Replies to post #265403 on Anavex Life Sciences Corp (AVXL)

[rayovacAAA]

FOR YEARS ANY PATIENT HAS HAD THE ABILITY TO ASK A DR. TO SUBMIT "A BIT OF PAPER-WORK" TO THE TGA FOR APPROVAL TO TAKE A2-73!!!!!!!!!!!!!!!!!

talked with someone at the TGA on Wednesday at in regards to blarcamesine and its 100% the truth = all Alz patients have access , just just need their DR. to fill-out the proper paperwork and submit to the TGA. A bit of paper-work as 2-73 isnt on the approved drug inventory, but people have access.

HOW MANY BITS OF PAPERWORK HAVE BEEN SUBMITTED AND APPROVED BY THE TGA TO TAKE A2-73 IN THOSE YEARS ?????????????????????




GOOD LUCK AND GOD BLESS!!!!!!!!!!!!!!!!!!!

[rayovacAAA]

WHAT % OF THAT 75% WERE EVENTUALLY APPROVED FOR ALZHEIMERS BY THE TGA?????????????

She also told me around 75% drugs eventually get approved after SAS, unless there are safety concerns while under SAS. People failing to realize 2-73 will be approved are kidding themselves and making a petty large mistake.

WHAT WAS HER NAME AND IS THERE DOCUMENTATION AVAILABLE FROM THE TGA ONLINE TO CONFIRM THE ACCURACY AND CONTEXT OF HER STATEMENT????

[falconer66a]

From a TGA insider:

...around 75% drugs eventually get approved after SAS, unless there are safety concerns while under SAS.

Thanks for this message, telling that someone inside the Australian Therapeutic Goods Administration (TGA) specifically made this statement.

Consider it.

First, if about three-quarters of drugs in the SAS eventually get approved, what, perchance, would keep the other 25% from being approved? Has to be at least one of two things: inefficacy (the drug doesn't really work), or unsafe (too many or too severe adverse events, side effects).

Efficacy. For Anavex 2-73 in treating Australians with Alzheimer's, in the earliest study, was the drug ineffective; failed to produce positive results? If that were so, why would so many in that study elect to continue to take the drug continuously, for many years, after the formal study was completed? If the drug did not produce real-world results for those in the early trial, there wouldn't have been a near 100% continuance of taking the drug.

Of course, those holding to accepted standards of drug approval studies will claim that data are still insufficient to render a confident positive on blarcamesine against Alzheimer's. "Not enough data to know for sure, yet."

That might be true. But it's not a factor in the recent Special Access Scheme (SAS), allowing Australian physicians now to petition the TGA to prescribe blarcamesine for their patients. Quibble over that as some sort of "approval" as various parties have, or not, the Anavex drug nonetheless will be available to patients, albeit initially for only a few.

Safety. The other, more important reason a drug now in the SAS might not gain eventual full approval would be lack of safety. The drug causes more harm than allowable. It's plainly unsafe, all other factors notwithstanding.

Fortunately, for blarcamesine, that is not a factor of concern. The drug has proven profoundly safe in all of the preclinical murine (lab rodent) tests, and also in all human trials. As noted, side effects of blarcamesine, when they occur, are only a bit of dizziness or headache. Compare those cautionary outcomes to the side effects noted in any of the TV advertisements for prescription drugs on any of the network evening news programs. Many of those, too, are for central nervous system (CNS) diseases. Side effects are severe, some being lethal. Blarcamesine works in the CNS, but has no lethal or other severe side effects. And side effects appear only at maximum doses, or are quickly resolved when dosages are reduced, or after a period of accommodation. The body gets used to the drug.

If these things are true (they are), looks like blarcamesine is highly likely to fall within the 75% of SAS drugs that subsequently get approved.

As with all the clinical blarcamesine data, in both murines and humans, the odds for eventual full approval are now very strong.

[Warby 3]

Thanks for sharing
Did this person tell you how long it takes now for approval?

[attilathehunt]

Just curious....Did you speak with Rebecca?

[Turner2017]

Thank you very much. We got Australia! Rest of the world soon!!!

[nidan7500]

Fireman

People failing to realize 2-73 will be approved are kidding themselves and making a petty large mistake.

Well said. It's too bad we have to go through this but (IMO) stealth is absolutely the correct strategy at this point. Let the science do the work, the rest will take care of itself.

Thankfully there are places in the world where science prevails over politics.

[sokol]

SAS designation of Blarcamesine in Australia is a smart move. Will early access be expanded to Europe? See the below excerpts from 3 articles with references.

