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  • Comment To The FDA Regarding The Lemtrada Appeal 76 comments
    Jan 7, 2014 11:36 AM | about stocks: SNY

    A citizen petition has been filed with the FDA regarding the application for the the new MS therapy Lemtrada. If you would like to comment on this matter, you can do so here. Before you submit your views, please read these tips for submitting effective comments.

    What comments would be especially helpful and relevant? So far, the process has been run by regulators whose specialties are far afield from the treatment of MS. To fill this hole, neurologists, especially those treating MS patients, should consider commenting. MS patients should also make sure that their voices are heard.

    Why now? The comment period began after the FDA's complete response letter (CRL) was issued on December 27th, 2013. There are two reasons. First, it is important that the record indicates the views of those most impacted by MS so that these views are taken into account in the future.

    Secondly, there is an appeal process. Where does the appeal stand? So far, the Lemtrada decision has been delegated to the Division director, who is an implacable foe of this drug and its sponsors. The Director of the Center for Drug Evaluation and Research, Dr. Janet Woodcock, will weigh in on the appeal. So, any views submitted in reaction to the Citizen Petition could make a difference when this appeal is taken up at the higher level within the FDA.

    Themes: Lemtrada, alemtuzumab, GCVRZ Stocks: SNY
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Comments (76)
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  • US Investor
    , contributor
    Comments (42) | Send Message
     
    Thanks Chris for initiating this. The MS patients voice matters.
    7 Jan, 12:29 PM Reply Like
  • TimeOnTarget
    , contributor
    Comments (2175) | Send Message
     
    Regarding writing persuasive comments, I have been on both sides of this particular exercise (although not with the FDA), so I thought I might share a couple of general thoughts in the hopes they are helpful.

     

    i) Bear in mind you are trying to persuade the people that will be reading the comments, not others who think like you do: Don't treat your comments like you would commenting on a Blog;

     

    ii) Things like insults, sarcasm, snide tone, etc. will not be persuasive and will likely have an effect opposite of what is intended;

     

    iii) Understatement is usually better than overstatement; and

     

    iv) Specificity regarding which part of the decisions you take exception to is very useful: Specific, well-considered criticisms will be most effective.

     

    And a couple of personal thoughts on comments on this matter in particular.

     

    People who are personally affected by MS should certainly convey that point. If you are frustrated with the FDA not allowing a legitimate treatment, that is certainly one point to make.

     

    Discuss your view of side effects and the FDA's apparent concern over them.

     

    For example, something along the lines of " Side effects can be considered and a cost/benefit analysis done on a case by case basis between patient and doctor: Simply not having the treatment available when it has demonstrated efficacy in some cases based on a concern over side effects indicates FDA's priorities are not weighted appropriately and that it fails to adequately consider the persons most concerned: Those affected by MS."

     

    I would think it might also be appropriate in this case to express frustration at the FDA's lack of responsiveness to patient concerns and note the approval in other countries and for other indications in this country. Given those things, fail to see what compelling interest the FDA has in withholding this treatment from MS patients.

     

    I think lack of adequate concern regarding those most impacted and the lack of input those persons have in the process may be effective points to make.

     

    The FDA is a creature of statute: It's role can be changed by Congress changing its enabling statutes. It can be effective to make that point, but it is probably better to do it somewhat indirectly.

     

    It would probably not be effective to say "I'll be asking my congressman to give the FDA a haircut it won't soon forget."

     

    It might be more effective to say something along the lines of "It is difficult to express the frustration I feel over a decision not to allow a treatment that is being utilized in other countries and for other purposes in this country, especially when that decision not only does not allow those who are most affected any meaningful input, but also has no meaningful appeal or review."

     

    In my experience, politicians are very responsive to concerns about lack of public input and lack of government responsiveness. Agencies are responsive to politicians. But it is probably more effective to let them reach the conclusion that they are going to have very unhappy people writing their representatives if they don't do something than it would be to say it directly.

     

    The "example" type comments I give above are rough--written quickly-- should be revised based on personal thoughts, experiences, and beliefs. Polite, sincere, and determined is usually a good tone.

     

    For what it is worth.
    7 Jan, 01:44 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » Excellent points. I certainly hope that you will be one of the people that the FDA will be hearing from.
    7 Jan, 01:47 PM Reply Like
  • TimeOnTarget
    , contributor
    Comments (2175) | Send Message
     
    I will be.
    7 Jan, 02:37 PM Reply Like
  • TimeOnTarget
    , contributor
    Comments (2175) | Send Message
     
    I forget where I originally found this (it might have been a link you provided Chris), but I liked it as an overview and thought it might be useful to others.

     

    "How the FDA approves drugs and regulates their safety and effectiveness."

     

    http://bit.ly/16ExMBy
    7 Jan, 02:50 PM Reply Like
  • Christopher Schons
    , contributor
    Comments (26) | Send Message
     
    Thank you for this, Chris.

     

    --Chris Schons
    7 Jan, 05:18 PM Reply Like
  • TimeOnTarget
    , contributor
    Comments (2175) | Send Message
     
    @Chris Schons --

     

    My pleasure.
    7 Jan, 05:29 PM Reply Like
  • Dean Plassaras
    , contributor
    Comments (102) | Send Message
     
    Would her planned retirement affect the appeal process?

     

    http://bit.ly/1ddUL8z
    7 Jan, 06:33 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » My understanding is that contrary to the press reports she is not planning on leaving. In any event, she plans on seeing this appeal while she is in office.
    7 Jan, 06:37 PM Reply Like
  • Dean Plassaras
    , contributor
    Comments (102) | Send Message
     
    O.k.

     

    Are you aware of this document? I am assuming you have seen it.

     

    http://1.usa.gov/KwuZzZ
    7 Jan, 06:45 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » Yes; thanks.
    7 Jan, 06:55 PM Reply Like
  • nmecmbtnt
    , contributor
    Comments (122) | Send Message
     
    Thank you for launching this.One area thatI thinkthat has not beenstated perse--is the potential value gainedfrom a largerbody of patientsfor the treatment.Surely this couldbe misstated& misconstrued.But the point is not to suggest some sortof post-approval"experime...

