Compassionate use allows any physician to petition to use any investigational drug that has not yet been approved. In other words, patients do not need to enroll or be qualified for a clinical trial in order to use the drug.
how do you decide to buy the Jun 3rd call with strike price of 10? why not Jun 3 call with a different strike price? there are so many choices how do you know which strike price to pick? thanks!
Since the stock price went up a little today -- don't you make a profit on this trade only if the stock goes above $40 for the June 17 calls?
Does this mean that if the price of the stock hits $35, you will make $25,000 ($10 * 2500)?
obender can you give an example? i'm trying to learn more about options since it seems like its the way to go.
I'm probably in the same boat as most -- mostly trading in stocks and only know the very basics about options, but never actually did a trade. Can you tell us exactly what trade you did (or a similar example) and what result you were hoping for? Thanks!!!!
The adcom questions were loaded and designed to get the most "No" votes as possible. They were so convoluted that there was no way to say "yes" to them.
While every drug is different and there should be some questions tailored to the drug in question, there also should be a set of standardized straightforward adcom questions for all adcom meetings such as:
1) Do you believe the medication is effective?
2) Do you believe the medicine is safe?
3) Do the benefits of this medication outweigh the risks?
This makes it fair for everyone.
Also, anyone who abstains needs to state their reason for abstaining, such as "I think the question is stupid" or "I'm too stupid to answer this question."
The 12 patient study was only a phase II trial; however, because of the positive results and the severity of the disease, the FDA encouraged SRPT to pursue AA as long as a confirmatory trial is in progress. So SRPT has already started a Phase III trial. The FDA has been advising SRPT what do to all along, so its funny how now at they end they are say "you should have done this and done that".
Per Ed Kaye, Sarepta drug was already showing production of dystrophin so patients were reluctant be in the placebo arm of a trial. They discussed this with the FDA and it was decided to proceed with historical controls for this exon with follow up placebo controlled trials for upcoming exon which have yet to show benefit. SRPT is correct that the FDA greenlighted this trial, but Farkas is now playing monday morning quarterback saying its all flawed.
Farkas may not like it, but the law allows for historical control trials.
CFR - Code of Federal Regulations Title 21:
"Generally, the following types of control are recognized:
(i) Placebo concurrent control. ...
(ii) Dose-comparison concurrent control. ...
(iii) No treatment concurrent control. ...
(iv) Active treatment concurrent control. ....
(v) Historical control. The results of treatment with the test drug are compared with experience historically derived from the adequately documented natural history of the disease or condition, or from the results of active treatment, in comparable patients or populations. Because historical control populations usually cannot be as well assessed with respect to pertinent variables as can concurrent control populations, historical control designs are usually reserved for special circumstances. Examples include studies of diseases with high and predictable mortality (for example, certain malignancies) and studies in which the effect of the drug is self-evident (general anesthetics, drug metabolism)."
[50 FR 7493, Feb. 22, 1985, as amended at 50 FR 21238, May 23, 1985; 55 FR 11580, Mar. 29, 1990; 64 FR 402, Jan. 5, 1999; 67 FR 9586, Mar. 4, 2002]
Watched Farkas him talk at the PPMD conference in June 2015 (on youtube). He seems thoughtful, but socially awkward, makes little eye contact, tends to mumble and close his eyes when talking. He's very methodical and didactical -- specifically states that the fastest path to approval is a placebo-controlled trial.
However, he does state that the federal regulations allow for dose comparison, active treatment and historical control trials as well. He blows off historical controls and doesn't even talk about this. However, he did talk about active treatment control trials, which is comparing two different drugs -- if one performs better than the other, then it clearly works. However, no conclusion can be drawn regarding the efficacy of the lower performing drug. He spent some time discussing this and I'm wondering if it would be possible for Sarepta to compare the results of epi to drisp? Would this be possible? Just looking at the dystrophin data, epi (0.9%) does make more than drisp (0.3%). How did the walk tests compare between the two? Was this discussed in the new Briefing documents?