Now that 2023 is upon us, now’s the time to gather some key intel from the past year: What did we learn from some of the biggest stories in life sciences? And how can we apply those learnings in the year ahead?
To gain some perspective, we tapped SMEs from across the Norstella brands to weigh in. Here’s what they had to say.
Amy Brown, Deputy Editor, Evaluate Vantage: To me, the biggest stories came from the latest innovations on the market. In obesity, we now have the first drugs that actually work, and not only on weight loss but other important metabolic and cardiovascular measures that could make a real difference to patients. We are waiting for more data in 2023 to see how Mounjaro is going to perform in other related conditions; one analyst has predicted that Mounjaro might become the first $100 million drug!
In Alzheimer’s, we’ve had the first unequivocally successful Phase 3 trial. And while these amyloid-targeting antibodies are not a cure, they might slow down disease progression, offering the first real hope in this space. There’s still much room for improvement and a lot of research needs to happen, but at the very least this could persuade more money into Alzheimer’s research, which has suffered from lack of progress.
Margaret (Meg) Richards, PhD, MPH, Executive Director of Solutions, Panalgo: One of the biggest life science stories of 2022 involves advancements in mRNA immunology and vaccinology. Messenger RNA (mRNA) is a single-stranded RNA molecule that complements DNA and tells your body how to make proteins. This technology played a prominent role in the creation of highly effective and safe COVID-19 vaccines. Now, leading biotech firms are betting on mRNA therapies to treat other viral diseases such as HIV, Lyme, Ebola, Zika and more. Moreover, because of mRNA’s effect on the production of proteins, mRNA may be able to combat diseases in which proteins are missing (like cystic fibrosis), which could radically alter the treatment landscape for these conditions. The enthusiasm for mRNA vaccinology has spilled over into other platforms and other conditions, including a vaccine to prevent fentanyl overdose.
Jayne Hornung, Chief Clinical Officer, MMIT: I hate to beat a dead horse, but the biggest story in life sciences in 2022 had to be the CMS decision not to cover Biogen’s controversial Alzheimer’s drug Aduhelm. The decisions sent waves through the drug industry, Wall Street, patient advocate groups and Congress. It shined light on the accelerated approval process and whether it functions as it should. Even though the FDA gave “provisional approval” to Biogen, allowing it nine years to provide evidence that the drug slows the progression of Alzheimer’s, CMS responded by exerting its own power and denying coverage of an expensive drug outside of a clinical trial. Drug companies, pharma investors and patient advocacy groups all responded with alarm and are worried this could happen to other expensive drugs on the accelerated approval path.
Jon Moser, Chief Innovation Officer, Norstella: This decade has seen huge scientific progress with new technologies like cell and gene therapy and mRNA, including “untreatable” diseases having significant breakthroughs (Alzheimer’s), and protein structures being released into the wild (AlphaFold). During 2023, I expect to see further leaps forward in all of these areas, and more curative therapies for conditions where people are suffering, with new technologies leveraging AI and proteomics to treat patients in better, more targeted ways.
Particularly, the focus on obesity as a medically treatable condition excites me. We’ve seen the first generation of meaningful obesity drugs arrive this year. Successfully treat obesity and people’s lives improve, hospital admissions reduce, healthcare bills reduce, and more money can flow to social care and to other medical unmet needs. This could be one of the most meaningful medical interventions in recent history from a societal perspective.
Mike Munsell, Director of Research, Panalgo: I am most excited about the use of real-world data (RWD) for trial design and the application of advanced methods, such as machine learning, for the optimization of study insights.
This year, the FDA kicked off the Advancing Real-World Evidence Program, in which they partner directly with drug makers to develop regulatory-grade RWD protocols prior to study initiation. The European Medicines Agency (EMA) also continued to make progress on their goal of establishing guidelines for the use of real-world evidence (RWE) in regulatory decision-making by 2025, with a proposed framework on data quality and governance. The creation of guidelines and programs such as these by regulatory bodies gives credibility for the use of RWD in regulatory submissions, which helps explain why the percentage of Phase IV trials using RWD has doubled since 2020—a trend that we can expect to see continue in 2023.
With the increased use of RWD, advanced methods that help to analyze unstructured and high-dimensional information, such as machine learning, will continue to gain traction in the industry. While many traditional statistical methods will continue to be the gold standard for establishing causality, machine learning will continue to find its place by optimizing various segments of the analytical pipeline, including target trial emulation, protocol optimization, trial risk management, and determining a technology’s value in the marketplace through retrospective analyses. In 2022, many professional organizations began to release guidelines for the use of machine learning in research, often highlighting the need for transparent and explainable applications. In 2023, we will see these recommendations begin to have an impact on study design and output.
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