     

    It isthis—a larger andmore diverse doctor& patient population usingthe protocol could foster:

     

    1) Saferexperience for worldwidepopulation using thetreatment
    2) Deeperinsight into howtreatment alters thedisease processes
    3) Wealth ofexperience with compoundand treatment, perhaps exploitable elsewhere

     

    There are certainlyserious potential downsides.However the perilsof ignoring avery promising treatment--despite the risksis shortsighted.

     

    (Case in point:the U.S. hasbeen left outof the racein stem cellresearch and manypotential benefits. Forthe singular reasonthat a small,but vocal minoritystifled all workusing anon-scientific thesis.) Normal 0 false false false EN-US X-NONE X-NONE /* Style Definitions */ table.MsoNormalTable {mso-style-name:"Table Normal"; mso-tstyle-rowband-siz... mso-tstyle-colband-size:0; mso-style-noshow:yes; mso-style-priority:99; mso-style-parent:""; mso-padding-alt:0in 5.4pt 0in 5.4pt; mso-para-margin-top:0in; mso-para-margin-right:... mso-para-margin-bottom:10.0pt; mso-para-margin-left:0in; line-height:115%; mso-pagination:widow-o... font-size:11.0pt; font-family:"Calibri",... mso-ascii-font-family:Calibri; mso-ascii-theme-font:m... mso-hansi-font-family:Calibri; mso-hansi-theme-font:m...
    7 Jan, 06:45 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » Formatting problems but good points. I hope that you submit your comment to the FDA.
    7 Jan, 06:56 PM Reply Like
  • nmecmbtnt
    , contributor
    Comments (122) | Send Message
     
    Thanks. Will do.

     

    Gah....re formatting....that's what I get for using Word and a cut/paste to the posting window. Here it is again.

     

    "Thank you for launching this. One area that I think that has not been stated per se--is the potential value gained from a larger body of patients for the treatment. Surely this could be misstated & misconstrued. But the point is not to suggest some sort of post-approval" experimentation.

     

    It is this—a larger and more diverse doctor & patient population using the protocol could foster:

     

    1) Safer experience for worldwide population using the treatment
    2) Deeper insight into how treatment alters the disease processes
    3) Wealth of experience with compound & treatment, perhaps exploitable elsewhere

     

    There are certainly serious potential downsides. However the perils of ignoring a very promising treatment--despite the risks is shortsighted.

     

    (Case in point: the U.S. has been left out of the race in stem cell research & many potential benefits. For the singular reason that a small, but vocal minority stifled all work using a non-scientific thesis.)
    8 Jan, 06:09 PM Reply Like
  • nmecmbtnt
    , contributor
    Comments (122) | Send Message
     
    Cleaned up, word-smithed & done. FDA comment receipt: 1jy-89r7-puog

     

    Here's to hoping reason prevails.
    8 Jan, 06:10 PM Reply Like
  • John DiStanislao
    , contributor
    Comments (121) | Send Message
     
    Chris Thanks!

     

    My life has been *&^&& by MS. FDA got Tysabri wrong, twice. Most doctors lack sound judgment, initiative and the desire to find best solutions if extra effort is involved.

     

    Life could have been very different for many if this drug was available or possibly Tysabri 7 or 8 years ago. For many of us but clearly not all living with MS is just not living.

     

    Now thanks to your work I can submit my comments to the FDA. Thanks John
    8 Jan, 12:39 AM Reply Like
  • TimeOnTarget
    , contributor
    Comments (2175) | Send Message
     
    @John --

     

    I have a feeling that your comments will be just exactly the kind that are quite effective and hard for anyone to not consider very carefully and weigh very heavily -- that is anyone who is not just zealous only about being a pernicious bureaucrat.
    8 Jan, 12:53 AM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » Please do submit your views. People such as you should drive these decisions.
    8 Jan, 07:55 AM Reply Like
  • PICOLOMINI
    , contributor
    Comments (545) | Send Message
     
    Please tell me that I am misreading =
    ------Where does the appeal stand? So far, the Lemtrada decision has been delegated to the Division director, who is an implacable foe of this drug and its sponsors. The Director of the Center for Drug Evaluation and Research, Dr. Janet Woodcock, will weigh in on the appeal.---------------...

     

    Are you telling the MS world that the verdict on Lemtrada was handled on Division level only?
    Are you telling the MS world that this Division is run by an implacable foe of this drug and its sponsors? On what grounds?
    Are you telling the MS world that only now the Director for Drug Evaluation and Research will weigh in? What does weighing in mean , power to overturn the verdict?
    Two people playing Godfather over the fate of thousands?
    No interviews with those responsible? No hearings?
    Where is the MS lobby? Why so silent?

     

    I beg to substantiate such assertions = implacable foe of Lemtrada and sponsors. The US Department of Health and Human Services owes to that much to the MS world!
    8 Jan, 02:48 AM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » The verdict on Lemtrada was handled on the Division level only.

     

    The Division is run by an implacable foe of this drug and its sponsors on the grounds that the statistics and review were distorted for the apparent reason that he disliked the trial design. Additionally, he disliked treatments that are dosed annually because that decreases the FDA's influence over the patients after they are treated.

     

    You are not misreading me and I stated my views because I thought them to be helpful and relevant. If any of this is of interest to you, I strongly suggest that you share any of your views with the FDA via the above opportunity to comment.
    8 Jan, 08:00 AM Reply Like
  • Dean Plassaras
    , contributor
    Comments (102) | Send Message
     
    Chris:

     

    This is a general question but what is the precedent of overturning FDA decisions?

     

    So say the appeal results in declaring the review harsh or capricious or disproportionate. What does this mean in terms of the next step?

     

    What could Genzyme reasonably expect to get out of this?

     

    Obviously Sanofi's strong card is Lemtrada's approval in Europe and elsewhere. But are we dealing with an FDA and EMEA hidden rivarly here? Are the 2 authorities cooperating or competing?

     

    http://bit.ly/19SRjjj
    8 Jan, 09:06 AM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » I am not familiar with a precedent for overturning an FDA decision. However, I am equally unfamiliar with a precendent for a review that has been turned into a weapon against the sponsors in the manner that this one has.

     

    We will know more about next steps in about a month.

