It is a known fact that clinical trials are very selective when creating their selection criteria for who to include. (That is not a grammatical sentence but its early in the day here.) What I am trying to say is that if you do not fit their criteria, you will not be included. I have so many ailments, I have never been included in a clinical trial. They don't want me.
Hop on over the clinicaltrials.gov and see if you would fit their criteria for any. They are very specific. Basically they are trying to determine toxicity, dosage and more. An abbreviated version of the official government description of the different stages are:
"Phase 0: Exploratory study involving very limited human exposure to the drug, with no therapeutic or diagnostic goals (for example, screening studies, microdose studies).
Phase 1: Studies that are usually conducted with healthy volunteers and that emphasize safety.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition).
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing. These including postmarket requirement and commitment studies that are required of or agreed to by the sponsor."
In Phase 2 and 3 trials, the most common ones we hear about and participate in, are where the drug manufacturers select their groups of patients who have the disease that they hope to treat. But they routinely exclude people with other issues who might not conflict with the trials or cause side effects that could be from the new drug or from the other issues. So you have to be perfect.
A growing problem is that with the aging population as the Boomer generation about to hit their 70s, older patients are being treated with new drugs that have not been tested on their age group.
"For at least 25 years, it’s been known that drugs that work one way in an otherwise healthy younger adult might not work the same way in the elderly. Many older adults are frail and might metabolize medications more slowly or suffer unforeseen drug interactions. Blood thinners, for example, might be riskier in someone with a tendency to fall. Medications to control blood pressure could be harder to dose appropriately in a patient with poor kidney function. In 1989, the FDA set guidelines encouraging drug companies to study how their drugs affect the elderly, with the goal that the population being studied prior to drug approval should resemble the population that would ultimately take the medications. But the guideline was not a mandate...."
"...one in five trials excluded potential participants based on their age alone. In half of the remaining trials, patients were excluded from participating because of conditions that disproportionately affect older adults, such as cognitive dysfunction or limited physical activity. And only one quarter of the studies looked at outcomes that might be relevant to older adults, such as quality of life or physical function."
So for all of us imperfect people out there, you can understand the issues here with multiple health issues, never mind age, which may cause us additional problems through treatment.
Showing posts with label clinical trials. Show all posts
Showing posts with label clinical trials. Show all posts
Wednesday, May 13, 2015
Monday, April 27, 2015
Wishes granted
"If wishes were horses, beggars would ride."
Just over 2 years ago, I blogged about Darlene Grant's video when she was dying of stage IV breast cancer and wanted the FDA to give permission for her to get Perjeta so she might live a little longer. Perjeta was not yet available and she needed special permission to get it.
When I blogged about it, my basis was that she was wishing for things that weren't there. There was no guarantee that it would work. Cancer patients are optimistic about potential treatments because sometimes it comes to grabbing at any straw that comes our way.
But I must say I am absolutely delighted to watch this video this morning. She got Perjeta and she is alive and well today. She has started a non profit to help breast cancer patients.
So while Darlene is clearly riding these days, she is not out of the woods yet. But she did get her wish and she is still here. She will be closely supervised for the rest of her life but looks to count her time in years and not in days as she did in 2012.
Just over 2 years ago, I blogged about Darlene Grant's video when she was dying of stage IV breast cancer and wanted the FDA to give permission for her to get Perjeta so she might live a little longer. Perjeta was not yet available and she needed special permission to get it.
When I blogged about it, my basis was that she was wishing for things that weren't there. There was no guarantee that it would work. Cancer patients are optimistic about potential treatments because sometimes it comes to grabbing at any straw that comes our way.
But I must say I am absolutely delighted to watch this video this morning. She got Perjeta and she is alive and well today. She has started a non profit to help breast cancer patients.
So while Darlene is clearly riding these days, she is not out of the woods yet. But she did get her wish and she is still here. She will be closely supervised for the rest of her life but looks to count her time in years and not in days as she did in 2012.
