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Clinical Trials: they are not designed only to be a “last resort”
Pancreatic Cancer | Last Active: Sep 13 7:47am | Replies (42)Comment receiving replies
Thank you for the explanation. Some of it makes sense, but some of it is troubling. You described two drugs that were efficacious in the healthier, stage II participants. Did these patients continue taking the drug and maintain results or was the treatment stopped because the drug was not able to miraculously cure sicker and less healthy patients? Theoretically, if a drug was able to cure a disease within a certain age, gender, or racial category but was less effective for the general population, would it be denied FDA approval based on statistical requirements? Younger and younger people are being diagnosed with early on-set cancers and they tend to be healthier than older and elderly patients who are diagnosed towards the end of their life spans, yet drug development, seeming based on traditional trial specifications and likely projected market sales, requires a significant cure-all for all patient populations. I strongly feel like there's a serious problem here.
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The individuals who did benefit from the drugs (RPI-613 and SM-88) which did not go on to becoming FDA approved remained on those drugs under a provision of the FDA called “Compassionate Need”. At the conclusion of the trail, the principal investigator of the trial writes an Individual Patient-Investigational New Drug (IP-IND) protocol and sends it to the FDA. The review is done within 24 hours of submission. After FDA approval, the developer of the drug provides it at no charge to the patient for as long as it works. So no patient that benefitted from an investigational new drug is deprived.
In the trial I was in, my drug was not submitted to the FDA for approval. The target of overall response was not achieved. I had an exceptional response to the drug and my oncologist wrote an IP-IND protocol submitted to the FDA which approved it in less than 24 hours. The drug manufacturer has provided the medication to me for almost 10 years and continues to do so. In return, data from my diagnostic tests and exams is shared with them. A new entity has purchased the rights to the drug so there is the possibility they could do further improvement of the drug and apply to the FDA for approval to market it in the future.