     

    What could Genzyme reasonably expect to get out of this? Maybe better public policy. Maybe – maybe – a slight chance of a different outcome to this drug’s future.

     

    Meanwhile, what should be done? Anyone with relevant and helpful views should submit them directly to the FDA.
    8 Jan, 09:21 AM Reply Like
  • Dean Plassaras
    , contributor
    Comments (102) | Send Message
     
    I am not a physician to have relevant views on the technical aspect of the approval.

     

    From an efficiency point of view and as a citizen seeking a greater public good, I might suggest that when we have a drug approved already in 3 other major economies(countries) of the center (in this case EU, Canada and Australia) then what the FDA ought to do is to overlap the 3 approval processes and focus on work left undone rather than pretend that we must re-invent the wheel on efficacy basis. It seems to me 3 other major countries have done such already and that life science transcends national boundaries because we are all the same humans.
    8 Jan, 12:29 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » My view is that your above comment would be a worthy part of the conversation and as such should be submitted to the FDA.
    8 Jan, 12:59 PM Reply Like
  • bigred2
    , contributor
    Comments (35) | Send Message
     
    I just read on cafe pharma that sanofi is going to sell campath in the U.S .

     

    How will this affect lemtrada sales?
    8 Jan, 09:55 PM Reply Like
  • nmecmbtnt
    , contributor
    Comments (122) | Send Message
     
    If it were used to treat MS off-label--it "might" count towards the sales goals in those milestones--but this is not certain.

     

    Campath is approved for oncological and transplant treatments in the U.S. But it is clearly called out in the CVR agreement that any sales of Campath or MabCampath that are for those two treatments do _not_ count towards sales milestones.

     

    However, it also does not say that sales of Campath for use unrelated to those two conditions are not counted towards sales milestones.

     

    So your mileage may vary--and may depend on a more precise reading of the CVR agreement--namely the specifics for what _can_ be credited towards the sales milestones.

     

    Opinion: SNY will not roll over and abandon a valuable, world class pharma franchise and astounding treatment for MS.

     

    Disclosure: Long GCVRZ with 50K units @$0.32. Will hold till facts on the street cave in for a reasonable NPV or SNY triggers a buyback on a failure scenario. If it rises--will let it run.
    9 Jan, 08:16 PM Reply Like
  • Matt Young
    , contributor
    Comments (11) | Send Message
     
    I think a point that has got misinterpreted is that the failure scenario gives SNY the option- but not the obligation- to buy back all CVRs at $0.5 (or lower based on previous trading). I think some people think that this sets a floor on the CVR price (and therefore guaranteed upside) but I disagree as the value of the option is to SNY not the holder.

     

    Disclosure: 1% position long GCVRZ @ $0.63 cost.
    10 Jan, 05:56 AM Reply Like
  • samuelfhuntermdphd
    , contributor
    Comments (3) | Send Message
     
    Genzyme took the CAMPATH brand off the market in 2012 (and choose to give it away for leukemia) to allow the code to expire because the cost and price is so different in MS than oncology. So off-label prescribing is not an option currently, but the FDA would really get upset if there was major use off-label and likely fine Genzyme mercilessly.
    13 Jan, 07:21 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » Teriflunomide in Multiple Sclerosis: Added Benefit Not Proven
    http://bit.ly/1irsPh9
    10 Jan, 01:17 PM Reply Like
  • Ruerd Heeg
    , contributor
    Comments (410) | Send Message
     
    Amazing, seems that the trials for teriflunomide (aubagio) were well designed ("adequate and well controlled") but that these trials did not proof any better efficacy than Rebif. And that a comparison of the side effects did not reveal any benefits either.
    10 Jan, 03:23 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » MS patient fights FDA over rejection of Genzyme’s Lemtrada: http://bit.ly/KaOwFN
    10 Jan, 02:51 PM Reply Like
  • Ruerd Heeg
    , contributor
    Comments (410) | Send Message
     
    "If Lemtrada is not approved, Burdick says she plans to go on the third drug by Biogen for MS, the recently approved Tecfidera. She worries, however, about side effects the company lists, including stomach problems."

     

    Stomach problems are common with many oral drugs that need to be used for many years or even decades. In The Netherlands they fear for a shortage of gastro-enterologists due to increased oral drug usage.
    10 Jan, 03:05 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » "Unexpectedly and disappointingly, alemtuzumab has not been approved by the FDA. In our opinion, this decision denies people with multiple sclerosis in the United States from an important treatment option." - Alasdair Coles http://bit.ly/1ktQrpR
    10 Jan, 04:04 PM Reply Like
  • Ruerd Heeg
    , contributor
    Comments (410) | Send Message
     
    Thanks for the link. He writes he is concerned at the quality of the FDA decision making.

     

    This man is from the famous Cambridge University in the UK. He is involved in the development of alemtuzumab. I suppose that alemtuzumab will be approved in the UK. But can you imagine how much he would be disappointed if NICE would refuse to reimburse alemtuzumab in the UK? What language would he use then?

     

    See also the link below Alasdair's reaction to the FDA CRL:
    http://bit.ly/UAPoWG
    They are organizing a trial where all patients get alemtuzumab and half of the patients get Kepivance as well. They want to test whether this extra drug reduces the risks of getting other autoimmune diseases.
    10 Jan, 04:18 PM Reply Like
  • samuelfhuntermdphd
    , contributor
    Comments (3) | Send Message
     
    I am a subspecialty neurologist who uses alemtuzumab/Lemtrada/C... in the USA and very experienced with it.

     

    I would appreciate more details about the person you identify as a foe of the medication. I would like to be sure my voice is heard and that I can identify which bureaucrat is responsible for errors. I will contact other within the legislature and Federal bureaucracy to make our views known.

     

    I can also disseminate the important players to me colleagues in the field. There are literally hundreds of physicians ready to sign petitions and write letters.

     

    Meanwhile, here is another citizen petition
    http://bit.ly/JVg1Uh
    13 Jan, 11:21 AM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » Ellis F. Unger: http://1.usa.gov/JVgfe4. He is anti-Lemtrada and anti-Genzyme generally.
    13 Jan, 11:23 AM Reply Like
  • TimeOnTarget
    , contributor
    Comments (2175) | Send Message
     
    Dr. Hunter --

     

    I expect that would be extremely useful. It strikes me that sometimes the only people that have sway over doctors are other doctors.

