It feels arbitrary because much of it is. Part of the issue is that choosing patients who are doing functionally "well" (defined by RECIST scores, which include things like : can you climb a flight of stairs? perform ADL's?) and have no signs of end organ damage or other comorbidities selects for patients who will produce fewer confounding symptoms and side effects that would worsen data. "Better" data means better study outcomes, which also means a higher likelihood of a medication being given a fast tract distinction and ultimately FDA approval. And that is money. The trials are really for the drug companies and for future patients, not for the patients currently enrolled in the trial. The patient actively in the trial has the opportunity to be helped as a side effect but not a primary endpoint. Though, if they can get into a phase 2 study with efficacy as an endpoint then it is trying to help them in order to help others, which is a better position to be in.
The truth is there is also a lot of arbitrariness. For example, brain mets are often used as an exclusionary factor for studies, because, although a drug is evaluating mets as part of asking "will this drug work for cancer throughout the body?" a drug company will LATER have a study that focuses on whether it works on brain mets, thus excluding a huge swatch of patients. If a cancer tends to met to the brain, it really doesn't need a different study. It needs to include real-world patient populations. But that's because the studies are meant to get a drug to market.
I feel strongly that policies need to change. The FDA plays a big role in making the process problematic (a longer point for another time), but, curiously, the NIH actually is one of the most functional (for patients) research centers.
Comments
It feels arbitrary because much of it is. Part of the issue is that choosing patients who are doing functionally "well" (defined by RECIST scores, which include things like : can you climb a flight of stairs? perform ADL's?) and have no signs of end organ damage or other comorbidities selects for patients who will produce fewer confounding symptoms and side effects that would worsen data. "Better" data means better study outcomes, which also means a higher likelihood of a medication being given a fast tract distinction and ultimately FDA approval. And that is money. The trials are really for the drug companies and for future patients, not for the patients currently enrolled in the trial. The patient actively in the trial has the opportunity to be helped as a side effect but not a primary endpoint. Though, if they can get into a phase 2 study with efficacy as an endpoint then it is trying to help them in order to help others, which is a better position to be in.
The truth is there is also a lot of arbitrariness. For example, brain mets are often used as an exclusionary factor for studies, because, although a drug is evaluating mets as part of asking "will this drug work for cancer throughout the body?" a drug company will LATER have a study that focuses on whether it works on brain mets, thus excluding a huge swatch of patients. If a cancer tends to met to the brain, it really doesn't need a different study. It needs to include real-world patient populations. But that's because the studies are meant to get a drug to market.
I feel strongly that policies need to change. The FDA plays a big role in making the process problematic (a longer point for another time), but, curiously, the NIH actually is one of the most functional (for patients) research centers.