Thank you for another outstanding piece following your thorough summary of the Vinay Prasad travesty. You might think that “science journalists” dedicated to the FDA beat might have at least a cursory understanding of trial design. What a pathetic display of ignorance. Thanks for making your voice heard.
Thank you. Let’s hope Marty and Jay don’t leave. Very very frightening and saddening. Your writing is very good and I appreciate all the time that you and the other Substack cardiologist who spend on non-cardiology issues and cardiology issues. Thank you for your time, talent, and treasure. Keep up the fight.
Why would a patient who has already progressed agree to be randomized? The randomized trial you (and the FDA) are suggesting will be hard to recruit for and maybe impossible. There’s also the ethical question of randomizing patients to standard of care therapy that is highly unlikely to work. It’s easy to tell the company to raise money for an additional trial but actually raising the money is much harder and very time consuming. Time is the one asset that these patients don’t have.
The FDA approves drugs based on non-RCTs all the time. The wise (and decent) decision would have been the decision that a Janet Woodcock led FDA surely would have made; approve the drug and require a post-marketing study. At the very least, instead of asking for a prospective, randomized trial, the FDA could have accepted historical controls as it sometimes does for rare diseases. No one thinks that historical controls are perfect but the advent of EMRs, especially at academic medical centers, has made segmenting these controls much better than it once was. For advanced PD-1 refractory melanoma, there are detailed and reliable data sets from academic centers and oncology networks that could be used to formulate a viable historical control group. It seems to me that the insistence on a prospective, randomized comparator is simply unethical in this particular case.
I think that there’s little doubt that the FDA in the previous administration would have approved this oncolytic virus therapy. They would have been right. The decision of the current FDA Administrator is ill-conceived.
By the way, it’s not just the Wall Street Journal and CNBC who are criticizing the FDAs decision on the Replimune drug, many of the physicians who treat these patients find the FDAs decision perplexing at best and irrational at worst.
Great piece.
This company is an abomination and an embarrassment. As are all the “journalists” carrying water for it.
Thank you for another outstanding piece following your thorough summary of the Vinay Prasad travesty. You might think that “science journalists” dedicated to the FDA beat might have at least a cursory understanding of trial design. What a pathetic display of ignorance. Thanks for making your voice heard.
Thank you. Let’s hope Marty and Jay don’t leave. Very very frightening and saddening. Your writing is very good and I appreciate all the time that you and the other Substack cardiologist who spend on non-cardiology issues and cardiology issues. Thank you for your time, talent, and treasure. Keep up the fight.
Wide spread virtually corruption in medicine
Worse part is the educational promotes “PROTOCOL’ and no critical thinking
Ray Graf MD Interventional cardiologist
Keep writing
Why would a patient who has already progressed agree to be randomized? The randomized trial you (and the FDA) are suggesting will be hard to recruit for and maybe impossible. There’s also the ethical question of randomizing patients to standard of care therapy that is highly unlikely to work. It’s easy to tell the company to raise money for an additional trial but actually raising the money is much harder and very time consuming. Time is the one asset that these patients don’t have.
The FDA approves drugs based on non-RCTs all the time. The wise (and decent) decision would have been the decision that a Janet Woodcock led FDA surely would have made; approve the drug and require a post-marketing study. At the very least, instead of asking for a prospective, randomized trial, the FDA could have accepted historical controls as it sometimes does for rare diseases. No one thinks that historical controls are perfect but the advent of EMRs, especially at academic medical centers, has made segmenting these controls much better than it once was. For advanced PD-1 refractory melanoma, there are detailed and reliable data sets from academic centers and oncology networks that could be used to formulate a viable historical control group. It seems to me that the insistence on a prospective, randomized comparator is simply unethical in this particular case.
I think that there’s little doubt that the FDA in the previous administration would have approved this oncolytic virus therapy. They would have been right. The decision of the current FDA Administrator is ill-conceived.
By the way, it’s not just the Wall Street Journal and CNBC who are criticizing the FDAs decision on the Replimune drug, many of the physicians who treat these patients find the FDAs decision perplexing at best and irrational at worst.