Europe votes for clinical trial transparency(alltrials.net) |
Europe votes for clinical trial transparency(alltrials.net) |
http://en.wikipedia.org/wiki/Bad_Pharma
http://en.wikipedia.org/wiki/Bad_Science_%28book%29
I wonder if you can get "I am a Nutritionist!" T-shirts?
[Edit: Thanks for pointing out the T-Shirt pretty much does exist].
Congrats to ben goldacre and everyone else who worked on this!
Getting all the data on drug efficacy, availability, correlate it with chemical structure, combine it with the information on other drugs could potentially help us a lot in predicting better working future drugs and the workings of existing drugs.
Also, transparency. No more picking out those trials that worked and conveniently forgetting about those that did not work.
[1] https://www.sciencemag.org/content/321/5886/263.abstract
One of the oft repeated justification for the absurd levels of IP protection and patent extensions that pharma enjoys is the huge cost of trials. But if huge development costs are the reason for these protections to exist, guess what, these costs will continue to remain huge. Its just self perpetuating.
Patents are primarily a preemptive measure against a hypothetical scenario. The hypothesis is that unless market pressures are cordoned off, innovation will stop. It is a believable hypothesis but not a quantitative one, and this exactly where we need a quantitative one. How many years exactly does one need to subvert the market so that a satisfactory level of innovation is maintained ? Nobody knows. In these scenario what we need is a feedback system, and pretty much the only scalable and fair apparatus that we have for such feedback on economic affairs is the market.
So rather than subverting the market, market should be explicitly and actively involved to work out this trade-off. How exactly this is to be done needs to be worked out.
There could be a futures market on patents on drugs undergoing testing: competitors can promise to buy the patent to the (drug,usecase) pair at a particular price (even if its a dud) and if the owner decides not to sell the owner pay a particular compensation. Such measures will spread the risk. There should be mechanisms for a company to raise money for testing in lieu of rights over the product.
The initial period of validity of a patent should be short, like 3~6 years, following which the patent is on the market. Entities can bid to bring the patent to public domain. The owner(s) bids to retain it. Whoever wins gets the balance. Of course the "bring it to public domain" bidders will bid less because profitability is less when its in the public domain, but market decides who gets to own it. If the owner(s) think they can still extract lots of profit from the drug, bid high. We should stop handing out blank checks without a feedback loop, that is just bad design.
There are some rules trying to get all new trials registered, but the rules aren't followed at all and the FDA doesn't enforce them.
1. You are right that typically you need two registrational trials for approval and yes you can have many trials that show it doesn't work.
2. You are incorrect that you don't need to show it works against a disease. Sometimes you can show that you are impacting a biomarker (which I assume is what you mean by "some number"), but there must be evidence that changing the biomarker impacts the disease. Without that evidence you will not get approval.
3. You are incorrect you only need to show its effective for up to six weeks. If that were true, why would drug companies be running multi-year trials for cholesterol drugs? You typically need to show its effective for however long the drug is taken, but a follow-up period is also required.
That is not correct. When you submit an NDA to the FDA, you need to include every piece of clinical trial data you've obtained so far. This includes trials that you've either ended early or failed to show any effect at all.
The FDA doesn't look kindly upon a company that hides any clinical trial data.
How many years exactly does one need to subvert the market so that a satisfactory level of innovation is maintained ?
We have some data on that, but we can't predict the future. I can't find the stat right now, but only about 1 in 3 drugs approved by the FDA actually produce an overall positive return.
I'm pretty sure a "trial" is a statistically significant study of many patients. So in this case, your analogy to the coin is flawed. A better analogy would be x groups of 10,000 coin flips, and to take the average of each group in x. Now, no such average would favor the less probably side of the coin.
Good luck with that. Explaining complex cellular mechanisms to a lay person without any scientific nor statistical background is a very difficult task. When you consider modern medicine, you need basic knowledge in genetics, biochemistry, biology, proteins, drug formulation, pharmacology, pharmadynamics, toxicity, clinical studies design, to have a proper understanding of what a study means. We're not talking about aspirin class of medicines anymore, and more and more drugs are now in the class of large molecules.
Even required publication is no cure for this; a result saying "we inadvertently broke the blind" or "the protocol was poorly implemented" is perfectly possible even in good faith.
Next comes mom and then apple pie.
