Update From The God Himself - Dr. Takashi Tsuji

[Dogma]

Dogma is just pissed I took him up on a bet for his house lol so now the coward is trying to set up new terms to cover his ***

No I’m not setting up new terms, I’m sticking to the terms that I have been talking about since the inception of this forum and I began participating in The discussion expressing my vehement doubts of a full commercialized release by 2020, I have made this explicitly clear it’s not going to happen and so has another member bboy. If you think the conditional seven year access, under the new PMD act allows products to be given early after safety and efficacy clinical trials are proven, is full commercialization you just don’t know what I’m talking about, full commercialization is not just the Japanese market full commercialization means that we can all book an appointment to receive the treatment, this requires a phase 3. I already conceded and in fact never disagreed that it might be possible for a few people to benefit from the new Japanese regenerative medicine regulations by 2020 if they complete most of a phase II to qualify, again I’ve said many times that is possible however full market release with clinics around the world trying to purchase their rights to sell the treatment is not going to happen by 2020. In fact look at the interview with Dr. Tsuji he says the same thing, it’s not gonna happen. And again if you want to follow the pedantic definition of commercialization not “Full commercialization“ which are two different terms within business The money that they receive courtesy to the PMD act during a seven-year conditional period they have to continue on to complete a larger phase 3 trial, and they have to continuously provide data and information to the regulatory board in Japan, the money they make from The conditional release in the “Japanese market“ is almost guaranteed to go to paying for the phase 3 clinical trial ( which costs a ridiculous amount of money), and collecting all of that data and providing it to the regulatory board, therefore it’s not revenue, it’s more along the lines of fundraising helping the product reach actual full commercialization which is literally the purpose of the new regulations for regenerative medicine in Japan! And again even if it was giving them a small profit, it’s not full commercialization whilst being: conditional, temporary and isolated to the Japanese market under strict supervision and regulations you dotard

 

[Dogma]

Hopefully with Replicel not have to wait so long.....

Bro they have to go through the same process, and are under the same regulations however if I’m not mistaken they have started a phase II A little while ago in Japan, but again even in accordance with their new regulations as of 2014 it would still only give early access to the Japanese market. As for foreigners gaining access I have no idea, I think it’s a safe assumption Japanese market means Japanese citizens, but also accept foreigners into the early conditional release could be against the second act. I don’t know that for sure, I’m just saying that that could be within the legislation

 

[Dogma]

the way i understand it, after the initial trial that could start next year if it is successful there will be a conditional market release for a small number of people but it probably won't be acceptable to us right away. during that time they can gather further data and work on their infrastructure. if nothing bad happens after a while(not sure abut how many years but at max 7) they can apply for full approval without a phase 3 study. so potentially we are looking at long time for us to get it. if it indeed works like that, balding guys from the US and europe might have to wait until 2025-2026 for it to be available IF the results are good. if not we are looking at 2030 release for us. f*****g moronic that it takes this long

but could someone figure this out? what happens during this initial commercialization and how long/how many people and from where can take part? the way i see it now it's just another looong clinical trial phase but they can make money along the way? there has to be a difference?

They are starting a phase 1 trial in humans! Still got a ways to go before they can qualify for conditional release within the Japanese market thanks to the new regulation ( requires phase 1 and 2 clinical results proving efficacy and safety). And you are unequivocally, incontrovertibly, blatantly wrong about them not having to complete a phase 3! First of all under the second act the conditional release, which is isolated to the “Japanese market” has a time limit seven years, during that time they are to complete moving forward trying to complete larger more in depth clinical trials a.k.a. a phase 3, and they have to provide data and information on the progress to the Japanese regulatory board throughout the process. If they are unsuccessful after seven years they have to go back to the drawing board or shut down, but their conditional product release is now revoked.

Just to give you a Little perspective, this is what it is like for pretty much all western countries, Japan used to operate pretty much the same way until they implemented the brilliant idea of keeping pharmaceuticals and cellular based therapies separate and overseen by different administrations. The requirements and stipulations for each phase are likely the same, nothing in the new regulations for regenerative medicine states that the clinical trial phases will be shortened, and/or that these stipulations and requirements will be more lenient. The first act makes the R&D phase shorter , The second act just allows conditional release after completion of a phase 1 and phase 2 showing efficacy and safety.

- Phase 0 of a clinical trial is done with a very small number of people, usually fewer than 15. Investigators use a very small dose of medication to make sure it isn’t harmful to humans before they start using it in higher doses for later phases.

- During phase I of a clinical trial, investigators spend several months looking at the effects of the medication on about 20 to 80 people who have no underlying health conditions. 70% make it through this phase.