Tuesday, April 7, 2015
So far good news but
Pfizer has developed a new drug called palbociclib (until it gets renamed by some team of marketing geniuses through some in depth market research and then gets a color, fancy packaging and more) that has provided some positive results in a small phase two clinical trial. I am not writing this in terms of an overwhelmingly positive result because it isn't.
It is for one of the most common breast cancers - ER positive, Her2 negative - when they have become recurrent or metastatic. In some headlines the media has gotten hold of this and pumped up the results to be something else but we must remind ourselves of a couple of things before we run in circles thinking that it is that huge a game changer:
Breast cancer specialists who were not involved in the study are cautiously optimistic, as am I. It was not a blind study meaning that the doctors who ran it knew who received the new drug. And it was so small.
It is for one of the most common breast cancers - ER positive, Her2 negative - when they have become recurrent or metastatic. In some headlines the media has gotten hold of this and pumped up the results to be something else but we must remind ourselves of a couple of things before we run in circles thinking that it is that huge a game changer:
- It was a clinical trial of 165 women.
- Of those 165, many had their dose reduced because of side effects 13% dropped out of the study because of side effects.
- But if you ask the doctor who ran the study, the drug was generally well tolerated.
Breast cancer specialists who were not involved in the study are cautiously optimistic, as am I. It was not a blind study meaning that the doctors who ran it knew who received the new drug. And it was so small.
"A big question is whether Pfizer will be able to win approval of the drug based on this study. The Food and Drug Administration normally requires larger Phase 3 studies, but sometimes makes exceptions for drugs for cancers and other life-threatening illnesses.
If Pfizer can get early approval, the drug could probably reach the market next year. If the company must complete a Phase 3 study, which is already underway, approval might be delayed a couple of years, according to the ISI Group.
Garry Nicholson, president of Pfizer’s oncology division, told analysts on Sunday that the company “can envision the possibility” that the data from the Phase 2 trial would be sufficient for approval. He said the company had not gotten far enough in its discussions with the F.D.A. to be able to decide whether to seek approval now, however."
Pfizer wants the approval so they can start marketing it asap. And their investors aren't happy as it didn't live up to the initial expectations of 26.1 months vs. 7.5 months presented halfway through the trial. So now they have more pressure to get it on the market.
The FDA has previously given approval for drugs after Phase 2 trials but not always successfully? Avastin is a good example. It was approved for breast cancer after Phase 2 trials and then pulled from the market three years later, in 2011, because it showed it did not work after all. On the other hand, other companies have other breast cancer drugs in development.
So while I am happy to learn about this good news, I am hesitant because I firmly believe it needs more testing. This result is just too small to show us enough. Its not ready yet. We must have patience here.
Tuesday, January 20, 2015
Exclusivity of clinical trials
I understand why pharmaceutical companies are insistent on their strict criteria. They want to make sure if they take a group of similar people they can monitor for effectiveness and adverse effects. If they have a very diverse group, it can be much harder to determine the results.
The FDA is interested in changing this policy and forcing companies to include patients with chronic conditions (possibly me?) in clinical trials.
"Pharmaceutical companies routinely exclude the sickest patients from studies, fearing complications they may suffer from the drug candidates, but, as a result, the studies don’t provide a glimpse of the treatment’s “real world” effect.
To counter this, the FDA recently issued a memo to its new drug reviewers asking them to work with drug manufacturers to include a broader population in trials more regularly.
“The whole point of this is that if there really are differences in response — either favorable or unfavorable — among subsets of the population, what clinicians need is to know about it, so they can either watch for them or use a different drug,” Bob Temple, MD, deputy director for clinical science at the FDA’s Center for Drug Evaluation and Research, told reporters Friday. “This adds to the information available.”"
I find it to be a shame that so few Americans participate in clinical trials but then so many are told that they are eligible. With my medical history, no one ever wants to include me. I for one am looking forward to this change.
Sunday, January 18, 2015
Wishes granted
"If wishes were horses, beggars would ride."
Just over 2 years ago, I blogged about Darlene Grant's video when she was dying of stage IV breast cancer and wanted the FDA to give permission for her to get Perjeta so she might live a little longer. Perjeta was not yet available and she needed special permission to get it.
