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Expanded Access Summit @Expanded_Access

Ax-S Pharma, producer of the annual global conference on integrating pre-approval access into clinical drug development.

Joined April 2018
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Expanded Access Summit@Expanded_Access
29 Jul 2021
Congressional Hearing on funding for #ExpandedAccess in #ALS taking place right now. We'll be fact checking the whole way. energycommerce.house.gov/com…

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Business Insider
@BusinessInsider
5 Aug 2021
Despite some criticism, business owners said the overwhelming majority of people applauded them for requiring vaccines upon entry. businessinsider.com/business…
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Congressional Hearing on funding for #ExpandedAccess in #ALS taking place right now. We'll be fact checking the whole way. energycommerce.house.gov/com…

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more replies
Research trials need to be diverse, but not broadly inclusive in terms of numbers of patients. The clinical research enterprise was not created to provide a treatment option for whole populations. That's what #ExpandedAccess is for.
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More questions from Committee members about how to make #ClinicalTrials more inclusive. FDA missing a huge opportunity to educate lawmakers about #ExpandedAccess channel, which is the intended channel for humanitarian widening of eligibility for investigational treatment.
Rep Sarbanes, re Section 610 of PDUFA 6, requiring examination of trial inclusion/exclusion criteria. Ax-S Pharma: We authored some of the key language in this law to make sure the focus remained on utilizing #ExpandedAccess to increase overall inclusivity.
Cavazzoni: It's a very special disease with particularly challenging factors in clinical development. Also much greater tolerance for risk in the patient community wrt benefit/risk tradeoff.
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Castor: Guidance advises larger, secondary cohorts to explore differential response. How has industry responded to this?
Rep Burgess; is FDA utilizing data generated by the Federally funded ALS Registry? A: not prepared to answer
Rep Mastui about legislation to greater utilize "patient experience data" in neurodegenerative diseases
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Cavazzoni answer: We commented on this in the ALS Drug Dev Guidance. More patient reported outcomes. Ax-S Pharma comment: Need to explore secondary data generated from very large #ExpandedAccess programs. Not just the widening of endpoints in research trials.
@Expanded_Access: What is needed is funding for Medicare coverage of identified Investigational Treatment options in diseases like #ALS. #CostRecovery #ExpandedAccess #Healthcareinnovation
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Cavazzoni discussed more funding for biomarker research. Take that how you want.
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Upton cited ALSA recommendations for greater funding for FDA. Ax-S Comment: That's not pivotal to greater utilization of #ExpandedAccess
Butterfield: Duke ALS Clinic director has treated over 3000 patients, most of whom could not enter research trials; many of which go overseas or embark on self experimentation. Why not more encouragement of #ExpandedAccess ?
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Cavazzoni: We encourage #ExpandedAccess, but drug companies often say no.
Cavazzoni recognizes higher threshold for therapeutic uncertainty ("risk") considering the lack of treatment options and consequences of inaction.
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