- Phase II of a clinical trial involves several hundred participants who are living with the condition that the new medication is meant to treat. They’re usually given the same dose that was found to be safe in the previous phase. ( in Japan they would need to be pretty much near completion of this phase for when they can begin offering treatment early to the lucky few, keep in mind this means no adverse reactions, no investor fallout, etc. All of which can lengthen any part of a clinical trial indefinitely). Investigators monitor participants for several months or years to see how effective the medication is and to gather more information about any side effects it might cause. 30% make it through this phase.

- Phase III studies involve randomized and blind testing in several hundred to several thousand patients. This large-scale testing, which can last several years, provides the pharmaceutical company and the FDA with a more thorough understanding of the effectiveness of the drug or device, the benefits and the range of possible adverse reactions. 70% to 90% of drugs that enter Phase III studies successfully complete this phase of testing. Once Phase III is complete, a pharmaceutical company can request FDA approval for marketing the drug.

- Phase IV studies, often called Post Marketing Surveillance Trials, are conducted after a drug or device has been approved for consumer sale. Pharmaceutical companies have several objectives at this stage: (1) to compare a drug with other drugs already in the market; (2) to monitor a drug's long-term effectiveness and impact on a patient's quality of life; and (3) to determine the cost-effectiveness of a drug therapy relative to other traditional and new therapies. Phase IV studies can result in a drug or device being taken off the market or restrictions of use could be placed on the product depending on the findings in the study.
Sources: https://www.healthline.com/health/clinical-trial-phases#phase-i

https://www.centerwatch.com/clinical-trials/overview.aspx/

 

[byebyehair]

After a phase 3 yes, but during the conditional seven-year marketing period after phase II confirms safety and efficiently it clearly states it will Allow the product access to the “Japanese market“, that is a direct statement from more than one source but I’m going to assume that the Japanese market pretty much unanimously consists of Japanese citizens. I would consider that a fair and rational assumption. However we all know damn well money talks, if you have multi millions you can pick any clinical trial right now for any condition bring all of the equipment and surgeons down to the Bahamas where they have very little medical regulation but great facilities and have the experimental therapy performed… So is it crazy to assume that if you are capable and willing of paying quadruple the standard price or make an offer they cannot refuse they will not allow a foreigner to receive early access in the Japanese market? The only question is if that would be antithetical to the PMD act, and thereby damage their full approval process well they are in the conditional phase.

Laws and regulations are commonly bound to specific regions (geographically ) and not to specific populations. Therefore i would say your assimption is quit questionable. My assumption is if you want to get the treatment as a foreigner you have to ensure them to return after a year for a follow up investigation.

But that is just my assumption based in my knowledge of laws.

 

[Dogma]

Laws and regulations are commonly bound to specific regions (geographically ) and not to specific populations. Therefore i would say your assimption is quit questionable. My assumption is if you want to get the treatment as a foreigner you have to ensure them to return after a year for a follow up investigation.

But that is just my assumption based in my knowledge of laws.

This is a fair observation, but one would have to read the specific rules and requirements within the PMD act, there are a few doctors who have expressed their concern about allowing early access even after a phase 2 clinical trial, and the fact is the Japanese government put a seven-year contingency in place for the “conditional early access“, and they have to continuously update the Japanese regulatory board on what is transpiring, while continuing on with the larger trials. All of these contingencies are in place for a reason, there is concern when it comes to cellular research, and these new regulations apply to genetic therapies as well. Like I said I don’t know I just made an assumption, it just states early release within the Japanese market, either way it’s going to be primarily Japanese citizens.

 

[RolfLeeBuckler]

Here The understanding of Lee Buckler:
http://www.cj-partners.com/en/news/files/000025_en_01.pdf

 

[Dogma]

I haven't read all yet but the 7 year figure is already wrong, it's maximum 7 years, most treatments under pmd (all of them so far) have a period no longer than 5 years for conditional approval. and yeah so by what you are saying, did lee buckler lie when he suggested people from across the globe can get rch-01? enlighten me, why are they talking about or did talk about a 2018 release, why did they even see it as something feasible if they would have to conduct another phase 3 trial?

because if what you say is true, if only half of it is true, then we have pretty much a decade to go for tsuji and rich-01 to come out for the vast majority of people. but i wouldn't be surprised, seeing how pathetic the medical research community has become when it comes to hairloss

OK I have read each one of your ‘rebuttals’ and I’m not sure if you just didn’t read the entirety of the source you provided, or you completely misinterpreted the information that I cited (potentially assumed that I was just speaking from my opinion instead of quoting directly from articles about the reforms made in Japan, back in 2014). So I will try and go through everything you said chronologically:

I haven't read all yet but the 7 year figure is already wrong, it's maximum 7 years, most treatments under pmd (all of them so far) have a period no longer than 5 years for conditional approval

- from your source In regards to the The 7 Year Conditional Approval Period under the PMD act (note that is the literal headline of this section in your source “7 Year Conditional Approval Period”): A conditional approval will be time-bounded and include requirements for further studying the safety and efficacy of the regenerative medical product during the conditional approval period. The conditional approval period may be as long as seven years or fewer, as determined at the discretion of the Japanese regulators. At the end of the conditional approval period, a re-application must be submitted to continue marketing the regenerative medical product and the PMDA/MHLW review and decision process will be repeated. The regenerative medical product's marketing authorization may be revoked if the safety and efficacy of the product has not been corroborated.

