Clinical Trials: they are not designed only to be a “last resort”

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.

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.

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.

I'm in a clinical trial at the NIH in Bethesda, MD for the drug Olaparib ( or Lynparza) for my acinar cell pancreatic cancer. Started early June and the drug has had a positive effect on the remaining CT visible tumor on my liver. I asked the Dr. in charge of the 2 yr. trial that if I continued to see progress until the end of the trial, and that if Olaparib had still not been approved by the FDA for acinar cell pancreatic cancer treatment, would I still be able to get the drug? The Dr. told me that she could write some type of letter to the VA, (I'm a disabled veteran), that would allow the VA to issue me the drug. So, as I understand your post, she would actually send the IP-IND letter the FDA on my behalf? I'm just trying to understand the process before I head back up the NIH on Sept. 24. Thank you for your time and any input.

I was the first patient in the US on the RucaPANC trial conducted from 2014-2016 testing the biosimilar PARPi to Olaparib called Rucaparib (Rubraca). I have a germline BRCA2 mutation and acinar cell carcinoma. I was the longest person in the trial and when the trial was terminated, had a complete response from this PARPi. The Principal Investigator who conducted the trial wrote an “Individual Patient-Investigational New Drug” protocol and applied to the FDA to allow me to continue on the drug under “Compassionate Need”. The request was approved in less than 24 hours resulting in no interruption in dosing. The company that was developing the drug sought approval for ovarian and prostate cancers for which it was approved. With a small market for pancreatic and Astra Zeneca having the pancreatic cancer market, the company did not look to spend limited resources on pancreatic when their next targeted market was breast cancer.

The company declared bankruptcy and the rights to Rubraca was bought by a company based in Europe. The former company and now the new company has been providing the PARPi since the trial ended in April 2016. I have taken it since and is very well tolerated. October 10 will make 10 years on the drug.

Your situation may even be simpler. Physicians can prescribe a medication approved for another use as “Off-label drug” use mwaninf that a drug that’s been approved by the FDA for one purpose is used for a different purpose that has not been approved. The provision in FDA regulations allows a doctor to prescribe the FDA approved drug for another purpose. The FDA regulates the testing and approval of drugs, but not how doctors use drugs to treat their patients.

The difference in my situation is PARPi’s we’re just being tested for the first time with pancreatic cancer and the trials were accepting both PDAC and PACC forms and somatic and germline BRCA mutations. Now there is history of it working in PDAC and limited data with PDAC because it is such a rare form. I was likely the first PACC patient it was tested on and was successful.

You're a weath of information, sir. I'm feeling more assured that if I'm still here in June of '26, the end of my NIH trial, I'll be able to continue using the Olaparib. And if anyone reading this forum has pancreatic acinar cell cancer, and also has the BRCA2 mutation, the clinical trial I'm in is still looking for participants. They are looking for 20 patients but so far I'm the only one in the trial. The NIH protocol title for the trial is "000596-C: Phase II study of Olaparib in subjects with advanced Pancreatic Acinar Cell Carcinoma". The contact doctor is Christine Alewine. I don't have her ph. no. handy. I was concerned that, since this study is government funded, they might pull the plug on it due to lack of participants. Dr. Alewine said, "That's not going to happen." I'm hoping some others come forward.

I’m in the Natural History study of Dr. Alawine. I’ve talked to one of the M.D.’s in her group and a couple of nurses and they have been reviewing the medical records of my case history for the factors that may have led to the longevity and being considered an “exceptional responder” that resulted in cure. At some point in the future I will heading down to the NIH campus.

The concern in pulling the plug on a study is not the funding as that was already allocated for the study by the NCI. What results in a trial being terminated is failure to accrue the pre-determined number of participants necessary to obtain enough statistical data to make valid conclusions.

"What results in a trial being terminated is failure to accrue the pre-determined number of participants necessary to obtain enough statistical data to make valid conclusions." That's actually what I suspected when I asked Dr. Alewine the question about the trial being canceled. If that's so, I'm hoping there are enough folks reading this forum to where a few are afflicted with this rare cancer and will apply. Thanks for the input, sir.