Post by icemandios on Apr 22, 2016 3:04:45 GMT
Lots of strange stuff going on in the biotech world these days. Lots of strange stuff going on in the world today.
A fair amount of discussion in the shout box about SRPT which will vanish b/c that's what the shout box does.
While a discussion of SRPT would be exquisitely appropriate in this "Other Stocks" venue, we have a discussion of Theranos.........which, of course, is not a stock.
Moving on, was it coincidence that this evening on the lame stream media TV I saw a heart wrenching video of a young boy afflicted with Duchenne MD and the prognosis of death by year 20?
Further, since there appears to be no news attributed to SRPT which explains the vicious plunge in pps, I googled SRPT and found a Zack's commentary. I'm no fan of Zack's, but this is maybe worth reading:
"Shares of Sarepta Therapeutics (SRPT - Analyst Report) are down a staggering 45% today. Investors initiated a selling frenzy after the medical research and development company received some bad news from the FDA regarding its Duchenne muscular dystrophy drug.
The drug, known as “Eteplirsen,” looks like it will be fighting an uphill battle to gain FDA approval. Back in January, Sarepta requested that the FDA review its original assessment of the drug, citing errors in its report. Today, the FDA review board announced its decision:
“[W]e do not agree with the applicant's characterization of inaccuracies in the initial FDA briefing document... Although FDA is prepared to be flexible with respect to a devastating illness with no treatment options, we cannot approve drugs for which substantial evidence of effectiveness has not been established.”
As we know, research-based drug companies depend heavily on their ability to develop new treatments and get them on the market. Of course, this depends on the FDA's approval. Sarepta has pushed hard to get this treatment approved, but it is now clear that the regulatory body is not satisfied with the results.
While today's news is certainly not great, it is not exactly the end of the road for Sarepta and eteplirsen. The company announced today that the FDA is still expected to conduct an outside advisory panel next week."
Now we get the FDA saying no......but. No, but we'd like to hear what some real doctors who work in the field have to say. That's the meaning of no, but. And, while I'm not some expert on FDA hearings, nor do I claim to have a world of experience in monitoring these things, I can say I don't recall a no, but happening before.
(See the apparent coincidence cited above).
Somewhere in this vast repository of DD and related threads, I believe I posted something about how the medical practitioners viewed the FDA. Surveys going back several years, taken among different specialties of practice each year, produced a picture of general dissatisfaction with the FDA. IE, doctors on the front line of healing, in the majority, felt that the FDA did more harm than good when it came to treating people who were subjects of their particular specialty.
And at the bottom of that article, instead of just bitching about things, there was a suggestion which was based upon an observation.
The observation derived from the comments of practitioners was that the government had set up the FDA as the King of Medicine. One does not do anything that the King does not approve. And the reason why the FDA was given this kingly authority sprung from the Thalidomide debacle. Prior to Thalidomide, the FDA only was charged with safety. Some old (now deceased) Senator whose name escapes me just now passed a bill that expanded the FDA's authority to include verdicts on efficacy.
Suddenly bureaucrats were in the position to decide what drugs could come to market based on statistics. If the stats weren't better than placebo, or if the stats weren't better than existing drugs, bureaucrats could make the call to deny. Then came the ADCOM hearings where actual doctors could "advise" the bureaucrats.
This still left the problem of people who died or suffered. If only 10 of 100 people benefited from drug X, but 20 people benefited from placebo, the FDA would deny. And that, of course, is stupid. That means 10 more people will die b/c the drug was disapproved.
So there was a modest suggestion. Instead of letting the FDA be the "doctor" for the country, and thereby having a monopoly on the menu of drugs available to treat conditions, let the FDA be more like Underwriters Laboratory or Good Housekeeping. IOW, if the drugs are safe, or relatively safe as compared to what (if anything) is also available, let the doctors who treat people say "this drug is not recommended by the FDA, but I think it's what we need to try.
And just a personal note or two:
1) In a previous life, I worked at an EPA testing facility for imported cars. We did emissions testing to ensure compliance with EPA emission standards. After a while, I discovered that the EPA had college students who worked part time (2 days on, 4 days off, etc) making these approvals of our submissions.
2) Being invested in MNKD (to my eventual dismay), I listened to an ADCOM webcast wherein the FDA people (who sounded pretty damned young to my ear) started asking questions in a challenging way about the design of the studies for Afrezza. At which point, one of the doctors who sat on the ADCOM (who sounded not so young) spoke up and said something like "Wait a minute. Didn't you people agree on the design of this study? Was it not conducted under a SPA? And haven't you been working with these people for 10 years?" Well, that ended any discussion of the design of the study. Point being, the FDA people seemed to be there to deny, seemed to be unfamiliar with the history. Seemed to be reminiscent of the part-timers I had to deal with in the EPA.
Laws, rules, regulations rarely tell you what you can do. Most tell you what you can't do. That is the nature of the thing. It is negative....can't is negative.
So we get back to this odd situation where the FDA says no to SRPT, a heart wrenching video of a young boy with Duchenne MD crops up on TV this evening, and the FDA wants the ADCOM to weigh in.
What I'd like to see is the above suggestion. Let the FDA recommend things. Let the doctors use their education, experience and skill decide things. Let the statiticians do their computations, but if you want statistics......
consider an 88 yr old woman who had two double by-pass operations in the '90s, a radical mastectomy for breast cancer in 2000, has COPD and requires nebulizer treatments every 4-5 hours, oxygen (though she only does it at night b/c it might make her look old), hip replacement last week, had a heart attack in the hospital while waiting for hip surgery, who is in rehab now. And she's fighting to get back to going here and there, playing cards with the old ladies, scrounging at yard sales and a bunch of other gadfly activities.
