Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) (Olivier Douliery/Sipa USA/Sipa via AP Images)

Un­like­ly duo leads bi­par­ti­san Sen­ate bill to ex­pand Right to Try in­to MD­MA and psilo­cy­bin

Sens. Cory Book­er (D-NJ) and Rand Paul (R-KY) like­ly don’t share much in com­mon when it comes to pol­i­cy views, but that didn’t stop the pair from in­tro­duc­ing a new bill this week that would al­ter the con­tro­ver­sial Right to Try Act to al­low ter­mi­nal­ly ill pa­tients to ac­cess Sched­ule I drugs af­ter a Phase I tri­al has been com­plet­ed.

Build­ing off its pre­de­ces­sor, the bill would skirt around the FDA’s ex­pand­ed ac­cess pro­gram and the DEA’s reg­u­la­tions around con­trolled sub­stances, and al­low pa­tients to po­ten­tial­ly ac­cess Sched­ule I drugs — like MD­MA and psilo­cy­bin — much more eas­i­ly, as the sen­a­tors say these drugs “have shown ex­cep­tion­al promise in treat­ing a va­ri­ety of men­tal health con­di­tions, in­clud­ing sui­ci­dal de­pres­sion, anx­i­ety, and PTSD.”

But as with Right to Try, the FDA is not the bar­ri­er that many as­sume it to be when it comes to ac­cess­ing in­ves­ti­ga­tion­al drugs. The agency signs off on more than 99% of all ex­pand­ed ac­cess re­quests it re­ceives. It al­so re­mains un­clear how many have at­tempt­ed and failed to gain ac­cess to Sched­ule I sub­stances out­side of clin­i­cal tri­als or FDA’s ex­pand­ed ac­cess pro­gram.

Al­i­son Bate­man-House

Al­i­son Bate­man-House, as­sis­tant pro­fes­sor at New York Uni­ver­si­ty and an ex­pert on ex­pand­ed ac­cess, ex­plained to End­points News how this bill builds on oth­er state ex­pan­sions of RTT leg­is­la­tion, even as any doc­tor with the ap­pro­pri­ate li­cens­ing can pre­scribe con­trolled sub­stances.

“This is not an at­tack on the FDA, but just on reg­u­la­tions in gen­er­al, as they don’t care who’s reg­u­lat­ing when it comes to bar­ri­ers to ac­cess,” Bate­man-House said.

But she said she has heard that there’s been a lot of in­ter­est from pa­tients around clin­i­cal tri­als test­ing MD­MA and psilo­cy­bin, al­though some of the larg­er play­ers in this space al­ready have ex­pand­ed ac­cess pro­grams set up.

The Wis­con­sin-based Usona In­sti­tute, for in­stance, pro­vides cGMP psilo­cy­bin for clin­i­cal stud­ies and has a process set up where­by el­i­gi­ble aca­d­e­m­ic re­searchers can ac­cess the sup­plies to ad­vance sci­en­tif­ic and med­ical un­der­stand­ing of psy­che­delics.

“Re­cent stud­ies sug­gest that MD­MA and psilo­cy­bin could rep­re­sent an enor­mous ad­vance­ment in men­tal health and psy­chophar­ma­col­o­gy,” Book­er said in a state­ment. “Un­for­tu­nate­ly, many el­i­gi­ble pa­tients who ur­gent­ly need care do not cur­rent­ly have ac­cess to these promis­ing ther­a­pies.”

Com­pan­ion leg­is­la­tion will be in­tro­duced in the House by Reps. Earl Blu­me­nauer (D-OR) and Nan­cy Mace (R-SC).

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sanofi, Re­gen­eron boast PhI­II win with Dupix­ent in COPD, clear­ing first bar for ex­pan­sion

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.

Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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Alaa Halawa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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In­cyte wins ac­cel­er­at­ed ap­proval for PD-1 in rare skin can­cer

Incyte touted an accelerated approval for its PD-1 retifanlimab in a rare skin cancer on Wednesday, roughly a year and a half after the drug suffered a rejection in squamous cell carcinoma of the anal canal (SCAC).

Retifanlimab, marketed as Zynyz, was approved for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC), a fast-growing skin cancer typically characterized by a single, painless nodule. It’s roughly 40 times rarer than melanoma, according to the nonprofit Skin Cancer Foundation — but incidence is growing, particularly among older adults, Incyte said in its announcement.

FDA re­jects Ab­b­Vie's in­fu­sion ther­a­py for Parkin­son's, re­quests more in­fo on pump de­vice

The FDA rejected AbbVie’s 24-hour infusion therapy for Parkinson’s, saying it needs more information on a device used to administer the treatment before it can clear it.

The Chicago-area drugmaker said in a press release that the complete response letter from the agency didn’t include any requests for more efficacy or safety trials related to the drug, known as ABBV-951. The company said it aims to “resubmit the NDA as soon as possible.”

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A new study finds that many patient influencers are sharing prescription drug experiences along with health information.

So­cial me­dia pa­tient in­flu­encers ‘danc­ing in the gray’ of phar­ma mar­ket­ing, more clar­i­ty need­ed, re­searcher says

It’s no surprise that patient influencers are talking about their health conditions on social media. However, what’s less clear is what role pharma companies are playing, how big the patient influencer industry is, and just how is information about prescription drugs from influencers relayed — and received — on social media.

While University of Colorado associate professor Erin Willis can’t answer all those questions, she’s been researching the issue for several years and recently published new research digging into the communication styles, strategies and thinking of patient influencers, many of whom partner with pharma companies.

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Drug short­age so­lu­tions brought be­fore Sen­ate Home­land Se­cu­ri­ty com­mit­tee

With more than 300 active drug shortages, the Senate Committee on Homeland Security and Governmental Affairs had its hands full on Wednesday with multiple experts testifying on drug shortages and possible solutions.

A picture of the shortage situation. presented by Erin Fox, an adjunct professor at the College of Pharmacy at the University of Utah, explained how some patients have died due to drug shortages, including with medication errors when substitutes were dosed incorrectly or when an emergency product was not available.

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