"First, Do No Harm". This was ingrained in their medical training. Let's look at how ETEP fits this Treatment Concept. 1) Pristine Safety Data, 2) A Likely Degree of Potential musculoskeletal and Cardio-Respiratory data and Testimonials, 3) Untreated, 100% NH clinical regression, morbidity and mortality ( i.e. 100% Type 2 Error). Type 2 Error is 100% inherent if children are left untreated. Type 1 actually has very little effect in the area of harm...perhaps just a small unlikely percentage. Summary, Type 2 = Harm, Type 1 = the drug may not be as efficacious as thought, but will probably still be safe. I.e., Confirmatory can easily patch a Type 1 error with little chance of harm. So far, we have nearly 4.5 years demonstrating that type 2 is a far greater threat to the physicians gold standard..of.. "First, Do No Harm!" Again, Type 1 is fixable, Type 2 is not!
Many phrases were intentionally left out at ADCOM, such as FADASIA language: "Clinically meaningful", "Clinically Likely", "Reasonably Likely", "Risk/Benefit Analysis", "Totality of Evidence", "Rolling NDA", "Approval Based on Confirmatory Data"....NADA...Nothing of the sort!
I think behind closed doors, without the Lime Light of the public Eye (no pun intended), where pride and scientific showmanship will be shifted towards a less dogmatic, indoctrinated mind set, to a more pragmatic consensus. This is obviously my hope and opinion.
Will turn the final voting discussion towards a Risk/Benefit discussion, as opposed to the Study criteria (power, "N", design etc..) that was so ridiculously and nauseously scrutinized in the BD's and ADCOM. If J.W. can redirect and align their thinking within the frame work of FADASIA, using the Risk/Benefit analysis in an unmet disease state with NO other options, I believe there is a strong chance for at least a conditional approval. It will be a waste of time to rehash the BD's and ADCOM, except to point out that the questions were complex and confusing and the tenner of discussion was absent the language and intent of FADASIA. If J.W. and Callif are successful in realigning their mind set to FADASIA and the totality of evidence, safety and nature of disease state, then by June 1st we should see ETEP approved.
My problem with your statement, was calling the ADCOM DMD experts...they absolutely were not. As equally biased as you claim the SRPT experts were, was the hand picked non qualified DMD array of multi disciplinary question devised pigeon holed FDA biased puppets. The panel was determined to design questions that were not fair balanced and lacked FADASIA verbiage and jurisdiction.
Maybe, maybe..for a slowing of progression in a disease state, but NOT a REVERSAL after 4 plus years in all 10 boys. A Reversal in Physical function, LOA, QOL, ADL's, Falls, Cardio-respiratory Parameters and Safety. Saying this is a placebo effect is truly a Farkass cried stretch!
bottom line, I just don't think she will throw her staff and the outside adcom experts under the bus without additional proof of real efficacy -- i.e., beyond a potential placebo effect
This obviously could happen given the nature and history of all the obstacles ETEP has had to endure. However, your statement and sentiment above is abysmal. DMD experts thrown under the bus?? How about 36 REAL DMD experts, 50 positive testimonials, a company with an approved NDA and data to back it, congress, Senate, Parents, Children...FADASIA, Unmet need, No other options and pristine safety..thrown under the bus. Seriously, a 6 to 7 vote, confused and unqualified panel, unfair questions, absentees galore (again, confusion). Bottom line, You think J.W. is worried about 7 unqualified and confused EYE doctor types vs. the litany of circumstantially abused proponents. Put the Bus in drive and do the right thing.
There are plenty of worthless drugs that the third party has to pay for. Opiates for one. They costs billions in detox/rehab therapy for addicted patients. Many physicians hand them out like candy and we are paying for the prescription, then the addiction. Then there is the ED industry, #$%$ etc...
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