When I blogged about it, my basis was that she was wishing for things that weren't there. There was no guarantee that it would work. Cancer patients are optimistic about potential treatments because sometimes it comes to grabbing at any straw that comes our way.
But I must say I am absolutely delighted to watch this video this morning. She got Perjeta and she is alive and well today. She has started a non profit to help breast cancer patients.
So while Darlene is clearly riding these days, she is not out of the woods yet. But she did get her wish and she is still here. She will be closely supervised for the rest of her life but looks to count her time in years and not in days as she did in 2012.
Just over 2 years ago, I blogged about Darlene Grant's video when she was dying of stage IV breast cancer and wanted the FDA to give permission for her to get Perjeta so she might live a little longer. Perjeta was not yet available and she needed special permission to get it.
When I blogged about it, my basis was that she was wishing for things that weren't there. There was no guarantee that it would work. Cancer patients are optimistic about potential treatments because sometimes it comes to grabbing at any straw that comes our way.
But I must say I am absolutely delighted to watch this video this morning. She got Perjeta and she is alive and well today. She has started a non profit to help breast cancer patients.
So while Darlene is clearly riding these days, she is not out of the woods yet. But she did get her wish and she is still here. She will be closely supervised for the rest of her life but looks to count her time in years and not in days as she did in 2012.
Tuesday, January 13, 2015
Only perfect people need apply
It is a known fact that clinical trials are very selective when creating their selection criteria for who to include. (That is not a grammatical sentence but its early in the day here.) What I am trying to say is that if you do not fit their criteria, you will not be included. I have so many ailments, I have never been included in a clinical trial. They don't want me.
Hop on over the clinicaltrials.gov and see if you would fit their criteria for any. They are very specific. Basically they are trying to determine toxicity, dosage and more. An abbreviated version of the official government description of the different stages are:
"Phase 0: Exploratory study involving very limited human exposure to the drug, with no therapeutic or diagnostic goals (for example, screening studies, microdose studies).
Phase 1: Studies that are usually conducted with healthy volunteers and that emphasize safety.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition).
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing. These including postmarket requirement and commitment studies that are required of or agreed to by the sponsor."
In Phase 2 and 3 trials, the most common ones we hear about and participate in, are where the drug manufacturers select their groups of patients who have the disease that they hope to treat. But they routinely exclude people with other issues who might not conflict with the trials or cause side effects that could be from the new drug or from the other issues. So you have to be perfect.
A growing problem is that with the aging population as the Boomer generation about to hit their 70s, older patients are being treated with new drugs that have not been tested on their age group.
"For at least 25 years, it’s been known that drugs that work one way in an otherwise healthy younger adult might not work the same way in the elderly. Many older adults are frail and might metabolize medications more slowly or suffer unforeseen drug interactions. Blood thinners, for example, might be riskier in someone with a tendency to fall. Medications to control blood pressure could be harder to dose appropriately in a patient with poor kidney function. In 1989, the FDA set guidelines encouraging drug companies to study how their drugs affect the elderly, with the goal that the population being studied prior to drug approval should resemble the population that would ultimately take the medications. But the guideline was not a mandate...."
"...one in five trials excluded potential participants based on their age alone. In half of the remaining trials, patients were excluded from participating because of conditions that disproportionately affect older adults, such as cognitive dysfunction or limited physical activity. And only one quarter of the studies looked at outcomes that might be relevant to older adults, such as quality of life or physical function."
So for all of us imperfect people out there, you can understand the issues here with multiple health issues, never mind age, which may cause us additional problems through treatment.
Hop on over the clinicaltrials.gov and see if you would fit their criteria for any. They are very specific. Basically they are trying to determine toxicity, dosage and more. An abbreviated version of the official government description of the different stages are:
"Phase 0: Exploratory study involving very limited human exposure to the drug, with no therapeutic or diagnostic goals (for example, screening studies, microdose studies).
Phase 1: Studies that are usually conducted with healthy volunteers and that emphasize safety.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition).
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing. These including postmarket requirement and commitment studies that are required of or agreed to by the sponsor."
