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State's "Right To Try" laws vs. Expanded Access

  • 1.  State's "Right To Try" laws vs. Expanded Access

    Posted 19-Mar-2015 11:40
    Nearly 10 states have passed so-called "right to try" laws and 20+ are in process.  When challenged that these state laws are adding hoops to the existing federal (FDA) process (i.e. extra items and witnesses in the consent form, needing 2 physicians to diagnose the terminal illness etc) , Goldwater Institute (the organization that is pushing for these laws in all 50 states) state that this is a separate process outside of Expanded Access that the manufacturer does need an IND/IDE for nor does an institution need IRB approval for, They state the supreme court allows patients to determine their healthcare and thus (in so many words) the FDA has no jurisdiction over state's Right To Try laws.  Of course, the FDA tells me differently but Goldwater counters with that this is too new for the FDA to digest and they are just fighting for a power grab. When i asked if there were manufacturers shipping Phase 2-3 investigational products outside of clinical trials without an IND/IDE, they sated yes but would not tell me the names for me to contact them to ask what their thinking is on this.

    1) Has anyone actually shipped (or determined that you could ship) investigational products under the auspices of a state "Right To Try" act and if so, what is the justification for not needing an expanded access IND/IDE + IRB approval?     

    2) To the corollary, has anyone seriously looked at this and determined "no way am I doing that"?

    3) Has anyone shipped for a terminal illness in a "Right To Try" state and followed federal law but NOT followed the state law when it requires items above and beyond the federal regulations (i.e. Louisiana and Arizona requiring 2 physicians to attest there are no other options, Colorado and Michigan's extra items in the consent form above FDA's Part 50 requirements)?

    I am chairing a session at both ACRP and DIA on this topic (and maybe RAPS will let me do something) so your feedback is definitely appreciated


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    David Vulcano RAC
    Assistant Vice President, Clinical Research
    Hospital Corporation of America (HCA)
    Nashville TN
    United States
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  • 2.  RE: State's "Right To Try" laws vs. Expanded Access

    Posted 24-Mar-2015 04:20
    I hadn't heard anything about this, but I appreciate your posting about it, as I think it's very interesting.

    I wonder how many physicians are at liberty to forego IRB approval, just because state law says they can.  Many medical institutions have their own ideas about what their IRBs should approve, which physicians are bound to by virtue of their affiliation with the institution.  I wonder also who is going to assure that the informed consent meets the state law requirements, if no IRB is involved. Physicians don't exactly have a great track record when it comes to preparing consent forms that contain all the required elements.

    As for industry, I don't know how it might react.   It's not clear to me that there is much benefit in these laws for industry. In that case, it would probably depend on how they assess risk.


    I'm sorry I can't answer any of your specific questions, but I would be very interested to hear what others have to say about this topic.  I think you will get some very useful feedback at ACRP and DIA.  It would be great if you could come back and share it with us.


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    Julie Omohundro RAC
    Durham NC
    United States




  • 3.  RE: State's "Right To Try" laws vs. Expanded Access

    Posted 24-Mar-2015 10:57
    Thanks for starting this discussion, David. If anyone would like to find more information about their States' "Right to Try" laws, you can find out more in Regulatory Focus. I've assembled a pretty comprehensive list of laws and proposed legislation that people can read through.

    http://www.raps.org/Regulatory-Focus/News/Right-to-Try/

    And in case anyone needs a refresher on Expanded Access, they can find that here: http://www.raps.org/Regulatory-Focus/Expanded-Access/ 

    Best, 

    Alec
    -------------------------------------------
    Alexander Gaffney
    Manager, Regulatory Intelligence at RAPS
    agaffney@raps.org
    -------------------------------------------




  • 4.  RE:State's "Right To Try" laws vs. Expanded Access

    Posted 24-Mar-2015 11:11
    I'm really interested to hear if anyone is convinced that they can ship without an expanded access IND/IDE. I am not but the advocates are convincing state legislators you can and the press is relaying this.

    -------------------------------------------
    David Vulcano RAC
    Assistant Vice President, Clinical Research
    Hospital Corporation of America (HCA)
    Nashville TN
    United States
    -------------------------------------------





  • 5.  RE: State's "Right To Try" laws vs. Expanded Access

    Posted 24-Mar-2015 13:21
    David,

    Based on the supremacy clause, federal statutes/US code rule over the State laws.  In this sense, FDA will/is likely to claim it is under our authority/jurisdiction over an expanded access IND/IDE, requiring FDA review/approval.

    HOWEVER,

    Based on the "Practice of Medicine" exercised by medical doctors, patient waivers to bring suits against the doctors, and "Right to Try" State law provisions, how is the FDA going to prosecute anyone if they want to?

    If you look at the recent DOJ action (http://wp.me/P5vHi0-TR) indicting 14 people in connection with compounding pharmacy, we may get some idea on criminal charges.

