The main reason this therapy isn't available is because the FDA has decided that the risks of gene therapy aren't worth the benefit of curing color blindness. I agree with that evaluation, but I also think mavericks should be allowed to try curing their own colorblindness (assuming they had informed consent and paid for the therapy themselves).
But huge changes like a major color might overcome that. Humans learn to recognize new major objects they've never seen before.
If colourblindness glasses (e.g. EnChroma®) work for you, then I suppose the answer is trivially "yes, because it already does" – and we should expect gene therapy to be useful. If there are people for whom those glasses don't do anything at all, I'd be more cautious.
I mean, what's stopping you? This guy cured his own lactose intolerance: https://www.youtube.com/watch?v=J3FcbFqSoQY
But I'm guessing — in part because you say "paid for the therapy" — that you don't really mean "should be allowed to cure their own colorblindness"; but rather "should be allowed to buy a colorblindness treatment from a vendor of such" or maybe "should be allowed to request a prescription of a colorblindness treatment from a doctor who acquires it from a vendor of such."
And you're already also allowed to do that — in theory. The FDA doesn't care about people buying medical treatments. It cares about companies selling them. Because companies are profit-focused, and the quickest way to money in medicine is to lie.
For you to be able to get what you want, there'd have to be an intermediate category that stops just shy of the current "FDA-approved medical therapy" category. For a treatment to qualify for this category, it would still have to pass both safety and efficacy studies. The only thing that would be different, is that the FDA wouldn't weigh the safety vs. the efficacy of the treatment at the end; but instead just put an absolute minimum threshold on both safety and efficacy, and leave the prescriber to determine whether the safety risk outweighs the efficacy.
But think about that word "prescriber." This would certainly allow people motivated to do this to go to their doctors and ask for such treatments specifically. But it would also enable doctors to choose such therapies for their patients, without the patient being previously aware of the treatment option.
And doctors can be bought. (See: doctors prescribing on-patent drugs when equally-effective generics are available; doctors adding on unnecessary adjunct therapies because a sales rep convinced them they should "give it a try"; etc.)
Do you want to live in a world where there's no ability for regulatory bodies to set a safety:efficacy-ratio threshold on a doctor's ability to prescribe drugs to patients? A world where a (bad) doctor might prescribe a drug that has a huge risk for very little chance of success, and all they have to do is put a consent form in front of the patient for the huge risk — without even having to explain the low-efficacy part?
(Honest question. Some people might very well prefer such a world.)
My wife is a neurologist. She deals with this. I’m an anesthesiologist; I just give more as needed because my patients don’t take their own drugs. Almost everything I give is generic, and the exceptions are when those fail.
You can make a very good living as a physician in the US without being a whore. Not worth my moral sense to make a few more dollars that won’t go to me directly anyway. Sure as hell not worth it to her; she’s a hospital employee.
https://www.pharmacytimes.com/view/debunking-a-common-pharma...
Still, these measured %ages should be much more explicit in the actual delivered drugs and in the range of values that are determined safe. There are plenty og drugs where double dose is fine and the therapeutic dose is "let's experiment on every patient evey day to see what works".
Antiepileptics are a very special category of drugs in that respect - they really do need to be right on target every time. I know a neurologist with epilepsy - not my wife - and he takes branded Zonegran. Thyroid hormone supplements are similar although the consequences are much lower (you're not going to accidentally kill anyone in a car wreck if your thyroid hormone is low).
Certainly, but I was mostly referring here not to doctors who actually make more money due to pharma companies; but rather to doctors who are "lobbied" / "direct marketed" to by pharma companies, using steak dinners, free samples (implicitly, to dispense directly to patients without insurance coverage — big in e.g. psychiatry), exclusivity arrangements with HMO boards, and so forth.
There's also the propaganda level: full-page ads in industry publications, biased (but technically unaffiliated) guest speakers presenting at industry conferences as a condition of sponsorship of said conferences, etc.
(My own wife is an optician. If you ignore frames and just focus [heh] on lenses, the optical industry is essentially a specific instance of the "medical device" industry. The number of people from the lens companies who come in to "educate" staff on "relevant use-cases for" their new technologies, but who end up instead trying to feed them specific marketing spiels, is ridiculous. The quantity of submarine product pitches I see disguised as factual content when I flip through her trade magazines is also ridiculous. And the industry-sponsored content that she had to wade through as part of her degree was downright abominable.)
