Rep. Diana DeGette (D-CO) (Alex Brandon/AP Images)
Reps. Fred Upton (R-MI) and Diana DeGette (D-CO) on Tuesday unveiled the second iteration of their mammoth 21st Century Cures Act, a wide-ranging, mostly pro-pharma law signed by former President Obama that pushed the FDA in certain directions on real-world data and other hot button issues.
This time around there’s a provision to fund a $6.5 billion (on par with Biden’s request) research center at NIH to speed new treatments and make riskier investments. Similar to DARPA, the new NIH division to be known as ARPA-H, would be run by a small group of program managers with more latitude to pursue high-risk, high-reward projects.
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For years, paper-based processes and individual point solutions dominated the clinical research landscape, and patient participation in clinical trials was largely an in-person engagement. But when the COVID-19 pandemic took a stronghold, traditional clinical trial methods emerged as inadequate, putting clinical trials and the life sciences industry at a crossroads. Practically overnight, the industry had to rapidly shift to decentralized clinical trial methods, while maintaining data quality and regulatory compliance.
Al Sandrock (Biogen via Youtube)
Two years after Al Sandrock jumped from CMO to the top post in R&D — and just months after the hyper-controversial approval of the experimental Alzheimer’s drug aducanumab (Aduhelm) — Sandrock is planning to step out of his long career at Biogen.
Late Monday evening the big biotech put out word that Sandrock, a longtime fixture in the company after a 23-year stint, is hitting the exit.
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A few weeks after Jennifer Doudna introduced CRISPR/Cas9 genome editing to the world, one of her old students decided to take the central part of the biology-altering invention and kill it.
CRISPR/Cas9, as the name implies, is a two-part system: a string of letters called a guide RNA, that says where to cut the DNA. And an enzyme, Cas9, that does the cutting. Often compared to molecular scissors, it was the first system that allowed researchers to cut DNA with ease and precision, promising potential cures for genetic diseases such as sickle cell and cystic fibrosis.
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The House Energy & Commerce Committee began marking up a dozen bills on Wednesday morning including one that would require the FDA to craft a five-year action plan for fostering the development of drugs that improve or extend the lives of people living with rare neurodegenerative diseases.
Rare neurodegenerative diseases, like amyotrophic lateral sclerosis or ALS, have been historically very difficult to treat. But this bipartisan bill, introduced by Rep. Mike Quigley (D-IL), will provide $100 million for each of fiscal years 2022 through 2026 to help HHS award grants to facilitate access to investigational drugs that diagnose or treat ALS.
Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Valeriano Di Domenico)
Following an oral explanation held at the November meeting of the EMA’s human medicines committee, Biogen received ‘a negative trend vote’ on its marketing application for its controversial Alzheimer’s drug aducanumab, the company said Wednesday morning.
The setback is just the latest in a string of negatives — from an anemic launch to rejection of coverage from the VA — since the surprising June approval of the drug by the FDA that led to multiple resignations from an advisory committee that unanimously rejected it.
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Chiquita Brooks-LaSure (Photo by Caroline Brehman/CQ Roll Call via AP Images)
Although sales of Biogen’s expensive new Alzheimer’s drug have been anemic since the approval in June, the prospect of CMS eventually paying for it opens up a billion-dollar can of worms, and already has the agency defending some premium and deductible increases for seniors.
CMS explained late Friday that Medicare Part B will have to increase its standard monthly premium — from $148.50 in 2021 to $170.10 in 2022 — in part because of the massive spending that could occur should the agency sign off on a national coverage decision for the drug, known as Aduhelm, and its $56,000 annual price tag next year.
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Patients and families tell their Parkinson’s disease stories in new Acadia and StoryCorp campaign (via Acadia/StoryCorps))
Acadia Pharmaceuticals is putting the power of storytelling to work for Parkinson’s disease. While storytelling in general is often used in pharma marketing to help explain the realities of health conditions, Acadia is teaming up with the storytelling powerhouse StoryCorps for the new campaign.
Called ‘Yours, Truly,’ the work centers on pairs of people in poignant conversations relaying the stories of their own or their loved ones’ Parkinson’s experience.
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The US Securities and Exchange Commission has launched a probe into claims that Cassava Sciences, an Austin-based drug developer, manipulated data key to its case for its experimental Alzheimer’s drug simufilam, the Wall Street Journal reported Wednesday.
The report comes just two days after Cassava in an SEC filing revealed that ‘certain government agencies’ had asked the biotech for documentation. It wasn’t clear which agencies were inquiring or what information they sought, and Cassava went out of its way to say the requests weren’t accusations of wrongdoing.
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A cell containing smallpox viruses, as captured by transmission electron microscopy
Smallpox was declared eradicated in 1980 by the World Health Organization, after an effort to vaccinate the globe. But several vials labeled as the disease were found at a Pennsylvania vaccine research facility belonging to Merck, when a laboratory worker was cleaning out a freezer.
There’s no indication that anyone was exposed to the vials, the CDC told CNN in an email. The lab worker was wearing gloves and a face mask, and the FBI is now investigating.
https://endpts.com/cures-2-0-bipartisan-house-bill-would-allow-rwe-to-satisfy-confirmatory-trial-requirements-for-accelerated-approvals/