     

    Plus, with you having actual experience with the medication, I would expect your opinion to draw very considered evaluation. I doubt that there are many doctors in the U.S. with any experience.

     

    One topic apparently important to them was apparently the lack of their standard model for a blinded trial. I understand the reason for those types of evaluations, but the fact that it is difficult to impossible to meaningfully design such a trial for a specific medication should not preclude approval of the medication. Your knowledge of side effects, etc. might be useful information to further the discussion on that point.

     

    Thanks.
    15 Jan, 10:24 AM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » These boards -- http://bit.ly/1aiZpiU -- are useful to get the pulse of the sales reps. In the case of Lemtrada, it is currently pretty glum. They do not necessarily know more than we do, but they tend to have great gossip networks.
    13 Jan, 09:16 PM Reply Like
  • nmecmbtnt
    , contributor
    Comments (122) | Send Message
     
    Ouch.... seems to be mostly sales folks sounding off. So a bipolar response seems about right with livelihoods at stake.
    16 Jan, 08:33 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » Sanofi Seeks to Reach Compromise With FDA on Lemtrada MS Drug

     

    According to Bloomberg this morning, Sanofi (SNY) will work on reaching a compromise with U.S. regulators who rejected Lemtrada last month. The company’s top research executive said that,

     

    “hopefully, the FDA will listen to our appeal, and we will come to a compromise.”
    14 Jan, 08:57 AM Reply Like
  • Dean Plassaras
    , contributor
    Comments (102) | Send Message
     
    o.k. now we are talking. It's a leadership issue not a drug issue.
    14 Jan, 09:16 AM Reply Like
  • bazooooka
    , contributor
    Comments (2323) | Send Message
     
    Chris, have you found any way to handicap the appeal process? Would a 10% chance of success seem high (or low)?

     

    Also assuming another turn down, do you expect new trials to appease the FDA or would you assume the US market is a non-starter?
    14 Jan, 06:57 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » 6% would be average. I think that this is better than average. 10% is probably reasonable.

     

    I do not believe that Sanofi should or will conduct new trials to appease the FDA. The US market is probably a non-starter.
    14 Jan, 06:59 PM Reply Like
  • Matt Young
    , contributor
    Comments (11) | Send Message
     
    Chris do you know what a 'compromise' would look like? Could it be enough to trigger the $1 milestone payment. Many thanks.
    15 Jan, 09:17 AM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » We'll know more about details by the end of the month.

     

    Yes.
    15 Jan, 09:23 AM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » My Experience on Lemtrada: http://bit.ly/1aGf8ZT
    15 Jan, 11:36 AM Reply Like
  • Ruerd Heeg
    , contributor
    Comments (410) | Send Message
     
    Thanks for the link.

     

    Apparently it can work for people in a more advanced stage of MS as well, and apparently it makes sense to give more than 2 treatments if symptoms come back after a few years.
    15 Jan, 01:07 PM Reply Like
  • John DiStanislao
    , contributor
    Comments (121) | Send Message
     
    Rued, after reading I was also surprised. Thinking the med was only for the newly diagnosed. There are plenty of JCV positive MS patients where Tysabri is no longer an option.

     

    FDA like most if not all government agencies are corrupt, ineffective, often harmful, political. Government agencies like the FDA are protected from accountability or producing cost benefit measurable results.
    15 Jan, 03:33 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » I hope that you take the opportunity to comment directly to the FDA to ensure that your view is taken into account.
    15 Jan, 03:35 PM Reply Like
  • John DiStanislao
    , contributor
    Comments (121) | Send Message
     
    Chris I will! Funny (not really) my MS fatigue has been so horrible lately. it’s difficult to write meaningful content. Updating my blogspot, sending out emails with anything meaningful has been near impossible. But too my surprise my recent DWCH article was selected as a pro article. "This Next Generation Analytics Will Disrupt The Business Intelligence Industry".

     

    Sharing my thoughts and experience makes me still feel relevant even if everything else was taken away.

     

    I don't care for hits as NO money will be taken.SSI is my destiny.

     

    Random thought, It's likely if Tysabri was available when made available in Europe, years before FDA approval (I think). It’s possible I could continue working paying close to 50% taxes for cumulative NYS, Federal, and local taxes.

     

    Thank goodness we live in such a free country with capable, caring, over worked, under paid, anemic benefits, job insecurity,but still dedicated public servants / government bureaucrats. Exactly like those government workers at the bloated FDA. /sarcasm.
    15 Jan, 08:12 PM Reply Like
  • Ruerd Heeg
    , contributor
    Comments (410) | Send Message
     
    John, as you have experienced the economic impact of MS is enormous.

     

    See also http://bit.ly/19foahy
    and http://bit.ly/KkOPxH
    16 Jan, 03:36 AM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » Did the agency wrongly emphasize unfavorable results? http://bit.ly/1d5gptf
    15 Jan, 12:00 PM Reply Like
  • nmecmbtnt
    , contributor
    Comments (122) | Send Message
     
    Only 4 -- Comments Received* on the petition to date.

     

    *This count refers to the total comments received on this docket, as of 11:59 PM yesterday, from Regulations.gov and alternate means. All comments including the bulk submissions received for this docket may not be posted at this time; therefore, the counts may differ between: total comments received and posted, as well as the counts shown on the Docket Folder Summary page.
    15 Jan, 04:49 PM Reply Like
  • John DiStanislao
    , contributor
    Comments (121) | Send Message
     
    nmecmbtnt,

     

    I will post in the link Chris provided us. I wanted to get it right. What is the dead line for comments? I could just post my heartfelt thoughts and explain how with products like software development you need to release imperfection or it will never be of any benefit and see improvements to the masses. Close doctor's care (blood, mri, etc) as they do with tysabri is the reason Tysabri patients are not having concerns with PML in USA.

     

    This is the petition location, dead line? Thanks!

     

    http://1.usa.gov/1apZdLV;D=FDA-2013-P-1637-0001
    15 Jan, 08:31 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » In practice, the deadline is right away. They will make the key decisions this month and this petition will get attention or it won't. So, if you have something to say, then please say it.
    15 Jan, 08:47 PM Reply Like
  • nmecmbtnt
    , contributor
    Comments (122) | Send Message
     
    I think the deadline is upon us. As mentioned--whether they ingest & fairly consider the input may is a question.

