Regulatory affairs

The pharmaceutical industry is grappling with the consequences of a significant data breach at Cencora, the prominent drug distributor formerly known as AmerisourceBergen. Recent cybersecurity rules from the U.S. Securities and Exchange Commissions (SEC), which came into effect in December 2023, have also played a role in a wave of data breach reports, highlighting the…

In 2023, the American Society of Anesthesiologists (ASA) issued guidelines advising patients to discontinue GLP-1 receptor agonists (GLP1-RAs) a week before surgery, citing concerns over potential operative complications, leading to delayed or rescheduled surgeries across the U.S. Dr. David Klonoff, a diabetes researcher and Endocrinologist at Mill Peninsula Medical Center, thought it was curious that…

Strategically selecting the right outsourcing models can accelerate the cost and time curve of drug development by maximizing quality, operational success, and financial efficiencies. To help meet increasingly demanding timelines, organizations are looking more to functional service partnership models to outsource specific functions of clinical trials (e.g., clinical operations, pharmacovigilance, medical writing, etc.). The increasing…

The success of any organization is dependent on consistent, clear, and reliable information sharing among all internal stakeholders. In the pharmaceutical industry, where there are multiple complex layers within manufacturing processes, communication among teams is especially critical. If information-sharing gaps occur among these layers, vulnerabilities could result in costly errors impacting all levels of the…

The global counterfeit drug trade has exploded into a deadly public health crisis, with the rise of online pharmacies and increasingly sophisticated criminal networks fueling the trend. A 2020 analysis from the Organisation for Economic Co-operation and Development (OECD) and the European Union Intellectual Property Office (EUIPO), found that the problem is global in scope…

The FDA’s Center for Drug Evaluation and Research (CDER) recently unveiled the launch of the CDER Center for Clinical Trial Innovation (C3TI), which aims to drive clinical trial innovation. When asked to assess the potential impact of the center, Charles Fisher, founder and CEO of Unlearn, said: “The truth is, I don’t know. I’m sort…

Deloitte’s 14th annual report on pharma innovation reveals a modest uptick in R&D returns for 2023, with the projected internal rate of return (IRR) for the pharma sector rising to 4.1% after hitting a low of 1.2% in 2022. Yet the report emphasizes the need for biopharma companies to adapt to ongoing challenges and leverage…

On March 6, 2024, the U.S. Securities and Exchange Commission approved a rule to require some companies to report some of their carbon emissions (Scope 1 and 2, but not Scope 3)1. While some feel this rule doesn’t go far enough, it certainly demonstrates the need to better understand our respective carbon footprints and develop…

In the rapidly evolving landscape of drug development, real-world evidence (RWE) is making significant strides, finding its way into the early stages of the process. No longer confined to just the peri- and post-approval phases, RWE is now being tapped for external control arms (ECAs), aiding in health technology assessment (HTA), and informing payer discussions.…

Cell and gene therapies are upending the treatment of a growing number of diseases by addressing the underlying causes of genetic disorders. Yet the high costs associated with these therapies, sometimes costing multiple millions of dollars for a single treatment, pose significant challenges for patients, payers and healthcare systems. To address this matter, a growing…

The recent emergence of H5N1 avian influenza in humans and many other animals has intensified global efforts to prepare for a potential pandemic. Public health agencies and international organizations are collaborating with pharmaceutical companies and academic institutions to develop vaccines, treatments, and strategies to mitigate the impact of an outbreak. The CDC, for instance, has…

[Updated May 10, 2024] In the face of rising R&D costs and growing pricing pressures from payers, the pharma and biotech sectors continue to transform to adapt to an evolving landscape. While workforce reductions persist in 2024 for some companies, major players like AbbVie, AGC Biologics, Amgen, Novartis and Thermo Fisher Scientific are demonstrating confidence…

