Drug and FDA News, May 2013

Yesterday, the CFIDS Association formally announced what had been rumored to be on the way: they are preparing to file an Investigational New Drug application. The first step will be a pre-IND meeting with FDA. This kind of meeting is a chance for the Association and its partner, Biovista, to seek FDA’s expertise and guidance in designing the clinical trial. To my knowledge, this is the first formal FDA action on an ME/CFS related potential treatment since the Drug Development Workshop in April. The Association will have to file an IND application before beginning the clinical trial, and FDA has 30 days to review and approve the application. Since this is a drug repositioning trial – meaning that the drug in question is already approved for another purpose – this will probably be a straightforward process. I don’t know what drug is involved, and as a Patient Representative at FDA, I cannot participate in the trial or have other involvement in it or I risk the appearance of a conflict of interest that would preclude me from serving on an Advisory Committee down the road. It is incredibly exciting to see treatment development move forward, and let’s hope more sponsors will follow suit.

There are a few other FDA-related news items to share with you:

  • On June 14th, FDA will hold the next in the series of Patient Focused Drug Development meetings. This meeting will focus on the impact of HIV on daily life and currently available therapies, and patients’ views on issues related to HIV cure research. The format appears to be similar to the ME/CFS meeting, although the questions for patients are more extensive and detailed. The meeting will be webcast, and registration closes June 5th.
  • On June 28th, FDA will hold a PFDD meeting focused on lung cancer. Again, the meeting will be webcast, and registration closes June 19th.
  • Irritable bowel syndrome is an overlapping condition with ME/CFS. The FDA will hold an advisory committee meeting on July 10th to discuss the risk evaluation and mitigation strategies in place for the IBS drug Lotronex. Lotronex is used to treat severe IBS in women, and was withdrawn from the market in 2000 after several deaths and severe complications were reported. The drug was reintroduced in 2002 with more safety restrictions on its use. The purpose of this advisory committee meeting is to consider whether the risk strategy in place assures safe use without placing undue burdens on the patient or health care delivery system.
  • FDA is inviting public comment on two draft industry guidance documents: Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers and Guidance on Charging for Investigational New Drugs – Questions and Answers. Both comment dockets close on July 8, 2013.
  • Finally, ME/CFS patients tend to take numerous prescription medications including pain medications. FDA has posted information on the safe disposal of medications, including certain drugs that should be flushed.


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4 Responses to Drug and FDA News, May 2013

  1. Sasha says:

    Thanks, Jennie. It’s interesting that the CAA has got this IND application in. Is there any reason why they can’t tell us all which drug it’s about? So that patients can consider whether they want to try it at their own risk, if that’s possible (I don’t know the US laws on prescription)?

    • Jennie Spotila says:

      I don’t think the Association can identify the drugs until the IND application is accepted and they can start the clinical trial. People not in the study may not be able to try the drugs on their own. Even if they can convince doctors to try it, it will be an off label use. That means they would be taking the drugs for a condition that is not approved by the FDA, and insurance may not cover it. It could also disqualify those patients from participating in a larger clinical trial of those drugsdown the road.

  2. Sasha says:

    Thanks, Jennie. That’s an interesting situation and it’s striking how at odds all this regulation (if that’s the constraint rather than some contractual thing) is with the sense of urgency that patients have to try things and get well.

    I’m not blaming the CAA for this – it sounds like their hands are tied.

    • Jennie Spotila says:

      One of the things that has fascinated me about FDA is how constrained everyone is in the process. The regulations around the testing and approval of drugs are remarkable, and FDA is as affected by them as everyone else. It’s interesting, but not very compatible with impatience!

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