     

    Nevertheless, any advocacy, for so many MS patients stymied without this protocol, is imperative.

     

    Thanks!
    16 Jan, 09:18 PM Reply Like
  • John DiStanislao
    , contributor
    Comments (121) | Send Message
     
    I have something to say. Tonight it will be submitted, far from perfection but its important.

     

    I will do my best to eliminate my real thoughts on FDA incompetence and if they were competent they wouldn't be working for the government.
    15 Jan, 08:51 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » Excellent!
    15 Jan, 08:52 PM Reply Like
  • TimeOnTarget
    , contributor
    Comments (2175) | Send Message
     
    Done.
    16 Jan, 09:59 AM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » One recent submission to the FDA:

     

    The FDA’s rejection rationale for MS therapy Lemtrada deserves a through rebuttal. That rebuttal requires a deeper look at each individual responsible for the FDA decision. Then ask why Europe, Canada, Australia approved. Europe has the world’s highest prevalence per 100,000. Furthermore, medical experts using real world patient data supported Lemtrada’s approval. Lemtrada’s risk and cost compared to tangible real benefits was a factor driving approving countries.
    I was motivated to respond to the FDA even if it won’t be complete. Furthermore I have Multiple Sclerosis so for me even these small efforts to organize my thoughts can be a struggle. It’s easy to rationalize putting off my response but a challenge is needed. So having said that let me make a few comments. I hope time is available (FDA deadlines) and personal energy to submit a more complete rebuttal.
    I’m a MS patient on Tysabri. Unfortunately I remember the FDA removed Tysabri twice while patients were benefiting.
    The rationale was illogical and motivation questionable. In my opinion it was lack of initiative and patient concern that was absent to recognize what was best as an aggregate. Patients made their feelings known to the FDA. Those patients would assume potential risks. A basic freedom we often assume is real now removed by a few individuals with no transparency into their motivation.
    Tysabri was and is safe. Why because by combining close observations via frequent office visits, blood work to test for JVC virus and semiannual MRI’s has made risk of PML statistically nonexistent. This rigorous observation will be with Lemtrada. FDA used Europe’s cases with PML to immediately rationalize removing Tysabri. As you must know MS tysabri users were mixing treatments with Avonex coupled with other unrelated Tysabri factors. The initial lack of rigor with MRI, blood work can be assumed to be potential contributing factors. But at the end of the day it’s the patient’s decision with his or her medical advisors of choice. Not a hand full of federal employees. The key word is freedom of choice.
    Every advancement or life enhancing product is the direct result from freedom of choice. Its individual risk taking using their chosen “consultants” then followed by trial and error period that leads to an improved product for society. Anyone can observe this phenomenon with the internet or software that has dramatically improved our lives. At first Software changes are never perfect or even worth the effort and money. It’s without fail there will be frustration, unexpected substantial costs incurred, customers lost, and faulty conclusions drawn that negatively impact lives of other people. But this is the unavoidable process of continuous improvement as feedback from those informed risk takers are part of creating a better solution/result that benefits everyone.
    Tesla, Iphone, Kahn Academy, Microsoft, or Pharmaceuticals latter found to provide other uses. For example, Propecia originally used to treat Prostate enlargement now used for male pattern baldness. Viagra prescribed for heart disease now used for erectile dysfunction. You get the idea.
    I don’t know who or how many FDA employees have hands on MS neurological expertise were directly responsible for the rejection of Lemtrada. Those individuals are in the minority to the rest of the world that see benefits and rationale for choice.
    In conclusion we must consider human bias, massive industry pressure, political influence, lack of expertise/experience unfortunately concentrated in the hands of a few federal employees. Existing FDA MS approved treatments are concentrated in about 3 or so companies.
    Its rationale to ask do FDA decision makers have connections, ever worked with or received funding from these few MS drug companies. The FDA process is long overdue for independent auditing and creating more public transparency. Pressure not to approve could come from Biogen that in Feb 2013 paid 3.25 Billion to receive the remaining drug rights for Tysabri from Elan. Biogen also sells the FDA approved MS therapy Avonex. The life time cost using Lemtrada versus Tysabri or Avonex is not only less expensive but has potential to be a life altering drug for MS patients and society.
    Please reconsider your decision.
    Respectfully but emotionally involved
    John DiStanislao
    16 Jan, 02:37 PM Reply Like
  • US Investor
    , contributor
    Comments (42) | Send Message
     
    I dont know if this was mentioned earlier. Kudos to DeMuth Sr and Jr. for the opinion article in WSJ

     

    The FDA Nixes a Pathbreaking Drug for MS
    http://on.wsj.com/LcN46r
    16 Jan, 09:42 PM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » Status of CVR Product Sales Milestone #1: http://www.bit.ly/1hfhcci
    21 Jan, 03:47 PM Reply Like
  • Ruerd Heeg
    , contributor
    Comments (410) | Send Message
     
    From this status document one of the risks of the CVR becomes clearer. This risk is that the drug will not be reimbursed in a country yet after commercial sales have started in that country.

     

    From the document it seems that if the US approves during the fall of 2015 then that's late enough to reach the sales milestone #1.
    21 Jan, 04:16 PM Reply Like
  • TimeOnTarget
    , contributor
    Comments (2175) | Send Message
     
    Regarding the document that Chris provided a link to on the Status of CVR Product Sales Milestone #1, if that is their simplification, I'm really glad they didn't try to say something confusing.
    21 Jan, 05:57 PM Reply Like
  • TimeOnTarget
    , contributor
    Comments (2175) | Send Message
     
    Chris --

     

    Regarding GCVRZ --

     

    I got curious about this because best I can tell it is just exactly the kind of BS I hate from government agencies. Anyway, decided to look at what the substance of the appeal might shake out like. I include a couple of links and a couple of quick thoughts below. Probably not adding anything to what you already know, but I actually came away liking the appeal's chances better than before, i.e., before I drilled down on the substantive language that I thought would likely govern the decision.

