval because in some cases there’s a great unmet medical need,” said Nicholas Downing, a Yale School of Medicine student and first author of the study.
Approval of a drug after limited trials is reasonable “if your treatment of the new drug is leaps and bounds better than the existing therapy,” (or there is no existing therapy) Downing said. The first AIDS medications were good examples, although they were not included in this study, because there were no drugs when the disease was discovered and people were dying.
“Very specifically, over one-third of drugs that were approved in our study period (were) approved on the basis of one single pivotal trial,” Downing said. “Forty percent of approvals involved ... comparing a new drug to an existing drug.”
“Most people presume that when a drug is approved for us that it’s safe and effective,” Ross said. “There’s always uncertainty in the drug-approval process.”
What patients should do is hold a conversation with their doctor that takes into account not only what a drug has been approved for but how the studies were done to determine whether it’s the best treatment for that patient, Ross said.
FDA spokeswoman Sandy Walsh said via email, “The agency applies the same statutory approval standards of safety and efficacy to all drugs, but uses regulatory flexibility in applying those standards.
“Some drugs may be tested in clinical trials that enroll hundreds of participants while others, particularly those seeking to treat rare diseases, may be tested in trials that enroll only a handful of participants.
“In all cases, however, the statutory standards of safety and efficacy must be met in order for the drugs to be marketed in the United States.A drug’s prescribing information (drug label) and patient Medication Guide contain important safety and efficacy information that patients and health care professionals can use to weigh the benefits and risks of a drug.”
Walsh continued, “The FDA has a robust program for postmarketing surveillance to help ensure the benefits of marketed drugs continue to outweigh their risks. When safety issues arise, we work quickly and transparently to inform patients and health care professionals so they can make informed decisions about treatment.”
So if I am reading this correctly - irrespective of HOW (FDA Frequently Asked Questions: Breakthrough Therapies) the FDA (speedily?) approves NNZ-2566 because there are no existing therapies for Retts - the FDA rightly put the onus on the drug company to provide a drug’s prescribing information (drug label) and patient Medication Guide containing important safety and efficacy information that patients and health care professionals can use to weigh the benefits and risks of a drug.”
So then - if NNZ-2566 is fastracked to market in 2015 then the "FDA has a robust program for postmarketing surveillance to help ensure the benefits of marketed drugs continue to outweigh their risks."
To me this supports the chairman strategy.
NEU Price at posting:
11.5¢ Sentiment: Buy Disclosure: Not Held
(20min delay)