Just a few points on the seven-year conditional approval peroid in my comment:
1. Similarly, the PMD Act introduces a specific regulatory framework for regenerative medicine products. Under the PMD Act, conditional and time-limited marketing approval can be given to a regenerative medicine product after exploratory clinical trials have demonstrated probable benefit and proven safety (aka phase I and II). According to these new laws, once a company has demonstrated safety and basic efficiency data in humans and has the cell product manufactured to the standards described within the Pharmaceutical and Medical Devices (PMD) Act, the cell therapy can be given conditional approval for up to seven years. This allows for commercial use with data reporting requirements and potential for national insurance coverage.

2. The intent of the laws is to accelerate the commercialization of cell therapeutics within Japan by allowing companies to benefit from conditional marketing authorization. Therefore, cell therapies that show safety and probable efficacy during Phase I and Phase II trials can get conditional approval for up to seven years...

3. During the seven-year conditional approval period, companies must continue to submit clinical trial data to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), and subsequently apply for final marketing approval or withdraw the product within seven years.
Source: https://bioinformant.com/japan-accelerated-approvals-of-cell-therapies/#869

I’m literally perplexed do you not see what I cited!? Throughout my entire comment it clearly states that it is a maximum of seven years for the temporary conditional approval, after seven years there needs to be a resubmission for approval and there will be a revaluation . Did you assume that these citations were stating it was a minimum of seven years that a conditional approval had to be in place before they could move forward or something? Every time the seven year time period is mentioned it is preference with: “for up to seven years“, “ apply for final marketing approval or withdrawl the product within seven years“. You stated that the seven-year figure was wrong, and that it was a “maximum of seven years”… Well yes I thought that was blatantly evident, I’m not sure what you were reading? Although you did preference with saying I didn’t read everything. Might better next time.

 

[Dogma]

and yeah so by what you are saying, did lee buckler lie when he suggested people from across the globe can get rch-01? enlighten me, why are they talking about or did talk about a 2018 release, why did they even see it as something feasible if they would have to conduct another phase 3 trial?

Can all of us go to a relatively nearby clinic, or even Japan to receive rich-01 as it stands today Sunday, March 17, 2019? I wouldn’t call it a lie per se, I would call it marketing, or if I wanted to be more pessimistic/cynical overzealous expectations of their capabilities.

 

[Dogma]

now that I read more of it, a lot of what you say is complete and utter bullcrap holy sh*t. no, they do NOT need two phases to quality for conditional release, I literally talked to a dude yesterday who got a treatment that is under such a scheme right now and they have only done a 12 month 20 person trial. you make so much sh*t up, it's literally in the bill

https://www.caladrius.com/wp-content/uploads/2018/05/Sietsema-et-al.-2018-RAPS.pdf


read this and then look at the graphic on page 14, that should disprove this nonsense. only one trial is required in which both things are tested at once, this is unique to regenerative stem cell treatments but think about it in the case how tsuji, how deranged would it be to make two trials after one another one where you see if the hair grows and another one in which you check efficiency. obviously that nonsense and not required and if you actually read this bill you'd know that. but you haven't

Well first of all I didn’t make anything up, I provided three sources about articles on these new regenerative therapy regulations in Japan as of 2014, and I quoted them. So let us get more scrupulous:

- Page 2 of your source where it explains the logic behind separating pharmaceuticals from regenerative therapy’s, treating the regulatory process different, hopefully hastening advanced cellular therapies, etc.: ...products are approved through the Clinical Trial Notification (CTN) system, as are pharmaceuticals and medical devices. Consultations, required for regenerative medicine products prior to submission of a CTN, are also managed in a similar fashion as those for pharmaceutical products and medical devices, although the consultation may be more detailed for regenerative medicine products. It is possible for regenerative medicine products comprised of heterogeneous components to receive conditional approval with a limited clinical data set, as long as the data demonstrate a safety profile of the new product consistent with the severity of the disease or condition being treated along with efficacy data predicting probable patient benefit. Probable patient benefit may be demonstrated with data sets from exploratory clinical trials and may be based on predictive surrogate endpoints. In practice, a conditional approval with a limited data set will be granted after consultation with the PMDA to discuss the design of the trial and the data set to be considered for getting conditional approval. More than one consultation from early stages of development will likely be needed to achieve agreement on the study design. Once data generation is complete, a Japan New Drug Application (JNDA) is prepared and submitted and subjected to a review process similar a review for pharmaceutical products (Figure 1).5 The JNDA is first reviewed by the PMDA and a report is written which may recommend for or against approval of the product. The report may set out conditions for

