WASHINGTON — A key Republican lawmaker released a new version of so-called “right to try” legislation around midnight Friday, a pivotal next step in the effort to help dying patients get access to experimental therapies.
The legislation, from House Energy and Commerce Committee Chairman Greg Walden (R-Ore.), is more limited than an earlier measure championed by Sen. Ron Johnson (R-Wis.) that passed the Senate last fall. Walden’s version would ensure that the Food and Drug Administration has more oversight of the process. Like the Senate bill, it includes liability protections for drug companies and providers who decide to use the process. Walden’s bill also limits the process to patients who are likely to die “within a matter of months” or “severely premature.”
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Maybe there are others who simply lack experience in how to make the right to try process work but we have used it allot and it works fine as written
The FDA’s EA system is a 4 armed horse that once each arm gives a thumbs-up, the horse rides. The PharmaCo is arm one they must agree to provide the drug the next is the MD they must be willing to accept the use of the drug and the new bill will help with that since it removes their liability exposure something we haven’t been concerned about up till this point. The next arm is the patient who must be given a FULL informed consent. Once those arms are intact the FDA basically signs off and it can happen in as short as 60 minutes but no longer then 24 hours if all others have signed off their paperwork sections
Once the final FDA stamp is applied the drugs get shipped and that is it
The REAL issue is this entire process is on the shoulders of the PharmaCos
Not only do they NOT get paid (non-approved drugs can’t be charged for) BUT if something happens during the experiment it could derail the drug permanently or surely for a long time while they build new clinical trials to demonstrate safety and efficacy in enough random people to overshadow the issue in the one-off sample
I personally have a rare disease and about 2 years ago we asked a PharmaCo to allow me access to their soon to be approved drug. They were more than willing to assist me as I had aided them in the past the issue was that the drug was in the time period between trials and final approval and their risk was WAY too high to risk it on a one-off trial not absolute proof that it would help me at all
After the drug was released I did gain access to it thru paid for methods and it is working OK but surely not so well so as to risk the entire drugs wellbeing for the OK results I am getting
ALL in all, we need to ask since the FDA only gets like 1K requests per YEAR do we really need a whole new LAW created by non-medical professionals to overshadow the concept and get everyone all excited for what amounts to pie in the sky possibilities. The article mentions:
“This bill will provide millions of terminally ill Americans an additional pathway to access promising investigational treatments before it’s too late” Where are these patients? I don’t see them! I see MANY people who PRAY they find such a magic elixir of life before it gets too late but there are minimal actual patients who can benefit from a drug that is enough thru the pipeline to be deemed safe enough for human testing while the patient is too sick to actually become part of a clinical trial which would give the open access to the drug
Also, who is going to pay for this access? Today it is free to them because the Emergent Access system and then in 6 months they have to pay for it. What about the InsCo who is pretty much stuck paying for it after approval and the PharmaCo before.
The fact that the PharmaCos can’t even recover costs makes them less exciting to step up and rightfully so based on the potential risk
Dr. Dave
My wife is the first pt. to testify for RTT. See ” Lorraines’ Story ” on the Goldwater RTT website. She almost died while dealing with the slow, small , ” compassionate use ” FDA system . Today she is 6 yrs. cancer free. Whatever ” deal ” Congress comes up with , my bottom – line is : The FDA should not delay for even one day a dying pt.’s chance at Life.
As a former hospice Nurse I witnessed how many doctors would not put their patients on hospice. ( prognosis of less than 6 months ) Reasons? Therefore I believe that a terminal illness, such as als, should qualify those affected with no limits on life expectancy. Who would define that? See recent studies on new thinking about how als is different in each person. The congressional bill gives me great concern. Saddened Senator Johnson’s bill was changed. He worked so passionately on that bill. Another concern is the FDA. Think about this. How many drugs are on the market today after going thru clinical trials for years and now multiple class action lawsuits? Approved by the FDA. People with als don’t have years to wait for drugs. Our system is a failure and needs to function for our American citizens. Frankie’s mom, Carol.
Dear Carol,
It appears you may be a parent of a child suffering with a rare disease. I am sorry for this struggle in your child’s life and yours. Across this country, independent advocates for patient safety believe that those with life-threatening, rare diseases, for whom no other treatment option exists, should have access to potentially effective experimental treatments. However, FDA oversight is NOT a roadblock to that access. Through the existing pathway of the FDA’s Expanded Access program, 99.7% of the approximate 1,000 requests received each year are approved. Further, FDA involvement provides protection against false claims of potential efficacy as well as placing caps on the amount drug companies and physicians can charge for this access. Right to Try legislation has typically not provided any of these protections for a population that already lives with great suffering. Advocates for patient safety believe access should be possible, but not without the minimum safeguards already in place through Expanded Access. Contrary to the opinion of those pushing the Right to Try legislation, the current pathway works. Not only is Right To Try legislation unnecessary, the proposed solutions raise the risk of increased harm and suffering for patients and families who already carry a heavy burden.