This article discusses the issues surrounding allowing patients with horrific prognoses to take unproven, or weakly proven, drugs. It makes me think we need to move from a black-and-white system where a drug is either approved or not to something more complex. For example, one tier for solidly proven treatments, and another for treatments with limited evidence, but no better substitutes. I support the right of people with horrific prognoses, such as Alzheimer's disease, ALS, or ME, to try experimental treatments as long as they're informed of the risks. And yes, ME has a horrific prognosis. Some would consider being imprisoned in your home for half a centry, sometimes in pain or unable to leave your bed, while the whole world refuses to even recognize you're sick, to be just as bad dying of cancer.
This sentence stands out for me: 'Still, I’m deeply concerned about what I see as a trend toward prioritizing access to unproven medical products over gathering evidence that they safely work.' The solution to the problem of drugs not being tested rapidly and reliably is to get rid of the patent system. Most drugs arise from independent academic research and then get bought by companies that test and license them only when it suits them and block others from doing it. Rather than messing about with laws about what companies can do governments should be funding the trials themselves rather than pouring money into the pockets of industry. But as things are there is no place for making things easier to provide untested treatments. Doing trials is perfectly possible if you really want to know if the treatment works.
You bring up a good point I missed. If we give early access more liberally, drug companies may never bother proving their treatments actually work, which will hold back medicine in the long run. I'm personally uncomfortable with Aduhelm and Relyvrio receiving approval in the way they did. Full FDA approval is seen as an endorsement, and these drugs don't deserve that. I followed Aduhelm more closely, and the evidence for that one is weak as wet cardboard. These decisions have costs. Here in the US, the government takes some money out of your disability or retirement check to pay for your healthcare, and it's higher this year because they expected to have to pay for Aduhelm.
I get it - the bigger issue is open information where such things have been taken and by whom, trials focus on homogenous somewhat and if you were trying a hail Mary without a trial then it is quite different if a clinician can find out how x number of people in similar circumstances to yourself have fared from taking something. What is the state of this type of information exchange? I know a worry with trials is only reporting the positive, but I would assume/hope that in actual clinical situations for most conditions (I doubt this for ME where inaccurate stuff is written due to the dodgy BPS license allowing for 'interpretation' of physical phenomena) bad is reported as much as good it re: outcomes and side effects? We absolutely do need some sort of register for ME in the absence of yellow card and due to the heterogeneity I think in order for this to be possible and possibly give a clinician something to hang their hat on if someone is asking them to take such a gamble?
Your check personally? I'm sorry I don't know what these drugs are, are they ME-related and so they just took it out assuming you might ask for it?
No, these drugs aren't ME-related. One is for Alzheimer's and another is for ALS. Not my check personally. The fact that the gov't expected to have to give millions of people Aduhelm caused the Medicare part B premium to increase significantly for everyone this year. Awkwardly, Medicare later decided they won't pay for the drug outside of trials so hopefully it will go down again next year.
