With the Trump Administration’s FDA being more supportive of lowering the barriers to introducing new meds do you think this novel approach will get a second chance?
The company stopped its trials of CYP 1020 (BL-1020) as it didn’t meet its end points but it passed Phase 2 and looks like a very interesting combination of both GABA agonists and D2 antagonists.
I’m not sure the FDA had much to do with it, if the company thinks it’s not gonna work, that would be the company’s decision.
There might be some improvements in how the FDA handles drug approvals, but they still have to work and the company still has to want to invest the $ in trials.
If another company thinks it could work they could possibly license it. That does happen, Denovo Biopharma has done that with a phase 3 failed glutamate modulator, they think they can develop biomarkers for determining which people it will help.
Yes Bio-markers seem to be the way BigPharma is moving atm in an attempt to pass stringent FDA clinical trials and produce more targeted therapies. I know there are a number of Alz clinical trials that are being run only on patients that meet certain bio-markers. It fines tunes the effect - an interesting development.
I wonder if there are differences in bio-markers based on gender, race or …class ( ie ability to pay . …opps down paranoia down! ) .
Heres the plan for “Operation Resub and repurpose” (jpeg below) LoL …I guess it makes sense to get maximum benefit from all the clinical trials that have been done. I wonder what else they are going to “extract” from all this stored data and apparently “failed trials” and how many proprietary tools are going to be unearthed and monetized.
Got to admire the BigPharma industry - they do play a Long Game… :)
Hopefully that “someone” is actually representative of the disease being addressed.
I thought I was being overly paranoid (not unusual …lol) but others do share some of my concerns …
" However, shifting away from gold standard endpoints toward an evolving
set of biomarkers also raises the prospect that the FDA will approve new
drugs that eventually prove that they don’t actually do anything to
affect the course of the disease, raising hopes and costing billions
without any real benefit."
There’s a couple ways of looking at biomarkers and how to use them. One is in identifying which patients would be helped by which medication. It would be like holding a clinical trial to test iron tablets for the symptom of weakness and tiredness, without understanding that only patients who were iron deficient would be helped. However, if you knew who was iron deficient, you could successfully treat the people who could be helped. So we have iron anemia blood tests and generally it’s not reccomended to just take excess iron if you’re tired.
Then there’s prevention over the course of decades, biomarkers make sense for this use as well. According to the article “… they’re likely going to continue with a new gold standard that will focus on long-term cognition alone,” frankly that doesn’t sound so bad. Probably would be useful to track other biomarkers, and I’m sure they will, but when it comes down to it, cognition is the biggie after all.
I guess I am just being a devils advocate and trying to get my head around the issues .
I hope that the FDA have in place some process that ensures the ethical linkages between bio-markers and underlying pathology - not just approving drugs on the basis of them modifying bio markers. I can see companies salivating at the thought of dusting off a bunch of failed trials and resubmitting them based on the biomarkers of the patients that responded well against the ones that didnt respond. So now we have small molecules with “new indications” - in effect new sub classes of disease that are based around the drug rather than a drug based on an existing disease.
Look I agree with concept of bio markers if they are generated from a pure science perspective - ie take a disease like Schizophrenia and survey a bunch of bio markers/genes - provide clustering/ classification based on these in relation to therapies so targeted. Cool bananas - we are on the way to Personalized Medicine - roll on brave new world its about time !
I just am a bit worried/unsure of companies retrofitting past trial data and gaining FDA approval based on rejigging statistical analysis on these bio markers.
Dunno… I cant put my finger on it…I think in some way this bio marker approach could be momentous.
Using your Iron / tiredness analogy it may not be Schizophrenia anymore - it could be “Schizophrenia - subclass biomarker xyz” which happens to be retrofitted to a drug. Also “Psychosis - Parkinson - subclass biomarker xyz” may have great overlap so we now have a new sub disease - specific to this small molecule…“Schizophrenia/Parkinson subclass biomarker xyz”.
Its inevitable really isnt it ? Look at the existing overlap of drugs for various indications. NVM …just trying to see how all this pans out …doing my head in lol …
Thanks for explaining. You are right ofcourse. Its all good…
What about the patient controls? I wonder if the Pharma companies are going to cluster the biomarkers of the placebo successes?
“Ah Miss we see you have the biomarkers of someone who responds well to a placebo - here take this brightly colored fluorescent sugar tablet that jiggles and wiggles in your mouth (patented ofcourse ) …and be cured …”
The great social battle will be as to why placebo tablets cost five times as much in the united states as elsewhere…lol