1.Early Access Programs
Governments worldwide have created provisions for granting access to drugs prior to approval for patients who have exhausted all alternative treatment options and do not match clinical trial entry criteria, these are so called Early Access Programmes (EAPs) or labelled Compassionate Use Schemes (CUSs). Some markets regulation allows patients to access drugs that are approved outside of the region, but not yet in their home countries. EAPs are governed by guidelines and legislation that vary by country, defining access criteria, data collection, supply and control of the drug distribution. Some countries (e.g. Canada and Australia) have well defined EAP, Special Access Program (SAP) and Special Access Scheme (SAS), respectively. EAPs can be put in place at any stage of development post-phase II and can run in parallel with phase III clinical trials, until market authorisation is granted. Reporting data about efficacy, safety and occurrence of adverse events to the responsible health authority are usually mandatory requirements. Mostly it is the treating physician that is responsible for initiating the request, monitoring and reporting any output coming for the utilisation of the unauthorised drug (in clinical trials, it is the sponsors responsibility). Regulations differ widely among countries, due to differences in national medical practices, resources available, product funding, hospital structures and national insurance systems.

While patients, hospitals and/or national insurance systems bear the costs in some countries, the sponsor is expected to provide Compassionate Use products free of charge. An important consideration is that if a drug is charged for, then the obtained price may be used as future benchmark for pricing and reimbursement committees.

Conclusions

Advances have been made in fast tracking medicines to patients with unmet needs. We have described some evolution by the regulators to create accelerated routes and health authorities allowing patients access to experimental or unapproved medicines. The developer needs to carefully evaluate these options before embarking on any of these routes, all while providing advantages to patients where certain limitations could apply.

Likewise, the decision to implement an EAP should be carefully considered and a sponsor should ask important questions such as when to offer access and for which patients, as there might also be many drawbacks tied to its implementation. Existing regulations do not force companies to offer access to drugs prior to approval or launch.

In addition to providing significant benefit to patients with unmet needs, EAPs can offer important benefits in terms of increased and earlier access to the sponsoring manufacturer. EAPs can be a part of a global market access strategy, generating development strategies that are increasingly innovative and global in scope.

https://www.huronconsultinggroup.com/insights/early-access-medicines-for-unmet-needs


2.Early access programs in Europe: a regulatory tool with pre-marketing impact

EAPs offer real marketing rewards

EAPs are a good means of testing the product in "real life" and convincing prescribers and patients of product efficacy before launch. One advantage is that "real life" data are obtained, reflecting a more clinically and ethnically diverse population than often encountered in clinical trials. This information gained pre-launch can also shape post-launch marketing messages. "Early Adopters" or brand advocates can be identified as a wider group of physicians gain experience with the drug, and physician loyalty may be developed. Furthermore, key opinion leaders often play a major role in discussions with regulatory agencies.

Equally with respect to patients, as well as providing them with access to a potentially life saving medicine, there is the opportunity for feedback, the development of patient loyalty and building relationship with patient advocacy groups and associations.

There is the potential for early revenues in countries where EAPs can be designed as "for-profit" programs, but whether or not a drug is made available free of charge, market penetration can be maximized pre-launch through an EAP, which can then translate to a successful launch and increased post-approval usage. In practice, a good rule of thumb is that first-year market penetration after employing an EAP strategy is equivalent to the second year market penetration of a regular launch.

Even when EAPs run at a loss or neutral returns in stand-alone financial terms, long-term benefits make them an attractive choice for companies. In fact, patient recruitment is generally faster for not-for-profit EAPs, so if these patients keep using the drug when it is sold post-launch, a not-for-profit scenario could in fact provide the most benefit to pharma/biotech firms.

3.Expanded access or compassionate use is the use of an unapproved drug or medical device under special forms of investigational new drug applications (IND) or IDE application for devices, outside of a clinical trial, by people with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial in progress.

These programs go under various names, including early access, special access, or managed access program, compassionate use, compassionate access, named-patient access, temporary authorization for use, cohort access, and pre-approval access.

Europe
In Europe, the European Medicines Agency issued guidelines that members may follow. Each country has its own regulations, and they vary. In the UK, for example, the program is called "early access to medicine scheme" or EAMS and was established in 2014. If a company that wants to provide a drug under EAMS, it must submit its Phase I data to the Medicines and Healthcare products Regulatory Agency and apply for what is called a "promising innovative medicine" (PIM) designation. If that designation is approved, the data is reviewed, if that review is positive, the National Health Service is obligated to pay for people who fit the criteria to have access to the drug. As of 2016, governments also paid for early access to drugs in Austria, Germany, Greece, and Spain.

Companies sometimes make use of expanded programs in Europe even after they receive EMA approval to market a drug, because drugs also must go through regulatory processes in each member state, and in some countries this process can take nearly a year; companies can start making sales earlier under these programs.

https://en.m.wikipedia.org/wiki/Expanded_access

[Steady_T]

My guess is that the 75% that do get approved also have P3 trials completed and then get approved.

I think the word "eventually" is the operative word here.