     

    I expect you have already looked at this, but if not here is a link to a page from which you can easily draw up 21 CFR Section 314.126, "Adequate and well-controlled studies."

     

    http://1.usa.gov/1aNFLf6...

     

    My assumption is that the key issue will be determining whether the requirements of Section 314.126 were satisfied. I haven't done enough research to really be informed on the issues or the exact procedural state (e.g., did they request a waiver of some requirements under Section 314.126 and, if so, was it denied and, if so, on what basis, etc.) but I was more pleased with the language of 314.126 than I expected to be.

     

    There are some procedural provisions (e.g., 314.200, etc.) that bear on the determination, but the substance appears to be Section 314.126 and Section 505(d) of the Federal Food, Drug, and Cosmetics Act.

     

    http://1.usa.gov/1aNFNUi...

     

    If the issues relate solely to whether the drug is effective the provisions of Section 505(d) prescribe that criteria to be "substantial evidence" and go on to define that term in some detail. (See Section 505(d)(5) in particular and the last part of Section 505(d))

     

    The text of Section 505(d) is as follows:

     

    (d) Grounds for refusing application; approval of application; “substantial evidence” defined
    If the Secretary finds, after due notice to the applicant in accordance with subsection (c) of this section and giving him an opportunity for a hearing, in accordance with said subsection, that (1) the investigations, reports of which are required to be submitted to the Secretary pursuant to subsection (b) of this section, do not include adequate tests by all methods reasonably applicable to show whether or not such drug is safe for use under the conditions prescribed, recommended, or suggested in the proposed labeling thereof; (2) the results of such tests show that such drug is unsafe for use under such conditions or do not show that such drug is safe for use under such conditions; (3) the methods used in, and the facilities and controls used for, the manufacture, processing, and packing of such drug are inadequate to preserve its identity, strength, quality, and purity; (4) upon the basis of the information submitted to him as part of the application, or upon the basis of any other information before him with respect to such drug, he has insufficient information to determine whether such drug is safe for use under such conditions; or (5) evaluated on the basis of the information submitted to him as part of the application and any other information before him with respect to such drug, there is a lack of substantial evidence that the drug will have the effect it purports or is represented to have under the conditions of use prescribed, recommended, or suggested in the proposed labeling thereof; or (6) the application failed to contain the patent information prescribed by subsection (b) of this section; or (7) based on a fair evaluation of all material facts, such labeling is false or misleading in any particular; he shall issue an order refusing to approve the application. If, after such notice and opportunity for hearing, the Secretary finds that clauses (1) through (6) do not apply, he shall issue an order approving the application. As used in this subsection and subsection (e) of this section, the term “substantial evidence” means evidence consisting of adequate and well-controlled investigations, including clinical investigations, by experts qualified by scientific training and experience to evaluate the effectiveness of the drug involved, on the basis of which it could fairly and responsibly be concluded by such experts that the drug will have the effect it purports or is represented to have under the conditions of use prescribed, recommended, or suggested in the labeling or proposed labeling thereof. If the Secretary determines, based on relevant science, that data from one adequate and well-controlled clinical investigation and confirmatory evidence (obtained prior to or after such investigation) are sufficient to establish effectiveness, the Secretary may consider such data and evidence to constitute substantial evidence for purposes of the preceding sentence."

     

    Since the effectiveness doesn't appear particularly amenable to attack, what it should boil down to in this case is determining whether there were "adequate and well-controlled investigations." That will essentially turn on how that term is defined in their rules, i.e., in 21 CFR Section 314.126. (Another link that may be direct: http://1.usa.gov/1aNFNUk... -- I'll tack on the text at the end.)

     

    I didn't spend much time looking at it, but I wasn't real wild about some of the procedural stuff I saw, including the "separation of functions" (I think that is the term they used) stuff in particular. Basically that allows the Division to woodshed the Commissioner prior to the hearing if they agree that there should be a hearing on an issue.

     

    One strategy that the Division might use if they are really married to their position against it is recommending that they be granted a hearing on the issue of effectiveness, thereby giving them the opportunity to consult with the Commissioner or his designated officer prior to notice going out. This might play out as them communicating along the lines of something like the following:

     

    "Well, we think you should give them a hearing because it has a lot of interest and they have some evidence of effectiveness, but we don't really think they investigations were "adequate or well-controlled" for the following reasons. Now we expect them to say ____, but what that doesn't really consider is _________. "

     

    I used to do a decent amount of litigation. I would ALWAYS, and I mean always, look for chances to do one of two things: i) poison the well; and ii) inoculate the judge (or decision maker if before an administrative agency).

     

    Poison the well -- concept is pretty simple, basically get info in front of the judge with your timing and terms and present it in a way that is going to make the judge unfavorable toward the the other side or the particular issue. Here one way they could poison the well is by saying something to the effect of "they had the opportunity to adequately control these investigations by doing X, but they chose to do Y instead, even though we warned them that we thought X was the way to go . . . . . [that last part obviously only if they did, in fact, warn them and document in the record in a way they can get before the judge.]

     

    Inoculation -- basically what you do is take whatever good argument the other side has and bring it up to the judge first, on your own terms, where you kind of steal the other side's thunder on the substance of the argument and give the judge reasons why the argument doesn't work or isn't as good as it might otherwise seem. What I shoot for is for the judge to basically already be bored of their argument by the time the other side presents it to him. In this case, I would expect them to try to so something to diminish the independent evidence they are going to present-- the MRIs in particular. I have no idea what they would say, except it could be something along the lines of "Yeah, they will say that the MRIs show ____, but really you can't tell what that is attributable to . . . . everyone is different, small sample, etc." Really the whole point behind inoculation is to show the judge you aren't afraid of that argument by bringing it up (we know that argument and it still doesn't get them where they need to be) and to steal the other side's thunder for when they do present it.

     

    Anyway, don't know the FDA, didn't look at this stuff all that closely, particularly not the procedural stuff, so . . . . caveat, caveat . . . .

     

    But, I was actually pretty encouraged about the substantive statutory and rule provisions -- could have been worse for sure.

