Home > Advocacy, Research > Will FDA Step Up?

Will FDA Step Up?

September 13th, 2012

The Federal Drug Administration held a conference call today to speak with CFS patients, advocacy groups and other interested parties. This is the first time in the history of this disease that FDA has communicated with the patient community in such a broad and direct way. I was fortunate enough to attend the conference call, and offer this summary of the discussion.

This call happened through the efforts of patients to secure an FDA Stakeholders Meeting. The meeting premise is modeled on a similar process between FDA, HIV patients and others in the 1980s and 1990s. Through sustained communication, FDA and HIV patients came to an understanding of the need for accelerated drug approvals balanced with FDA’s focus on safety and effectiveness. CFS patients and advocates have secured a commitment from Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research (CDER), to hold a similar meeting on ME/CFS. The goal of that Stakeholders Meeting is to discuss acceleration of treatments, access to treatments and evaluation of treatments. Understanding the burden of disease from the patient perspective and how to measure those effects are an essential part of developing new therapies.

Today’s call was led by Dr. Sandra Kweder, Deputy Director of the Office of New Drugs within CDER, and she provided some key information:

  • FDA considers CFS to be a serious or life-threatening condition. This means that drug applications can qualify for the Accelerated Approval Process. Accelerated Approval brings drugs to market after shorter clinical trials, and additional studies are done after that approval. This is how many HIV drugs have been approved, as well as drugs for cancer and other conditions.
  • FDA will offer a webinar on “Excellence in Advocacy” in November 2012. The purpose of the webinar is to show advocates how they can speed the development and approval of treatments through engagement of researchers and pharmaceutical companies. I find it a little ironic that FDA will be telling us how to be better advocates, but I think this will be an important session. FDA has been through this process with HIV and other serious conditions; they must have a perspective on what works and what does not.
  • Ampligen will be considered for approval at a public meeting on December 20, 2012. This meeting will feature an FDA panel, as well as public comments from clinicians, patients and others.
  • FDA will hold the 1.5 to 2 day stakeholder meeting in the spring of 2013. This meeting will bring together other federal agencies, researchers, clinicians, pharmaceutical companies and patients. It emerged during the call that a major goal of the meeting will be identifying consensus endpoints. In this context, endpoints are the reliable quantitative measures that will be used in clinical trials to evaluate whether a treatment is working.
  • FDA takes no position on the name issue of ME v. CFS. What they focus on is whether a treatment improves patient condition. They are using the term ME/CFS as a framework for that process but take no position on lumping vs. splitting in defining the illness.
  • There are currently 8 open applications for new drugs to treat CFS. Dr. Teresa Michele stated that many of those applications are for nutritional supplements, with small early trials. FDA expects that agreement on measurable endpoints will speed and increase research into other treatments.

Two issues emerged during the discussion through the comments made by patients on the call. First was the issue of case definition. While FDA insisted that it does not take a position on definition or name, many patients pointed out that case definition shapes research results. It’s been well established that some criteria sweep in people with idiopathic chronic fatigue or depression. In order to evaluate if a drug helps people with ME/CFS, then the case population must be carefully defined. FDA seems to recognize this as an issue, but there was no clarity on what requirements they might make in study design.

The second issue was endpoint measurements: how do we know if a treatment is working? What can be measured in clinical trials to determine effectiveness? In a condition like high blood pressure, it’s clear that the desired endpoint is a lower blood pressure measurement. But in our disease, what can be reliably and quantitatively measured as endpoints? Multiple suggestions were made, including VO2max, natural killer cells, and actimetry data. Debra Waroff offered the “salad” endpoint: whether she is well enough to make a salad. Dr. Kweder seemed to like that measure, as it is a measurement of functional improvement in patients’ lives. Identifying a consensus around endpoints will be a major feature of the 2013 meeting.

Overall, I think this call was very positive. I would have liked to have heard more on FDA’s processes for orphan drugs and accelerated approvals. I also want to know more about how the 2013 meeting is being planned, and how patients can add the most value to that process. But I give FDA a lot of credit for holding today’s conference call and appearing to be so open to our input. If this is a sign of things to come, then I think we can have high expectations for the meeting in 2013.

 

  1. Joyce Shaffer
    September 13th, 2012 at 16:30 | #1

    Jennie, thanks for sharing what you learned today on the conference call. I have heard that Ampligen is a successful treatment. It is nice to hear of the 12/20/12 date! And I am encouraged to hear about new drugs/supplements. Those of us suffering from ME/CFS are ready to try anything. Thank you!

