Author Topic: Right to try: Compassionately cutting through pharmaceutical bureaucracy  (Read 574 times)

markt

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http://www.foxnews.com/opinion/2018/03/13/right-to-try-unapproved-drugs-advances-personal-freedom-but-risks-must-be-taken-into-account.html

So, I sort of believe this to be novel idea.  I thought it would be interesting to discuss here... as seemingly, this concept would benefit a lot of Sjogrens patients.  Most of the systemic treatment options (Biologic's, et. al) being developed specifically for Sjogrens are in fact post Phase-II, have demonstrated some efficacy, and possess some level of product maturation and licensing for production, etc.

Say what you want about this man, but I appreciate that folks working for him are using his office to make medications coming to market an imperative... for those who desperately need the help.  Not sure if anything will come of this, or if the insurance industry will play along though. 

Any who, it is certainly interesting.

Regards,

Mark
« Last Edit: March 13, 2018, 12:56:44 PM by markt »

irish

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One thing is for sure, the pharm companies need to be reined in. My drug costs last year were horrible. I know that many people are watching grocery shopping in order to get their needed meds. Irish

markt

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Another plus to this legislation (and in in 38 States, State Law), patients that are sero-negative for Sjogrens would be able to submit "right to try/compassionate use" claims through their provider for next generation investigational medications (that they could have normally received through participation in clinical trails, if not for the fact they were excluded for being sero-negative). 

warmwaters

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I've been following this law, and to me the unanswered question is how treatment would be paid.  Obviously, no insurer is going to pay for a not yet approved medicine. And even if the pharmaceutical company donates the drugs (though that may cost them a lot of money), someone will still need to pay for the cost for administering and monitoring the outcomes.  For example, if it's a biologic administered by infusion, patients will probably have to pay for the cost of the infusion center, and any procedures that need to be due to reactions.

So while somewhat encouraging, I'm not sure this will open new medicines to most.
Primary Sjogrens, dx June 2009, Immunoglobulin deficiency, axial spondylosis arthritis, IBS, autonomic neuropathy
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markt

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All too true on the above.  I wrote several providers and their practices a message asking how the current State law plays out at their offices... maybe we can get some more resolution shortly.  In the meantime, it really is in everyone's interest to engage their representatives on this matter.

Speaking of representation,  I wonder what the SSF is doing for us on that front?!

markt

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I suppose an incentive for pharmaceutical developers is that more folks (large numbers typically excluded due to study design criteria) would be able to participate in ongoing trials due to this law, and thus more data on clinical end-points...and possibly quicker times to market where they can begin to recoup investments.

Carolina

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  • —if it ain't one thing, it's another." Roseanne R.
There will be no reining in of any corporations by the Federal Government in the next 3 years.  Do not hold your breath.

Regards, Elaine
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markt

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Come now, that's a bold statement considering the previous administration's FDA allowed Mylan Pharmaceuticals to price-fix the Epi-Pen (epically at that).   Or what about the FDA's lack of challenge to Allergan transferring to the Saint Regis Mohawk Tribe the rights to Restasis (Cyclosporine) in a deal to protect it from patent challenges and keep prices inflated.  The  Obama era FDA wasn't exactly friendly to a lot of patient communities, but that is neither here nor there.  Atleast today, they are trying to make Right to Try Federal Law (if Senate Dems will eventually allow it)... for that I am thankful.  The President spent about 10 minutes advocating for it during his State of the Union...  so it does have some forward inertia.

https://www.reuters.com/article/us-allergan-patents-mohawk/allergan-pays-mohawk-tribe-to-protect-patents-from-challenges-idUSKCN1BJ2DK

The number one rule I have learned from being a Naval Officer:  You will never be successful if you put your fate or conditions of success solely in other peoples hands.  (i.e. Own your circumstances as much as possible, and advocate for yourself, your own interests (in this case, our health, etc.).  Otherwise, you will be on the sideline lamenting, watching others and wondering why others have it better.

Edit*  On another note, I contacted the SSF to see what there level of awareness and advocacy for this legislation is.  Unfortunately, it's next to nil... and that should not be.  All of the folks I made inquires to did not even know who to pass me on to for further discussion, and had had little awareness of the State Law/Federal Bill.  So that's unfortunate to know that the folks who are supposed to be advocating for us, are missing a big opportunity.  Again, that goes back to my previous statement.  (Until SSF leadership catches on). 
« Last Edit: March 21, 2018, 08:26:11 AM by markt »

Linda196

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I'm afraid this discussion is skirting political content, so please keep comments pertinent to the types of drugs becoming available and what you see as a need for them. the developing companies responsibilities and rights, and those of the patients, without comment on the political background or undercurrent if possible.

If this thread deteriorates to a political comparison discussion, it will be removed.
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SjoGirl

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As requested politics aside, there seems to me a need for better advising regarding drug options in general. I have SjS, positive markers, sero-negative RA, OA, neuropathy, etc. It's not just one drug I need to know about, but combinations of drugs and alternative treatments that might prove beneficial.

My rheumy is, fortunately, working with me on drug choice and treatment as are my PCP and neurologist. That said I still feel like the systems are too bi-frucated, docs don't talk to each other (even my PCP and rheumy who are with the same organization), I have to keep each of them informed. I even had to go to my PCP during a recent horrific pain flair to ask which drug  or drugs I should take (many options on the table).

I would also argue that we need better medication advising. Years ago I heard a person speak who had the title Consulting Pharmacist. He is a pharmacist who advises people about drug options that have been recommended by docs, provides info about other drugs that might work or be available, etc.

Finally, I know there are compounding pharmacies because I use one. I would love to know which drugs might be able to be combined so I don't have to take so many different ones.
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warmwaters

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I suppose an incentive for pharmaceutical developers is that more folks (large numbers typically excluded due to study design criteria) would be able to participate in ongoing trials due to this law, and thus more data on clinical end-points...and possibly quicker times to market where they can begin to recoup investments.
Actually, the data for those who didn't meet the study design can't be included, unless they are negative results. If you are trying a new drug and lots of people have bad side effects, that's worth noting. But you can't count the positives, because you are not starting from the same baseline of characteristics.
Primary Sjogrens, dx June 2009, Immunoglobulin deficiency, axial spondylosis arthritis, IBS, autonomic neuropathy
Omeprazone DR 40 mg, mobic 15 mg, Plaquenil, LDN, B1, B6, B12, D, fludrocortisone, gralise, various inhalers