Hey there Mr. Learner
To try and finally finish our discussion about the Rare-Disease-day Webinar I have what I hope is a last favour to ask of you - would you please have a look at 2 posts I made after the last live AGM (sankaido post 4192204 of 12/12/2019, and post 4364011 of 23/3/20). They are about a question I asked the Chairman at that AGM, and his response it to.
The reason I ask is that it seems clear to me that Mr. Moses' actions in recent weeks of 1. arranging for the Board to agree to appoint Dr. Gittleson as a Board Director, 2.announcing his decision to stand-down to allow her to become Chairman, and then 3. having it activated by the Board, are his responses to his undertaking to consider what I had raised with him (in his words "..leave it with me") I am asking because I value your opinion and know that we can discuss things we don't necessarily agree on without losing mutual respect. Also, as I noted, I asked the question on behalf of quite a number of other posters who had also expressed support on this forum that something be done for the boys, and would appreciate any views from them. I think arjay, imp (who quickly indicated his doubt that anything would result from it), Sam were among the supporters - please excuse my not having a full list.
From here on the decisions will obviously be made by the new Board/management - I hope the company will choose to stay headquartered in Australia. There have been some indications that this may eventuate, such as the R&D agreement with the Murdoch Foundation, and the change of their Melbourne office address from the previous location to the same place (not a typo).
The possibility of undertaking another trial in Australia in which the original 9 boys can again participate seems very remote, but as there is still no clarity as to where things stand with either the EMA or the FDA I haven't given it up, and there are some goods posts containing support information on the validity of natural history studies for instance being accepted as a tool for getting agreement for earlier access, and the participation of Gil Price in the PPMD project is another positive.
I think Mr. Moses actions have also, in effect, dealt with your media concerns, which as far as I can identify apply only to the arrangements made for generating investment in Asia/Australia which involved the issue of company shares in lieu of cash payment to Spark Plus, with the placement made under the joint lead management of Wilsons and Morgans.
In the end, possibly the most important impression that resonated with me from the Webinar was what a huge, complex organisation the FDA is. An agency within the U.S. Department of Health and Human Services with 18,062 employees (2020 estimate) whose activities involve long-term programs with a huge range of entities - other arms of government, companies, patients and patient groups, Congress, insurers etc. etc. including the provision of a massive variety of assistance services. That is the context within which our dealings with them, as a tiny foreign company seeking their approval to permit the sale of our drug within their country, take place. We have been informed that they are willing, even keen to co-operate with us in hopefully getting agreement to speed up the approval time, but we certainly will be in a long queque. But there are quite a number of references in the final Q & A session of the webinar which confirm that is one of their major objectives, and there appear to be quite a number of of similar programs underway, some of which have been operating for several years.
The following is panel member Dr. Jeff Shuren 's view of how they want to adopt some of the measures they implemented due to the PANDEMIC requirements to their regulatory procedures (note - it is rather long but has a reference to the Pharmaceutical Advisory Board that the PPMD is organising for the FDA which Dr. Price is joining)
Dr.JeffreyShuren is oneof the senior DEA executives who reports direct to Ms. Woodcock.
"Inspite of the pandemic and themassive workload that it entailed, we have continued to take actionsto help advance the availability of important medical technologiesfor individuals with rare diseases. So let me talk through some ofthose activities. Sofor example, over the past year, we have authorized the PlasmaDelipidation System, which is for individuals who have homozygousfamilyhypercholesterolemia.We also authorized Sonelete, which is an MR-guided high intensityfrequency ultrasound that is used in individualswho have an osteoma. Just a non-invasive way of treating them,particularly individuals who have developed intractable pain that isunmitigated with medications.