  2. Kathryn Stephens
    September 13th, 2012 at 18:30 | #2

    Thank you Jennie, for another excellent piece of work; your captioning and division of the points of interest were very helpful to me.

    Thank you, patients, who were able to be present for this meeting. I appreciate you so much.

    I have a question: Without appropriate testing, how in the world can treatments be assigned to any patient? I am of the opinion that we should request some of the testing that our esteemed researchers are using, such ans NK cell counts, HHV6, EBV, CMV, et al…plus, of course, whatever the Lipkin conference reveals.

    • Jennie Spotila
      September 13th, 2012 at 22:37 | #3

      Identifying consensus endpoints will be one of the goals of the 2013 meeting. So NK cell count, for example, may be considered a measurable endpoint for at least a subset of patients. A bad NK count could indicate a need for a particular treatment, and that treatment might be effective if the count improves.

  3. Jen Whitestone
    September 13th, 2012 at 18:52 | #4

    Jennie, So glad you were able to participate in the meeting and that you were willing to take the time to summarize the highlights. Great to hear about ampligen! Hopefully the December meeting will result in acceptance of this medication. Now wondering how long it will take to propagate through the medical community.

  4. Valerie Krumins
    September 13th, 2012 at 23:38 | #5

    I still believe this condition/ illness will turn out to have different triggers and therefore, different treatments, for various patient groups under the CFS/ M.E. umbrella. I definitely fall under post-viral syndrome definitions – I did NOT have “fatigue” until approximately three years ago. My doctors and I believe this fatigue is due to being allergic to a binder or other ingredient, in a medication I was prescribed. I have high pain levels due to central nervous system (CNS) damage that mimics Multiple Sclerosis – with an explanation of how that CNS damage may have taken place. I’m not like a lot of CFS patients – therefore, they’re not like me! I think this must be kept in mind. There will have to be numerous medications to treat all of us. I’m not wild about Ampligen, personally. So, there it is … Just feel like this has to be said. Thanks.

  5. Rich Carson
    September 14th, 2012 at 05:17 | #6

    I am happily surprised that they said, “FDA considers CFS to be a serious or life-threatening condition”. That is huge. Brilliant on the spot reporting from Jennie. Good job!

  6. Lilly C
    September 14th, 2012 at 15:07 | #7

    I really hope supplements are not going to be counted as part of a quota of research efforts purported to be for treatment. Are supplements offered as treatments for any other serious disease other than for specific deficiences? The only possible benefit from that would be that supplements might be prescribed and thus be covered by insurance, but they cannot be considered to be worth much as real treatment and calling them treatments will further trivialize our illness.

  7. Janelle
    September 14th, 2012 at 17:00 | #8

    @Lilly C
    actually, yes, supplements are used for other diseases including, for instance, mitochondrial disorders (the traditional ones). These diseases are considered very serious. People die from them at an early age.

    Even though these disorders [mitochondrial diseases] are long term and incurable, treatments are available. Early treatment of symptoms can reduce their impact and limit further disability. Avoiding certain medications and stressful situations that worsen symptoms is also helpful. Certain medications and supplements may improve mitochondrial disease-related symptoms — just as they do for other incurable diseases — such as diabetes and emphysema.

    http://www.umdf.org/site/pp.aspx?c=8qKOJ0MvF7LUG&b=7934635#supportive

    Having clinical studies for CoQ10, etc., would make sense from a clinical (though perhaps not financial) standpoint. Doing them with very small sample sizes doesn’t necessarily make sense.

    That’s an interesting point about insurance coverage. I wonder whether it would work like that. I think if it’s available over-the-counter, perhaps not, but that’s something to look into! If it were shown to be effective and our doctors said it was medically necessary, maybe something could be worked out.

  8. September 14th, 2012 at 17:45 | #9

    The FDA will teach us how to become better advocates. Looking forward to that webinar. Interesting times ahead.

  9. Latha Steinbronn
    September 14th, 2012 at 20:18 | #10

    Wow. It gives me hope.

  10. September 15th, 2012 at 04:59 | #11

    Thank you so mcuh for sharing all that in such a clear and digestable manner!

  11. September 15th, 2012 at 05:50 | #12

    thank you for your great summary. Fantastic you could attend this conference call. I hope you will not have a problem if I translate your testimony to spanish and upload it on the website of the Spanish National Platform for FM, CFS, MCS.
    It is great news and the spanish speaking people (affected and caregivers) really have to be informed. Many of them don’t even believe in the existence of CFS.
    Thanks again

    • Jennie Spotila
      September 15th, 2012 at 21:40 | #13

      You may translate my article and post on your website as long as you credit my name and link back to the blog. Thank you!

