Sunday, 2 November 2014

Fingers crossed for PPMS

The Primary Progressive Trial with Gilenya is over end of 2014, when will the results be announced to the stock market?..........academics will have to wait until next year to see the data no doubt.

Is it going to be good news and a game changer or more bad news for PPMSers. Fingers crossed that 2014 can draw to a close on a good note.

11 comments:

  1. http://www.novartis.com/investors/share-data-analysis/index.shtml

    just wait and see...
    J

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  2. It'e being compared to placebo. MDR,why do they do that? Why not compare to another drug? Surely,even a rubbish drug-which they all seem to be-would be a better choice then a sugar pill??

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    1. You are so correct and the FDA and EMA have to wake up

      Why do pharma compare drugs to placebo...its because the regulators let them get away with that and sometimes the people that advice the regulators want this.... Scandalous

      As there are no treatments for pPMS, they want to know if the drug works and it is a case of putting your best shot against nothing.

      However this approach is still being used in RRMS and it is unbelievable in this day and age that the regulators are letting pharma get away with this. (a) They test drug against placebo. In one trial that is ongoing 600 MSers are being asked to take nothing. Many of them will accumulate disability..scandelous

      Pharma move their trials further eastwards so they can find people without access to drugs so they justify in their minds that it is ethical..it isn't. It is about time that ethical review panels put a stop to this terrible practise.

      (b) Pharma test against the weakly effective drug so the beta interferon trial usually. (c) Rarely do the head to head with a highly effective drug. This should be a non-inferiority trial, where the aimis to shown my drug is no worse that the current best practise. So every one gets treated with an active drug and not a sugar pill :-)

      I will of course get chastised if we ever do a placebo controlled trial, Proximus is such a trial but there are no available alternatives at present, our symptom control trial is of a short duration and we are aiming for drug naive people who have the condition of interest but have not started taking drugs because of the side effects of the alternatives.

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    2. Yes, non-inferiority trial seems with naked eyes a better approach. However the large sample size required for claiming non-inferiority of the new drug compared with the current best practice in such a trial exposes many patients to a new drug that its safety and efficacy has yet to be tested. Sometime we need to choose between two evils: exposing reasonable number of patients to placebo or a large number of patients to a new drug which might turn out to be less safe than a placebo...

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    3. Spoken like a true pharmaceutical person:-). The people are going to be exposed to the test drug so if this is less safe than placebo it will happen the difference is whether you give people a drug that you know works or you given them nothing.

      Can you supply numbers to show that the trials have to be so huge for a non-inferitority using a drug that has high efficacy i.e. not beta interferons or copaxone. I have seen some calculations and I have seen studies powered and sizes are not necessarily as big as you may think and certainly less than......having 600 people on placebo which seems to me a pretty huge number and indeed bigger than most DMT trials.

      For phase III the safety profile at phase II should be known

      Sounds like a pole from ProfG is warranted.

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    4. Using a placebo in this trial is a moot point. There are no treatments for PPMS, so it doesn't matter. If however, they use a RRMS drug it could prove to be that the RRMS drug (even Interferons or GA) are more effective than Gilenya since I assume there are new endpoints for this progressive trial. In essence all of the previous PPMS trials are crap so you have to start from scratch.

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    5. A placebo control for a PPMS trial is absolutely warranted as we need to prove that the treatment works. For an RRMS trial it is different as we know some of the drugs are active. We know that interferons and GA are not active compared to placebo, unless people are relapsing progressive (gadolinium lesions).

      Based on MS history trial design evolves over time and eventually they will find a winning formula. I guess we will soon find out if it gilenya that is a game changer.

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    6. If you are a "have" would you risk taking nothing on the off chance that the grass is greener with the new test drug....I suspect not.

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  3. Axiously awaiting ProfG to voice his opinion...Primun non nocera is indeed the base of medical practice but often choosing between two evils is the only way to go...

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    1. Maybe he is too conflicted or already knows the answer?

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