> So if you have a legal, generic drug here, it might be a 120% one, and your next refill might be an 80% one - you just got a 1/3 reduction in dose without warning.
I think you're speaking here of cases where a generic medicine is prescribed directly, which can get resolved to any old (white-label, store-brand?) formulation of the drug on each refill, or especially if you switch pharmacy.
But I was (clumsily) trying to motion at the idea of doctors prescribing an on-patent drug (that can only be manufactured by one company) vs. prescribing any specific brand of a sufficiently-effective, off-patent drug (where the lack of patent creates competition as many major pharma companies release their own formulations, which then lowers the price for all brands' versions of the drug, not just for the generic / white-label ones.)
This is specifically relevant in the case of patent lifetime extension — i.e. the classic https://www.youtube.com/watch?v=I0VIOQ4OpD8 — though also then see https://www.reddit.com/r/patentlaw/comments/16dyvfi/drug_pat.... Regardless of who's responsibility this phenomenon is, it does happen — doctors prescribing (expensive) X+1 rather than (now-cheap) X, because X+1 is now the standard of care... even though X was fine last week.
In this case, though, it's:
> We have this new drug, whose strict required dosing regimen and annoying, long-lasting guaranteed withdrawal effects mean that your patients will be very unlikely to stop taking it... ever... or even to switch medications once they start on it, without your repeated insistence. (They can exponentially taper the drug, but we will never provide instructions on how to do that, nor fund studies on the success rate of doing that. Your medical education will have taught you a linear tapering procedure, that does not work at all for avoiding this drug's withdrawal effects.)
> Here's some free samples that you can use to hook your poorest patients on our drug — you know, the ones you'd normally prescribe generics so that their not-very-good insurance plans will cover them, or so that they can afford them out-of-pocket. We'll be back with more pretty frequently, so if you don't have very many patients, you may just be able to treat some of your poorest patients indefinitely from free samples alone. (But we know this won't work out for you in the long term, as we've calculated what would be enough for a prescriber operating at patient capacity, and we shoot for maybe 1/3rd of that. It'll only seem like a good idea when you're just starting a new practice.)
> Oh, and we also do have an Assistance Program, because we know the insurance carriers refuse to pay for our drug (since it's not considered to be any more effective than the generics... just yet. Our lobbying hasn't fully kicked in yet.) Our Assistance Program will allow those same poorest patients to continue to pay for the drug out-of-pocket once you've hooked them with the free samples, rather than having them convert to the generic. They'll be paying at a painful — but reduced to just doable! — rate. We'll cover the rest. (And still make a profit!)
> It is, of course, a needs-based Assistance Program. We aren't in the business of slicing into our own (huge) profit margin for charity. We're in the business of helping people get over medical stumbling blocks that might be preventing them from being productive, and thus preventing them from getting a high-earning job. Our data says that our Assistance Program acts as an amazing self-funded channel partnership for us, in that it manufactures high earners pre-primed to buy our drug. And of course, we can bilk them for as much profit as we want.
This might not reflect your specialty — this is from my experiences in psychiatry.
But should I be able to do that? Am I capable of evaluating the risks? Am I capable of even enumerating the various risk factors? This is not my field at all. Maybe the person making this for me is doing it in an unsafe manner, in a way that could result in dangerous contamination. How would I know?
This Reddit thread has some interesting insight into just how dangerous it could have been: https://www.reddit.com/r/videos/comments/7x8x3q/dude_uses_ho...
This is why I'm leaning toward a "no" here... I probably shouldn't be legally allowed to pay someone else to make me something like this, regardless of what sort of informed consent form I sign.
In what way do you agree with that evaluation then? The FDA certainly doesn't force anyone to undergo a treatment. And my understanding was the FDA doesn't decide who pays for a given treatment. In particular I don't think they define what medical insurance companies (or Medicare/Medicaid) have to pay for, do they? I think they just say whether the treatment is ethical to be performed at all. So when you say that mavericks should be allowed to try it, I think you're simply disagreeing with them?