AI
Saturday, December 10, 2022 5:13:59 PM
Thanks for your comment about Pazdur.
Pazdur is an exceptional source. He is leading the FDA’s OCE into a new era.
There is new guidance involving RWE including ECAs, but since you prefer not to consider draft documents, I have included the existing final guidance below first. The full-context FDA source link is provided followed by two paragraphs excerpted in full mentioning crossover and ECAs.
While historical ECAs are recognized by the FDA as valid where appropriate, it is noteworthy that the murcidencel P3 utilized contemporaneous or concurrent studies. The studies were matched by an independent expert firm. Some studies ended far earlier than the murcidencel study, but began in the same cohort timeframe.
https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/cfrsearch.cfm?fr=314.126#:~:text=314.126%20Adequate%20and%20well%2Dcontrolled,placebo%20effect%2C%20or%20biased%20observation
Notwithstanding the FDA’s recognition of ECAs, is important to note that murcidencel’s approval does not depend solely on ECA data. Furthermore, all RAs are likely to support this novel cell-based technology, but murcidencel does not depend on any single RA as there are many RAs worldwide.
Data collected from ECA comparison only comprise a fraction of the clinical efficacy data that exist for murcidencel today.
Murcidencel has been studied in three trials—only one of those three relied on an ECA. All clinical data are relevant to a regulatory application including those data gathered from other studies, interim analyses, and compassionate use. All three trials have produced substantial safety and efficacy data, and two of the three clinical trials ran for over a decade with survivors alive today.
Below are some additional sources. Read as much as you can to get an accurate sense of the FDA’s stance overall as a public service institution. They are careful, but also want drugs to be accessible to those who need them.
Aside from the well-established existing FDA guidance cited above, Real-World Evidence is a vastly significant topic to research and explore. The FDA’s shift toward RWE includes a renewed and enlightened position on ECAs which were and are already recognized as valid in settings where ECAs are the most feasible and ethical option. I hope we will bring further light to this topic in our discussions on iHub.
https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-use-real-world-data-and-real-world-evidence-support-regulatory-decision-making-drug
https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/cfrsearch.cfm?fr=314.126#:~:text=314.126%20Adequate%20and%20well%2Dcontrolled,placebo%20effect%2C%20or%20biased%20observation.
https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track
https://www.fda.gov/drugs/news-events-human-drugs/fda-approval-demonstrates-role-real-world-evidence-regulatory-decision-making-drug-effectiveness
https://jamanetwork.com/journals/jamaoncology/fullarticle/2798847
Pazdur is an exceptional source. He is leading the FDA’s OCE into a new era.
There is new guidance involving RWE including ECAs, but since you prefer not to consider draft documents, I have included the existing final guidance below first. The full-context FDA source link is provided followed by two paragraphs excerpted in full mentioning crossover and ECAs.
While historical ECAs are recognized by the FDA as valid where appropriate, it is noteworthy that the murcidencel P3 utilized contemporaneous or concurrent studies. The studies were matched by an independent expert firm. Some studies ended far earlier than the murcidencel study, but began in the same cohort timeframe.
https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/cfrsearch.cfm?fr=314.126#:~:text=314.126%20Adequate%20and%20well%2Dcontrolled,placebo%20effect%2C%20or%20biased%20observation
Notwithstanding the FDA’s recognition of ECAs, is important to note that murcidencel’s approval does not depend solely on ECA data. Furthermore, all RAs are likely to support this novel cell-based technology, but murcidencel does not depend on any single RA as there are many RAs worldwide.
Data collected from ECA comparison only comprise a fraction of the clinical efficacy data that exist for murcidencel today.
Murcidencel has been studied in three trials—only one of those three relied on an ECA. All clinical data are relevant to a regulatory application including those data gathered from other studies, interim analyses, and compassionate use. All three trials have produced substantial safety and efficacy data, and two of the three clinical trials ran for over a decade with survivors alive today.
Below are some additional sources. Read as much as you can to get an accurate sense of the FDA’s stance overall as a public service institution. They are careful, but also want drugs to be accessible to those who need them.
Aside from the well-established existing FDA guidance cited above, Real-World Evidence is a vastly significant topic to research and explore. The FDA’s shift toward RWE includes a renewed and enlightened position on ECAs which were and are already recognized as valid in settings where ECAs are the most feasible and ethical option. I hope we will bring further light to this topic in our discussions on iHub.
https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-use-real-world-data-and-real-world-evidence-support-regulatory-decision-making-drug
https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/cfrsearch.cfm?fr=314.126#:~:text=314.126%20Adequate%20and%20well%2Dcontrolled,placebo%20effect%2C%20or%20biased%20observation.
https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track
https://www.fda.gov/drugs/news-events-human-drugs/fda-approval-demonstrates-role-real-world-evidence-regulatory-decision-making-drug-effectiveness
https://jamanetwork.com/journals/jamaoncology/fullarticle/2798847
Bullish
Believe carefully. This is the greatest and most powerful lesson that I have learned since arriving on Earth. Examine what you believe about yourself most importantly, and then believe carefully as you interact with the world.