I think your analysis of this test is a bit ambiguous as the text itself Is. “ receive conditional approval with limited clinical set, as long as the data demonstrates a safety profile of the new product consistent with the severity of the disease or condition being treated along with efficacy data predicting probability patient benefit.“ “ in practice, a conditional approval with a limited data set will be granted after consultation with the PMDA to discuss the design of the trial and the data set to be considered for getting conditional approval. More than one consultation from early stages of development will likely be needed to achieve agreement on the study design. “

So it seems the focus is conditional approval, as we have discussed above the seven year Period, clearly they emphasize limited clinical data, but are emphatic about the clinical dabta demonstrating a safety profile, at bare minimum this requires a small size phase 0 and maybe even phase 1, and even a phase 2 ( all three of these phases are primarily for safety a phase 2 is when efficacy begins to actually become a priority, naturally any kind of beneficial outcome no matter what phase will be recorded but a phase 0 end phase 1 are focussed on gathering safety data, phase 2 is for safety and efficacy ) not to mention the animal research before ( which we know for this specific work is concluded, unless their results do not replicate within humans.) The safety data needs to be consistent with the severity of the disease/ condition Sakigake designation is for terminal conditions as described in your source and one of mine as well, within this designation very little clinical data is required for obvious reasons. With a very superficial, cosmetic condition safety is naturally going to be of far more concerned, ergo needing more safety data. Also it clearly states the efficacy data predicting the probability of benefit is taken into account as well so unless you have stellar results in a very limited trial with not any adverse affects in human patients and a very high probability of benefit by this description, you’re going to need to access a larger population size, double blinded placebo in order to get the safety and/or efficacy data a.k.a. phase 2.

- Once data generation is complete, a Japan New Drug Application (JNDA) is prepared and submitted and subjected to a review process similar a review for pharmaceutical products (Figure 1).5 The JNDA is first reviewed by the PMDA and a report is written which may recommend for or against approval of the product. The report may set out conditions for approval. The report is then submitted to the MHLW for consideration. An advisory committee may be engaged to assist MHLW in its deliberations. Aconditional approval will be time-bounded and include requirements for further studying the safety and efficacy of the regenerative medical product during the conditional approval period. The conditional approval period may be as long as seven years or fewer, as determined at the discretion of the Japanese regulators. At the end of the conditional approval period, a re-application must be submitted to continue marketing the regenerative medical product and the PMDA/MHLW review and decision process will be repeated. The regenerative medical product's marketing authorization may be revoked if the safety and efficacy of the product has not been corroborated.

So the initial review process is similar to pharmaceuticals, and then it has to undergo its own share of bureaucratic stipulation going through two different regulatory boards, who can derail the entire process by requiring an advisory commission. Who are both fully capable of outright denying the approval, or designing strict stipulations. Either way according to the text just as my sources said as well during conditional approval further studying the safety and efficacy of the regenerative product is obligated to continue, ergo people are now able to buy the product within the Japanese market but clinical trials and data gathering do not stop, nor do their expenses.

We have analyzed your source now let’s take a look at what I provided from a different source summarizing this legislation:

1. New regulations accelerating the approval of regenerative therapeutics in Japan took effect November 25, 2014. The significance of these regulations is that they allow companies to receive conditional marketing approval and commercialize regenerative medicine products while clinical trials continue through the later stages.
2. Similarly, the PMD Act introduces a specific regulatory framework for regenerative medicine products. Under the PMD Act, conditional and time-limited marketing approval can be given to a regenerative medicine product after exploratory clinical trials have demonstrated probable benefit and proven safety. According to these new laws, once a company has demonstrated safety and basic efficiency data in humans and has the cell product manufactured to the standards described within the Pharmaceutical and Medical Devices (PMD) Act, the cell therapy can be given conditional approval for up to seven years. This allows for commercial use with data reporting requirements and potential for national insurance coverage.
3. The intent of the laws is to accelerate the commercialization of cell therapeutics within Japan by allowing companies to benefit from conditional marketing authorization. Therefore, cell therapies that show safety and probable efficacy during Phase I and Phase II trials can get conditional approval for up to seven years, during which time:

1) Larger-scale, later-stage clinical trials are performed
2)Revenue from the cell therapy is pursued within the Japanese market

During the seven-year conditional approval period, companies must continue to submit clinical trial data to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), and subsequently apply for final marketing approval or withdraw the product within seven years.
https://bioinformant.com/japan-accelerated-approvals-of-cell-therapies/

