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KarinCA
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I'm an independent trader/investor whose primary interests are in the stem cell and biotech arena. My secondary interest is in oil/energy speculation. I do venture elsewhere at times. There’s an option to follow me at the top of any article I write. Click it and you’ll also see an option to get... More
  • $CUR President And CEO Garr On "Right To Try" Bill 0 comments
    Mar 5, 2014 1:43 AM | about stocks: CUR

    $CUR - President and CEO, Richard Garr, blogs about the "Right to Try" bill

    I'm copying over a blog due to the importance of this to everyone. More people need to see this. - KarinCA

    http://www.neuralstem.com/neuralstem-ceo-blog/208-every-man-gotta-right-to-decide-his-own-destiny

    "Every man gotta right to decide his own destiny" - Bob Marley

    Posted: December 3rd, 2013

    The "Right to Try" bill is making its way through the (now infamous) Arizona State Legislature. The basic premise of the Bill is that patients with fatal and incurable diseases should be able to choose for themselves whether or not to try an experimental drug. The Bill's authors state that they should not be constrained by the FDA's approval process.

    Now the Arizona State Legislature is the incubator of some of the worst laws in the history of bad laws, embodying obviously hateful and penal opposition to all manner of groups. But I suspect that this bill may resonate broadly both in and outside of Arizona.

    The Bill was drafted by the libertarian leaning Goldwater Institute, and is based on the premise that the FDA's approval process denies citizens the right to choose their own medical path. The Bill stipulates that it only applies to fatal diseases, and only to medicines that have passed a FDA Phase I safety trial, and further to medicines that are currently in (at least) a FDA Phase II trial; so there is some effort to restrict the scope of this program to drugs that have had a good level of FDA scrutiny. There must still be a licensed medical doctor involved to prescribe the experimental drug; and the law states that companies cannot be forced to provide the drug if they so choose. Finally, there is language that looks to allow for companies to charge for these treatments. The provision is vague but seems to reference cost reimbursement language.

    At Neuralstem we are keeping a sharp eye on this legislation. As we move into the final stretch of transplantations for our Phase II therapy for ALS (the dosing of all patients should be complete early this summer) we are keenly aware of all of the patients who cannot be included in the trial; both because of the small number of patients in the trial, and also because of the exclusive nature of inclusion/exclusion criteria the trial is built around. And as we begin to design the next trial, a trial which we hope will lead to approval by the FDA, we are thinking more and more about ways to broaden access to our cells. (Spoiler alert: Dr. Eva Feldman will be giving interim Phase II data in mid-April at a conference; I have not seen the data, and will be hearing it for the first time when she presents. The fact that we are working on the design of the next trial should not be taken for an early signal on that data beyond the confidence we have already publicly expressed). Further, ALS is exactly the type of indication the Bill is talking about; and our therapy the type of experimental treatment the bill is aimed at.

    So in general, we support the intent of this bill. One does not have to be a fire breathing libertarian to wish that cutting edge technology could get to its intended recipients sooner and with less red tape. Although, in the end I suspect that the FDA will claim that this is an area of Federal Preemption. That is, an area where the states cannot create laws in conflict with the Federal authority. The FDA already has the ability, should it so desire, to make drugs available sooner and quicker through programs like Accelerated Access, and Breakthrough Designation, and others. What the authors of this bill are trying to do is take away the FDA's role as the sole arbiter of whether or not a drug should be made available early in the process.

    We now live in an always on, always logged in world, where vast amounts of information on drugs in development is widely available, and where patients in trials are blogging and in constant communication with others about their trial experiences. If a drug is showing any hopeful signs of helping patients with rare diseases, the entire community knows almost instantly. One can understand the urgency behind efforts to make these drugs more widely available, sooner. A program that has the safeguards of using only FDA (Phase I safety trial) passed drugs is making a good start. The bill raises more questions than it answers with respect to its implementation of course, but that is not a reason not to proceed with it.

    The FDA and indeed the entire medical community is evolving as they try to cope with the incredible pace of change occurring in the basic science involved with drug development and testing. What is perhaps a surprise is that not the least of these changes taking place is in the living rooms of patients and their families, in front of computer screens, not in the lab.

    The information age and social media have brought in unexpected and often quite dramatic changes in many areas of society. I believe they are going to be powerful forces driving public policy in the rare disease treatment arena also. There are going to be conflicts with many existing norms and institutions, and so moving the decision making power, even a little, from institutions to individuals is going to play out over years, not months. The medical profession is not immune to the social and political forces restructuring our society. Right to Try is a good idea with lots of flaws; but a good idea none the less, and an idea worth supporting and pursuing. -

    Disclosure: I am long CUR.

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