Have you ever heard of the Abigail Alliance?
Abigail Alliance
Abigail was a lovely 21 year old woman who had cancer, but current therapies weren't effective for her. As awful as that was, the really painful twist is that the medical establishment victimized her further; she was denied a drug that blocked EGFR which was in trials while she was fighting for her life-and her tumor expressed EGFR. This drug was already proven to be safe in stage I/II trials- but it wasn't yet through the stage III trials to prove it was effective.
Her doctor tried to get her in the trial but she didn't fit the entry criteria. And the FDA doesn't allow people to have safe (proven in stage I/II trials) but not yet proven effective (stage III) drugs or treatments.
She died waiting for the drug.
Does this remind you of the struggle to get CCSVI treatment?
Currently medical policy makers think the responsibility to is protect our concept of providing what is termed "evidence based medicine", meaning the system makes therapies available only after the trial system has proved it's safety then usefulness. In this arrangement the keepers of this evidence are the people who carry out studies (mostly researchers funded by PhRMA) and the people who review it after it's done (FDA and journals). We have granted these bodies the right to decide for everyone else if/when research meets the quality standard and the findings are reliable.
The system is set up so that we don't allow patients access to therapies until all stages of trials are finished; stage I to prove safety, stage II for safety and initial reveiw of efficacy, and stage III with larger numbers of trial participants to prove efficacy followed by approval. At the end of this process we assume others with the same diagnosis will have results similar to the "average" person in the trial.
If doctors try to apply treatments outside this system for an individual patient they are discouraged with both regulatory sanctions from the FDA, sanctions through their local medical board, and pressure from peers.
This culture encourages conformity with the evidence based approval process no matter the cost to individual patients even if they die when no approved treatment works for them. This is considered the unfortunate price we pay as a society for safe and effective medicine for the population as a whole.
The culture also ignores concerns about the lack of objectivity or bias in the commercial evidence base that skews the findings in favor of commercial products. There acts as if the evidence base is utterly objective and can predict what every single patient should take for their disease. And it acts as if these treatments are always available in a timely manner.
This creates a system that is also hostile to paradigm shifts and may be inhibiting or even suppressing investigation of non-commercial but potentially promising treatments.
Patients are discouraged from daring to question the system and admonished that only evidence based mediciine is permitted. They are told questioning the wisdom of a system that insists patients are all alike and standard treatments are always effective is somehow "unscientific."
But patients who are deteriorating in spite of the best treatments available know very well that "proven" treatments don't always work. They are not sanguine about quietly slipping through the EDSS while they wait for science to work through drawn out processes especially when something is already known to be safe but its barrier to use is that it has not proven effective in a large group trial.
One option for these patients is the provision the FDA has made under "Compassionate" and "Humanitarian" Use--This would allow for use of a device in an off-label manner, for a patient who is without other options. A case could be made for this patient if certain conditions are met. For more information- link
Things are changing all over the medical world and people are asking if lack of science proving efficacy is really a good reason to deny people the right to try it and see if it helps them. Since people fail standard therapies all the time--and these therapies are supposedly proven "efficacious" -- why not let such people try something "outside the box" when their back is against the wall?
Is not the ultimate goal of all this science to make a difference to individuals? And when individuals have run out of options, is it really society's business to say "You can't have that safe therapy because WE don't agree it could help you?" Isn't it always up to the patient and his doctor to decide if something was effective for THIS person in THIS situation, whether proven for that diagnosis or not?
In light of this, think about venoplasty treatment--something that is relatively safe and readily available in the US for blood flow issues with millions of procedures done worldwide every year- but not yet approved for MS because we don't know if it will reduce disability (and we won't know that for years).
Arguably, autologous (your own) stem cells as an MS treatment is the same boat because stem cell transplants are commonly used in cancer treatment (thus safe) but using stem cells to aid in regenerating nerve tissue has not been proven effective for MS in FDA approved trials.
Safe therapies that have not yet been proven efficacious in new applications like these might benefit from a bill advanced by the Abigail Alliance that has been languishing in congress; HR 4732.
Link to HR 4732
Let's keep this bill front and center and ask our congress people to support this. Adults who are losing their battle with a life altering disease deserve to have all posible options available to them. Everyone agrees that in a perfect world all therapies would be fully and perfectly tested before use, but since the only trial that REALLY matters is the one in which the patient tries a therapy himself, shouldn't all safe therapies be on the table?
Marie