Literally summarizes everything in the source you provided, it literally reiterates everything. At one point it states how cell therapies will show safety and probable efficacy data through phase 1 and phase 2 trials, as Long as it is not within the Sakigake designation looking to treat terminal conditions with no affective treatment currently, this is a fair statement, it is almost guaranteed a cellular-based product outside of that designation Will require a phase 1 human trial, depending on how well that trial goes they may or may not have to gather more data potentially requiring more patients and different methodology: double blinded-placebo for example, a.k.a. a phase 2… This will be at the discretion of the PMDA and MHLW ( also potential he at the discretion of their advisory committee if they request). This is a multifaceted decision making process, analyzing each individual condition, regenerative product, team of researchers, pharmaceutical company backing, and the data that is gathered and presented of course. If you think that both regulatory organizations will never ask for two phases of clinical data and analysis you are delusional, regenerative research especially in regards to genetic therapies are a new frontier of medicine..

 

[Dogma]

read this and then look at the graphic on page 14, that should disprove this nonsense

Oh how cute a diagram You are aware of that page 14 is after the introducion of the third aspect of this new regulation in Japan the Sakigake Designation ( The diagram on page 3 comparing regenerative and pharmaceutical treatments is more appropriate for the more common regenerative therapy is under the new regulations especially PMD) , Under this designation if the criteria is met this is when regenerative research can be extremely expedited ( they provide an example at the end of your source, the only actual example with any specificity, and it’s a very extreme condition I will refer to later), but for good reasons. Let’s review some of the specifics:

The criteria for SAKIGAKE designation are:

• Innovative product for a disease in dire need of innovative therapy; can include new indications, new target diseases, new drug delivery systems which are expected to greatly improve the efficacy.

• Prominent effectiveness (i.e., radical improvement compared to existing therapy) can be expected based on the mechanism of action from non-clinical study and early phase clinical trials.

• Intended to treat a serious disease with significant impact on life or a disease with chronic debilitating conditions.

• Addresses an unmet medical need for the target disease.

• There is no approved drug(s) for the condition or the new product contributing to a significant improvement of efficacy/safety compared to drug(s) already approved for the condition.

• There is intent for the product to be introduced in the Japanese market first or at least in parallel with release in other markets. It is to be a therapeutic agent for which domestic development is progressing steadily or an agent fulfilling either or both of the following conditions: First-in-human (FIH) study was conducted in Japan. Proof of Concept (POC) study was conducted in Japan.

Requirements for submitting a SAKIGAKE designation request are designed to be efficient. Specific forms and instructions are provided in a notification by the MHLW. The forms are slightly different depending on whether the product is a medical device, an in vitro diagnostic or a regenerative medicine product. For example, for a regenerative medicine product, Form 1-3, limited to one page, collects information about the sponsor and the product and calls for justifications that the product is innovative and addresses an unmet medical need for a life threatening, serious disease (Table 2). It is also necessary to provide justification of clinical evidence regarding the effectiveness of the product and also to confirm the product will be developed in Japan ahead of the rest of the world. Preferred dates for a hearing with the PMDA are also provided along with contact information. There is an attachment in which further detail can be provided for each of the requirements (Table 3).

Once a SAKIGAKE designation is awarded, PMDA will assign a concierge, who will organize a preliminary meeting and work with the sponsor to interface with PMDA departments as necessary and to help the sponsor negotiate a pathway to approval in an expedited and efficient fashion.

Speed and efficiency: The concept behind the conditional approval pathway is that a product can be approved based on data confirming safety and predict efficacy and the pivotal confirmatory trial(s) are allowed to occur after the product is marketed and made available to patients in Japan. This is shown graphically in Figure 2 ( Page 14’s diagram that you are relying on) Via the conventional approach, one or more pivotal registration studies were needed before making an application for marketing authorization. In the new approach, a conditional marketing authorization may be granted based on a single, small-scale trial. Once more, the sponsor can market the product while performing one or more confirmatory studies. The duration of time between initial conditional approval and re- application can be as long as seven years, although this period may be shorter as determined by Japanese regulators.

Example of very small study granting conditional approval: The primary data set for conditional approval of HeartSheet® was from a Japanese clinical trial in which seven patients with severe heart failure due to ischemia were transplanted with the product. In addition, supportive data was provided for four patients with end-stage dilated cardiomyopathy, seven patients with dilated cardiomyopathy and eight patients with ischemic cardiomyopathy. Thus, this conditional approval for heart failure due to ischemia was granted on a very modest size trial (seven patients) plus a modicum of supportive data in related indications (19 patients).

