Congressional Hearing on funding for #ExpandedAccess in #ALS taking place right now. We'll be fact checking the whole way.

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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.

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.

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.

We have a special channel called #ExpandedAccess to accommodate the massive range of patients in therapeutic area who are OUTSIDE the eligibility criteria of research trials. Bending the inclusion/exclusion of research trials will never be a meaningful solution for inclusiveness

Reg Griffith (VA) to Dr. Koroshetz (NIH), why are we so slow recruiting in #ALS trials? A: This disease area is actually pretty good in trial enrollment. Ax-S Comment: The problem isn't speed of enrolling research trials. It's about the 90% of pALS who are study-ineligible.

Castor: Guidance advises larger, secondary cohorts to explore differential response. How has industry responded to this?

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.

Rep Castor (FL) Why did FDA develop specific Guidance for ALS drug development?

Rep Burgess; is FDA utilizing data generated by the Federally funded ALS Registry? A: not prepared to answer

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.

Rep Mastui about legislation to greater utilize "patient experience data" in neurodegenerative diseases

Cavazzoni discussed more funding for biomarker research. Take that how you want.

@Expanded_Access: What is needed is funding for Medicare coverage of identified Investigational Treatment options in diseases like #ALS. #CostRecovery #ExpandedAccess #Healthcareinnovation

Upton cited ALSA recommendations for greater funding for FDA. Ax-S Comment: That's not pivotal to greater utilization of #ExpandedAccess

Rep Upton referencing 21st Century Cures Act. Neighbor died of #ALS. What can we do make treatments available faster? So that CURRENT patients aren't languishing and dying in wait.

Cavazzoni: We encourage #ExpandedAccess, but drug companies often say no.

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 ?

Cavazzoni recognizes higher threshold for therapeutic uncertainty ("risk") considering the lack of treatment options and consequences of inaction.

Rep Butterfield to Dr. Cavazzoni, why the need for new confirmatory trial in quick killer disease (#ALS) for therapeutic candidates which have demonstrated efficacy. Where is the regulatory flexibility?