    If the matter goes on war between the States and the FDA, how is the FDA going to show/prove "intent" and potential crimes against the doctors who acted in accordance with the State's "Right to Try" beyond reasonable doubt? Who would the jury listen to and go with at the end?

    I don't believe FDA will try to come after each State and the doctors against the State "Right to Try" provisions.

    Accordingly, all things considered, I am convinced that the doctors and firms may/should work under the State's Right to Try provisions as the FDA will have to exercise "enforcement discretion" over the matter in lieu of going on war with all the States by trying to enforce the supremacy clause.  

    CAUTION: it should be noted that there may be some room for abusive use and practices over some drugs/devices for unjustifiable gains. Such abusive discretion/practices should be prevented.  





  • 6.  RE: State's "Right To Try" laws vs. Expanded Access

    Posted 25-Mar-2015 21:37
    David, I can't see FDA going after the physicians, because FDA doesn't regulate the practice of medicine.

    As far as I know, the only time FDA can pursue physicians for anything is when the physician has signed something (eg, 1572, financial disclosure) that opens the door for FDA to pursue them, and then only within the narrow constraints of what they have signed.  I don't think any physician has ever signed anything that says they won't treat patients with products not approved by the FDA.  The only time physicians are restricted in using an unapproved product is if they are an investigator in a trial of that product.  The onus is on the manufacturer to control their unapproved product and keep it out of the hands of physicians who have not signed an Investigator's Agreement.

    Nor can I see FDA going to war with the States over anything.  If anyone takes this up with the States, I would expect it to be Congress, not FDA. If anyone were to go to war with the physicians, I would expect that to be DOJ, not FDA.

    I would expect the FDA to focus its efforts where it always has...on manufacturers.  I would expect FDA to take the position that it is a federal agency charged with various responsibilities under federal law (eg, the FD&C Act), and that it will continue to enforce applicable federal law until such time as Congress amends the law and tells it to do otherwise.  Therefore, I expect that FDA would continue to take action against manufacturers shipping unapproved product without an IND/IDE.  In other words, I would expect FDA to ignore the "right to try" laws, rather than go to war over them, and stick with business as usual.

    This would leave it to the manufacturers to fight with FDA over this.  I'm skeptical that manufacturers are likely to see this fight as being in their shareholders' interests.

    The States do have jurisdiction over the practice of medicine.  The "right to try" laws allow physicians licensed in a given State to treat terminally ill patients with therapies not approved by the FDA.  Unless the manufacturer is in the same State as the physician, this would seem to be mostly theoretical, since physicians can't use drugs they can't get, and I don't think a State has any jurisdiction over manufacturers in another States.

    I note that the model law says manufacturers may make an unapproved product "available." It does not say manufacturers may introduce them into interstate commerce.  I would guess this wording was carefully chosen.


    -------------------------------------------
    Julie Omohundro RAC
    Durham NC
    United States




  • 7.  RE:State's "Right To Try" laws vs. Expanded Access

    Posted 25-Mar-2015 21:42
    Do you forsee any manufacturers shipping without an IND/IDE?



    -------------------------------------------
    David Vulcano RAC
    Assistant Vice President, Clinical Research
    Hospital Corporation of America (HCA)
    Nashville TN
    United States
    -------------------------------------------





  • 8.  RE: State's "Right To Try" laws vs. Expanded Access

    Posted 26-Mar-2015 16:57
    regardless of a state's 'right to try' law, there is still a need to have the legal authority to ship a product in interstate commerce to get it to the treating physician (the infamous commerce clause).  a company would still need an IND/IDE to ship the investigational product.  however, remember, FDA has for years authorized emergency or compassionate use of a product (emergency IND) over the phone with immediate shipment, so this part is not the problem.

    the bigger issue I think, is whether states can authorize this 'right to try' and overrule the Feds when FDA already has a process to handle expanded access, even in emergency situations (Federal preemption).

    in the end, if a company does not want to ship a product to a physician for a patient to 'try' no one can force them to do it.  Waiving liability etc. is not going to be a convincing argument in court if someone is harmed.


    -------------------------------------------
    Michael Hamrell, Ph.D., RAC, FRAPS
    MORIAH Consultants
    Huntington Beach CA
    United States
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  • 9.  RE:State's "Right To Try" laws vs. Expanded Access

    Posted 26-Mar-2015 17:57
    I agree with you Michael. The FDA is quick to quote where this was tested in federal court less than 10 years ago (the Abigail alliance case) resulting in there should be no shipments without an IND/IDE. The representative I met from Goldwater Institute says manufacturers are shipping within an IND/IDE (but claimed he could not share their names) but I have not heard of any that have dust my reaching out.