Given the limited powers of the parliament do they carry any more weight than politicians' pre-election promises?
I don't know where you live 3 billboards on my way to work are now taken up with advertisements on behalf of our local MEP. She had been all but invisible previously.
As I said in a comment lately she has recently been on the radio here claiming credit for another measure that promises much but has been criticised for being easily sidestepped.
>Good luck with that. Explaining complex cellular mechanisms to a lay person without any scientific nor statistical background is a very difficult task.
I don't think that the cellular mechanisms, or generally speaking the mode of action, are what has to be explained understandable to a lay person. What has to be explained is the result of the trial.
Also, what do they tell people who take part in trials - I'd be pretty annoyed if I took part in a trial and someone told me "we can't tell you because with your humble CS education you wouldn't understand it".
Its sort of like in the bad ol' days of computing - you used to have to know all kinds of arcane things just to be able to get some numbers crunched. Nowadays, if you want, you can look at the source for your OS .. this is the same effect that needs to happen in the pharmaceutical industry, and I for one look forward to the reduction in sheer Power over peoples Lives that the pharma mega-industry has had, as people start to realize that there is a lot of smoke and mirrors in what they're being told 'is a solution to their problems .. for $99/month' ..
Still hard, obviously, and plenty of space for semantic whitewashing.
You can explain everything if you have the patience and will to break it down to pieces for others to understand.
Here[1], try and see if you can understand it. I'm sure you can, I'm sure your grandmother can too. Also if you have any specific questions about pharmacology, molecular biology or anything related feel free to ask, I might be able to come with a reasonably simple answer.
3) C.f. SSRIs, and Robert Whitaker's book anatomy of an epidemic.
The best example I can give you right now are the drugs in development to treat Duchenne's muscular dystrophy. These drugs work by increasing the levels of a biomarker called dystrophin. The FDA recently came back and said "nope, not enough evidence that increasing dystrophin will improve patient's conditions, therefore we will not approve your drug".
As for the length of depression trials, it has been established that 6-week trials provide enough evidence to support an improvement in patients. [1]
Therefore, a 12-week trial is not necessary for all older patients; rather, the degree of improvement in the first 4 to 6 weeks identifies patients who are highly likely to benefit from continuing antidepressant treatment as well as those who very probably should have their treatment regimen altered at that point.
However, the depression drugs also run trials for long-term use: "Cymbalta is indicated for the treatment of major depressive disorder (MDD). The efficacy of Cymbalta was established in four short-term and one maintenance trial in adults [see Clinical Studies (14.1)]."
Of course the layman is not supposed to understand that, that is no explanation in layman's terms. You can make these sentences understandable by generalizing it: "We already knew before that the effect of these types of drugs is very specific to the specific genetics of a patient. Now in this trial we tried to find out what specific drug works for what specific genetic combination. The result is that Drug A works better than B for a combination that is called XY: Whether or not that applies to you can be found out by <test method>."
In other words, it won't help laymen in the end.
At one point, if you really want to understand the clinical trial results, you need to go in details, and laymen won't be able to do that. So I'm not sure what they are trying to accomplish here.
Plus, you don't need that laymen language in the first place. Isn't that the job of journalists who cover scientific discoveries ? I'm afraid we hit another hurdle here, with the deplorable state of clinical trials results reporting in the general public by the media. That says a lot about what you can expect from laymen in terms of scientific comprehension.
Yes, that's the problem with study subjects as complex as humans :) My academic background is in educational science and things are even fuzzier there: there never is a simple treatment->result relation because the persons receiving treatment reflect on that treatment, they are an active part in it.
> In other words, it won't help laymen in the end.
I think I know what you're meaning, but that's the point where I disagree: In my opinion this is no either/or situation but rather a question of relative amount of insight that can be given to laymen: Currently they will usually understand 0% of the scientific abstract. If we assume that a laymen's version of that abstract loses 80% of the information but is 100% understandable, that would still be a win.
> Plus, you don't need that laymen language in the first place. Isn't that the job of journalists who cover scientific discoveries ?
Absolutely, as journalists are already used to do exactly that. In practice the result will probably be exactly that: the institution will have science journalists who will produce these laymen's abstracts.
Plus, phase I trials are really limited in size, so they are meaningless versus the total population of people who go on clinical trials for phase 2 and 3 - and these are patients always.