Suggestions for Drug Approvals
14/09/10 14:41 Filed in: GenomeWeb Daily Scan
Submitted by S. Pelech - Kinexus on Tue, 09/14/2010 - 14:41.
A more adaptive system for regulatory approval of drugs has clear merit. However, it is possible that drugs used for treatment of chronic conditions might have much wider dispersion before it is realized that they can have very serious longer-term side-effects in a significant portion of users. The story with the eventually withdrawal from the market of COX-2 inhibitors like Vioxx (rofecoxib) exemplifies such a problem.
Personalized medicine is becoming more of a reality with improved genomic and proteomic tests with the discovery of larger panels of robust and reliable biomarkers. However, it will also become increasingly important to test combinations of complementary drugs that can simultaneously address the multiple defects that arise in cancer and other diseases. Monotherapy appears to produce only a transient relief for most forms of cancer, and eventual drug resistance is a common result. It appears that the opportunity for drug resistance is effectively mitigated with combination therapy, as has been so well demonstrated for the treatment of AIDS with three drugs used together.
Clinical testing of drug combinations will be extremely difficult as the number of possibilities escalates with the appearance of promising new drugs and the number of available patients for a specific trial declines with the identification of more specific genomic/proteomic profiles in which to stratify them. It may make sense for pharmaceutical companies to continue testing their drugs in large patient groups, but submit their drugs for approval with the data from those patients that shared biomarkers where there are clear efficacious results without deleterious effects.
Whatever changes in regulatory drug approvals may be forthcoming, they will need to permit a better assessment of combination therapy. Foremost, it should be clear that individual drugs are proven safe in healthy volunteers and cause no harm to intended patients. Thereafter, the beneficial synergistic effects of drug combinations applied in a rational manner can be determined.
While individual patients would still need to be tested for their unique responses to a combination of drugs, at least they can be treated based on their unique circumstances and not on findings for the general population.
Link to the original blog post.
A more adaptive system for regulatory approval of drugs has clear merit. However, it is possible that drugs used for treatment of chronic conditions might have much wider dispersion before it is realized that they can have very serious longer-term side-effects in a significant portion of users. The story with the eventually withdrawal from the market of COX-2 inhibitors like Vioxx (rofecoxib) exemplifies such a problem.
Personalized medicine is becoming more of a reality with improved genomic and proteomic tests with the discovery of larger panels of robust and reliable biomarkers. However, it will also become increasingly important to test combinations of complementary drugs that can simultaneously address the multiple defects that arise in cancer and other diseases. Monotherapy appears to produce only a transient relief for most forms of cancer, and eventual drug resistance is a common result. It appears that the opportunity for drug resistance is effectively mitigated with combination therapy, as has been so well demonstrated for the treatment of AIDS with three drugs used together.
Clinical testing of drug combinations will be extremely difficult as the number of possibilities escalates with the appearance of promising new drugs and the number of available patients for a specific trial declines with the identification of more specific genomic/proteomic profiles in which to stratify them. It may make sense for pharmaceutical companies to continue testing their drugs in large patient groups, but submit their drugs for approval with the data from those patients that shared biomarkers where there are clear efficacious results without deleterious effects.
Whatever changes in regulatory drug approvals may be forthcoming, they will need to permit a better assessment of combination therapy. Foremost, it should be clear that individual drugs are proven safe in healthy volunteers and cause no harm to intended patients. Thereafter, the beneficial synergistic effects of drug combinations applied in a rational manner can be determined.
While individual patients would still need to be tested for their unique responses to a combination of drugs, at least they can be treated based on their unique circumstances and not on findings for the general population.
Link to the original blog post.