Actually, a P value of .05 probability would equal 95% and 5% due to chance. A P value of .05 should be sufficient for this type of unmet FADASIA oriented trial...a P value of .01 or .001 seems a bit tough and leaves virtually no room for "Flexibility".
Of FDA deliberation, if the approval consensus can get within a 2 or 3 vote differential yes/no, then the top two or 3 FDA officials can over turn this relatively close margin via FADASIA and Risk/Benefit analysis. The perspective of analysis must be revisited to address risk/benefit...not absolute P value significance via 201/202 trial data alone. Bonus. I am not expecting this but it would be a pleasant surprise...SRPT comes in with additional supportive data from ongoing trials over the next few weeks.
Sorry..One more point. Congress/Senate/Leading DMD experts support at the very least Conditional Approval!
100% Chance Very Premature of Regression/Death without drug
TOP 3 FDA Hierarchy Supportive of FDASIA/AA ETEP
Unmet Disease State
6/7 vote on AA...top 3 officials may play tie breaker
Mixed ADCOM Votes
Confused ADCOM panel on many fronts i.e. abstentions, questions about qustions, qualifying votes, wrong button pushing
FDA and ADCOM made their point...so hands are now clean of Type 1 Error
Public Sentiment overwhelmingly strong
50 positive testimonials
Testimonials demonstrating REVERSAL after 4 years, with sustained function and LOA
HC was Special Protocol Agreed Upon by FDA
"N" number was accepted via NDA . If N=12 was an issue, the NDA would never have been accepted.
Dystrophin extremely difficult to quantify and correlate to clinical benefit as was shared by both the FDA and ADCOM
Safety data Pristine and Efficacy has Reasonable risk/benefit FDASIA basis
No other treatment options for years!!
Confirmatory trials will give the FDA a means to monitor safety and Efrficacy...which gives them a way out if necessary
MOA is well defined as agreed upon by the FDA and Panel
The shorts will be out of the picture...finally. They made their Hay and will not go short with PDUFA any Monday between now and May 26th. Good BYE forever SRPT shorts!
Go to 3rd article down (A.F. article) in Yahoo Finance under the SRPT section...He provides an excellent link "where he tells you to click here" works great.
Go to yahoo finance and the link is attached to A.F. article....simply click the link in the SRPT section and you are there
Even many longs still believe the BD's will be relatively unchanged, mostly negative and maybe nothing more than very slightly better than the Jan. BD. The shorts may be playing the BD report due to not much upward price risk and a good chance of a significant temporary drop. In other words, the BD play is in their risk/reward favor. However, I would think there will be a huge amount of covering on the other side of the BD. ADCOM may be very challenging for the shorts and PDUFA will be a mystery all the way to the end.
Sorry You feel that way. This is the first time I have ever reached outside the message board to convey a message. I do realize, It may have been a waste of time, but I am determined to do what ever I can.. no matter how you want to condemn me! I will NEVER GIVE UP for this final Chance for the DMD families..#$%$!!
I Just Forwarded My post to Dr. J.W and staff. I hope she reads it, because it is from the bottom of my heart. It took me two Hours to find her E-mail..How Embarrassing..I am not the most computer savvy at my age. Anyway I sent it for all it's worth. I doubt if she reads it, but if she does..I'll feel better about the two hour adventure. I need to hand these tasks off to you savvy Young bucks and Buckettes...Take Care. KGR
New ADCOM Panel
Strong Insider Buying
F.W. stronghold supporter of Parental testimony
Positive Respiratory Data
No other options for years
LOA data approaching 4.4 years
Charitable Huge Support
Congressional Huge Support
Congressional Huge Support
DMD Experts Huge Support
Confirmatory data in Place for monitoring
Other Exon trials in Place for Monitoring
A DISEASE State 100% DESTINED TO MORTALITY
Pristine safety with 200 plus ETEP exposures for various duration of time
J.W. Willing acceptable of Parental testimonials
Keep in mind, ETEP has not yet been fine tuned for efficacy and Safety..Via Maximal Tolerated Dosing (MTD). Once on the market and the Confirmatory trials show continued safety and moderate efficacy, they may push the dosage and frequency to a higher level in order to not compromise safety, but to maximize efficacy. I do not Believe we are at the Peak Efficacious/safety ultimate combination of this compound yet. It must first hit the conditional approval market, then titration and frequency dosing in a gradual manner can seek out MTD. I believe by getting ETEP on the market, is only the beginning due to conservative safety concerns: due to the initial approval process. Add to that, other DMD compounds that can have a synergistic combination approach to ETEP without compromising safety issues. Get ETEP approved, and infancy may turn into compelling maximum maturity in the DMD State
You may be Correct Pears. However, I am a believer in a new lead ADCOM representative with at least a Neurological specialty that can handle the 36 DMD experts. Certainly not fit for Farkas...We will see How adaptable and prudent are FDA can adjust to the changing tides from so many fronts. BD errors, New 4 year data, new overall data and 36 experts endorsing a safe a at least somewhat effective DMD drug for Kids with No other chance, under the Auspices of FADASIA?AA Unmet Disease State...with a confirmatory for back up. Please tell me how a pristine safe drug can miss on these marks.
Mw, thank you for the clarification. However, If Farkas is not on the ADCOM, why was he so influential in the first BD process and publication?. Secondly, If he has a vote in the final PADUFA, then that makes me even more worried. I am here for ETEP and the kids, so nothing will change on my end. However, Farkas's role in DMD is quite disconcerting no matter how the algorithm plays out. The BD's, ADCOM and PDUFA are all separate entities....though intricately and politically impacted by each other..Let's Hope one Entity gets FDASIA/ AA right.
I may be wrong, However, I think the next BD will be lead Authored by at least a Neurologist on the ADCOM Panel. Especially, in light of the 36 DMD experts endorsing approval. It would not make sense if an Ophthalmologists Lead/Authored the BD and rebuking this expert group. If that does happen, I will be astoundingly ashamed of the assignment representation leading into a Neurological FADASIA/AA unmet and complicated Disease State. Farkas, under his own volition should relinquish this role and pass it on to a more qualified panalist. If not, then we do know Arrogance, Ego and Pride are certainly more important than doing the right thing.