In Phase 2 and 3 trials, the most common ones we hear about and participate in, are where the drug manufacturers select their groups of patients who have the disease that they hope to treat. But they routinely exclude people with other issues who might not conflict with the trials or cause side effects that could be from the new drug or from the other issues. So you have to be perfect.
A growing problem is that with the aging population as the Boomer generation about to hit their 70s, older patients are being treated with new drugs that have not been tested on their age group.
"For at least 25 years, it’s been known that drugs that work one way in an otherwise healthy younger adult might not work the same way in the elderly. Many older adults are frail and might metabolize medications more slowly or suffer unforeseen drug interactions. Blood thinners, for example, might be riskier in someone with a tendency to fall. Medications to control blood pressure could be harder to dose appropriately in a patient with poor kidney function. In 1989, the FDA set guidelines encouraging drug companies to study how their drugs affect the elderly, with the goal that the population being studied prior to drug approval should resemble the population that would ultimately take the medications. But the guideline was not a mandate...."
"...one in five trials excluded potential participants based on their age alone. In half of the remaining trials, patients were excluded from participating because of conditions that disproportionately affect older adults, such as cognitive dysfunction or limited physical activity. And only one quarter of the studies looked at outcomes that might be relevant to older adults, such as quality of life or physical function."
So for all of us imperfect people out there, you can understand the issues here with multiple health issues, never mind age, which may cause us additional problems through treatment.
Wednesday, January 7, 2015
So far good news but
Pfizer has developed a new drug called palbociclib (until it gets renamed by some team of marketing geniuses through some in depth market research and then gets a color, fancy packaging and more) that has provided some positive results in a small phase two clinical trial. I am not writing this in terms of an overwhelmingly positive result because it isn't.
It is for one of the most common breast cancers - ER positive, Her2 negative - when they have become recurrent or metastatic. In some headlines the media has gotten hold of this and pumped up the results to be something else but we must remind ourselves of a couple of things before we run in circles thinking that it is that huge a game changer:
Breast cancer specialists who were not involved in the study are cautiously optimistic, as am I. It was not a blind study meaning that the doctors who ran it knew who received the new drug. And it was so small.
It is for one of the most common breast cancers - ER positive, Her2 negative - when they have become recurrent or metastatic. In some headlines the media has gotten hold of this and pumped up the results to be something else but we must remind ourselves of a couple of things before we run in circles thinking that it is that huge a game changer:
- It was a clinical trial of 165 women.
- Of those 165, many had their dose reduced because of side effects 13% dropped out of the study because of side effects.
- But if you ask the doctor who ran the study, the drug was generally well tolerated.
Breast cancer specialists who were not involved in the study are cautiously optimistic, as am I. It was not a blind study meaning that the doctors who ran it knew who received the new drug. And it was so small.
"A big question is whether Pfizer will be able to win approval of the drug based on this study. The Food and Drug Administration normally requires larger Phase 3 studies, but sometimes makes exceptions for drugs for cancers and other life-threatening illnesses.
If Pfizer can get early approval, the drug could probably reach the market next year. If the company must complete a Phase 3 study, which is already underway, approval might be delayed a couple of years, according to the ISI Group.
Garry Nicholson, president of Pfizer’s oncology division, told analysts on Sunday that the company “can envision the possibility” that the data from the Phase 2 trial would be sufficient for approval. He said the company had not gotten far enough in its discussions with the F.D.A. to be able to decide whether to seek approval now, however."
Pfizer wants the approval so they can start marketing it asap. And their investors aren't happy as it didn't live up to the initial expectations of 26.1 months vs. 7.5 months presented halfway through the trial. So now they have more pressure to get it on the market.
The FDA has previously given approval for drugs after Phase 2 trials but not always successfully? Avastin is a good example. It was approved for breast cancer after Phase 2 trials and then pulled from the market three years later, in 2011, because it showed it did not work after all. On the other hand, other companies have other breast cancer drugs in development.
So while I am happy to learn about this good news, I am hesitant because I firmly believe it needs more testing. This result is just too small to show us enough. Its not ready yet. We must have patience here.
Subscribe to:
Posts (Atom)