- These conditions obviously meet the criteria for Sakigake, life-threatening, no effective treatment for any kind of heart muscle damage, 100% Japanese research and clinical trial. So cannot be compared to a less severe condition being clinically studied for treatment under the new regulations, especially when speaking in reference to cosmetics.

• what my source says: The Japanese want to be the regenerative medicine center of the world,” said Colin Lee Novick, managing director of the CJ Partners biotech consulting firm in Tokyo. “To be able to do that, they need to entice companies to come to Japan, and they need to entice their own pharmaceutical companies to license in and obtain the best.”
• The “regen” boom in Japan is based on a series of reforms and new laws for pharmaceuticals, but especially the landmark Pharmaceuticals and Medical Devices Act (PMD Act) and the Act on the Safety of Regenerative Medicine. The result is the ability to offer conditional marketing approval for a regen so it can move more quickly to commercialization – an approval process widely seen as the fastest in the world. Regens also benefit from what is known as the Sakigake Package of policy changes, which speed up the introduction of innovative medical products. The package focuses on everything from basic research to clinical research/trials, approval reviews, safety measures, insurance coverage, improvement of infrastructure and the environment for corporate activities, and global expansion. It includes a designation system that promotes R&D in Japan aimed at early practical application for innovative pharmaceutical products, medical devices, and regenerative medicines. It also includes a scheme for rapid authorization of unapproved drugs, which speeds the practical application of unapproved/off-label use of drugs for serious and life-threatening diseases. Source: https://hbr.org/sponsored/2018/02/h...ities-in-the-field-of-regenerative-medicine-2

In summary this is a very intricate matter yes without question some regenerative treatments will get conditional approval based on very little data only a phase 0, not even a phase 1, these will almost always be within the Sakigake indication, others however at the behest of the regulatory boards may require a phase 1 And even a phase 2 or at least part of a phase 2. And no matter what is all of the sources both his and my own have made clear conditional approval’s can be cancelled at any point, require consistent updates, and larger clinical trials are to continue even if Conditional approval is granted.

 

[Dogma]

- non-clinical research is relatively the same outside of human patients, although the first act of the new regulation helps expedite this process.

- phase I-II clinical trials vs small scale clinical trial to confirm safety and probability of efficacy. If the titles at the top of the diagram didn’t give it away this only pertains to certain regenerative therapy’s, regardless if all of these pages haven’t made one thing clear the length of this “small scale clinical trial“ fluctuates and the decision-making behind its design based on the regulatory board is multifaceted, as one of my sources pointed out it could be no different then a phase 1 and/or phase 1 and a phase 2 ( or at least part of a phase 2) it just depends how the research goes over, any adverse affects, amazing results, ineffective, what kind of condition, what the product is, the data presented etc.

- phase III vs conditional marketing authorization + approval of conditional release and continuing further evaluation of safety and efficacy + re-application if necessary. ( this is where I think Japan’s new regulations and separating pharmaceutical from regenerative therapy’s is genius, A phase 3 can take five years to decades, but my concerns in regards to dr. Tsuji’s work being widely available by 2020 have no relevance at this point, because I know for a fact that it is at this point in the process a certainty that the new regulations will benefit and hasten the arrival of his work likely avoiding a massive phase 3 in any sense compared to what the FDA would require).

- Market release, obviously pharmaceuticals are easier to manufacture than cellular products for now but technology is developing rapidly. And administration of pharmaceuticals is more simplistic but tends to be quotidian.

 

[Dogma]

also just a side note, everyone can take sh*t off of google and paste it in here. your "knowledge" about phase 3 for example is extremely rudimentary, it's highly dependent on what kind of product you are talking about, procedures and devices are being treated differently than drugs. and even with drugs, for example cassiopeia plans to only do a 6 month phase 6 trial

Yeah but I thought I made it blatantly obvious where the information was coming from… You know by me providing the source and quoting it directly. in terms of it being rudimentary, no not at all not to most people, but despite that Depressing reality I do agree with you in fact I believe it to be so self-explanatory that any kind of product going through clinical trial depends on a multitude of factors such as the condition, the researchers, the people that happen to be the regulatory commission and their biases, the kind of drug or cell, The proceeds of adverse affects and their severity, etc. etc. that part is rudimentary to say the least. But I’ll tell you right now, go on a stroll and ask the average individual how many phases A new synthetic drug has to pass through I would be shocked if the majority knew the answer, now asked them to give a quick laconic explanation of each phase maybe one out of 10 people, I myself would have some trouble to be absolutely confident in my summarization.