Give me a statistical probability of that.
A fair amount of discussion in the shout box about SRPT which will vanish b/c that's what the shout box does.
While a discussion of SRPT would be exquisitely appropriate in this "Other Stocks" venue, we have a discussion of Theranos.........which, of course, is not a stock.
Moving on, was it coincidence that this evening on the lame stream media TV I saw a heart wrenching video of a young boy afflicted with Duchenne MD and the prognosis of death by year 20?
Further, since there appears to be no news attributed to SRPT which explains the vicious plunge in pps, I googled SRPT and found a Zack's commentary. I'm no fan of Zack's, but this is maybe worth reading:
"Shares of Sarepta Therapeutics (SRPT - Analyst Report) are down a staggering 45% today. Investors initiated a selling frenzy after the medical research and development company received some bad news from the FDA regarding its Duchenne muscular dystrophy drug.
The drug, known as “Eteplirsen,” looks like it will be fighting an uphill battle to gain FDA approval. Back in January, Sarepta requested that the FDA review its original assessment of the drug, citing errors in its report. Today, the FDA review board announced its decision:
“[W]e do not agree with the applicant's characterization of inaccuracies in the initial FDA briefing document... Although FDA is prepared to be flexible with respect to a devastating illness with no treatment options, we cannot approve drugs for which substantial evidence of effectiveness has not been established.”
As we know, research-based drug companies depend heavily on their ability to develop new treatments and get them on the market. Of course, this depends on the FDA's approval. Sarepta has pushed hard to get this treatment approved, but it is now clear that the regulatory body is not satisfied with the results.
While today's news is certainly not great, it is not exactly the end of the road for Sarepta and eteplirsen. The company announced today that the FDA is still expected to conduct an outside advisory panel next week."
Now we get the FDA saying no......but. No, but we'd like to hear what some real doctors who work in the field have to say. That's the meaning of no, but. And, while I'm not some expert on FDA hearings, nor do I claim to have a world of experience in monitoring these things, I can say I don't recall a no, but happening before.
(See the apparent coincidence cited above).
Somewhere in this vast repository of DD and related threads, I believe I posted something about how the medical practitioners viewed the FDA. Surveys going back several years, taken among different specialties of practice each year, produced a picture of general dissatisfaction with the FDA. IE, doctors on the front line of healing, in the majority, felt that the FDA did more harm than good when it came to treating people who were subjects of their particular specialty.
And at the bottom of that article, instead of just bitching about things, there was a suggestion which was based upon an observation.
The observation derived from the comments of practitioners was that the government had set up the FDA as the King of Medicine. One does not do anything that the King does not approve. And the reason why the FDA was given this kingly authority sprung from the Thalidomide debacle. Prior to Thalidomide, the FDA only was charged with safety. Some old (now deceased) Senator whose name escapes me just now passed a bill that expanded the FDA's authority to include verdicts on efficacy.
Suddenly bureaucrats were in the position to decide what drugs could come to market based on statistics. If the stats weren't better than placebo, or if the stats weren't better than existing drugs, bureaucrats could make the call to deny. Then came the ADCOM hearings where actual doctors could "advise" the bureaucrats.
This still left the problem of people who died or suffered. If only 10 of 100 people benefited from drug X, but 20 people benefited from placebo, the FDA would deny. And that, of course, is stupid. That means 10 more people will die b/c the drug was disapproved.
So there was a modest suggestion. Instead of letting the FDA be the "doctor" for the country, and thereby having a monopoly on the menu of drugs available to treat conditions, let the FDA be more like Underwriters Laboratory or Good Housekeeping. IOW, if the drugs are safe, or relatively safe as compared to what (if anything) is also available, let the doctors who treat people say "this drug is not recommended by the FDA, but I think it's what we need to try.
And just a personal note or two:
1) In a previous life, I worked at an EPA testing facility for imported cars. We did emissions testing to ensure compliance with EPA emission standards. After a while, I discovered that the EPA had college students who worked part time (2 days on, 4 days off, etc) making these approvals of our submissions.
2) Being invested in MNKD (to my eventual dismay), I listened to an ADCOM webcast wherein the FDA people (who sounded pretty damned young to my ear) started asking questions in a challenging way about the design of the studies for Afrezza. At which point, one of the doctors who sat on the ADCOM (who sounded not so young) spoke up and said something like "Wait a minute. Didn't you people agree on the design of this study? Was it not conducted under a SPA? And haven't you been working with these people for 10 years?" Well, that ended any discussion of the design of the study. Point being, the FDA people seemed to be there to deny, seemed to be unfamiliar with the history. Seemed to be reminiscent of the part-timers I had to deal with in the EPA.
Laws, rules, regulations rarely tell you what you can do. Most tell you what you can't do. That is the nature of the thing. It is negative....can't is negative.
So we get back to this odd situation where the FDA says no to SRPT, a heart wrenching video of a young boy with Duchenne MD crops up on TV this evening, and the FDA wants the ADCOM to weigh in.
What I'd like to see is the above suggestion. Let the FDA recommend things. Let the doctors use their education, experience and skill decide things. Let the statiticians do their computations, but if you want statistics......
consider an 88 yr old woman who had two double by-pass operations in the '90s, a radical mastectomy for breast cancer in 2000, has COPD and requires nebulizer treatments every 4-5 hours, oxygen (though she only does it at night b/c it might make her look old), hip replacement last week, had a heart attack in the hospital while waiting for hip surgery, who is in rehab now. And she's fighting to get back to going here and there, playing cards with the old ladies, scrounging at yard sales and a bunch of other gadfly activities.
Give me a statistical probability of that.