In August 2024, MDMA, which is better known as ecstasy, faced a setback as a potential therapy for PTSD—a complete response letter from the FDA. While its developer, Lykos Therapeutics, plans on launching a new Phase 3 trial with tighter scrutiny, another psychedelic company, MindMed, is readying a pivotal study of LSD for generalized anxiety…
Important ERP considerations for biotech manufacturers
Manufacturers in the biotech sector face unique challenges ranging from stringent regulatory requirements to complex production processes. Implementing an ERP (enterprise resource planning) system tailored to their needs is crucial for ensuring compliance, efficiency, and scalability. Whether you are a life sciences CMO (contract manufacturing organization), a pharmaceutical manufacturer with an R&D department, or a…
The future of RWD and RWE in healthcare decision-making: Applications of novel real-world data collection methods for healthcare decision-making
Real-world evidence and real-world data The use of real-world evidence (RWE) to support regulatory and reimbursement decision-making received increasing attention over the past decades. The US FDA provides a definition of RWE as “the clinical evidence about the usage and potential benefits and risks of a medical product”1. RWE can be used across the entire…
Key toxicology considerations for large molecule therapies
Large molecule therapies hold immense promise for treating an array of diseases, but their development presents unique challenges in preclinical testing. Toxicology studies form a cornerstone of this process, providing essential data on the drug’s biological effects. While small molecule drugs have been the focus of drug development for decades, large molecule therapies present unique challenges…
See how much Medicare plans to cut drug prices for Eliquis, Jardiance and more
Medicare is taking its scalpel to drug prices, excising billions in costs for popular medications like Eliquis and Jardiance. The Biden-Harris Administration announced significant agreements with drug makers that would cut list prices by up to 79% for the first 10 drugs selected under the new Medicare drug price negotiation program. CMS announced last year…
4 waves of NLP techniques and how to stitch them together
While clinical trials and regulatory filings offer a semi-structured view of drug safety, a large amount of insights lie in sources ranging from patient support programs (PSPs) to social media posts. As Natural Language Processing (NLP) evolves, a growing number of tools are becoming available to unlock this potential. Deepanshu Saini, Director of Program Management…
From social media to safety signals: How AI and NLP are transforming drug safety monitoring
The pandemic, the rise of decentralized trials and a wave of technological advances have each played a role in reshaping clinical trials and safety reporting. While these events have been changing the industry, the internet’s growing convenience and accessibility has also empowered and transformed the patient experience. Patients are increasingly turning to online platforms, particularly…
Phesi’s digital twin study uses 2,000+ patient records to model cGvHD treatment outcomes
Phesi, a Connecticut-based clinical development analytics company, has published research in Bone Marrow Transplantation showing the potential of digital twins to replace standard-of-care control arms in clinical trials. The study focused on chronic graft versus host disease (cGvHD), a serious complication affecting 30–50% of the 50,000 cancer patients who receive hematopoietic cell transplantation (HCT) each…
Q&A: Pharma’s revenue strategy overhaul in the IRA era
The pharmaceutical industry has long faced something of a perfect storm of regulatory upheaval and technological disruption. But now is different. The Inflation Reduction Act represents a unique challenge for pharma companies as the U.S. government can force them to negotiate drug prices for some Medicare drugs. And despite its name, some pundits believe the…
Experts endorse MDMA-assisted therapy for PTSD amid FDA adcomm setback
A group of prominent researchers and clinicians have published a consensus statement backing the use of MDMA-assisted therapy for post-traumatic stress disorder (PTSD). This statement comes as FDA is reviewing a New Drug Application for midomafetamine (MDMA) for the condition. “On the basis of the safety and efficacy data we have seen, assuming the integrity…
How BMS aims to close the LGBTQ+ data gap in clinical trials
In terms of treatment outcomes, the LGBTQ+ community continues to face significant disparities. For instance, a recent study in JAMA on breast cancer outcomes reported that those from sexual and gender minority groups were more likely to face delays in diagnosis. LGBTQ+ patients were also more likely to decline oncologist-recommended therapies while experiencing a three-fold…
New SEC rules and Cencora breach result in surge of pharma data breach disclosures
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…
How real-world evidence challenged anesthesia guidelines for diabetes patients on GLP-1 meds
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…
Five keys to successfully implement functional service partnerships
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…
Best practices to overcome communication breakdowns in pharma manufacturing
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…
How pharma companies can fight back against the rise of fake drugs
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…
Pharma’s cultural barriers could blunt AI progress despite pressures for more efficient drug approvals.
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…
Pharma R&D returns rebound in 2023, but regulatory challenges and AI advances demand further strategic adaptation
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…
How much does a clinical research associate impact a company’s carbon footprint? More than you might think!
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…
Real-world evidence use is expanding. What’s behind the change?
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.…
From Novartis to Pfizer: A closer look at novel cell and gene therapy pricing and reimbursement strategies
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…
Public vs. private: Who’s leading the charge in H5N1 preparedness?
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…
New pharma and biotech manufacturing facilities and expansions announced worldwide for 2024
[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…
How the FDA approval of J&J’s Opsynvi could simplify treatment and improve outcomes for PAH patients
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…
Clinical trials have a diversity Problem. Real-world data could be part of the solution
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…