 

[Dogma]

Here The understanding of Lee Buckler:
http://www.cj-partners.com/en/news/files/000025_en_01.pdf

I agree with his assessment assessment, japan’s regulatory change in regards to cellular therapy and gene therapy is, well could be groundbreaking! There’s no reason that pharmaceuticals and stem cell treatments let alone a gene therapy should be under the same regulatory commission, this chains only took place in 2014 but it is groundbreaking because it’s the first developed country to create its own program for regulating regenerative therapies which are the next generation of medical innovation! The Japanese intelligentsia and their prime minister are focussed on making Japan the bastion of cellular-based medical Technology. Their new regulations, especially as they encounter hurdles and develop and upgrade will likely become a paragon for regulating regenerative therapies that other developed countries replicate. Go through all of the tax, within the laws you can see how focusEd The Japanese are on making sure Japan leads the way in cellular innovation, Some of their stipulations require all of the research and clinical trials to be conducted within Japan with Japanese doctors, scientists, cellular biologist etc. However there are a few doctors who express concern about them going into the ‘unknown waters’ under expedited regulation ( prime example was the invention of antibiotics, penicillin, it was exalted as one of the greatest medical innovations ever at the time, Ian Fleming was a genius, now as we look at methicillin resistant staphylococcus aureus, vancomycin resistant enterococcus, and other super bugs some Infectious disease doctors have a grim view of the future).

However as it stands right now I have nothing but good things to say about what Japan is doing, and it will greatly expedite many cellular therapies including Dr. Tsuji’s and others however I am beyond confident that the vast vast majority of people that suffer from androgenic alopecia will not receive his treatment in 2020, members with A relatively comfortable net worth of $1 million USD, or are Japanese citizens May be lucky enough to benefit from the conditional marketing provided there are no adverse effects in the early patients treated in the initial phase 0 and the probability of efficacy is undeniable, making the PMDA and the MHLW Sign off on a conditional market release in 2020 for the lucky few, Will they continue to conduct larger clinical trials to eventually get full market approval. However given that they are just beginning a phase 1/0 depending on how many people, duration of observation, etc. in 2019 no real specific date given yet March was alluded to, I guarantee full market release is out of the question even for the Japanese market in 2020 or in Korea, China, etc. end it is possible they do not even get approved for an early conditional market release before 2020 is over. Hopefully I am wrong, but in the interview this website conducted with Dr. Tsuji , He and his team expected to start human trials in 2018, go look at the interview. And there are many threads on here about how they ran into a “hurdle“ with their initial research now hopefully they have overcome it. Time will tell.

I also vehemently believe Sakigake is not only practical but morally right, I for one would have signed off on the potential of death at the onset of my spinal injury to receive state of the art stem cells in early clinical research, hands down, all catastrophic disabilities and terminal illness should have that right, I mean many of us are going to die, many with catastrophic disabilities wish they would die even to see minor sensory or motor function returns can be life-changing in such a catastrophic situation. And on the more sombre side of things, you are going to sort out the duds, for example with Cellular therapies targeting cardiomyopathy if it ends up causing the specialized muscle tissue for constant contractions to deteriorate even farther or to lose its specialization within its cells then the people that weren’t going to live very long die and researchers discover a fruitless endeavor. Plus Sakigake Has a condition where if it makes the patient worse they may be entitled to some sort of reimbursement, most likely for the family… I know it’s grim but it is what it is. Either way long story short I agree with the man’s assessment

 

[MrV88]

"Japanese market full commercialization" just means you have to go to Japan, because it ain't available somewhere else nothing more...wow this forum could really make sane people completely insane

 

[RolfLeeBuckler]

"Japanese market full commercialization" just means you have to go to Japan, because it ain't available somewhere else nothing more...wow this forum could really make sane people completely insane

What do You think about The 7 year Limited phase (conditional Approval) in which Tsuji will Treat 10.000 People each year? Will it be possible for foreigners go to Japan and get it done?

 

[MrV88]

What do You think about The 7 year Limited phase (conditional Approval) in which Tsuji will Treat 10.000 People each year? Will it be possible for foreigners go to Japan and get it done?

Everybody talks about the 10.000, but it ain't sure or real at the moment, so don't care about that number and could expand to 7 years...that means they can progress for 7 years, but the don't have to, so if they're done and everything is just like they imagined/wanted they could complete it in several months to one year (I'm only guessing), but don't forget that Kyocera is developing machines for an automated process.

 

[RolfLeeBuckler]

Everybody talks about the 10.000, but it ain't sure or real at the moment, so don't care about that number and could expand to 7 years...that means they can progress for 7 years, but the don't have to, so if they're done and everything is just like they imagined/wanted they could complete it in several months to one year (I'm only guessing), but don't forget that Kyocera is developing machines for an automated process.

So what do You think about foreigners who Wants to get the Treatment in The conditional Approval Phase?

 

[MrV88]

So what do You think about foreigners who Wants to get the Treatment in The conditional Approval Phase?

