The whole world watched in awe as the first two Covid-19 vaccines quickly made their way through clinical trials, offered stellar efficacy results and were quickly ushered across the FDA finish line in record time.
As a result, leaders of the FDA’s Oncology Center of Excellence wrote recently in the Cancer Journal that now is the time to leverage the lessons learned from that high-speed race and translate them into new opportunities for future oncology drug development.
Among the highlights worth repeating in cancer trials: frequent, open, clear communication and information sharing across FDA, academia, and community/industry partners, as well as the speed and agility of trial initiation, and the “collective sense of necessity,” all of which may benefit the development of new cancer therapies, Donna Rivera, associate director of pharmacoepidemiology in the FDA’s OCE, and colleagues wrote.
Offering the example of the UK’s RECOVERY trial, which swiftly brought conclusions on the merits of several Covid-19 treatments, the OCE authors called for more adaptive trial methods, common protocols and common control arms in oncology, as well as more on platform and basket trial designs, on which the agency recently offered guidance.
The FDA also explained how there’s now “ample evidence that protocols have become more complex over time and that a conscious effort to simplify will make participation more feasible for investigators, particularly when trials are conducted in the context of clinical care.”
And while cancer drug development has been slow to adopt decentralized trial methods and telehealth, due to concerns about data variability, OCE authors called to increase the flagging of remote assessments at the case-report form level to allow analyses to unlock further information on the potential effects of decentralized modifications on data variability.
The second iteration of the 21st Century Cures Act is also making its way through Congress and seeks to expand the use of real-world evidence in confirmatory trials for certain accelerated approvals.
Rick Pazdur, head of the FDA’s OCE, told Endpoints News in a statement, “Use of RWE for aid in regulatory decision-making would depend on the strength of the available evidence and its applicability to the patient population.”
The OCE authors also noted that while the use of real-world data informed and guided the pandemic response, “there is a significant distinction” between the use of RWD for descriptive purposes and using RWD to generate evidence that determines whether a drug works.
“The pandemic provides the field with a clear lesson that the benefit of RWD availability and faster analytics must be balanced with the importance of careful design and methods to create the high-quality RWE necessary to support evidence-based intervention strategies,” they wrote. “The pandemic can become a watershed moment to forge unprecedented change in health care delivery and clinical trials through a dynamic modernization effort supported by adequate infrastructure funding and carefully coordinated global stakeholder collaboration.”
Disturbed or impaired sleep has been the subject of increased research attention in recent years, as both a primary disorder and in cases where disturbed sleep is a symptom of another disease. Disturbed sleep is common in people with arthritis, atopic dermatitis, Alzheimer’s Disease, Parkinson’s Disease, and many other conditions. Research is focused on measuring and diagnosing these sleep disorders, as well as the development of new treatments to improve sleep across a broad range of indications. Wearable devices provide a low-burden and remote approach to objectively quantify people’s sleep in their real life, often revealing meaningful insights that might not be available with polysomnography or self-report data. While sleep architecture and sleep staging are difficult to estimate with accelerometry alone, actigraphy is sensitive to treatment effects in previous studies of sleep disorders.1
Back in July, Merck’s pneumococcal vaccine Vaxneuvance got the seal of approval from the FDA to treat adults, with analysts predicting it could be a blockbuster product and secure more than $1 billion in peak sales. A few months later in December, the FDA granted Priority Review for an sBLA for Vaxneuvance to be potentially indicated for minors and infants.
And the federal agency gave the big pharma a PDUFA date of April 1, 2022. But rather than come to a decision Friday, the FDA instead pushed out the PDUFA date by three months to July 1.
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Congress is reportedly close to securing new federal funding for Covid-19 vaccines, testing and treatments, but the sum will be significantly lower than the White House’s initial ask.
Legislators are nearing a deal to provide $10 billion in new Covid-19 funding, the AP was among those to report, setting up a possible final approval next week. The total is less than half of President Joe Biden’s request for more than $22 billion in early March, and also lower than a $15.6 billion deal that was scuttled a few weeks ago.
In a marathon hearing Wednesday, outside FDA advisors recommended to the agency that a single randomized study did not provide conclusive proof over whether an experimental ALS drug is effective.
Panelists voted 6-4 in the negative, substantiating the agency’s concerns about the robustness of data in a Phase II study run by Amylyx Pharmaceuticals — by the slimmest possible margin. The drug, known as AMX0035, produced a statistically significant reduction on a functional rating scale, but the effect proved unconvincing to the committee.
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US regulators could have full access to auditing reports from many of the 200-plus China-based companies listed in New York as soon as the middle of 2022, according to a Bloomberg report.
Currently in the drafting stage, the framework from the China Securities Regulatory Commission and other national agencies would let most Chinese firms keep their US listings, Bloomberg said, citing anonymous sources. But those that hold sensitive data could still be delisted, the news service reported.
Roger Perlmutter has reeled in another big fish.
Just a week after luring Roy Baynes from Merck Research Laboratories to his new startup Eikon Therapeutics, Perlmutter struck again Thursday in bringing Merck’s ex-CEO Ken Frazier onto Eikon’s board of directors. The move will become effective starting Friday — and the team Perlmutter is assembling is no April Fool’s joke.
“It is a great honor to welcome Ken as our first independent board member,” Perlmutter, who serves as Eikon’s chair and CEO, said in a statement. “Our company will benefit enormously from Ken’s deep knowledge of the pharmaceutical industry, and from his broad perspective on matters of business, law, and the needs of society.”
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Three months after BridgeBio’s TTR amyloidosis candidate suffered a major — and according to CEO Neil Kumar, “baffling” — Phase III fail that wiped out $4 billion in market cap, Endpoints News has learned that the company is swinging the budget axe, all while lying low as best it can during the restructuring.
After receiving a call and tracking social media posts regarding a number of layoffs at BridgeBio, Kumar confirmed that the company has made cuts without specifying just how many.
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While referring to himself as living in the proverbial “overtime” of life at the age of 70, newly Senate-confirmed FDA chief Rob Califf said his hope, at the end of his second run at the agency, is to leave the system in a way that enables people to better find out the truth about the risks and benefits of products the FDA regulates.
The famed clinical trialist from Duke, who also recently spent time at Google’s parent company Alphabet, made clear that his vision is to fairly evaluate the real value and worth of new products, and to quickly dispose of investigational products that aren’t useful.
The FDA on Wednesday rejected Akebia Therapeutics’ vadadustat, once a frontrunner to treat anemia due to chronic kidney disease, due to safety concerns.
In issuing a CRL, the company, which saw its stock price fall by more than 65% this afternoon, said in a statement that the FDA has requested an additional trial and that the safety questions centered on the drug’s “failure to meet non-inferiority in MACE in the non-dialysis patient population, the increased risk of thromboembolic events, driven by vascular access thrombosis in dialysis patients, and the risk of drug-induced liver injury.”
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The University of Kansas
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