drug development Archives - Medical Design Sourcing

Real-world evidence use is expanding. What’s behind the change?

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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.

To maintain their competitive edge, biopharma executives need to develop a comprehensive early data strategy, noted executives from Evidera, part of the PPD clinical research business of Thermo Fisher Scientific, in a recent Q&A. This strategy should focus on carefully selecting and effectively harnessing real-world data (RWD) to support regulatory and HTA approvals.

The use of RWE, however, is not without challe…

In 2023, Roche and Novartis led the pack in drug pipeline scale

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When reviewing R&D spending trends for 2023, Merck & Co. is a clear outlier given its decision to count its $10.3 billion Prometheus acquisition as an R&D charge. In all, the company committed more than half of its revenue to R&D. But Swiss giants Roche and Novartis remain frontrunners in terms of their pipeline of potential new medications. Both have more than 190 drugs in their pipelines.

Roche’s diversified approach ranges from oncology to ophthalmology

Roche’s focus spans oncology, immunology, neuroscience, and cardiovascular diseases. The company’s oncology portfolio stands out with candidates such as inavolisib and giredestrant that target novel mechanisms in breast cancer. Additionally, Inavolisib, a PI3K inhibitor, targets the PI3Kα isoform, involved in the pathways that drive cell growth and survival. It shows potential not only in breast cancer but also i…

Could LSD change the game in anxiety treatment?

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A once-controversial psychedelic substance could potentially be a promising treatment for generalized anxiety disorder (GAD). That’s the view of Dr. Rakesh Jain, a psychiatrist with extensive experience in clinical practice, research, and education, affiliated with Texas Tech University School of Medicine. Jain expressed optimism in LSD-based therapy while acknowledging the challenges inherent in such a radical shift, in a recent MindMed analyst briefing on the heels of its MM-120 winning a breakthrough designation for an LSD-based treatment for GAD, based on promising topline data from a phase 2b study.

A once-controversial psychedelic substance could hold the key to treating Generalized Anxiety Disorder (GAD). This is the view of Dr. Rakesh Jain, a prominent psychiatrist who serves as a clinical professor at the Department of Psychiatry at Texas Tech University School of Medicine. While ack…

Are Big Pharma giants getting the right ROI on their R&D investments? A visual exploration

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While annual reports show broadly similar R&D strategies among Merck & Co., Pfizer, Johnson & Johnson and AbbVie, their 2020–2023 financial metrics reveal a concerning trend. Merck & Co. may be the new top dog of Big Pharma, but the firm’s 1.4% revenue growth in 2023 represents a significant slowdown. Pfizer’s 41.7% revenue decline from its COVID-inflated $100.3 billion peak in 2022 is even more stark. Similarly, J&J’s modest 4.2% growth and AbbVie’s 6.4% revenue dip also signal a departure from previous patterns.

In raw numbers, R&D spending in the pharmaceutical industry has surged over the past few decades, jumping from about $30 billion across the industry to more than $200 billion annually by the 2020s. Despite record-breaking R&D spending hitting $161 billion in 2023, marking a nearly 50% increase since 2018, as IQVIA has noted, …

Moving the needle on diversity in clinical trials: Where do we go from here?

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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 pregnant and lactating, pediatric and elderly patients, people with disabilities, LGBTQ+ individuals, and racial/ethnic groups specifically, Black/African American, American Indian/Alaska Natives, Hispanics/Latinos, Asians, Native Hawaiian, and other Pacific Islanders.

As a result, the U.S. government has taken increased measures with the passage of the Food and Drug Omnibus Report Act of 2022 (FDORA), which will require sponsors to submit diversity plans to the U.S. Food and Drug Administration (FDA) for all late-stage st…

From gatekeeper to strategist: The evolution of the CISO role in drug development

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There’s an old joke about chief information security officers (CISOs) being gatekeepers of new technologies and initiatives – the infamous “Department of No.” Imagine a bouncer who, strangely, doesn’t let anyone in, saying the club is already too full, even when it’s clearly empty.

But that image is outdated — especially in risk-focused industries like financial services where CISOs are integral to digital transformation projects and the broader risk management considerations.

From CIS-‘no’ to risk maestro

“Drug development is a risk-focused industry as well,” said Daniel Ayala, chief security and trust officer for Dotmatics. “There is a huge amount of risk.” Consequently, CISOs working in pharma contexts are increasingly expanding their roles from technical experts to risk-aware business leaders who happen to have deep technical expert…

Off with the training wheels: AI-based patient characterization can improve clinical trial performance without large data sets

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Only 12% of new drug candidates that enter phase 1 clinical development ultimately receive FDA approval. This dismal success rate leaves millions of patients with unmet medical needs and drives up the costs for the small number of drugs that make it to market. More frustratingly, it leaves untold numbers of potentially transformative therapies back-burnered or discarded entirely, not because they don’t actually provide benefit, but because they were tested in trials that weren’t effectively designed to demonstrate benefit. The true failure hasn’t been in drug innovation but in identifying the patient traits that govern clinical trial outcomes.

