4 ms·
The big way they are figuring out how to repurpose old compounds that fell out of the pipeline is by using biomarkers. We are now much more equipt at finding b
by dikdik 10y ago
The big way they are figuring out how to repurpose old compounds that fell out of the pipeline is by using biomarkers.
We are now much more equipt at finding biomarkers, generally through gene sequencing. Drugs that failed, often failed because they were applied to an entire population of a disease (ie multiple sclerosis). Now, we can subset the MS population by different biomarkers and test just that small subset for efficacy.
While testing anyone that happens to have MS would show a massive failure rate, testing 5% of the population that has specific biomarkers, we may be able to show a huge increase in efficacy and thus the drug would be approved for MS patients that fit the exact bio marker profile.
- akiselev 10y agoThere is another consequence: the FDA may allow you to classify the compound as an orphan drug based on a much smaller market than the entire disease pool. This greatly reduces the burden of getting a drug approved because orphan drugs have too few patients to justify the expense of the usual approval process and the FDA gives them special treatment for humanitarian reasons and to promote a wider field of research than just the drugs with large profit potential.
- hprotagonist 10y agobiomarker validation is just as annoying as drug testing, so you're just punting the problem one node down. it's hard work: http://blogs.sciencemag.org/pipeline/archives/2013/12/04/cancer_cell_line_assays_you_wont_like_hearing_this http://blogs.sciencemag.org/pipeline/archives/2013/12/04/can... nominal biomarkers can be wholly spurious: http://blogs.sciencemag.org/pipeline/archives/2012/04/10/biomarker_caution http://blogs.sciencemag.org/pipeline/archives/2012/04/10/bio... biomarkers that we believe to be proxies for disease conditions (like LDL) can be targeted to no effect: http://blogs.sciencemag.org/pipeline/archives/2016/12/14/simple-right http://blogs.sciencemag.org/pipeline/archives/2016/12/14/sim... etc.