  12. Justin Reilly
    September 15th, 2012 at 20:59 | #14

    We should produce a webinar for them on “How to be an Effective Regulatory Agency.”

  13. Justin Reilly
    September 15th, 2012 at 21:03 | #15

    Still, for what it was (“just talk” so far), I was very pleased with the call.

  14. Justin Reilly
    September 15th, 2012 at 21:04 | #16

    “very pleased” not just because i got to hog extra minutes bashing CDC. : )

  15. Laura Willging, RN,patient
    September 16th, 2012 at 12:41 | #17

    So delighted to hear of this meeting. Thank you Jennie for being our voice. I, too, wonder about measurable outcomes and sorting of the objective possibilities for measuring in certain populations of us with CFS/ME. This will be challenging but so necessary. This is the opening of a new door, one that has been closed too long. Was surprised to see FDA listed it as a serious or life-threatening condition and it is just now being brought to the table in this fashion. I am thankful that it is; don’t get me wrong. Thanks again, Jennie, for attending and for summarizing. I look forward to hearing about the next step(s). Amen!

  16. Laura Willging, RN,patient
    September 16th, 2012 at 12:51 | #18

    Regarding supplements: they are not FDA regulated and the manufacturers do not make them exactly the same and there can be no guarantee as to the quality and concentration (per capsule, etc) of the supplements. We are so far away from these being studied and considered an adequate ‘treatment’-and big Pharma would not like this, I must add. I have to research supplements on my own and try them one by one. We really do need help and supplementation at the cellular level-mitochondria, ATP, etc. Not so sure if there are many (any) medicinal options for this? I’m just glad to see this get off the ground. It is serious and it’ll take much time.

  17. SKIP GOOD
    September 17th, 2012 at 21:23 | #19

    Yeah I’m impressed that the FDA is coming on board with the situation, ME/CFS, and it’s very exciting. finally it seems like were gonna go somewhere with it. yes it looks to me like There just listing a lot of protocols that you would have on any other disease or Protocol on how they would outline The natural progression of research that have to do with chronic illness ,your new diseases such is mentioned about HIV. Although it looks like a lot of protocol has been listed on how they would go about the research with new diseases. it does again like I said show that we are making progress and we in the community should really be excited about this meeting. with the FDA in 2013.

  18. SKIP GOOD
    September 17th, 2012 at 21:26 | #20

    SKIP GOOD :
    Yeah I’m impressed that the FDA is coming on board with the situation, ME/CFS, and it’s very exciting. finally it seems like were gonna go somewhere with it. yes it looks to me like There just listing a lot of protocols that you would have on any other disease or Protocol on how they would outline The natural progression of research that have to do with chronic illness ,your new diseases such is mentioned about HIV. Although it looks like a lot of protocol has been listed on how they would go about the research with new diseases. it does again like I said show that we are making progress and we in the community should really be excited about this meeting. with the FDA in 2013.

    SKIP GOOD :
    Yeah I’m impressed that the FDA is coming on board with the situation, ME/CFS, and it’s very exciting. finally it seems like were gonna go somewhere with it. yes it looks to me like There just listing a lot of protocols that you would have on any other disease or Protocol on how they would outline The natural progression of research that have to do with chronic illness ,your new diseases such is mentioned about HIV. Although it looks like a lot of protocol has been listed on how they would go about the research with new diseases. it does again like I said show that we are making progress and we in the community should really be excited about this meeting. with the FDA in 2013. Its about damn time.

  19. September 18th, 2012 at 12:32 | #21

    This inspires hope for the CFS and ME communities. After reading what they are doing overseas, treating Lyme’s patients as though they are psychiatric patients, this is a breath of fresh air that renews my faith in our system. My only wish is that they adopt the Canadian Consensus Criteria for diagnosis. One shoe should fit all, especially since there are already subgroups of CFS, otherwise all research can be skewed.

    Laura, I completely agree with what you are saying regarding the mitochondria and supplements. This is an issue for FM patients too.

    In healing and hope, Celeste

  20. Firestormm
    September 29th, 2012 at 03:07 | #22

    Thanks Jennie.

    I don’t suppose a recording of this conference is available? Have just been reading Cort’s summary as well as your own again and wondered if I might listen to the original. Now that I have some more time.

    Prohealth: http://www.prohealth.com/library/showarticle.cfm?libid=17241

  21. Firestormm
    September 30th, 2012 at 01:16 | #24

    Many thanks Jennie :)

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