The big challenges of big data methodologies

Artificial intelligence (AI) holds great promise in improving this success rate by providing data-driven approaches to identifying traits and their combinations that enable more effective paradigms to enrich patien…

Rice Biotech Launch Pad plans to make Houston a top-tier biotech hub

Rice Biotech Launch Pad: A collaboration hub at TMC’s Helix Park. [Image courtesy of TMC Helix Park | Texas Medical Center]

Houston boasts many world-class assets that have made it a formidable player on the global stage. From the world’s largest medical complex to mission control for the cosmos, few other cities can compete with its diverse strengths. Houston is also home to the prestigious Rice University, renowned for its leading science and engineering programs.

Houston, the nation’s fourth largest city, has not traditionally been a leader in biotechnology, but Rice University’s new Rice Biotech Launch Pad aims to change that. As Omid Veiseh, director of the Launch Pad, explains, “What we wanted with the Launch Pad was something different.” The goal is to capitalize on Rice’s research strengths and the vast medical expertise at the nearby Texas Medical Center.

Ho…

Could an ‘extreme’ hibernating ground squirrel unlock new obesity treatments?

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In late 2023, Eli Lilly, whose stock is now up close to 80% over the past year, inked a deal with the Emeryville, California–based Fauna Bio potentially worth $494 million that focuses on the discovery of novel drug targets for treating obesity. In 2020, Fauna entered into an obesity-focused collaboration with Novo Nordisk, Lilly’s primary rival in the obesity treatment market. Coincidentally, Novo Nordisk’s stock is up more than 50% over the past year, thanks in large part to strong sales of the GLP-1 drugs Ozempic and Wegovy.

In the hunt for the next obesity blockbuster, both Lilly and Novo Nordisk are turning to Fauna Bio’s expertise in extreme mammal genomics. Among Fauna Bio’s focus areas is the 13-lined ground squirrel, an example of the company’s emphasis on “extreme mammals.” The squirrels are known for their remarkable metabolic transformations during hibernation that make them…

Biotech bounces back at JPM 2024 on optimism, breakthroughs and calculated bets, but uncertainties persist

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At the dawn of 2024, there’s a sense of renewed optimism in the biotech sector despite a downturn that has lasted for more than two years. At the JP Morgan Health Care Conference, deal-making activity showed signs of strength. For instance, Merck agreed to acquire cancer drug developer Harpoon Therapeutics for roughly $680 million, highlighting continued interest in oncology cancer therapies. Meanwhile, Novartis announced plans to acquire Calypso Biotech, a deal involving an upfront payment of $250 million with potential milestones worth up to $175 million.

“I feel like in the last two or three weeks, we’ve almost made up for 50% of the deals that didn’t happen in 2023,” quipped Jen Nwankwo, CEO of 1910 Genetics, a company specializing in computational biology and automated laboratory technologies. Pointing to recent clinical successes and FDA approva…

Core trends in 2023 FDA drug approvals: Oncology, neurology and hematology dominate

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 blockbuster from the 2023 cohort with expected peak sales of $1.9 billion. Alzheimer’s drug Leqembi could also be a major success, making up for the tepid demand for Aduhelm, which won conditional approval in 2021. While Aduhelm’s sales have sputtered, Leqembi could see peak revenue of $4.8 billion according to analysts.

Oncology continues to be a hot specialty with the most competition among the newly approved drugs, as the FDA continues to green light new entrants in crowded target classes such as PD-1 inhibitors.

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How a J&J exec found her calling in autoantibody drug development

Katie Abouzahr

Dr. Katie Abouzahr’s career, which began in the wards of the UK’s National Health Service (NHS) before extending into management consulting, paved the way for her leadership of autoantibody and maternal fetal medicine programs at Johnson & Johnson. “It’s not a typical pharma executive’s straight line path,” she acknowledged. “Careers can often be jungle gyms as opposed to ladders that you climb one rung at a time. It’s always a mixture of opportunity, timing, serendipity, and persistence.”

Those qualities helped pave the way for her current focus on autoantibody-based conditions marked by high unmet need. Drawing on her experience across clinical medicine, consulting, and drug development, Abouzahr focuses on advancing medicines for autoimmune and autoantibody-driven diseases, which are marked by high unmet need. The broader immunology area is a central …