     

    **************

     

    § 314.126 Adequate and well-controlled studies.
    (a) The purpose of conducting clinical investigations of a drug is to distinguish the effect of a drug from other influences, such as spontaneous change in the course of the disease, placebo effect, or biased observation. The characteristics described in paragraph (b) of this section have been developed over a period of years and are recognized by the scientific community as the essentials of an adequate and well-controlled clinical investigation. The Food and Drug Administration considers these characteristics in determining whether an investigation is adequate and well-controlled for purposes of section 505 of the act. Reports of adequate and well-controlled investigations provide the primary basis for determining whether there is “substantial evidence” to support the claims of effectiveness for new drugs. Therefore, the study report should provide sufficient details of study design, conduct, and analysis to allow critical evaluation and a determination of whether the characteristics of an adequate and well-controlled study are present.
    (b) An adequate and well-controlled study has the following characteristics:
    (1) There is a clear statement of the objectives of the investigation and a summary of the proposed or actual methods of analysis in the protocol for the study and in the report of its results. In addition, the protocol should contain a description of the proposed methods of analysis, and the study report should contain a description of the methods of analysis ultimately used. If the protocol does not contain a description of the proposed methods of analysis, the study report should describe how the methods used were selected.
    (2) The study uses a design that permits a valid comparison with a control to provide a quantitative assessment of drug effect. The protocol for the study and report of results should describe the study design precisely; for example, duration of treatment periods, whether treatments are parallel, sequential, or crossover, and whether the sample size is predetermined or based upon some interim analysis. Generally, the following types of control are recognized:
    (i) Placebo concurrent control. The test drug is compared with an inactive preparation designed to resemble the test drug as far as possible. A placebo-controlled study may include additional treatment groups, such as an active treatment control or a dose-comparison control, and usually includes randomization and blinding of patients or investigators, or both.
    (ii) Dose-comparison concurrent control. At least two doses of the drug are compared. A dose-comparison study may include additional treatment groups, such as placebo control or active control. Dose-comparison trials usually include randomization and blinding of patients or investigators, or both.
    (iii) No treatment concurrent control. Where objective measurements of effectiveness are available and placebo effect is negligible, the test drug is compared with no treatment. No treatment concurrent control trials usually include randomization.
    (iv) Active treatment concurrent control. The test drug is compared with known effective therapy; for example, where the condition treated is such that administration of placebo or no treatment would be contrary to the interest of the patient. An active treatment study may include additional treatment groups, however, such as a placebo control or a dose-comparison control. Active treatment trials usually include randomization and blinding of patients or investigators, or both. If the intent of the trial is to show similarity of the test and control drugs, the report of the study should assess the ability of the study to have detected a difference between treatments. Similarity of test drug and active control can mean either that both drugs were effective or that neither was effective. The analysis of the study should explain why the drugs should be considered effective in the study, for example, by reference to results in previous placebo-controlled studies of the active control drug.
    (v) Historical control. The results of treatment with the test drug are compared with experience historically derived from the adequately documented natural history of the disease or condition, or from the results of active treatment, in comparable patients or populations. Because historical control populations usually cannot be as well assessed with respect to pertinent variables as can concurrent control populations, historical control designs are usually reserved for special circumstances. Examples include studies of diseases with high and predictable mortality (for example, certain malignancies) and studies in which the effect of the drug is self-evident (general anesthetics, drug metabolism).
    (3) The method of selection of subjects provides adequate assurance that they have the disease or condition being studied, or evidence of susceptibility and exposure to the condition against which prophylaxis is directed.
    (4) The method of assigning patients to treatment and control groups minimizes bias and is intended to assure comparability of the groups with respect to pertinent variables such as age, sex, severity of disease, duration of disease, and use of drugs or therapy other than the test drug. The protocol for the study and the report of its results should describe how subjects were assigned to groups. Ordinarily, in a concurrently controlled study, assignment is by randomization, with or without stratification.
    (5) Adequate measures are taken to minimize bias on the part of the subjects, observers, and analysts of the data. The protocol and report of the study should describe the procedures used to accomplish this, such as blinding.
    (6) The methods of assessment of subjects' response are well-defined and reliable. The protocol for the study and the report of results should explain the variables measured, the methods of observation, and criteria used to assess response.
    (7) There is an analysis of the results of the study adequate to assess the effects of the drug. The report of the study should describe the results and the analytic methods used to evaluate them, including any appropriate statistical methods. The analysis should assess, among other things, the comparability of test and control groups with respect to pertinent variables, and the effects of any interim data analyses performed.
    (c) The Director of the Center for Drug Evaluation and Research may, on the Director's own initiative or on the petition of an interested person, waive in whole or in part any of the criteria in paragraph (b) of this section with respect to a specific clinical investigation, either prior to the investigation or in the evaluation of a completed study. A petition for a waiver is required to set forth clearly and concisely the specific criteria from which waiver is sought, why the criteria are not reasonably applicable to the particular clinical investigation, what alternative procedures, if any, are to be, or have been employed, and what results have been obtained. The petition is also required to state why the clinical investigations so conducted will yield, or have yielded, substantial evidence of effectiveness, notwithstanding nonconformance with the criteria for which waiver is requested.
    (d) For an investigation to be considered adequate for approval of a new drug, it is required that the test drug be standardized as to identity, strength, quality, purity, and dosage form to give significance to the results of the investigation.
    (e) Uncontrolled studies or partially controlled studies are not acceptable as the sole basis for the approval of claims of effectiveness. Such studies carefully conducted and documented, may provide corroborative support of well-controlled studies regarding efficacy and may yield valuable data regarding safety of the test drug. Such studies will be considered on their merits in the light of the principles listed here, with the exception of the requirement for the comparison of the treated subjects with controls. Isolated case reports, random experience, and reports lacking the details which permit scientific evaluation will not be considered.
    [50 FR 7493, Feb. 22, 1985, as amended at 50 FR 21238, May 23, 1985; 55 FR 11580, Mar. 29, 1990; 64 FR 402, Jan. 5, 1999; 67 FR 9586, Mar. 4, 2002]
    23 Jan, 08:13 PM Reply Like
  • TimeOnTarget
    , contributor
    Comments (2175) | Send Message
     
    By the way, I haven't researched any judicial decisions concerning a decision by the Commissioner (the Statute says "Secretary" but I think that has been essentially delegated to the FDA Commissioner, who will undoubtedly designate a hearing officer --didn't really look at all the procedural nuances). I may take a look if I have some time tomorrow.