    -------------------------------------------
    David Vulcano RAC
    Assistant Vice President, Clinical Research
    Hospital Corporation of America (HCA)
    Nashville TN
    United States
    -------------------------------------------





  • 10.  RE: State's "Right To Try" laws vs. Expanded Access

    Posted 26-Mar-2015 18:55
    At federal level,

    First, FDA can't act on its own to invoke a legal action, but it has to go through DHHS and DOJ.

    If the FDA wants to go after the manufacturers for shipping without an effective IND/IDE, it can do so.  But under the circumstances, I was stating that the FDA can exercise an enforcement discretion as long as a firm(s), doctor(s) or both don't engage in abusive activity/conduct.

    At State level,

    Legal claims against the doctors, or hospitals are based on State claims.  The State's legislation will give rights for doctors to exercise their discretion without having fear of being sued by patients as a result of using/applying/administering drugs/devices/biologics under the emergency use/compassionate use as long as they exercise sound and reasonable judgement in a justifiable manner. 

    I would like to recommend that the FDA should take initiatives to sign an "Understanding of Memorandum" or (MOU) with each State so that firms can ship their unapproved drugs/biologics/devices without fear of getting prosecuted by FDA/DOJ for illegal shipping/mailing in violation of an "interstate commerce" clause. 

    At the State level, the State's "right to try" will serve the purpose of protecting the doctors who participate in treating the patients under the emergency circumstances.

    In summary, the FDA has to agree to exercise "enforcement discretion". The State's new law protects the doctors from being litigated by the patients, assuming the doctors will exercise their sound judgment and discretion with no abusive practice.




  • 11.  RE: State's "Right To Try" laws vs. Expanded Access

    Posted 27-Mar-2015 15:12
    I agree David.  I would like to know of any actual example of someone shipping drug without an IND.

    -------------------------------------------
    Michael Hamrell, Ph.D., RAC, FRAPS
    MORIAH Consultants
    Huntington Beach CA
    United States
    -------------------------------------------




  • 12.  RE: State's "Right To Try" laws vs. Expanded Access

    Posted 24-Mar-2015 14:21
    David,

    Based on the supremacy clause, federal statutes/US code rule over the State laws.  In this sense, FDA will/is likely to claim it is under our authority/jurisdiction over an expanded access IND/IDE, requiring FDA review/approval.

    HOWEVER,

    Based on the "Practice of Medicine" exercised by medical doctors, patient waivers to bring suits against the doctors, and "Right to Try" State law provisions, how is the FDA going to prosecute anyone if they want to?

    If you look at the recent DOJ action (http://wp.me/P5vHi0-TR) indicting 14 people in connection with compounding pharmacy, we may get some idea on criminal charges.

    If the matter goes on war between the States and the FDA, how is the FDA going to show/prove "intent" and potential crimes against the doctors who acted in accordance with the State's "Right to Try" beyond reasonable doubt? Who would the jury listen to and go with at the end?

    I don't believe FDA will try to come after each State and the doctors against the State "Right to Try" provisions.

    Accordingly, all things considered, I am convinced that the doctors and firms may/should work under the State's Right to Try provisions as the FDA will have to exercise "enforcement discretion" over the matter in lieu of going on war with all the States by trying to enforce the supremacy clause.  

    CAUTION: it should be noted that there may be some room for abusive use and practices over some drugs/devices for unjustifiable gains. Such abusive discretion/practices should be prevented.  




  • 13.  RE: State's "Right To Try" laws vs. Expanded Access

    Posted 25-Mar-2015 08:27
    Hi folks.

    I think another key point to this discussion is something that many non-industry people overlook in this situation.  If the drug/device is currently approved for marketing for ANY indication a physician/HCP can use the drug/device for treating ANY condition regardless of whether it is an approved condition for the product or not.  So this is another layer to the discussion ongoing here.  If your IDE/IND is for say an extension of your indication on label, then you are likely completely in the clear legally from the standpoint of the state-level RtT laws.  Effectively, FDA has acknowledged for years that they do not control the practice of medicine and therefore once a product is approved for marketing, the FDA has itself also acknowledged that they no longer have jurisdiction (except when marketed for off-label use - and even that is questionable based on more recent Supreme Court decisions) for uses of the product outside the context of "approved" labeling.

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    Victor Mencarelli
    Sr. Manager - Regulatory Affairs
    United States
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  • 14.  RE:State's "Right To Try" laws vs. Expanded Access

    Posted 25-Mar-2015 08:42
    I wholeheartedly agree on the off label issue and that has never been a problem for us. I am exclusively concerned about drugs and devices that are not yet approved for any indication. If anyone has shipped, or has seriously considered shipping such a product outside of the IND/IDE process because of the new state laws, please let me know as I am interested in your thinking on this.

    -------------------------------------------
    David Vulcano RAC
    Assistant Vice President, Clinical Research
    Hospital Corporation of America (HCA)
    Nashville TN
    United States
    -------------------------------------------