Think about it like Tesla, the product is new and they have problems to scale the whole thing up, so if you want thousand of people, but can't due to the new product you can't treat more than a specific number of people. This will rise in time and especially when the process is automated, so the number of 100 or 10.000 is just pure bullshit, totally depends what they are going to achieve and how fast they will scale this up, same for the price. Will be high at the beginning and drop later due to the same reasons. Just as I said, it will be only available in Japan, but nobody said anything about Japanese citizen especially after reading the last comments. The other limitations will provide enough problems, so don't think about some crap that some guys quote from all over the internet, keep it basic and logical, think like people that want to earn money and don't have a super secret F22 program

 

[bboy]

Dogma's problem here is he keeps comparing the new regulation to the traditional 3 stage trial. So when it's said "safety and likely efficiency" needs to be proved he smuggles in a (aka phase I and II). But that's not what the regulations say, and it looks like that's not how it's been done.

What appears to happen is that you meet with the regulatory body and they tell you what kind of data they need to give you conditional approval, and you go about designing your trail around this, I suppose this could take 2 (or theoretically any amount) phases, but it's nowhere stipulated that it should. And obviously it looks at though they are more preferential to 1, as would make sense with the acceleration spirit of the regulation.

Then after you've finished your trial you meet with the regulatory body again and they look at your data at decided whether or not to give you conditional approval.

Then you can go out and market it, but you have to submit data showing that your tech actually does work within a given time frame or they pull your approval. Obviously this is so you can't just go on selling a product that doesn't work with the stamp of medical legitimacy. That time frame will obviously be on how long they think is reasonable to collect enough data to make a statistically sound judgement.

All Dogma's talk about this will take 2 trials and will require more safety data because it's a cosmetic condition is pure speculation that seems to based on little more than pessimism. How long it will take and what kind of data they will requite for safety will obviously be worked out by the board up on application. You could as easily argue that this procedure is super low risk (I don't know this) so will require very little proof of safety and almost guaranteed to work, so pretty much a slam dunk in terms of proving basic efficiency, so it could all be done with one tiny trial.

And also seeing as Tsuji et al having be saying in public 1 year for clinical trials, and they obviously have the best idea about any of these questions than anyone else, it's a good to fair bet that it will in-fact be 1 year.

It does look like, if the most recent statement is to be believed, that the timeline has possibly slipped by about a year though.

I just want to see the results of the human study though, all of this other stuff is just academic until then.

 

[byebyehair]

Dogma's problem here is he keeps comparing the new regulation to the traditional 3 stage trial. So when it's said "safety and likely efficiency" needs to be proved he smuggles in a (aka phase I and II). But that's not what the regulations say, and it looks like that's not how it's been done.

What appears to happen is that you meet with the regulatory body and they tell you what kind of data they need to give you conditional approval, and you go about designing your trail around this, I suppose this could take 2 (or theoretically any amount) phases, but it's nowhere stipulated that it should. And obviously it looks at though they are more preferential to 1, as would make sense with the acceleration spirit of the regulation.

Then after you've finished your trial you meet with the regulatory body again and they look at your data at decided whether or not to give you conditional approval.

Then you can go out and market it, but you have to submit data showing that your tech actually does work within a given time frame or they pull your approval. Obviously this is so you can't just go on selling a product that doesn't work with the stamp of medical legitimacy. That time frame will obviously be on how long they think is reasonable to collect enough data to make a statistically sound judgement.

All Dogma's talk about this will take 2 trials and will require more safety data because it's a cosmetic condition is pure speculation that seems to based on little more than pessimism. How long it will take and what kind of data they will requite for safety will obviously be worked out by the board up on application. You could as easily argue that this procedure is super low risk (I don't know this) so will require very little proof of safety and almost guaranteed to work, so pretty much a slam dunk in terms of proving basic efficiency, so it could all be done with one tiny trial.

And also seeing as Tsuji et al having be saying in public 1 year for clinical trials, and they obviously have the best idea about any of these questions than anyone else, it's a good to fair bet that it will in-fact be 1 year.

It does look like, if the most recent statement is to be believed, that the timeline has possibly slipped by about a year though.

I just want to see the results of the human study though, all of this other stuff is just academic until then.

I remember I read a classification list for stem cell treatments. Unfortunatly I cant remember if the classifiacations where made for the new regulation pathways in japan or if i found them in a different context.

There are 3 classes of stem cell treatments from 1 - 3 is from low risk to high risk.
-A non autologe treatment is allways a pretty high risk. (which tsuji is not).
-Another fact that increases the risk is if the cells are manipulated. (The setting of the hair diameter is not manipulating just sizing of the germ. But there are rumors that tsuji could give you your desired hair colour which could be done by manipulating.)
-The third fact was the potency of the used cells. (the cells are classifide as medium risk.)

Tsuji is class 2