     

    But, the point I wanted to mention is that "substantial evidence" generally has a term of art type meaning in administrative law cases. Normally you see it in the context of a reviewing court saying something to the effect that "there was substantial evidence supporting the agency's decision." In that context, it means more than just a tiny bit, more than just some, but not necessarily a "preponderance" of the credible evidence. In other words, substantial evidence in the administrative law context can be less than the majority of the evidence.

     

    What is interesting is the use of the term in the context they use it in this statute regarding an appeal of the efficacy determination. To me that potentially bodes well because based on the info I have read they would certainly have substantial evidence of the efficacy. Hence, that really boils down to the fight over the "adequacy and well-controlled" aspect.

     

    And, at least to me, when you are talking about adequacy and well-controlled, you have to consider not only the size, design, etc., but also how much efficacy was demonstrated. In other words, a small flaw in the design might be critical in my (and this is my personal thought) evaluation of a drug with only slight efficacy, but if there was a whopping huge demonstration of efficacy, I would be less concerned about minor issues with controls in the design.

     

    If it was me making the argument, based on the little I know now, I would come out swinging with "this drug is HUGELY effective and there were very reasonable controls that were thoughtfully considered and put in place because you couldn't do a normal double-blind placebo because of the side effects it wouldn't have been reliable and undercut the entire idea of why you use the placebo control in the first place . . . .
    23 Jan, 08:16 PM Reply Like
  • Elliot Eisenberg
    , contributor
    Comments (14) | Send Message
     
    I note that Fox Business Channel host Neil Cavuto is an MS patient. Has anyone asked him to address the subject, either on air or by letter vis a vis official comment?
    4 Feb, 10:51 AM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » I sent him a note via Twitter but never heard back. I don't have any connection to him that would allow for a more promising channel. If anyone else does, they should use it.
    4 Feb, 10:54 AM Reply Like
  • John DiStanislao
    , contributor
    Comments (121) | Send Message
     
    Yes I forgot Neil Cavuto.

     

    Neil was a very kind soul. He took time to speak with me about his MS and my concerns after his show. Neil C would be a big advocate to stand up against the FDA few bureaucrats that make health decisions for the entire population. I forgot send a note via email to Neil using the Fox website, I think.

     

    He will reply or his people will pass along to Neil. He will reply and would be very interested as this is not only MS its freedom of choice and government over reaching hands.
    4 Feb, 11:16 AM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » Would you please reach out to him? There could be a story here for him, I would think.
    4 Feb, 11:49 AM Reply Like
  • John DiStanislao
    , contributor
    Comments (121) | Send Message
     
    Chris, I will give it a try now.
    4 Feb, 11:59 AM Reply Like
  • Chris DeMuth Jr.
    , contributor
    Comments (4041) | Send Message
     
    Author’s reply » Excellent; thanks! -C
    4 Feb, 12:19 PM Reply Like
  • bazooooka
    , contributor
    Comments (2323) | Send Message
     
    John,

     

    Did the Cavuto team ever respond to your inquiry?
    3 Mar, 05:47 AM Reply Like
  • John DiStanislao
    , contributor
    Comments (121) | Send Message
     
    Bazooooka, I reached out to my contacts when original comment was posted . They did get back to me after reaching out to their Fox and Bloomberg contacts. They responded “we will research”.

     

    Because of your comment today I searched for an email and just sent an lengthy email to

     

    foxnewstips@foxnews.com

     

    Hopefully it will get a response. If and when Fox confirms their receipt I will let you know. BTW when I was at my neurological visit I spoke with the PA to the neurologist. They were SURPRISED Lemtrada was not approved. I don’t have any additional information. John
    3 Mar, 01:52 PM Reply Like
  • PICOLOMINI
    , contributor
    Comments (545) | Send Message
     
    Perhaps there is a chance to have a discussion on a more abstract level. My email of Jan 18 to Dr Hamburg has wide ranging implications, the answers affect millions of patients and billions of investments.
    Why is there no cross Atlantic coordination?

     

    Dr Margaret Hamburg
    Commissioner of the Food & Drug Administration

     

    Dear Dr. Hamburg,
    Safeguarding and betterment of health is a human right, so is access to medicine.
    To safeguard the health of its citizen is the mission of FDA as well as its counterparts in Europe, Canada, no difference, no conflict.

     

    The FDA decision on Lemtrada, which may be scientifically well founded is the diametric opposite of the affirmative decisions by its counterparts.

     

    The MS world has the right to know why a permitted treatment of MS is more risky on one side of the ocean than on the other. The MS world has the right to know why the residual risk -----and it can be only a residual risk otherwise the FDA counterparts acted in gross negligence---why such residual risk decision is not up to the doctors and the suffering patient to decide.
    FDA owes complete transparency to the MS world and the medical profession. Also the full disclosure of interactions between these agencies before the final verdict is in order.

     

    Rolf Kleinlein
    4 Mar, 10:32 AM Reply Like
  • John DiStanislao
    , contributor
    Comments (121) | Send Message
     
    Rolf thanks for that Dr Hamburg email. I also responded to the FDA’s denial. One of my concerns is lack of transparency. It’s frustrating not having a response.

     

    Rolf could you share emails, or websites to send our comments and concerns.

     

    This was the end of my email to the FDA .

     

    <<In conclusion we must consider human bias, massive industry pressure, political influence, lack of expertise/experience unfortunately concentrated in the hands of a few federal employees. Existing FDA MS approved treatments are concentrated in about 3 or so companies.

     

    Its rationale to ask do FDA decision makers have connections, ever worked with or received funding from these few MS drug companies. The FDA process is long overdue for independent auditing and creating more public transparency. Pressure not to approve could come from Biogen that in Feb 2013 paid 3.25 Billion to receive the remaining drug rights for Tysabri from Elan. Biogen also sells the FDA approved MS therapy Avonex. The life time cost using Lemtrada versus Tysabri or Avonex is not only less expensive but has potential to be a life altering drug for MS patients and society.
    Please reconsider your decision.

     

    Respectfully but emotionally involved>>
    John DiStanislao
    4 Mar, 02:07 PM Reply Like
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