Wehave also taken steps to further advance the availability of medicaltechnologies for children with rare disease. About thirty millionAmericans today have a rare disease, and about half of them arechildren. But there are a lot of challenges in being able to assesstechnologies. And for that reason, we see very little innovation inthe med-tech space when we are dealing with our children. One otherthing we have been involved in is helping to co-found apublic/private partnership called the Systems of Hospitals forInnovation and Pediatric Medical Devices, or SHIP-MD, that is anetwork of institutions, primarily pediatric academic medical centerswho, rather than individually going to places trying to findpediatric patients to recruit, they come together as a network topool their resources and expertise, to then recruit patients toclinical trials, conduct those clinical trials, then vet thetechnology to see if there is actually a good potential forassessing. And now, they are working on a single signature contractto really streamline the ability to set up and conduct a clinicalstudy. And one of the next steps is trying to bring in the door ofthe state Medicaid directors, because 40 percent of children in theU.S.receive their healthcare through Medicaid. Andhopefully, this way we find greater guarantees around reimbursement.And this combination of activities, we hope will be a shot in the armfor greater investment in medical technologies for our children,particularly children with rare diseases. Andthen, we have been taking steps to also advance the role of patientsand their care. One of those efforts is to better understand theirpreferences.Sofor example, the study underway with UC San Francisco Stanford Centeron understanding the preferences of children with heart failure, tothen informthe development of new technologies and patient reported outcomes. Wehave established a network of patient organizations called thePatient Caregiver Connection. And that provides us, really, withpatient experts to serve as advisors to the FDA and steps we shouldtake and help inform some of our decisions. And that network has 16members; we are almost at our 17th,and includes some of the organizations representing individuals withrare diseases, like the National Organizations of Rare Disorders andthe
MuscularDistrophy Association.Andone of our big strategic priorities for the Center is a creation ofsomething called collaborative communities. Now we engage incollaboration all the time. But often, it is government in thedriver's seat in one-off activities. A collaborative community iswhere the key stakeholder groups in that community come together tosolve shared problems and achieve shared outcomes in an ongoingfashion through a continuing forum, and
where government - FDA - has a seat at the table asamember of the table. Wedo not drive it. We do not run it. But we act as a member.And if the community comes up with a solution, and that is where theywant to go, and it is in the best interest of patients, and it is notcontrary to our statutory mandates, weare likely to adopt it as our solution.So really putting the community in the driver seat. Already, we havesigned up for 10 of these communities. We have many more in thehopper. And now, some of them arestarting to engage in work that can impact rare disease, such as theone that has been established for thalamic imaging. Lastly,let me close with some of the lessons learned out of COVID. Because Ido think it would be a terrible tragedy from this pandemic if we didnot.
Firstoff is regulatory flexibility. You know, when the pandemic hit, wewere able to take advantage of our emergency use authorizationauthorities. And it allowed us that flexibility - allowed us to trulytailor our approach to the technologies - to those balances - we gotsafe and effective devices out there, but also very timely patientaccess. And we think this, applied in the rare disease space,is criticallyimportant. Andsecondly, is engagement. Taking our approach in the breakthroughdevices program, with a lot of early and frequentengagement - but putting it on steroids where developers literallywere engaging with us onareal- or near-real time basis, submitting data on a rolling view. We hada 1-800 24/7 hotline setup, and 16 e-mail boxes, and a variety ofother actions. That engagement with developers, I think, helped leadto, along with regulatory flexibility - the development intechnologies like tests, within - you know, weeks, and validationand authorization in literally weeks, ratherthan what would take months to a year or longer. Andagain, that kind of approach advanced in the rare disease space, wethink can be a game-changer
(there is quite a lot more info like the above in the 4th Q&A panel which cannot be summarized by my brain - there is a written record of the complete webinar available on the FDA website which is a hell of a lot easier and quicker to read than watching it)
Last thing I will mention is a post by Chilunsun no.53923031 on 18/621 referring to "Devil in the detail" about advanced stage clinical trials, which inter-alia, contains info on conducting trials in foreign locations which was posted by someone I think back around early 2020 and may have given some of us (myself included) the impression that another trial in Australia was possible i.e. if the PPMD for instance were able to send some boys over to make a larger trial here. I sat in on a PPMD webinar hosted by Mary a few months ago in which she asked one of the company reps presenting how much it would cost all up to send a boy to participate in a trial in " Australia, for instance".
