Sequoia
Senior member
- Joined
- Sep 19, 2010
- Messages
- 841
- Reason
- PALS
- Diagnosis
- 08/2010
- Country
- US
- State
- Washington
- City
- Seattle
How about giving PALS the same benefit that the cancer community enjoys?
Eliminate the placebo arm. Heck you can use data from current trials of other ALS drugs where there is a placebo arm...if those patients continue to disintegrate when compared to Gilenya patients you have ample demonstration of efficacy.
Or, make the Gilenya trial and other trials an active control trial using Rilutek as the principal alternative...should be easy to demonstrate efficacy when using data from the many past years of Rilutek data.
Patients deserve to be treated humanely.
Our discussions with the FDA confirms their willingness to consider ALS treatment in a manner similar to how they handled HIV and cancer.
How about meeting with the FDA and Treat Us Now volunteers?
1- "By contrast, cancer clinical trials—for practical and ethical reasons—rarely are blinded or include placebo control groups. Most conventional chemotherapy agents have distinctive, often severe side effects that make blinded trials impractical. Moreover, many patients and researchers assert that in trials for a life-threatening condition such as cancer, placebos are inappropriate and that all participants should receive active treatment.
Indeed, placebos are rarely used in most early-phase trials, which are not usually randomized studies and are often first-in-human studies of new agents. Mary Schwartz of Newport News, VA, was diagnosed with stage IV adrenal cancer in 2004 at the age of 35. She has now been cancer free for 6 years after being treated with an investigational chemotherapy regimen in a phase II clinical trial at the NIH Clinical Center. Would she have considered enrolling in a randomized placebo-controlled trial had one been available and offered to her? Absolutely not, she said.
“Because of the rarity of my cancer and the almost certainty of fatality, I would not have chosen to participate if there had been a possibility of getting a placebo,” she said. “I would have wanted to know I was getting the investigational treatment.”
NCI Cancer Bulletin for May 3, 2011 - National Cancer Institute
2-"To satisfy ethical concerns, active-controlled trials offer the principal alternative to placebo-controlled trials. The comparison of a new treatment to one with known efficacy is valid and always interpretable provided that the objective is to demonstrate superiority in efficacy or a decrease in toxicity
Eliminate the placebo arm. Heck you can use data from current trials of other ALS drugs where there is a placebo arm...if those patients continue to disintegrate when compared to Gilenya patients you have ample demonstration of efficacy.
Or, make the Gilenya trial and other trials an active control trial using Rilutek as the principal alternative...should be easy to demonstrate efficacy when using data from the many past years of Rilutek data.
Patients deserve to be treated humanely.
Our discussions with the FDA confirms their willingness to consider ALS treatment in a manner similar to how they handled HIV and cancer.
How about meeting with the FDA and Treat Us Now volunteers?
1- "By contrast, cancer clinical trials—for practical and ethical reasons—rarely are blinded or include placebo control groups. Most conventional chemotherapy agents have distinctive, often severe side effects that make blinded trials impractical. Moreover, many patients and researchers assert that in trials for a life-threatening condition such as cancer, placebos are inappropriate and that all participants should receive active treatment.
Indeed, placebos are rarely used in most early-phase trials, which are not usually randomized studies and are often first-in-human studies of new agents. Mary Schwartz of Newport News, VA, was diagnosed with stage IV adrenal cancer in 2004 at the age of 35. She has now been cancer free for 6 years after being treated with an investigational chemotherapy regimen in a phase II clinical trial at the NIH Clinical Center. Would she have considered enrolling in a randomized placebo-controlled trial had one been available and offered to her? Absolutely not, she said.
“Because of the rarity of my cancer and the almost certainty of fatality, I would not have chosen to participate if there had been a possibility of getting a placebo,” she said. “I would have wanted to know I was getting the investigational treatment.”
NCI Cancer Bulletin for May 3, 2011 - National Cancer Institute
2-"To satisfy ethical concerns, active-controlled trials offer the principal alternative to placebo-controlled trials. The comparison of a new treatment to one with known efficacy is valid and always interpretable provided that the objective is to demonstrate superiority in efficacy or a decrease in toxicity