Pulmonary arterial hypertension (PAH), a rare, progressive and life-threatening blood vessel disorder, affects some 500 to 1,000 new patients each year in the U.S. FDA recently approved Opsynvi, a first-of-its-kind once-daily single-tablet combination therapy from Johnson & Johnson. “With this approval, our portfolio now includes treatments that address all three guideline-recommended pathways,” said a J&J…

While diversity in clinical trials has won attention in recent years, uneven representation of diverse populations in clinical trials remains a core barrier to global healthcare equity. Real-world data (RWD) paired with artificial intelligence techniques can almost instantly analyze how well drugs work in diverse subpopulations once they hit the market. RWD can also help…

The startup Unlearn embodies several trendy AI characteristics. Generative AI company? Check. San Francisco headquarters? Check. Aims to disrupt drug development (specifically, clinical trials) with AI? Check. Prominent AI leadership? Check. Mira Murati, the high-profile CTO of OpenAI, joined Unlearn’s board of directors in 2023. But the company’s pedigree is unique. The firm was founded…

The recent FDA approval of a cardiovascular risk reduction indication for Wegovy (semaglutide) could point toward a significant opportunity for pharma companies seeking to reshape payer perceptions and expand coverage for next-gen metabolic therapies. This regulatory shift, allowing Wegovy to be prescribed for reducing the risk of major adverse cardiovascular events such as heart attack…

The FDA continues to signal openness to psychedelic-based therapies. Following its recent Breakthrough Therapy Designation (BTD) for a potential LSD-based anxiety treatment, the agency has extended the same status to Cybin Inc.‘s CYB003 for major depressive disorder (MDD). The move marks the first FDA Breakthrough Therapy Designation for an adjunctive, psychedelic-based Major Depressive Disorder treatment.…

Enhancing patient diversity in clinical trials has become a key priority in drug development. The main concern is that critical data that includes underrepresented patient populations is being left out as many clinical trials do not reflect all populations that may eventually take a therapy. These underrepresented groups consist of women, including those who are…

In 2023, a year of accelerated regulatory success, a significant number of biotech labs sat empty in major hubs like San Francisco and Boston. The FDA approved 55 novel therapies in 2023, including Leqembi for early Alzheimer’s and Zurzuvae for postpartum depression. The approval number marked the second highest count in three decades (see graph…

The increasing complexities in clinical trials, including expanded patient populations, decentralized trials and new technologies, are directly impacting the amount of data and information available to clinical trial sponsors. Though this provides significant value to the industry, it presents a new challenge of consolidating disparate and siloed systems, improving standardization and unifying the explosion of…

2023 was a big year for hematology, neurology and oncology, with the medical specialties seeing the most FDA approvals. In terms of sponsors, Pfizer had the most approvals with six total, followed by UCB and Chiesi, each with three apiece. When looking at commercial prospects, AstraZeneca’s respiratory syncytial virus antibody Beyfortus could be the biggest…

In late 2022, we published a series of predictions, which, among other things, projected that 2023 would be a “massive showcase” for machine learning (ML) in drug discovery and development. And in many ways, 2023 was a pivotal year for AI in pharma. This evolution in AI and ML applications in pharma built upon the…

In July, the life sciences services company Eversana revealed it was partnering with AWS to tap generative AI (gen AI) for pharma and other life science customers. It aimed to “pharmatize” gen AI, as the company put it then. Now, the company has revealed the first technology from that partnership – a regulatory review automation…

Abstract The vast majority of vaccines are prophylactic in nature. As a result, the demonstration of their efficacy paradoxically requires the infectious disease to occur among non-diseased study participants randomized between investigational vaccine and appropriate control groups. The statistics of vaccine efficacy (VE) calculation are nearly entirely and solely based on the number of observed…

Drug development is often a vast and intricate journey. Each phase signifies an advancement in the process, always with an eye toward patient safety and efficacy. But before any therapeutic finds itself on the bedside tables of hopeful patients, it faces a formidable challenge: preclinical toxicology testing. As the gateway to clinical trials, this early…