Expanded Access Summit

Congressional Hearing on funding for #ExpandedAccess in #ALS taking place right now. We'll be fact checking the whole way. energycommerce.house.gov/committee-acti…

@maxwellsmith In the U.S., people won't get vaxxed because they're told it's good for them or good for public health. They'll do it because it's required at every workplace, store, airport, or gathering. As you say, it's a choice, not a mandate.

@maxwellsmith Liberty is not counterpositioned to safety in this matter. A true libertarian supports an organization's right to protect its workforce, customers, guests, and attendees by denying entry to unvaxxed people. The public health "mandate" concept is misleading and problematic.

@bsw5020 @US_FDA Here's how EA helps research trials, as we presented at NEALS #ALS forums 2013-2016. #ExpandedAccess programs, if made visible in advance, assure research trial participants that they'll have option to continue w/ active drug upon completion of study. Recruitment AND Retention.

In seeking to slow down #ACTforALS, @US_FDA has repeated the mantra that allowing expanded access will harm clinical trials for promising therapies. There’s only 1 problem with that mantra: it’s not true. Don’t take my word for it, listen to 2 leading ALS clinical trialists👇.

@momwithwheels @Michell62614519 @bsw5020 @US_FDA @HELPCmteDems @SenBlumenthal @SenatorHick @SenJackyRosen @timkaine @SenSanders @SenTinaSmith @SenatorHassan @SenatorLujan @ChrisMurphyCT @DrWoodcockFDA FDA's position and @DrWoodcockFDA position on #ExpandedAccess has been excellent. The OP may be referring to the oft-repeated motto "the surest way to bring a new treatment to the most people is through well-controlled clinical trials", which (falsely) implies conflict with EA.

@Michell62614519 @bsw5020 @US_FDA @HELPCmteDems @SenBlumenthal @SenatorHick @SenJackyRosen @timkaine @SenSanders @SenTinaSmith @SenatorHassan @SenatorLujan @ChrisMurphyCT Why does the @US_FDA keep saying that Expanded Access hurts clinical trials in #ALS if they then turn around and offer it to treat other diseases? Why is ALS being discriminated against by the FDA? @DrWoodcockFDA

Despite some criticism, business owners said the overwhelming majority of people applauded them for requiring vaccines upon entry. businessinsider.com/business-owner…

@gregggonsalves Although, comparing to seatbelts in cars is vulnerable to a libertarian counterargument, because failing to buckle up only harms the decision-maker. In contrast, failing to get SARS-COV2 vax harms the rest of us by prolonging the pandemic & putting other individuals at high risk

@gregggonsalves @RonDeSantisFL @GovAbbott We absolutely agree on this one!

@ProfRobHoward @gregggonsalves @US_FDA @icer_review @MadhavThambiset Yes, our name reflects our dedication to access - to modern healthcare, to investigational treatment options, and to the learning that can be generated by integrating research into widespread care practice. Call us crazy, but we think access is the key to a lot of good things.

@gregggonsalves @Expanded_Access @US_FDA @icer_review @MadhavThambiset It’s worse than that. I guess a clue to this account’s motivation lies in the name.

@gregggonsalves @US_FDA @icer_review @MadhavThambiset @ProfRobHoward Interpret it how you want. Misrepresent it if you want. But our comments are clear, and they're consistent with the law and with FDA's flexibility around treatment access in diseases where flexibility is needed most.

@gregggonsalves @ProfRobHoward Luckily the second is not a precondition for the first. Nevertheless we know the history, and there are many cases of EAP-generated datasets contributing substantially to the body of knowledge on the particular treatment and disease, perhaps more-so in cancer EAPs than AIDS.

@nadia_sethi Ha! It took me a while too. Here is the trick: Don't pronounce all 7 syllables. Say: Neuro-De-GenRative. We should have provided this to the Committee members today!

Feeling like we need an easier-to-say term than “neurodegenerative”.

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.

Congressional Hearing on funding for #ExpandedAccess in #ALS taking place right now. We'll be fact checking the whole way. energycommerce.house.gov/committee-acti…

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.

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