Flash, I'm recycling my post over the current collaboration with the FDA on the Rett Syndrome IND. I'm sure that there is some deliberateness driven by an effort to be overly cautious with the patient population of young girls. However, Drs Kaminsky and Missling have been associated for some time and the safety concern will have been in the forefront of their discussions of the trial. Add to this that the 2-73 P2 will follow directly on the completion of the Nueren "trofintide" P2 so, Dr Kaminski will have worked through participant selection in great detail recently.
I believe that discussions with CDER over the trial architecture may involve the possibility of a "Basket Trial" expansion covering one or more of the Orphan Diseases of Fragile X, Angelman's and Infantile Spasms in future. These were mentioned by CM in the Noble presentation and have the common end point of reducing spasms, tremors and uncontrolled limb movements.
Thus, IMHO the collaboration over the IND will lead to a more effective and possibly broader reaching trial.
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BIO, Reread the correspondence/testimony to Congress by Dr Woodcock(letter supplied in m last post) and Commissioner Gottlieb. The drug development charter of the FDA and CDER under the 21stCCA is to get safe and effective drugs to the American public faster. That is why the ACTC has been created along side the Cancer Center of Excellence. The IND process is now significantly a more collaborative process designed to assist drug companies to design their trials along targeted precision medicine guidelines. If I can make a simple analogy... it is like a a water channel "lock" rather than a regulatory wall to be scaled. Through direct consultation during the IND submission the FDA/CDER raises the drug submission to a precision level of testing. Certainly the new 21stCCA methodology is not mature from the regulatory side so there will be delays as the government and pharmaceutical industry work through the process. But, in the future there will be fewer drug failures because of trial design and more success because of targeted disease subgroups/endpoints.
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